Podcasts about qaly

public health diabetes guidelines mash therapies breadth health economics metabolic disease cost effectiveness disease burden qaly qalys louise campbell

Play Episode Listen Later Apr 11, 2024 1:58

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Should I donate my kidney or part of my liver?, published by Bob Jacobs on April 11, 2024 on The Effective Altruism Forum. I've been talking with my hospital about donating my kidney and it's been going rather well. However, one piece of unfortunate news they told me is that I can't donate both my kidney and a piece of my liver (and that I can't do this in another hospital either). So people that want to donate are faced with a dilemma of which one to choose. I asked the doctors whether they had literature on this, but unfortunately they didn't know of any that compared the two. I've looked at some papers, and the side effects for both kidney donation and liver donation seem to be negligible for the donor (way less than 1 QALY). That leaves us with the question of what has the bigger impact for the recipient. I've looked for papers that compared them directly, but couldn't really find anything. It seems like for kidneys: The average donation buys the recipient about 5 - 7 extra years of life (beyond the counterfactual of dialysis). It also improves quality of life from about 70% of the healthy average to about 90%. Non-directed kidney donations can also help the organ bank solve allocation problems around matching donors and recipients of different blood types. Most sources say that an average donated kidney creates a "chain" of about five other donations, but most of these other donations would have happened anyway; the value over counterfactual is about 0.5 to 1 extra transplant completed before the intended recipient dies from waiting too long. So in total, a donation produces about 10 - 20 extra quality-adjusted life years. Liver donation seems to generate less QALYs, though the estimates vary a lot. So I'm currently leaning towards donating my kidney. Does anyone have any more insights into this? Does anyone know of an analysis that compares the two? (If someone is/wants to write one, I'd be glad to help) Please share your thoughts. Thanks for listening. To help us out with The Nonlinear Library or to learn more, please visit nonlinear.org

speech ea jacobs liver kidney rationalist qaly qalys

S5 - E4.2 - The Breadth Of MASLD Disease Burden And Cost Effectiveness Of Therapies

Surfing the Nash Tsunami

Play Episode Listen Later Mar 3, 2024 10:45

Initially, this conversation focuses on how cost-effectiveness issues relate to the MASLD Disease Burden. In the process, Zobair Younossi provides education on some of the metrics and concepts pivotal to drug value assessment.Roger Green starts off asking how the economics of treating MASH stack up against hypercholesterolemia at the birth of statins in the 1980s, where the medical benefit was clear but economic was harder to manage. Zobair proceeds to describe the process by which the cost effectiveness of drugs is measured, computation of Quality Adjusted Life Years, or QALYs, and how different countries vary in the level of QALYs they consider cost effective. He also notes that within the US, at least, we may be willing to accept five times greater cost per QALY than for another. He also points out that cost effectiveness grows as new therapeutic options include price competition into a market.Louise Campbell shares the specific US cost numbers from Zobair's article, which she describes as “frightening,” particularly given the rate of growth in the disease and society's lack of efficacy in shifting the curve on this. Zobair responds by saying that one goal of the article was to create awareness that regular surveillance of diabetic patients for MASLD could have a significant economic impact in the US. As the conversation winds down, Jörn Schattenberg comments that all this is a team effort and Zobair agrees heartily.

QALY Ban Bill Takes Leap Forward

drop forward senate leap senior fellow bill smith qaly pioneer institute

Play Episode Listen Later Feb 12, 2024 17:20

This week's podcast is all about the QALY and the bill going to the Senate that would ban its use. The QALY is a metric used to determine how cost-effective a treatment is based on its ability to return a sick person to perfect health. Even if a treatment helps a chronically ill person to live longer, it will not be categorized as valuable as treatments are aimed at diseases that affect younger and healthier populations. To help us understand how the QALY negatively impacts those with chronic illness, we're joined by Michael Riotto, a member of Patients Rising, and Bill Smith, a Senior Fellow at the Pioneer Institute. Need help? The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at the Patients Rising Helpline. Have a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent? Drop us a line: podcast@patientsrising.org The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising, nor do the views and opinions stated on this show reflect the opinions of a guest's current or previous employers.

QALY Ban Passes House

house senate senior fellow disabilities house committees bill smith chronic illnesses qaly pioneer institute

Play Episode Listen Later Feb 8, 2024 4:00

On Wednesday morning, the House passed H.R. 485, the Protecting Care for All Patients Act, which bans the use of Quality-Adjusted Life Years (QALYs) in federal health programs. Bill Smith, a Senior Fellow at the Pioneer Institute, discusses what this means for patients and what the bill could mean for the future. Patients Rising has been pushing for a QALY ban for a long time and remains committed to working with all parties in the Senate to ensure all patients are valued equally in the eyes of the federal government. House Committee on Energy & Commerce Press Release: House Votes to Ban Metric Used to Deny Care for People with Disabilities and Chronic Illnesses

QALY Ban Bill Progresses

house congress senate committee patient advocate qaly

Play Episode Listen Later Feb 6, 2024 3:42

Michael Riotto, a Patient Advocate and member of Patients Rising, tells us how the exciting developments with the QALY ban bill could affect him and his health if it moves forward. Riotto discusses how the QALY negatively affects him and others in similar situations and what it would mean if it were banned. Patients Rising has been pushing for a QALY ban for a long time and the possibility of this going to the House floor is a big step in the right direction. Patients Rising is also submitting a letter to Congress signed by patients from all 50 states advocating for the passage of this bill. Senate Committee on Rules Announcement: Announcement for H.R. 485 – Protecting Health Care for All Patients Act of 2023 Patients Rising Now Webpage: More Than 100 Patient Advocacy Organizations In Support H.R. 485 – The Protecting Healthcare for All Patients Act

Willingness to Pay, wat zijn we bereid te betalen: waar komt het vandaan en waar staan we nu

Oncologie Up-to-date

Play Episode Listen Later Jan 15, 2024 23:56

In deze podcast bepreekt Koos van der Hoeven met Hans Severens over de Willingness to Pay Threshold. Aan bod komen onder andere begrippen als incremental cost-effectiveness ratio (ICER), quality adjusted life year (QALY), wat is de Willlingness to Pay Threshold in Nederland, hoe is deze gevormd en hoe wordt deze kosteneffectiviteitsdrempel gehanteerd, en is deze drempelwaarde ooit aangepast sinds introductie?Referentie 1. Kiezen en delen: rapport van de commissie Keuzen in de zorg (Commissie Dunning) (1991). SDU Uitgeverij. Disclosures Dr. Hans Severens: Alexion, Amgen, AstraZeneca, Boehringer-Ingelheim, Daiichi Sankyo, Gilead, GSK, Hikma, Idorsia, MSD, Nova Nordisk, Pierre Fabre, Recor Medical, Sanofi, Takeda. Prof. dr. ir. Koos van der Hoeven: Astellas, Bayer, BMS, Daiichi Sankyo, Gilead, Novartis, Pfizer, Seagen, voorzitter Oncomid, lid Adviescollege VIG en lid RVT DICA.Klik hier voor een vijftal korte vragen zodat wij u in de toekomst nog beter van dienst kunnen zijn.NL-UNB-0610

EA - 1/E(X) is not E(1/X) by EdoArad

Play Episode Listen Later Nov 9, 2023 2:45

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: 1/E(X) is not E(1/X), published by EdoArad on November 9, 2023 on The Effective Altruism Forum. When modeling with uncertainty we often care about the expected value of our result. In CEAs, in particular, we often try to estimate E[effectcost]. This is different from both E[costeffect]1 and E[effect]E[cost] (which are also different from each other). [1] The goal of this post is to make this clear. One way to simplify this is to assume that the cost is constant. So we only have uncertainty about the effect. We will also assume at first that the effect can only be one of two values, say either 1 QALY or 10 QALYs with equal probability. Expected Value is defined as the weighted average of all possible values, where the weights are the probabilities associated with these values. In math notation, for a random variable X, where x are all of the possible values of X.[2] For non-discrete distributions, like a normal distribution, we'll change the sum with an integral. Coming back to the example above, we seek the expected value of effect over cost. As the cost is constant, say C dollars, we only have two possible values: In this case we do have E[effectcost]=E[effect]E[cost], but as we'll soon see that's only because the cost is constant. What about E[costeffect]? which is not 1E[effectcost]=C211$QALY, a smaller amount. The point is that generally 1E[X]E[1X]. In fact, we always have 1E[X]E[1X] with equality if and only if X is constant.[3] Another common and useful example is when X is lognormally distributed with parameters μ,σ2. That means, by definition, that lnX is normally distributed with expected value and variance μ,σ2 respectively. The expected value of X itself is a slightly more complicated expression: Now the fun part: 1X is also lognormally distributed! That's because ln1X=lnX. Its parameters are μ,σ2 (why?) and so we get In fact, we see that the ratio between these values is ^ See Probability distributions of Cost-Effectiveness can be misleading for relevant discussion. There are arguably reasons to care about the two alternatives E[costeffect]1 or E[effect]E[cost] rather than E[effectcost], which are left for a future post. ^ One way to imagine this is that if we sample X many times we will observe each possible value x roughly P(X=x) of the times. So the expected value would indeed generally be approximately the average value of many independent samples. ^ Due to Jensen's Inequality. Thanks for listening. To help us out with The Nonlinear Library or to learn more, please visit nonlinear.org

speech ea inequality cost effectiveness 1x rationalist 1e expected value qaly qalys lnx

QALY Update

director patients revised rare diseases bill smith icer cathy mcmorris rodgers qaly pioneer institute

Play Episode Listen Later Sep 28, 2023 4:48

Bill Smith, Director of the Life Sciences Initiative at the Pioneer Institute, dives into the so-called update to ICER's value assessment framework; Congresswoman Cathy McMorris Rodgers explained earlier this year why she was against the QALY measurement; and Patients Rising Now along with several other groups have issued statements denouncing the ICER update. Pioneer Institute Webpage Patients Rising Podcast Episode: Healthcare Protections for Rare Diseases with Rep. Cathy McMorris Rodgers Patients Rising Now Webpage: Patients Rising Now Statement – ICER's ‘Updates' are Thinly Veiled Strategy to Continue Discriminatory Tactics National Pharmaceutical Council Webpage: NPC Evaluates ICER's Revised 2023 Value Assessment Framework – A Missed Opportunity for Patients and the Field Biotechnology Innovation Organization Webpage: ICER's updated treatment assessment still ignores patient concerns

Gigi Foster estimates COVID lockdowns cost young people 116x any benefits - EP205

Economics Explained

Play Episode Listen Later Sep 12, 2023 62:23

Professor Gigi Foster talks about her paper "COVID's Cohort of Losers" which argues that COVID lockdowns and other restrictions disproportionately imposed costs on young people with few offsetting benefits. Gigi is a Professor of Economics at the University of New South Wales, Sydney and was named the 2019 Young Economist of the Year by the Economic Society of Australia.Please get in touch with any questions, comments and suggestions by emailing us at contact@economicsexplored.com or sending a voice message via https://www.speakpipe.com/economicsexplored. About this episode's guest: Gigi FosterGigi Foster is a Professor with the School of Economics at the University of New South Wales, having joined UNSW in 2009 after six years at the University of South Australia. Formally educated at Yale University (BA in Ethics, Politics, and Economics) and the University of Maryland (PhD in Economics), she works in diverse fields including education, social influence, corruption, lab experiments, time use, behavioural economics, and Australian policy. Gigi's research contributions regularly inform public debates and appear in both specialised and cross-disciplinary outlets (e.g., Quantitative Economics, Journal of Economic Behavior and Organization, Journal of Population Economics, Journal of Economic Psychology, Human Relations). Her teaching, featuring strategic innovation and integration with research, was awarded a 2017 Australian Awards for University Teaching (AAUT) Citation for Outstanding Contributions to Student Learning. Named 2019 Young Economist of the Year by the Economic Society of Australia, Gigi has filled numerous roles of service to the profession and engages heavily on economic matters with the Australian community. As one of Australia's leading economics communicators, her regular media appearances include co-hosting The Economists, a national economics talk-radio program and podcast series premiered in 2018, with Peter Martin AM on ABC Radio National.What's covered in EP205Intro to the cost and benefits of lockdowns. (3:22)Quality adjusted life year (QALY) and WELLBY. (8:07)Fear and the crowd. (13:47)The history of the cordon sanitaire. (16:58)How many lives were saved? (22:14)The cost and benefits of lock-downs. (27:25)The economics of the lockdown. (34:24)How do we determine the severity of pandemics? (36:25)The difference between the 1918 flu and COVID-19. (41:18)Citizen juries. (46:35)New laws about misinformation and disinformation. (49:45)Health and good nutrition. (56:01)Links relevant to the conversationGigi's paper for CIS:https://www.cis.org.au/publication/covids-cohort-of-losers-the-intergenerational-burden-of-the-governments-coronavirus-response/Information on WELLBYs:HM Treasury's Wellbeing Guidance for Appraisal: Supplementary Green Book GuidanceFull transcripts are available a few days after the episode is first published at www.economicsexplored.com. Economics Explored is available via Apple Podcasts, Google Podcast, and other podcasting platforms.

Patients on Capitol Hill

Play Episode Listen Later Jun 13, 2023 4:54

Michael Riotto, a patient advocate from PA-01, says the QALY is top of mind for him as he attends the 2023 Patients Rising Now ‘We The Patients' Fly-In; Tomiyo Williams, a patient advocate from GA-6, shares how step therapy policies hurt her daughter's care. Patients will also be talking about the Dental and Optometric Care Access Act at the Fly-In; and Patients Rising awards eleven companies with its annual “Health Plan Hero” distinction, which recognizes companies with stellar health plans. Patients Rising Now Webpage: We The Patients Fly-In U.S. Representatives News Release: Carter, Clarke, Sessions introduce bill to Ensure Access to Quality Dental and Optometric Care Patients Rising Podcast Webpage: Better Care, Lower Costs Video: Stop Vision Benefit Manager Abuses Michael Riotto's Story

story patients ga capitol hill clarke dental qaly

Better Care, Lower Costs

washington dc drop capitol hill pbms better care lower costs qaly

Play Episode Listen Later Jun 12, 2023 19:04

It's here! The 2023 We the Patients Fly-In is THIS WEEK and we have several Health Plan Hero Winners on the podcast to discuss their health insurance success stories as they get ready to accept their awards in person in Washington D.C. Plus, Terry and Bob lay out all the policy priorities patients will be advocating for on Capitol Hill during the Fly-In, including reforms to PBMs, copay accumulator programs, and the QALY metric. Meet the 2023 Health Plan Hero Winners Need help? The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at the Patients Rising Helpline. Have a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent? Drop us a line: podcast@patientsrising.org The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising, nor do the views and opinions stated on this show reflect the opinions of a guest's current or previous employers.

CMS Alzheimer's Decisions + No Data

washington dc data decisions alzheimer's disease chief operating officer cms rsv acip qaly usagainstalzheimer

Play Episode Listen Later Jun 6, 2023 5:59

Russ Paulsen, Chief Operating Officer for UsAgainstAlzheimer's, explains how some recent coverage decisions from CMS are affecting thousands of patients a day; ACIP's public comment period RSV vaccines is officially open; Tony Newberne, a patient advocate from NC's 14th congressional district, discusses his policy priorities, including QALY reforms, as he gets ready for Patients Rising Now's Washington D.C. We The Patients Fly-In; and Patients Rising Now has submitted a letter to the Massachusetts Joint Committee on Health Care Financing supporting bill S754 which would mandate comprehensive coverage for obesity treatment. UsAgainstAlzheimer's Webpage: New Poll: Huge Majorities Want Medicare to Cover Cost of Alzheimer's Drugs CDC Webpage: ACIP Meeting Information Massachusetts Legislature Webpage: Bill S.754

EU Cost Effectiveness Models Threaten Vulnerable U.S. Patients

Vital Health Podcast

Play Episode Listen Later May 17, 2023 34:49

The Quality Adjusted Life Year, or QALY, was invented at the UK's University of York by Prof Alan Williams in the 1970s. Some currently engaged in the bitter trench warfare of America's drug pricing debate think it's high time for another British invasion, and the US should fully embrace the UK's use of QALYs. In this Vital Health Podcast, we have a discussion with William Smith, a Senior Fellow at the Pioneer Institute, about his recently published book, “Rationing Medicine: Threats from European Cost-Effectiveness Models to America's Seniors and other Vulnerable Populations.” William makes a strong case that the use of QALY for cost-effectiveness assessments within Medicare and Medicaid would violate several key provisions of the Americans with Disabilities Act.See omnystudio.com/listener for privacy information.

Why The WHO Shouldn't Grant Diet Drug Request To Be Added To Essential Medicine List - Part 3

Weight and Healthcare

Play Episode Listen Later Apr 15, 2023 24:50

This is the Weight and Healthcare newsletter! If you like what you are reading, please consider subscribing and/or sharing!In part 1 we talked about a request that has been submitted for the World Health Organization (WHO) to add diet drugs (specifically GLP1 agonists like Novo Nordisk's Saxenda and Wegovy) to their list of “essential medicines.” We discussed who was making this request and the justification that they were using. In part 2 we took a deeper dive into the research that they used to try to support this request, and in this final installment, we will look at the research around efficacy, harm, and cost-effectiveness.First I'll offer a summary for each issue and then I'll give a breakdowns of the research that they cite. Just a quick reminder that this request is asking the World Health Organization (WHO) to add these drugs to their list of “essential medications” globally.Before we get into the sections, I want to mention two overarching issues that are found throughout the entirety of this request and the studies that are used to support it.First, in general, a belief has been fomented (predominantly by those in the weight loss industry) that being higher-weight is so terrible then it's worth “throwing anything at the problem.” This leads to acceptance of poor, short-term, and/or incomplete data as “good enough” to foist recommendations onto higher-weight people, which means that part of weight stigma in healthcare is that higher-weight people are afforded less right to ethical, evidence-based medicine than thinner people.Second, is clinging to correlation (without any mechanism of causation) when it comes to weight, health, and health outcomes, including the abject failure to consider confounding variables. So throughout these studies “being higher-weight is associated with [health issue(s)]” stated uncritically in support of weight loss interventions. There is an utter failure to explore the idea that the reason for the outcome differences is not weight itself but, instead, exposure to weight stigma, weight cycling (which these medications actually perpetuate by their own admission,) and healthcare inequalities. Issues with research supporting effectiveness, harms, and benefitsStudy Duration:This is the main issue. While there was one study that went up to 106 weeks, the vast majority of the studies are between 14 and 56 weeks. We know that these drugs can have significant, even life-threatening side effects (earning them the FDA's strongest warning.) 14-56 weeks is not not nearly enough time to capture the danger of long-term effects, or to capture long-term trends around weight loss/weight regain.Study PopulationMany of the studies included have small samples. Many have study populations are overwhelmingly white, which is a huge issue when making a global recommendations.Small effect and overlapMany of the studies show only a bit of weight loss (often 15lbs or less) and often there was overlap in weight lost between the treatment group and the placebo group. Even using the “ob*sity” construct that this request is based on, for many people, this amount of weight loss wouldn't even change their “class” of “ob*sity.”Failure to capture adverse eventsMuch of the research they use to support their claims of safety didn't actually capture individual adverse events or serious adverse events. Often they only captured subjects who reported leaving treatment due to side effects.Issues with research supporting cost effectivenessThe cost-effectiveness analyses they cite are based on Quality Adjusted Life Years (QALYs). This is a measurement of the effectiveness of a medical intervention to lengthen and/or improve patients' lives.The calculation for this is [Years of Life * Utility Value = #QALY]So if a treatment gives someone 3 extra years of life with a Health-Related Quality of Life (HRQL) score of 0.7, then the treatment is said to generate 2.1 [3 x 0.7] QALYs.This is a complicated and problematic concept that deserves its own post sometime in the future, but looking just at this request I think it's important to note that they are working on two main unproven assumptions:1. That being higher weight causes lower health-related quality of life and/or shorter life span (rather than any lower HRQL being related to experiences that higher-weight people have including weight stigma, weight cycling, healthcare inequalities et al.) 2. That this treatment induces weight loss and/or health benefits that increase the life span and/or health-related quality of life of those who take it.I don't believe either of these assumptions are proven by the material cited in the request to the WHO. Specifically, it's very possible that it's not living in a higher-weight body, but rather the experiences that higher-weight people are more likely to have (weight stigma, weight cycling, healthcare inequalities) that impact their HRQL.Further, the short-term efficacy data available (and Novo Nordisk's own admission about high rates of regain) fall far short of proving any assumptions about these drugs ability to actually improve or extend life. Further, the failure of the literature to adequately capture negative side effects of the drugs, both short and long-term, means that this calculation cannot be properly made.Incremental Cost-Effectiveness Ratio (ICER)ICER is how QALYs are turned into a monetary value. It is calculated by dividing the difference in total costs by the difference in the chosen measure of health outcome or effect.[(Cost of intervention A -Cost of Intervention B) / (Effectiveness of Intervention A – Effectiveness of Intervention B)]The result is a ratio of extra cost per extra unit of health effect of a more vs less expensive treatment which can then be measured in QALYs.Again, this is worthy of its own post because there are all kinds of ethical issues around things like how we value life, how we define “healthy” and the ethics of determining whether or not prolonging someone's life is “cost effective.” I'm not going to do a deep dive into that today, but I do want to note that it is a serious issue in these kinds of calculations.In this specific case, even if one was to get past the ethical issues, an accurate calculation is impossible to make on both of the measures of the equation.Cost of these drugs varies wildly between countries and sometimes within countries because, for example, Novo Nordisk is a for-profit corporation whose goal is to create as much profit as possible. Per the WHO request letter, the monthly cost of liraglutide is $126 in Norway and $709 in the US. Semaglutide is $95 per 30 days in Turkey, but $804 per 30 days in US.When it comes to effectiveness of the treatment, again, there is virtually no long-term data. We do know that in Novo Nordisk's own studies, weight is regained rapidly and cardiometabolic benefits are lost when the drugs are discontinued and even when people stay on the drugs, weight loss levels off after about a year, at 68 weeks weight cycling begins, and at 104 weeks (when follow-up ended) weight was trending up. It's possible that these drugs are utterly ineffective over the long-term and/or that the prevalence of long-term side effects renders any treatment effects moot. We simply do not know.I do not think that this is a remotely appropriate basis from which to request that these drugs be declared globally essential by the WHO.Here are the citation breakdowns. These are not deep dives since there are enough issues with the research on a simple surface analysis.Breakdowns of evidence of comparative effectivenessEffects of liraglutide in the treatment of ob*sity: a randomised, double-blind, placebo-controlled study, Astrup et al.)This is a 20-week study funded by Novo Nordisk. It included 564 people on various doses of liraglutide and a placebo group who didn't get the drug and a group on orlistat. There were no more than 90-98 people in each group.The study explains “Participants on liraglutide lost significantly more weight than did those on placebo” by which they meant that those on the highest dose of liraglutide lose about 9.7lbs more than those on the placebo over the 20 weeks.III LEAD studiesThese are four studies that look at liraglutide in combination with other drugs for the treatment of Type 2 Diabetes that also included some information on weight changes. One was 52 weeks, the others were 26, the maximum amount of weight lost was only about 5lbs. The first [Liraglutide, a once-daily human GLP-1 analogue, added to a sulphonylurea over 26 weeks produces greater improvements in glycaemic and weight control compared with adding rosiglitazone or placebo in subjects with Type 2 diabetes (LEAD-1 SU), Marre et al] was a study that looked at the efficacy of adding liraglutide or rosiglitazone 4 to glimiperide in subjects with Type 2 Diabetes to test effects on blood sugar and body size.The study followed 1041 adults for 26 weeks. The study found that those on .6mg of liraglutide gained 0.7kg, those on 1.2mg gained 0.3kg, and those on 1.8mg of liraglutide lost 0.2kg, while those on placebo lost 0.1kg.The second [Efficacy and safety comparison of liraglutide, glimepiride, and placebo, all in combination with metformin, in type 2 diabetes: the LEAD (liraglutide effect and action in diabetes)-2 study. Diabetes Care, 2009. 32(1): p. 84-90. Nauck, M., et al.,]looked at the efficacy of adding liraglutide to metformin therapy for those with Type 2 Diabetes. They found that over the 26-week study those on liraglutide lost 1.8 ± 0.2, 2.6 ± 0.2, and 2.8 ± 0.2 kg for 0.6, 1.2, and 1.8 mg doses. Those on placebo lost 1.5 ± 0.3kg.The third [Liraglutide versus glimepiride monotherapy for type 2 diabetes (LEAD-3 Mono): a randomised, 52-week, phase III, double-blind, parallel-treatment trial. Lancet, 2009. 373(9662): p. 473-81. Garber, A., et al.,] This was a study of the comparative effectiveness of Liraglutide versus glimepiride for type 2 diabetes, with small weight loss as an ancillary finding. Those in the liraglutide group lost an average of 2kg.The final study [Efficacy and safety of the human glucagon-like peptide-1 analog liraglutide in combination with metformin and thiazolidinedione in patients with type 2 diabetes (LEAD-4 Met+TZD), Zinman et al.] was a 26-week study with 533 total subjects. The goal was to study the efficacy of liraglutide when added to metformin and rosiglitazone for people with type 2 diabetes. They found that those on liraglutide lost between 0.7 and 2.3kg (1.5lbs to 5.1lbs) in 26 weeks.Meta-Analyses and Systematic Review FindingsEfficacy of Liraglutide in Non-Diabetic Ob*se Adults: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Barboza, J.J., et al., None of the included studies were more than 56 weeks and one was only 14 weeks. One had as many as 3731 subjects, but one had only 40. Some had body weight loss as a primary outcome, but some did not. Maximum doses ranged from 1.8 to 3.0mg. The mean body weight reduction was 3.35 kg (7.4lbs) but in one study there was no difference in weight loss. The maximum difference was 6.3kg (13.9lbs)They also refer to Iqbal et al which we discussed in part 2.Effects of glucagon-like peptide-1 receptor agonists on weight loss: systematic review and meta-analyses of randomised controlled trials. Vilsbøll, T., et al.The included studies are between 20 and 53 weeks long, and include some of the studies they already cited individually above. Of the 25 included studies only 3 had “ob*sity” as the main inclusion criteria, the rest were Type 2 Diabetes.The mean weight loss for those on the highest dose of the drug was between 0.2kg and 7.2kg. For those in the control group it was 2.9 kg, so there was actually overlap between the treatment and placebo groups.Summary of evidence of safety and harmsThey begin with the claim “The safety profile of GLP-1 receptor agonists is also well studied”To support this they cite: Efficacy and Safety of Liraglutide 3.0 mg in Patients with Overweight and Ob*se with or without Diabetes: A Systematic Review and Meta-Analysis, Konwar, M., et al.,This included 14 total studies, many of which the authors of the WHO request had cited individually and were included in other systematic reviews and meta-analyses above. The smallest study included 19 people, the largest included 2,487. The total number of subjects was 4,142.Their conclusion was “Liraglutide in 3.0 mg subcutaneous dose demonstrated significant weight reduction with a reasonable safety profile for patients with overweight or ob*sity regardless of diabetic status compared to placebo.”Their methodology says that they omitted studies from analysis due to “short duration.” They included studies that had a minimum of 12 weeks and a maximum of 56 weeks of follow-up.While they included 14 studies, only 11 of them actually included information about adverse events.In terms of adverse effects (AEs,) they found that the pooled estimate of nine studies in nondiabetic patients and two studies in diabetic patients revealed a significant proportion of patients experiencing the adverse events in liraglutide 3.0 mg group when compared with placebo., and the pooled estimate of the eleven studies showed that liraglutide 3.0 mg had higher risk of AEs compared to placebo.When it came to “serious adverse events” they found that there was a similar risk level between the drug and placebo groups, but remember that's for only 12 to 56 weeks, and Novo Nordisk is recommending that people take these drugs for the rest of their lives. A few months to a little over a year is not enough time to capture long-term serious adverse events.The efficacy and safety of liraglutide in the ob*se, non-diabetic individuals: a systematic review and meta-analysis. Zhang, P., et al.,This included five RCTs (which were included in various of the above systemic reviews and meta-analyses) ranging in follow-up from 14 to 56 weeks.The only adverse event information captured was the number of people who withdrew from treatment due to adverse events (which they found was similar between drug and placebo) and nausea (which was experienced more by people on the drug.)So, in addition to being short in duration, this was far from a comprehensive list of side effects. They made no attempt to capture serious adverse side effects and their short-term nature would have made this difficult anyway.Association of Pharmacological Treatments for Ob*sity With Weight Loss and Adverse Events: A Systematic Review and Meta-analysis. Khera, R., et al.This looked at weight loss and adverse events with a number of different weight loss drugs. Interestingly liraglutide did not show the highest amount of weight loss but was associated with the highest odds of adverse event–related treatment discontinuation. It should also be noted that high drop-out rates of 30-45% plagued all of the trials which the study authors admit means that “studies were considered to be at high risk of bias.“Given that those who drafted the WHO request are asking that these drugs be considered essential globally, it is disappointing that they included this study and didn't bother to mention this issue in their written request.This included 28 RCTs (most of which were included in other citations above) and only 3 that included liraglutide. They didn't capture individual adverse events, but only “Discontinuation of Therapy Due to Adverse Events.” They only evaluated a year of data so, again, while it is likely that these studies would have captured common adverse events had they bothered to try, there isn't long enough follow-up to have any information about serious (possibly life-threatening) long-term adverse events.Association of Glucagon-like Peptide 1 Analogs and Agonists Administered for Ob*sity with Weight Loss and Adverse Events: A Systematic Review and Network Meta-analysis. Vosoughi, K., et al.,This study included 64 RCTs with durations from 12 to 160 weeks, with a median of 26 weeks. As is common in these studies, the majority of the sample (74.9%) was white.Like those above, they only looked at treatment discontinuation from adverse events, they did not capture specific adverse events (common or serious.) Of the seven GLP-1 drugs they tested, liraglutide was tied with taspoglutide for the highest discontinuation of treatment due to adverse events.The study authors also note that “Risk of bias was high or unclear for random sequence generation (29.7%), allocation concealment (26.6%), and incomplete outcome data (26.6%).”Breakdowns for Comparative Cost-effectiveness StudiesFirst, the WHO request authors themselves admit that when it comes to cost-effectiveness, “the analyses have generally been performed only for high-income countries.” This is significant since they are asking the WHO to consider these drugs essential for the entire world.It's also important to understand that none of the data looks at a comparison of cost effectiveness for weight-neutral health interventions to these drugs. Without that information there is no way to calculate actual “cost effectiveness” since it's possible that weight-neutral health interventions would have greater benefits with less risk and dramatically lower cost. NICE's guidance: Liraglutide for managing overweight and ob*sity Technology appraisal guidance [TA664]Published: 09 December 2020.Do recall that NICE is involved in the current scandal with Novo Nordisk for influence peddling.These guidelines are created based on a submission of evidence by Novo Nordisk. The committee's understanding of “clinical need” was based on the testimony of a single “patient expert” who “explained that living with ob*sity is challenging and restrictive. There is stigma associated with being ob*se.”Once again we see a rush to blame body size for any “challenges” and “restrictions” of living in a higher-weight body, accompanied by the immediate decision that those bodies should be subjected to healthcare interventions that risk their lives and quality of life in order to be made (temporarily, by Novo and NICE's own admission) thinner. There did not seem to be a patient expert to discuss the weight-neutral options.It was not immediately apparent if the patient expert was provided/paid by Novo Nordisk, but they certainly forwarded their narrative that simply living in a higher-weight body is a disease requiring treatment.It should be noted that while the trial Novo Nordisk submitted covered a wider range of people, they specifically submitted for this recommendation only the subgroup of that population who were diagnosed with “ob*sity,” pre-diabetes, and a “high risk of cardiovascular disease based on risk factors such as hypertension and dyslipidaemia.”So, even if we accept this guidance as true, the WHO Essential Medicines request applies to a population much wider than this and so this fails to justify the cost-effectiveness for that population.This guidance is also based on the costs associated with obtaining the drugs through a “specialist weight management service” since an agreement is in place for Novo Nordisk to give a discount to these services.In calculating the ICER per QALY gained, the recommendations note that “Because of the uncertainties in the modelling assumptions, particularly what happens after stopping liraglutide and the calculation of long-term benefits, the committee agreed that an acceptable ICER would not be higher than £20,000 per QALY gained”Again, this recommendation is based on a trial submitted by Novo Nordisk that included 3,721 people and lasted for three years, but only 800 met the criteria for this cost-effectiveness recommendation. The trial failed to show a significant reduction in cardiovascular events. Novo's calculation of risk reduction was based on surrogate outcomes, which NICE points out “introduces uncertainty because causal inference requires direct evidence that liraglutide reduces cardiovascular events. This was not provided in the company submission because of lack of long-term evidence.”The NICE committee admits “relying on surrogates is uncertain but accepted that surrogate outcomes were the only available evidence to estimate cardiovascular benefits.”I just want to point out that another option would be to refuse to experiment on higher-weight people without appropriate evidence.These cost-effectiveness calculations are based on someone using the drug for two years, with no actual data on reduction in cardiovascular events, and with the admitted assumption that “any weight loss returned to the base weight 3 years after treatment discontinuation.” Said another way, this committee decided that it was cost effective to spend up to £20,000 per QALY for people to take a weight loss drug with significant side effects for two years, with no direct evidence of reduced cardiovascular events, and with the acknowledgment that people will be gaining all of their weight back when they stop taking it.Those who wrote the request for WHO to consider these drugs “essential” chose to characterize this as “At the chosen threshold of £20,000 per quality-adjusted life year (QALY) gained, the report concluded that liraglutide is cost-effective for the management of ob*sity.” I do not think that is an accurate characterization of the findings.The request cites “A report by the Canadian Agency for Drugs and Technologies in Health (CADTH) found that compared to standard care, the ICER for liraglutide was $196,876 per QALY gained”For the US, they cite a study that found that to achieve ICERs between $100,000 and $150,000 perQALY or evLY gained, the health-benefit price benchmark range for semaglutide was estimated as $7500 - $9800 per year, which would require a discount of 28-45% from the current US net price.They also cite “Cost-effectiveness analysis of semaglutide 2.4 mg for the treatment of adult patients with overweight and ob*sity in the United States, Kim et al.Let's take a look at their conflict of interest disclosure (emphasis mine)“Financial support for this research was provided by Novo Nordisk Inc. The study sponsor [that means Novo Nordisk] was involved in several aspects of the research, including the study design, the interpretation of data, the writing of the manuscript, and the decision to submit the manuscript for publication.Dr Kim and Ms Ramasamy are employees of Novo Nordisk Inc. Ms Kumar and Dr Burudpakdee were employees of Novo Nordisk Inc at the time this study was conducted. Dr Sullivan received research support from Novo Nordisk Inc for this study. Drs Wang, Song, Wu, Ms Xie, and Ms Sun are employees of Analysis Group, Inc, who received consultancy fees from Novo Nordisk Inc in connection with this study.”Given that, you probably won't be shocked to learn that this concluded that Novo Nordisk's drug, semaglutide, was cost-effective. The reason I bolded the text above is that this study is based on modeling – they are taking what is, by their own admission, a “new drug” and making predictions for 30 years. Everything was simulated based on trial data (you know, those trials that we've been discussing that often have horrendous methodology…) and “other relevant literature.” The construction of the modeling and the interpretation of the results was directed by the company who stands to benefit financially from the findings, and carried out by that company's employees and consultants. Also, and I'll just quote again here since I don't think I can improve on their text “Cost-effectiveness was examined with a willingness-to-pay (WTP) threshold of $150,000 per QALY gained” I do not think that this WTP is based on a global assessment.In their (and by their I mean Novo Nordisk's) modeling they find that semaglutide was estimated to improve QALYs by 0.138 to 0.925 and incur higher costs by $3,254 to $25,086 over the 30-year time horizon vs comparators.And, again, this is without any kind of actual long-term data. I think that the best way to characterize this information is “back of the envelope calculations” at best.To sum up, I do not think that the research they cite comes anywhere close to proving that these drugs have levels of efficacy, safety, or cost-effectiveness that warrant their addition to the WHO list of essential medicines. I believe that if the WHO grants this request I think it will be an affront to medical science, it will cheapen the concept of “essential medicines,” and it will harm untold numbers of higher-weight people all over the world.Did you find this post helpful? You can subscribe for free to get future posts delivered direct to your inbox, or choose a paid subscription to support the newsletter and get special benefits! Click the Subscribe button below for details:Liked this piece? Share this piece:More research and resources:https://haeshealthsheets.com/resources/*Note on language: I use “fat” as a neutral descriptor as used by the fat activist community, I use “ob*se” and “overw*ight” to acknowledge that these are terms that were created to medicalize and pathologize fat bodies, with roots in racism and specifically anti-Blackness. Please read Sabrina Strings' Fearing the Black Body – the Racial Origins of Fat Phobia and Da'Shaun Harrison's Belly of the Beast: The Politics of Anti-Fatness as Anti-Blackness for more on this. Get full access to Weight and Healthcare at weightandhealthcare.substack.com/subscribe

Discriminatory Health Metrics, Inclusive VA Healthcare

business executive director healthcare afghanistan va economics fda inclusive cms hhs discriminatory patient access health metrics qaly columbia university professor

Play Episode Listen Later Apr 6, 2023 5:19

Neal Masia, Health Economist and Columbia University Professor of Business and Economics, discusses how the QALY discriminates against rare and chronic disease patients and calls for a more patient-focused health technology assessment; the Jax Act, named after Jax Scott, an Army Special Operations Warrant Officer, helps female veterans who served in cultural support teams in Afghanistan access healthcare through the VA; and MacKay Jimeson, the Executive Director of Patients Rising's Patient Access and Affordability Project, explains the challenge of getting CMS to cover drugs approved through the FDA's accelerated approval process. Patients Rising Now letter to the House E&C Committee The Jax Act Operation Warp Speed 2.0 for Rare Disease The Cold War at HHS

EA - It's "The EA-Adjacent Forum" now by Lizka

Play Episode Listen Later Apr 1, 2023 2:39

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: It's "The EA-Adjacent Forum" now, published by Lizka on April 1, 2023 on The Effective Altruism Forum. TL;DR: We're not really comfortable calling ourselves “EAs.” Moreover, we know that this is true for a lot of people in the EA community the eclectic group of people trying to make the world better who happen to use the Forum. So we're renaming the “Effective Altruism Forum” to be the "EA-Adjacent Forum" (“EA Forum” for short). We have some deep disagreements with EA Look, we run a forum focused on discussions about how to do the most good we can, and we work at the "Centre for Effective Altruism," but we're not really members of the EA community. We have some deep disagreements with many parts of the movement. (We don't even always agree with each other about our disagreements, we don't always think that the EA thing is the right thing (see also), and we even hosted an EA criticism contest to surface disagreements.) It's not just us We know that others who use the Forum also prefer to call themselves “EA-adjacent.” We're also somewhat worried that anything that someone posts on the EA Forum can be interpreted as representative of effective altruism. We think it's important to preserve nuance and be clear about the facts listed here, so we're rebranding. Impact of the rebrand, next steps It's already the case that “EA” often stands for “Ea-Adjacent,” and we don't think the rebrand will change much in terms of how the Forum will function. As always, we'd love to hear your feedback. You can comment here or contact us directly. (Thanks to [unnamed people] for suggesting this rebrand. We'd credit them directly, but some of them prefer to not associate so closely with EA.) The EA-Adjacent Forum team. Please note that not all teammates agree with everything written here (probably). Some example disagreements: 1) We disagree with a lot of people in the EA community about styling and font choices. 2) Most people in the EA community promote functional decision theory, but after spending many years making software for the forum, we've come to the conclusion that object-oriented decision theory is superior.3) We disagree with CEA about the spelling of “Centre” in “Centre for Effective Altruism.” It should be spelled “center” as Noah Webster intended.4) Many EAs appear to focus on scope sensitivity, but we think scope specificity is more neglected5) We think QALYs should be converted to their metric-system equivalent, such that 1 metric QALY is the amount of quality-adjusted life that can be supported by 1 joule of energy within a 1-cubic-meter box over 1 year at 0 degrees celsius. Thanks for listening. To help us out with The Nonlinear Library or to learn more, please visit nonlinear.org.

impact speech forum ea tl adjacent cea eas effective altruism noah webster rationalist qaly qalys ea forum

Patient Economics: How to Measure the Value of Care

High Truths on Drugs and Addiction

Play Episode Listen Later Mar 27, 2023 15:54

Get a look at the behind-the-scenes math that often determines whether or not insurance will cover the costs of treatments. Health technology assessments attempt to determine the value of a new treatment. But not all of these equations take into account patient voices, experiences, and needs. That includes the QALY - a discriminatory health care metric that's come under fire. But if it's banned, what replaces it? Economists Dr. Neal Masia and Dr. Darius Lakdawalla of EntityRisk explain a new model that takes into account patient needs when calculating the value of a treatment. You can share your support for banning discriminatory metrics like the QALY here. Plus, learn about the BENEFIT Act, and how it would inject more patient data into FDA approval process. Links Patient Access and Affordability Project: Health Technology Assessment Best Practices for Rare Disease Drugs Advocacy Masterclass Need help? The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at the Patients Rising Helpline. Have a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent? Drop us a line: podcast@patientsrising.org The views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising, nor do the views and opinions stated on this show reflect the opinions of a guest's current or previous employers.

health care drop patients economics fda measure qaly

Kick QALY to the Curb with Terry Wilcox

This is Growing Old

Play Episode Listen Later Mar 22, 2023 28:00

For more than 40 years, the quality-adjusted life year (QALY) has been devaluing the lives of older adults and patients with chronic medical conditions. Yet, thanks to organizations like Patients Rising, we may finally put these discriminatory assessments to rest once and for all. In light of the recent QALY Ban Bill introduced to Congress, we're joined by returning guest, Co-Founder, and CEO of Patients Rising, Terry Wilcox, to break down the impact of QALYs on patients whose quality of life relies on access to critical treatments.

ceo co founders congress kick curb wilcox qaly qalys

Episode #114 High Truths on Drugs and Addiction with Dr. Jason Doctor on Death Diary Research

Play Episode Listen Later Feb 27, 2023 89:51

Death Diaries is what I call the research I did with Dr. Jason Doctor. We reviewed data on every single person who died of an unintentional medical overdose and compared the drugs on autopsy to what was prescribed to the person. This list of medications were diaries that lead to death. The research changed my life as a doctor and how I prescribe medications. Dr. Jason Doctor is an Associate Professor in the School of Pharmacy and Faculty at the Leonard D. Schaeffer Center for Health Policy and Economics at the University of Southern California. His research program centers on decision-making in healthcare. Dr. Doctor specializes in applying behavioral economic methods within health and medicine and current leads a multi-site federally-funded multi-site cluster randomized clinical trial that evaluates behavioral economic approaches to improve physician adherence to comparatively effective treatments. In other federally-funded research, Dr. Doctor has developed Bayesian decision algorithms to identify errors in blood panels to improve patient diagnosis. He also maintains a research program in understanding preferences for health from a behavioral economic perspective. He has extended the quality-adjusted life year (QALY) preference model to accommodate preference for helping others in medical need (social preferences) and has developed mathematical representations in QALY calculations that accommodate cognitive limitations people have in abiding by rational principles in decision making. JAMA Letter: Effect of Prescriber Notification of Patient's Fatal Overdose on Opioid Prescribing

university death school doctors research addiction patients economics drugs southern california associate professor truths diary faculty pharmacy health policy bayesian opioid prescribing qaly

Healthcare Protections for Rare Diseases with Rep. Cathy McMorris Rodgers

ceo co founders vice president medicine drop healthcare representative reps ban burgess protections rare diseases house energy rare disease day cathy mcmorris rodgers qaly qalys

Play Episode Listen Later Feb 24, 2023 37:23

Ahead of Rare Disease Day, Congresswoman Cathy McMorris Rodgers (WA-05) joins the podcast to discuss new legislation that would ban the use of a discriminatory health care metric, quality adjusted life year, or QALY. Learn how the QALY hurts treatment access for patients with rare diseases and disabilities, and how a government ban on QALYs would create more equitable access to care. Plus, hear from Jean Baker, who shares her challenges getting coverage for her husband's rare form of cancer, anaplastic thyroid cancer. And our patient correspondents from the rare disease community talk about the health care issues that impact them. This is also our last Friday episode of the Patients Rising Podcast! You'll be able to catch the Patients Rising Podcast on Mondays, starting March 6th, in a brand new format. Hosts: Terry Wilcox, CEO, Patients RisingDr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public InterestGuests:Congresswoman Cathy McMorris Rodgers, Chair, House Energy and Commerce CommitteeJean Baker, Rare Disease Caregiver and Anaplastic Thyroid Cancer AdvocateMaggie Senese, Patient CorrespondentKaitlyn Trevathan, Patient CorrespondentTomisa Starr, Patient CorrespondentAvery Roberts and Kelly Berger, Patient CorrespondentLinks: Chairs Rodgers, Smith, and Reps. Burgess, Wenstrup Introduce Legislation to Ban QALYsContact your Representative and Urge Them to Ban the QALYRare Disease Day February 28, 2023 Health Technology Assessment Best Practices for Rare Disease DrugsNeed help?The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.orgHave a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent?Drop us a line: podcast@patientsrising.orgThe views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising, nor do the views and opinions stated on this show reflect the opinions of a guest's current or previous employers.

Is Icosapent Ethyl a QALY-ty Investment?

iForumRx.org

Play Episode Listen Later Dec 9, 2022 24:25

The landmark REDUCE-IT trial demonstrated that icosapent ethyl (IPE) 4 g daily significantly reduced cardiovascular events in high-risk patients when added to statin therapy, but is a fancy fish oil supplement really worth our coin? Guest Authors: Joseph Nardolillo, PharmD, BCACP and Vivian Cheng, PharmD, BCPS, BCACP Music by Good Talk

investment pharmd bcps good talk ipe bcacp qaly reduce it

EA - Carl Djerassi (1923–2014) by Gavin

kaizen con Jaime Rodríguez de Santiago

Play Episode Listen Later Nov 30, 2022 5:10

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Carl Djerassi (1923–2014), published by Gavin on November 29, 2022 on The Effective Altruism Forum. Carl Djerassi helped invent the synthetic hormone norethindrone, one of the 500 most important medicines (actually top 50 by prescription count). A large supply is a basic requirement of every health system in the world. Norethindrone is important for two reasons. First, it treats menstrual disorders and endometriosis, together 0.3% of the global burden of disease. More famously, it was a component of The Pill. People mix up the timelines, which is why he is sometimes called the 'Father of the Pill'. But "neither Djerassi nor the company he works for, Syntex, had any interest in testing it as a contraceptive" and it was only used for birth control 12 years after. As usual in industrial chemistry, Djerassi got no royalties from the blockbuster medicine he helped develop - but, surprise ending! - he bought cheap shares in Syntex and got rich when it became one of the most important medicines in history for two reasons. He also synthesized the third-ever practical antihistamine, and applied new instruments in 1,200 papers on the structure of many important steroids. He also worked on one of the first AI programs to do useful work in science. Achievements Epistemic status: little better than a guess. Not many inventions are fully counterfactual; most simple, massively profitable things which get invented would have been invented by someone else a bit later. So the appropriate unit for lauding inventors is years saved. And if I put a number on that I'd just be making it up. Here are the numbers I made up: About 4 million US users, so maybe up to 94 million world users at present. No sense of the endometriosis / contraception split. Call it 600 million users, 10% endometriosis use case. For menstrual disorders: on the market 65 years and counting. Counterfactual: on the market 3 years before the next oral progestogen was. It was the first practical oral progestogen, so we should compare to the injectable alternatives About 1/6 of Americans hate needles so much that they refuse treatment. Attrition and missed doses for needle treatments is higher than pill treatments. Endometriosis is about 0.25 - 0.35 QALY loss. So if it's 30% effective, around $30 / QALY, an amazing deal. For easy contraception: on the market 59 years and counting. The big gains (besides autonomy) are averting unintended pregnancies, abortions, and pregnancy-related deaths. Modern cost-effectiveness in Ethiopia is $96 / QALY. There's probably some additive effect for endometriosis sufferers (who would want contraception anyway). A full account would guess the Pill's effect on the sexual revolution and cultural attitudes toward women. But I've reached my limit. (You might also consider the role of the Pill in the ongoing decline of church authority: "1980: In spite of the Pope's ruling against the Pill and birth control, almost 80% of American Catholic women use contraceptives, and only 29% of American priests believe it is intrinsically immoral.") How many years did he bring the invention forward? Call it 5. Then split the credit three ways with Luis Miramontes and George Rosenkranz. So (largely made-up numbers) it looks like millions of QALYs for the treatment overall, and tens of thousands counterfactually for Djerassi. Artist After surviving cancer, he decided to become a writer. I was very depressed, and for the first time thought about mortality. Strangely enough I had not thought about death before... I realized that who knows how long I would live? In cancer they always talk about five years: if one can survive five years then presumably the cancer had been extirpated. And I thought: gee, had I known five years earlier that I would come down with cancer, would I have led a different life during these...

american father ai americans modern speech pope ethiopia ea pill endometriosis strangely attrition american catholics counterfactuals rationalist qaly qalys syntex carl djerassi

#144 ¿Cuánto vale una vida?

Play Episode Listen Later Oct 25, 2022 22:15

(NOTAS Y ENLACES DEL CAPÍTULO AQUÍ: https://www.jaimerodriguezdesantiago.com/kaizen/144-cuanto-vale-una-vida/)¿Para qué sirven los días?Los días son el lugar donde vivimos.Llegan y nos despiertan.una y otra vez.Existen para que nos alegremos:¿dónde vivir, sino en los días?Y para responder a esa preguntavienen el sacerdote y el doctorcon sus largos abrigosque aletean por el campo.El otro día me crucé con este poema de Philip Larkin que me gustó y que se conectó en mi cabeza con algunas cosas que he leído y escuchado en los últimos meses. De esas que te llevan a reflexiones que no imaginaste. El de hoy es uno de esos capítulos. Si llevas tiempo escuchando kaizen, ya sabes que suceden de vez en cuando. Y es probable que te deje con más preguntas que respuestas, pero creo que eso es lo que hace que esas conexiones sean interesantes.Tendemos a pensar que los problemas matemáticos más difíciles son los que para ser resueltos necesitan esos garabatos extraños que alguna vez vimos en el colegio o en la universidad. Nos imaginamos gigantescas pizarras llenas de símbolos extraños como las letras griegas, las raíces cuadradas o las culebrillas que simbolizaban las integrales. Parecen conjuros escritos en un lenguaje demoníaco. Sin embargo, algunos de los problemas más difíciles no necesitan nada de eso. A veces, de hecho, la pregunta es simple y el cálculo sencillo, pero la respuesta es muy, muy, muy complicada. ¿Para qué sirven los días? O, por ejemplo, ¿cuánto vale una vida?Responder a esa pregunta tan fría y tan dura inevitablemente nos va a llevar a hablar de la muerte y de algunas cosas tristes, así que si no tienes un buen día a lo mejor quieres dejarlo para otro momento. Pero como hablábamos en aquel capítulo de la temporada pasada sobre las cosas que los nietos deberían saber, la muerte es parte de la vida. Y es también parte de la sociedad aunque a veces miremos para otro lado.

sin nos pero existen una vida responders n'to llegan parecen tim harford tendemos philip larkin will macaskill qaly thomas schelling

Quais são os efeitos dos exercícios físicos em pessoas vivendo com Insuficiência Cardíaca?

Podcast POEMS BVS APS

Play Episode Listen Later Oct 3, 2022 11:06

Neste episódio, vou mostrar para vocês o resultado de uma revisão sistemática da Cochrane de janeiro de 2019, demonstrando os efeitos de exercícios em pessoas com insuficiência cardíaca. Aproveitarei para falar sobre estudos que avaliam intervenção e impacto na qualidade de vida, como QALY e DALY. Artigo disponível em https://www.cochranelibrary.com/cdsr/doi/10.1002/14651858.CD003331.pub5/full

neste quais pessoas cios daly vivendo exerc efeitos cochrane artigo qaly

EA - [Cause Exploration Prizes] Preventing stillbirths by Denise Melchin

Play Episode Listen Later Aug 29, 2022 11:42

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: [Cause Exploration Prizes] Preventing stillbirths, published by Denise Melchin on August 29, 2022 on The Effective Altruism Forum. This essay was submitted to Open Philanthropy's Cause Exploration Prizes contest. If you're seeing this in summer 2022, we'll be posting many submissions in a short period. If you want to stop seeing them so often, apply a filter for the appropriate tag! Author's note: This cause area exploration is far from the depth and clear conclusions I would want and is in parts clearly in draft mode. Open Phil encouraged me to submit regardless and publish properly later. I will do this to encourage myself to actually finish it at a later date and not forget the post in the drawer. This submission might change focus later: I could imagine improved antenatal and labour care to have wider benefits on child health than reducing stillbirths. Summary Two million babies are stillborn every year. A baby is called stillborn if they die after 28 weeks of pregnancy. Most charitable interventions focus on children already born despite babies being much more vulnerable in the last trimester. Almost half of all stillborn babies were still alive until labour started, passing away hours or mere minutes before they were born. Stillbirths are usually not given a DALY weighting so are ignored in cost-effectiveness analysis. Interventions reducing maternal and child deaths often reduce stillbirths as well, so they are more cost-effective than they first appear. Intragovernmental bodies as well as philanthropists have shown much less interest in reducing stillbirths compared to maternal and child deaths. This gives philanthropists the opportunity to save more lives by focusing on stillbirths. To a first approximation, children become less vulnerable as they get older. Adjusting for the length of the time period, the stillbirth rate is much higher than the infant mortality rate which in turn is much higher than the under 5's mortality rate. Although birth is the riskiest period. As philanthropists we have already noticed that focusing on young children is often most cost-effective. All recommended GiveWell charities above the GiveDirectly bar are focusing on the lives of young children. Trying to reduce stillbirths by improving care of pregnant women would just be going one step further in this direction. Uncertainties Major This submission primarily discusses stillbirths. But the potential interventions to reduce stillbirths have wider benefits. I do not know what fraction of the benefits of the interventions actually accrue as stillbirth prevention. Therefore it is possible a later publication will pivot into antenatal interventions and/or labour care more widely. As of now, his post is missing plenty of potential interventions, instead just giving a cursory overview, as well as lacking cost-effectiveness estimates. Therefore it is hard to say whether it is a plausible competitive cause area candidate. Minor Some data is a bit outdated. This seems particularly risky due to geographic shift in stillbirths. I have not paid enough attention to different risk factors by geography. Importance Two million babies are stillborn every year. A baby is called stillborn if they die after the 28th week of pregnancy but before they are born. If born in a rich country, most of these babies would live. In the UK, a baby born at 28 weeks is around 90% likely to survive up to virtually a hundred percent if full-term. This is especially true for the almost half of all stillbirths in which the baby only dies after labour has already started. A sharp discontinuity in health cost-effectiveness analysis such that a baby passing away minutes after birth should be given the full QALY weighting while a baby passing away minutes before should be ignored seems hard to defend.Babies passing away...

babies uk speech prizes exploration preventing ea adjusting interventions daly givewell givedirectly rationalist qaly open phil

Circulation August 16, 2022 Issue

Circulation on the Run

Play Episode Listen Later Aug 15, 2022 29:50

This week, please join author Philipp Lurz and Editorialist Daniel Burkhoff as they discuss the article "Characteristics of Heart Failure With Preserved Ejection Fraction Across the Range of Left Ventricular Ejection Fraction" and the editorial "HF?EF: The Mysterious Relationship Between Heart Failure and Ejection Fraction Continues." Dr. Carolyn Lam: Welcome to Circulation on the Run, your weekly podcast summary and backstage pass to the journal and its editors. We're your co-host. I'm Dr. Carolyn Lam, Associate Editor from the National Heart Center and Duke National University of Singapore. Dr. Greg Hundley: And I'm Dr. Greg Hundley, Associate Editor, Director of the Pauley Heart Center at VCU Health in Richmond, Virginia. Dr. Carolyn Lam: I just cannot wait to tell you about today's feature discussion, Greg. It's about heart failure with preserved ejection fraction and characteristics in the range of that ejection fraction that's above 50%. So, okay. I know, I know. It's like, "Oh my gosh. Wow. How much are we going to be talking about this ejection fraction thing?" But I'm telling you, everybody has to listen to this. It's really a big stride forward in our understanding of these patients with heart failure and a higher ejection fraction. Plus it includes one of my mentors from Mayo Clinic days, and I just can't wait for everyone to hear this. But before we go there, we've got really, really cool original papers in today's issue too. Would you like to start first? Dr. Greg Hundley: You bet Carolyn, and I can't wait for that feature discussion, especially this is a real area of your expertise. So listeners, hold on for that feature discussion. Well, my first paper, Carolyn, really pertains to physical activity. And Carolyn, the study is led by Dr. Don Hoon Lee from the Harvard T.H. Chan School of Public Health. And it evaluated a total of 116,000 adults from two large prospective US cohorts, the Nurse's Health Study and the Health Professional's Follow up Study that took place from 1988 to 2018. And they were examining self-reported leisure time physical activity and assessed the association between long term leisure time physical activity intensity and all cause and cause specific mortality. Dr. Greg Hundley: Now Carolyn, two types of physical activity were assessed. First, moderate physical activity and we're going to abbreviate that as MPA and that was 150 to 300 minutes per week, which is really recommended. Or second, vigorous physical activity for which it's really recommended that one performs 75 to 150 minutes per week. Now, Carolyn, as you know, some individuals accomplish both of these in their routine, some neither, some one or the other. And so it really remains unclear whether higher levels of long term vigorous or moderate are independently or perhaps jointly associated with lower mortality. Dr. Carolyn Lam: Oh, that's so interesting, Greg. Quick, quick, quick, tell us the results. Dr. Greg Hundley: Right Carolyn. So the nearly maximum association with lower mortality overall was achieved by performing approximately 150 to 300 minutes per week of long term leisure time vigorous physical activity or 300 to 600 minutes per week of long term leisure time moderate physical activity or an equivalent combination of both, so mixing those minutes. Also Carolyn, very interestingly, higher levels, suppose you go beyond those limits of either long term leisure time vigorous physical activity of more than 300 minutes per week or moderate physical activity of more than 600 minutes per week did not show clearly further lower all cause cardiovascular disease or non-cardiovascular disease mortality and nor did they show harm. So if you went above those thresholds, you really didn't experience greater harm. Dr. Carolyn Lam: Thanks, Greg. You know I'm going to be trying to apply that. That's so cool. All right. Well the next paper refers to the Cabana Trial, which I'll remind you is a trial in which catheter ablation did not significantly reduce the primary endpoint of death, disabling stroke, serious bleeding or cardiac arrest compared to drug therapy by intention to treat, but did improve quality of life and freedom from atrial fibrillation recurrence. In Cabana, the heart failure subgroup, ablation appeared to improve both survival and quality of life. The current paper led by Dr. Mark and colleagues from Duke Clinical Research Institute looked at the cost effectiveness of this ablation versus angio-rhythmic drug therapy in atrial fibrillation and which was a pre-specified Cabana secondary endpoint. Dr. Greg Hundley: Ah, Carolyn. So what did they find? Dr. Carolyn Lam: Well in this trial based economic evaluation, catheter ablation was estimated to cost $57,893 per QALY or Quality Adjusted Life Year gained compared to drug therapy in the overall cohort. And this was primarily driven by improvement in quality of life. It also cost $54,135 per QALY gained in the heart failure subgroup, driven by both gains in quality of life and survival. In summary, catheter ablation of atrial fibrillation was found to be economically attractive compared to drug therapy in the Cabana trial overall at present benchmarks of healthcare value in the US and based on projected incremental qualities, but not live years alone. Dr. Greg Hundley: Very nice Carolyn. Well, my next paper comes to us from the world of Preclinical Science and it's corresponding author is Dr. Swapnil Sonkusare from University of Virginia School of Medicine. So Carolyn, calcium signals in smooth muscle cells contribute to vascular resistance and control blood pressure. Now increased vascular resistance in hypertension has been attributed to impaired smooth muscle cell calcium signaling mechanisms. And in this regard, transient receptor potential vanilloid four or TRPV4 smooth muscle cell ion channels are crucial calcium entry pathways for smooth muscle cells. However, their role in blood pressure regulation has not been identified. Dr. Carolyn Lam: Wow. TRPV4 smooth muscle cells. Cool Greg. So what did they find? Dr. Greg Hundley: Right, Carolyn. So these authors provide the first evidence that TRPV4 smooth muscle cell channel activity elevates resting blood pressure in normal mice. A1AR stimulation activated TRPV4 smooth muscle cell channels through protein kinase calcium signaling, which contributed significantly to vasoconstriction and blood pressure elevation. Dr. Greg Hundley: Surprisingly, intraluminal pressure induced TRPV4 smooth muscle cell channel activity, opposed vasoconstriction through activation of calcium sensitive potassium channels indicating functionally opposite pools of TRPV4 smooth muscle cell channels. And so Carolyn, this team identified novel smooth muscle cell calcium signaling nano domains that regulate blood pressure and demonstrate impairment in hypertension. Dr. Carolyn Lam: Oh wow, Greg. Thank you so much for that. Well, let's cover the other articles in today's issue. There's a primer by Dr. Jaffe on High Sensitivity Cardiac Troponin and the 2021 AHA/ACC/ASE/CHEST/SAEM/SCCT/SCMR guidelines for the evaluation and diagnosis of acute chest pain. There's also Research [Letter] in there by Dr. Fordyce on the eligibility for non-invasive testing based on the 2021 American Heart Association and ACC guideline for the evaluation and diagnosis of chest pain, implications from the Promise trial. Dr. Greg Hundley: Great Carolyn. Well, I've also got an exchange of letters to the editor from Professors Benali and Professor Della Bella regarding a previously published article, “Does Timing of Ventricular Tachycardia Ablation Affect Prognosis in Patients with an Implantable Cardioverter Defibrillator, Results from the Multicenter Randomized Partita Trial.” And also there's a very nice Perspective piece from Dr. Udelson entitled “Glucose Insulin Potassium Therapy for Acute Myocardial Infarction, 50 years on and Time for a Relook.” Well, Carolyn, I can't wait to hear more of the discourse between you, the authors and editors on heart failure and preserved ejection fraction. Dr. Carolyn Lam: Yay. Here we go. I'm so excited about today's feature discussion. It's on my favorite topic, heart failure with preserved ejection fraction. Although thanks to one of the authors, Dr. Daniel Burkhoff of the editorial that accompanies it, I don't even know how to pronounce, it's Heart Failure question mark Ejection Fraction. I love it. How would you pronounce that? Dr. Daniel Burkhoff: Yeah, no, we debated how to pronounce it. HFQRef…a question mark? Dr. Carolyn Lam: HFQRef. Oh my goodness. What are we going to come up with next? Dr. Daniel Burkhoff: And we actually thought the editors would have a problem with that. Whenever we put something cute in the title, we always get knocked down. So we haven't yet got knocked down, so we'll see. Dr. Carolyn Lam: No, I'm not knocking you down. Dr. Daniel Burkhoff: But it just really emphasizes that we're in a very confusing time now as it comes to heart failure and ejection fraction, that we have to move on beyond ejection fraction. And although we have these strict buckets and upper and lower limits for each range, that maybe this is in a little bit of a way, doing us a disservice and doing the patients at disservice in terms of treatment. Dr. Carolyn Lam: That's really great. And everybody that was Dr. Daniel Burkhoff from the Cardiovascular Research Foundation in New York. He's the editorialist of today's feature paper. And I'm so excited that we have the corresponding author of today's feature paper as well, Dr. Philipp Lurz from Heart Center Leipzig in Germany. So first, I mean, or second, heartfelt congratulations on this beautiful paper, Philipp. If you could start with telling us all about what you did and what you looked at and what made Dr. Daniel Burkhoff call it now, Heart Failure, QREF? Dr. Philipp Lurz: Yeah. So thank you so much, first of all, obviously for having me for the kind introduction and your kind words about our work. The whole thing started with the observation that in recent drug trials, the response in HFpEF patients to drugs which were before proven to be quite successful in HFrEF actually showed a quite heterogeneous response in HFpEF patients. And that there were some patterns according to the range of ejection fractions. So it seemed that those HFpEF patients with the lower ejection fractions, they respond a little bit better to HFrEF medication than those with a higher ejection fraction. Dr. Philipp Lurz: And HFpEF studies, they included patients within and ejection fraction or 40 and above who normally would probably would consider HFpEF to start with 50 and above. But even in those truly HFpEF patients, so from 50 and anything above to 70, there was a suggestion that there might be differences. 50 to 60 might be something else and then 60 and above. And this is where we started to look into our cohort and to group them according to ejection fraction and see whether we can see some important and clinically meaningful differences in morphology, obviously in function, but then even also in terms of our biopsy results. Dr. Philipp Lurz: And that also implies that we did quite a deep phenotyping of our patients. So we use imaging, echo obviously. We use magnetic resonance imaging. We were able to acquire in a larger percentage of that cohort, by the way, it was 56 patients in total, we were able to get some left ventricular biopsies. And most importantly, when it comes to the functional properties of the left ventricle, we also acquired pressure volume loops. And that has the great advantage that we obviously we can look at low dependent parameters, but more important, also low independent in disease of both systolic and diastolic function. And that's pretty much what we did. Dr. Carolyn Lam: Oh my goodness. I mean, as an editor at Circulation, can I just first tell you that's what really, really stood out? It's the comprehensive, careful, in-depth characterization. It may be 56 patients, but MRI, echo, biopsies, exercise, PV loops. I mean, it was a lot, a lot that you did. Could you boil it down to what you found and maybe just to first clarify to the audience, how did you bend the ejection fraction? And then what you found? Dr. Philipp Lurz: We divided the cohort in two groups. One with an ejection fraction 50 to 60, and the other groups higher than 60% left ventricular ejection fraction. We ended up in lower group, 21 patients, in the higher group with 35 patients. And they were quite different. They had distinct features in terms of morphology, means that the lower injection fraction group, they had larger ventricles. That's probably what you would expect when you group them according to eject fractions. So not that surprising. Dr. Philipp Lurz: And at that point, and they had the same stroke volume. The low ejection fraction group, you could say that they had a certain degree of eccentric modeling, whereas those were the high ejection fraction group, that concentric modeling. When we then looked at the biopsies, the group with the low ejection fraction group, so 50 to 60, they had higher percentage of myocardial fibrosis at 15%. Dr. Philipp Lurz: And then on left ventricular biopsy, we saw that patients with a high ejection fraction group, they had less myocardial fibrosis, so more fibrosis in those with the lower ejection fraction group. And this is really interesting because when we then look at the real size of the pressure volume loop analysis, despite the fact that the high ejection fraction group had less fibrosis, they had the most stiff ventricles on pressure volume loop analysis. So that's already a very important point because it illustrates once again that we should not mistake fibrosis with stiffness, and also not the other way around. There are other parameters which can cause stiffness such as cellular stiffness, and it's not just the extracellular matrix. Dr. Philipp Lurz: So that's an important point. Those patients with high ejection fraction, they had the most stiff ventricles. They had the most relevant limitations in left ventricular filling, so the most marked diastolic dysfunction. We also looked at systolic parameters and there we found that they had a very high contractility. So this is the end systolic pressure volume relationship or also called elastics and that's a marker for contractility. So these ventricles with high ejection fraction, they can contract very well. They have high contractivity. But this is also a marker of systolic stiffness. So they stiff, both in terms of diastolic properties, but also systolic properties. And there, their most important limitation is the limitation in filling. Dr. Philipp Lurz: The other group, injection fraction 50 to 60, they do have some stiffness. Obviously we are talking about HFpEF. This is a disease of a diastolic dysfunction. So they have increased stiffness, but to a lesser extent. They can feel a little bit better, but what we see there is a reduction in contractility, so systolic properties. So you could argue that in some extent, that group 50 to 60, they behave a little bit more like HFrEF. Whereas those with high ejection fraction, they're completely different. Very stiff, both doing diastolically and sistolly. Dr. Carolyn Lam: Thank you so much. Now I really have to get the gurus insight into this. And of course by guru, I mean, Dr. Daniel Burkhoff. First, I'm going to take this opportunity to share a little private personal story that it's very hard for me to call Dr. Burkhoff, "Dan", although if you will allow me it's because I was a fellow when I first met Dr. Burkhoff. And one of my first papers was actually communicating with Dr. Burkhoff trying to draw these PV loops based on the noninvasive measurements that I was obtaining at the Mayo Clinic in the Olmsted County Cohort. And so this is just really making me smile. So Dan, if I may, what do you make of all this? And if you could give us what you think may be the clinical take home message. Dr. Daniel Burkhoff: Thank you so much, Carolyn. And also again, Philipp, congratulations on really a really important paper. I think as Carolyn was saying, one of the many things that impressed me was the multifaceted aspects of the thorough evaluation using multi modality characterization, which is in my mind, unprecedented, especially for this particular group of patients. And you summarized the main results very well. But to me, the thing that really struck me and that we really tried to emphasize in our editorial was the differential response to exercise, to hand grip exercise in this point. Dr. Daniel Burkhoff: As you already said, and I don't think this could be understated, that in the lower range, the 50 to 60, these patients were able to fill, the ventricle was able to fill more, the end diastolic pressure still went up significantly into an abnormal range, which is of course is one of the requirements for the diagnosis of HFpEF. But those patients, the end diastolic volume was able to increase and that helped them to increase their cardiac output. Dr. Daniel Burkhoff: In the higher EF group, the greater than 60, the end diastolic pressure went up, but the volume did not increase. So the end diastolic pressure volume relationship became higher elevated. And this of course, was reminiscent of what was identified in the early nineties by Dalane Kitzman. And really, he didn't make this distinction between the lower half and the higher half. And we had also in a paper that we did with David K and others, seen a similar finding a couple years ago. And I think this is really, to me, was the along with the apparently disparate findings on myocardial biopsies with fibrosis going the wrong way, if you will, as you already commented on. So this is at least for the higher EF group, is identifying what I would refer to as really true diastolic dysfunction. Dr. Daniel Burkhoff: That means that the patients can't fill, there really is some problem why the EDP can go up, but the volume does not increase. And this is obviously for future research, we have to understand what is the difference between these two groups. There are several speculations about why it might. For example, one thing that has been proposed in the literature is the pericardial restraints. If the heart is constrained in a tight pericardium, the volume is recruited from the venous system, which is another what I think is a very important part of this HFpEF phenotype, but the heart is already constrained. That would elevate both the CVP and the wedge pressure without concomitant increases in RV or LV volumes, and therefore limit the ability to increase the cardiac output. Dr. Daniel Burkhoff: Another alternative is delayed relaxation. That means a true abnormality of active relaxation. The tau if you will, but I believe in your study, if I remember correctly, the tau was not that abnormal and did not really change very much in either group. So it was harder to really pin it on an abnormality of active relaxation. So that was one really, I think, really important finding. Dr. Daniel Burkhoff: The second is now in the group 50 to 60 where they could fill, one of the limitations of the study that you pointed out was that there's no control group. So why this population, this HFpEF population, the EDP increased, but the EDV the end diastolic volume also increased, so what's different between those patients and normals? That we don't really have an answer for yet. So that would be one thing is to compliment these findings with results in a true control group that does not have HFpEF. Dr. Daniel Burkhoff: So we still have this mystery of why does EDP go up in this group? My perspective is not an abnormality of diastolly. It's not a diastolic dysfunction, even though it's a HFpEF. I've been trying to promote this idea for more than 20 years, that all HFpEF is not an abnormality of diastolic ventricular properties. There may be extra cardiac factors even beyond the pericardium in this group. So I think these results are just further telling us how complicated and individualized our approach to the pathophysiology of these patients should be. Dr. Daniel Burkhoff: And also just one final comment, making a strict cutoff at 60% or 57% as we saw from Valsartan Cuba trial, Valsartan study, is clearly also not going to do us justice. Let's say that we're dealing with anyway, patients with two different pathophysiologies. There's going to be a distribution of these different mechanisms and there's going to be an overlap of these distributions. So we need to start thinking about how we individualize and characterize the pathophysiology in individual patients. So maybe patients with EF of 55, certain patients with EF 55 will not respond to Sacubitril- Valsartan and maybe some patients with an EF of 62 will. So we need to go more deep into the clinical characterization. Dr. Daniel Burkhoff: And methods that you use in particular, the pressure volume loop, seem to separate, very nicely, these two different, at least two different subtypes. So I think it's very exciting, and I think people should really take notice of what you found and build on this as a foundation and understand that we need to go deeper on the clinical side to phenotype these patients. Dr. Carolyn Lam: Philip, what are your thoughts? Dr. Philipp Lurz: No, I think that the concept about stress and unstressed volume is extremely important and fascinating, but that's where it gets really complex because you could make the conclusion from our results that especially the high ejection fraction group, they are have a very high preload sensibility, which means that when we reduce pre-load too much in them, they drop the stroke volume very suddenly. So that's probably also the clinical question for the future. There are many patients, HFpEF patients in whom we should reduce stress volume and they can benefit from that. Dr. Philipp Lurz: But I also believe that there is a cord of patients, and those are probably more those with high ejection fraction. They actually, they could experience some harm if you reduce preload too much because of the severe filling restrictions, because of the inability to increase end diastolic volumes, especially as an adaptation to exercise. And I think if we find out ways to differentiate who will benefit from preload reduction and from decongestion and who actually might experience even harm from these interventions, then we are certainly one step further. Dr. Carolyn Lam: If I may, I just, I could go on forever, but I know that there's going to be this question that the audience is immediately thinking. Here we are going into the weeds, hemodynamics, we all love it. In fact, I think we're all kind of a little bit geeky about it, but then you've got, Emperor preserved, the Deliver Trial sort of being positive in heart failure with ejection fraction above 40. So, do we really need to understand the different hemodynamic? What do you think is happening that this sort of blanket benefit can occur? Dr. Philipp Lurz: I don't think that everyone will benefit. And I think that a better understanding of the underlying pathophysiology will help to even increase the rate of responders and dissect of those who will not respond or we need a different therapy. Obviously here we group patients according to ejection fraction. We just discussed that this is a very rough way to characterize patients. So the next step certainly would be to understand or to see distinct patterns in left ventricular functional behavior irrespective of ejection fraction. Because you might can skip ejection fraction at one day, but the conclusion is not, at least not in my opinion, that all heart failure is the same. Dr. Carolyn Lam: Nice. And Dan, what do you think? Dr. Daniel Burkhoff: Well, I think with STLT2 inhibitors, first of all, we're looking forward to actually seeing the results from Deliver what the details of it in terms of mortality versus hospitalizations and quality of life. But with regard to HSGL2 inhibitors, I don't think we know the mechanism. I mean, I've read about six papers that definitively talk about different mechanisms of action. I think we don't know. And even despite, let's say positive studies, there's still a significant residual risk that these patients have. So these are not the end of the story by any means. I think this is the beginning of the story and there's still going to be a lot to learn. Dr. Daniel Burkhoff: I think with SGLT2 inhibitors, I think it's difficult to identify who is or how do you define a responder on an individual patient basis? When we look at groups and you look at mortality and hospitalizations, yes, you can identify them. But that group does not overlap exactly with those who have an improvement in quality of life. So I think that we're just at the beginning, Dr. Daniel Burkhoff: I do think that a deeper phenotyping is going to be the way to go ultimately and I think we're going to need more therapies. Thanks again, Carolyn for inviting me to do the editorial and to participate in this. And I would really be remiss if I did not mention the extraordinary contributions for Mickey Brener and also Barry Borlaug who were equal contributors to this editorial and the evaluation of this paper. So I'm really indebted to both of them for this interpretations. And thank you again, Philipp, for just a wonderful paper. Dr. Carolyn Lam: Couldn't have said it better and I just want to thank you once again for providing that deeper phenotyping for opening the door. Many of us said it again and again, this is the beginning and we need more studies, frankly, in yours. I mean, I think Dr. Burkhoff wants you to send normal, healthy people for MRI biopsies, exercise, echo. I'm kidding, on PV loops, but it's true. We need that data. We need the same and the HFrEF and really just to understand the whole thing. I'm so excited about what this paper opens up. Dr. Carolyn Lam: I am thrilled to see your editorial, Dan. I'm sure it's going to be well received. Everyone who's listening to this, Pick up the paper, pick up the editorial. Thank you so much for joining us today. You've been listening to Circulation on the Run and from Greg and I, don't forget to tune in again next week, Dr. Greg Hundley: This program is copyright of the American Heart Association, 2022. The opinions expressed by speakers in this podcast are their own and not necessarily those of the editors or of the American Heart Association. For more, please visit hajournals.org.

EA - To WELLBY or not to WELLBY? Measuring non-health, non-pecuniary benefits using subjective wellbeing by JoelMcGuire

Play Episode Listen Later Aug 12, 2022 25:11

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: To WELLBY or not to WELLBY? Measuring non-health, non-pecuniary benefits using subjective wellbeing, published by JoelMcGuire on August 11, 2022 on The Effective Altruism Forum. This essay was written for the Worldview Investigations category of Open Philanthropy's Cause Exploration Prizes by staff at the Happier Lives Institute Summary Open Philanthropy recognises the need to measure benefits beyond health and income. We think that subjective wellbeing is the best tool for the task. Subjective wellbeing (SWB) is measured by asking people to rate how they think or feel about their lives. We propose the wellbeing-adjusted life year (WELLBY), the SWB equivalent of the DALY or QALY, as the obvious framework to do cost-effectiveness analyses of non-health, non-pecuniary benefits. As our previous work has shown that using WELLBYs can change funding priorities by giving more weight to improving mental health, compared to DALYs or income measures; and they may reveal different priorities in other areas too. The advantages of SWB over alternatives are fourfold. (1) SWB captures and integrates the overall benefit to the individual from all of the instrumental goods provided by an intervention. This avoids the challenging problem of assigning moral weights to different goods, makes spillover effects easier to estimate, and clarifies the importance of philosophy. (2) SWB is based on self-reports by the affected individuals whereas Q/DALYs rely on flawed predictions about how good or bad we think a malady will be for ourselves or others. (3) Using SWB will reveal previously under-captured benefits, such as it has already done for psychotherapy. (4) Measures of subjective wellbeing already exist, are easy to collect, and widely (and increasingly) used in academia and policymaking across an extensive array of circumstances and populations of interest. Furthermore, subjective wellbeing measures are reliable and valid instruments and the existing evidence supports consistent use across people. Having said that, SWB is not without its disadvantages. (1) There is little research on the comparability between SWB scales across people. (2) We don't know where the ‘neutral point' lies on SWB scales. (3) We're unsure how to choose the best measure of SWB (e.g., life satisfaction or happiness) or how to convert between them. (4) There are very few cost-effectiveness analyses using WELLBYs. Fortunately, we think these issues can be resolved and we are actively working towards doing so. 1. The problem and a solution Open Philanthropy's mission is to help others as much as possible. Its human-focused Global Health and Wellbeing grantmaking aims to save lives, improve health, and increase incomes. However, Open Philanthropy recognises that measuring changes to health or income does not capture all the benefits experienced by the recipients. So, they ask, how should they account for the effects of injustice, discrimination, empowerment, and freedom? To that list, we could also add crime, loneliness, and corruption. Whilst the standardised health metrics, QALYs and DALYs, make it easier to compare different health states in the same units, the broader challenge is to find a common currency that allows sensible trade-offs between health, wealth, and non-health, non-wealth outcomes. How could this be done? We take it that Open Philanthropy is interested in funding interventions that improve wellbeing. Therefore, reducing discrimination (or injustice, etc.) is good mostly because it increases wellbeing, any other reason is secondary. But what is ‘wellbeing'? Philosophers have three main theories: (1) positive experiences, (2) satisfied desires, and (3) a multi-item ‘objective list' that includes ‘objective' goods such as knowledge, achievement, and love. Conspicuously absent from this list are wealt...

health benefits speech wellbeing measuring ea philosophers measures global health daly subjective rationalist swb qaly open philanthropy conspicuously qalys

EA - Corporate campaigns for chicken welfare are 10,000 times as effective as GiveWell's Maximum Impact Fund? by Vasco Grilo

Play Episode Listen Later Jul 29, 2022 26:25

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Corporate campaigns for chicken welfare are 10,000 times as effective as GiveWell's Maximum Impact Fund?, published by Vasco Grilo on July 28, 2022 on The Effective Altruism Forum. Summary This analysis estimates the cost-effectiveness of corporate campaigns for chicken welfare (CCCW), and compares it with that of GiveWell's Maximum Impact Fund (MIF) (see Methodology). The results were obtained with this Colab, and the key ones are summarised in the table below (for more, see Results). Comments about how to interpret them are welcome. The mean ratio between the cost-effectiveness of CCCW and MIF is estimated to be 10 k (i.e. 10^4). The specific value depends on very uncertain parameters, such as the ones defining the badness of the conditions of factory-farmed chickens relative to the goodness of fully healthy chicken life, and the moral weight. However, it appears to be robustly larger than 1 (see Discussion). ResultMeanMedianImprovement in the conditions of broilers (cQALY/cyear)1.120.505Improvement in the conditions of laying hens (cQALY/cyear)1.410.806Improvement in the conditions of chickens (cQALY/cyear)1.370.783Improvement in the conditions of broilers (%)45.449.8Improvement in the conditions of laying hens (%)64.665.2Improvement in the conditions of chickens (%)62.863.4Moral weight of chickens relative to humans (QALY/cQALY)2.410.0180Cost-effectiveness of CCCW for broilers (QALY/$)64.40.0269Cost-effectiveness of CCCW for laying hens (QALY/$)2040.656Cost-effectiveness of CCCW (QALY/$)1510.494Ratio between the cost-effectiveness of CCCW and MIF12.2 k38.9 Acknowledgements Thanks to Cynthia Schuck-Paim, Kieran Greig, Lewis Bollard, and Saulius Simcikas. Introduction About 2 year ago Stephen Clare and Aidan Goth (S&A) published an analysis comparing the cost-effectiveness of The Humane League (THL) and Against Malaria Foundation (AMF). The analysis presented here has the same motivation of better allocating resources between different causes (namely, animal welfare and global health and development). In addition, it relies on data from the Welfare Footprint Project (WFP; overviewed here) to model more accurately the relative improvement in the conditions of broilers and laying hens. Methodology The ratio between the cost-effectiveness of CCCW and MIF was calculated via a Monte Carlo simulation in this Colab. All distributions were assumed to be independent, and their parameters were estimated from quantiles as explained here. The methodology to determine the cost-effectiveness of CCCW and MIF, and assess room for more funding are presented below. Cost-effectiveness of corporate campaigns for chicken welfare The cost-effectiveness of CCCW was based on its cage-free campaigns, and determined from the product between: The cost of improving the conditions of chickens (cyear/$). The improvement in the conditions of chickens (cQALY/cyear). The moral weight of chickens relative to humans (QALY/cQALY). Cost of improving the conditions of chickens The cost of improving the conditions of chickens was supposed to follow a lognormal distribution with 5th and 95th percentiles in cyear/$ equal to: For broilers, 0.2 and 90. For laying hens, 12 and 160. For both combined, 9.6 and 120. These are the lower and upper bound of the 90 % subjective confidence interval estimated for the cost-effectiveness of "broiler and cage-free campaigns" in this analysis from Saulius Simcikas. Potential concerns with those estimates are discussed here. Improvement in the conditions of chickens The improvement in the conditions of chickens were calculated assuming weights of: 12 % for changing broilers from a conventional scenario (CS) to a reformed scenario (RS). 88 % for changing laying hens from conventional cages (CC) to cage-free aviaries (CFA). The fractions above were computed with the Guessti...

cost chicken corporate speech results ea cs campaigns welfare cc rs methodology vasco cfa monte carlo colab maximum impact grilo givewell rationalist mif impact fund qaly

EA - Comparison between the hedonic utility of human life and poultry living time by Vasco Grilo

Play Episode Listen Later Jun 9, 2022 20:05

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Comparison between the hedonic utility of human life and poultry living time, published by Vasco Grilo on June 8, 2022 on The Effective Altruism Forum. Summary This analysis estimates the negative utility of poultry living time as a fraction of the utility of human life, under total hedonic utilitarianism (classical utilitarisnism). The results are presented by country in this spreadsheet (see tab "TOC"). The conclusions are very sensitive to the moral weight of poultry birds relative to humans, and the quality of their living conditions in factory farms relative to fully healthy life. However: In expectation, the (mean) negative utility of poultry living time seems much larger than that of human life. Realistically, the (median) negative utility of poultry living time seems comparable to that of human life. You can use this Guesstimate model to select your preferred inputs. Acknowledgements Thanks to Cynthia Schuck-Paim, Jason Schukraft, Lewis Bollard, Matt Sharp, Michael St. Jules and Scott Smith for feedback. Methodology The logic of the calculations is illustrated in this Guesstimate model. The tabs mentioned throughout the following sections refer to this spreadsheet. Utility of human life The utility of human life (QALY/person/year) was determined from the ratio between the healthy and full life expectancy. This is imperfect, but allows to estimate the utility of human life via a linear regression on life expectancy at birth: The utility of human life was calculated through the mean life expectancy at birth of the years between 2025 and 2100 (see tab "Future utility of human life"). Data about the future life expectancy at birth was taken from OWID, and is in tab "Future life expectancy". The linear regression coefficients were calculated based on data about the healthy and full life expectancy at birth from 1990 to 2016 (see tabs "Utility of human life and life expectancy" and "Past utility of human life"). Data about the past healthy life expectancy at birth was taken from OWID, and is in tab "Past healthy life expectancy". Data about the past life expectancy at birth was taken from OWID, and is in tab "Past life expectancy". Utility of poultry living time The utility of poultry living time per capita (-QALY/person/year) is the product of: The poultry living time per capita (pyear/person/year), which is the product between: The poultry production per capita (kg/person/year). The ratio between poultry living time and production (pyear/kg). Moral weight of poultry (QALY/pQALY). Quality of the living conditions of poultry (-pQALY/pyear). The factors defining the poultry living time per capita were modelled as constants, and the ones regarding the moral weight and quality of the living conditions of poultry as distributions. The following sections provide further details. The calculations of the utility of poultry living time per capita by country were performed in this Google Colab program, and the respective results uploaded to the tab "Utility of poultry living time per capita". Poultry production per capita The poultry production per capita between 2025 and 2100 (kg/person/year) was determined from the time-weighted average of those of the following periods: 2025 to 2050: mean between the poultry production per capita in 2025 and 2050. 2050 to 2100: half of the poultry production per capita in 2050. The poultry production per capita in 2025 and 2050 was estimated from the ratio between the poultry production and population. The poultry production was calculated considering: The poultry production by country in 2018 from OWID (see tab "Poultry production"). The poultry production annual growth rate by region, which was estimated by adding the following (see tab "Poultry production annual growth rate"): Poultry population annual growth rate between 2005/2007 an...

future data speech comparison moral ea utility vasco human life poultry toc scott smith hedonic grilo michael st matt sharp rationalist qaly google colab

Updates from the Frontlines of Medical Research

co founders executive director vice president medicine drop als banks front lines medical research patient advocate chief research officer jenny jones muscular dystrophy association qaly

Play Episode Listen Later May 6, 2022 32:54

In recognition of ALS Awareness Month, we get a report from the frontlines of ALS research, including new breakthroughs that impact patients. Dr. Sharon Hesterlee, the Chief Research Officer at the Muscular Dystrophy Association, gives us the big picture on ALS research and other neuromuscular diseases. Plus, Terry and Dr. Bob discuss new health legislation, including a bill that would ban the QALY, a discriminatory health care metric that hurts patients with rare or chronic diseases. And Kate speaks with Jenny Jones, an advocate for Familial Adenomatous Polyposis (FAP), about her advocacy work for this rare genetic condition. Hosts: Terry Wilcox, Executive Director, Patients RisingDr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public InterestKate Pecora, Field CorrespondentGuests: Sharon Hesterlee, Chief Research Officer at the Muscular Dystrophy AssociationJenny Jones, Patient Advocate and Author of Life's A PolypSara Healy, Patient CorrespondentJana Healy, Patient CorrespondentLinks: Terry Wilcox Applauds QALY Ban Bill - Patients Rising NowAmyotrophic Lateral Sclerosis (ALS) - Diseases | Muscular Dystrophy AssociationRodgers, Banks, & Wenstrup Lead QALY Ban to Affirm Every Person's Life has Value - Energy and Commerce CommitteeQALY Bill | H.R.7634 - Protecting Health Care for All Patients Act of 2022ICER to Assess Treatments for Amyotrophic Lateral SclerosisMOVR Data Hub (neuroMuscular ObserVational Research) | Muscular Dystrophy AssociationLife's A Polyp - Youtube Channel Need help?The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.orgHave a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent?Drop us a line: podcast@patientsrising.orgThe views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.

EA - Kidney stone pain as a potential cause area by Dan Elton

pain prof speech substack ea readers kidney elton kidney stones thinking fast karl popper open society rationalist medical physics its enemies emilsson will macaskill qaly qalys

Play Episode Listen Later May 3, 2022 22:55

Welcome to The Nonlinear Library, where we use Text-to-Speech software to convert the best writing from the Rationalist and EA communities into audio. This is: Kidney stone pain as a potential cause area, published by Dan Elton on May 3, 2022 on The Effective Altruism Forum. This is a cross-post from my Substack (original post here). Note: At the Effective Altruism Global: San Francisco conference in 2017, Prof. Will MacAskill implored the audience to “keep EA weird”. As the EA movement grows, it's important to keep EA's original spirit of exploration alive. To help do that, I'm planning to write several articles on potential new — and weird — cause areas. Effective altruists want to figure out how to do the most good per dollar spent. “Good” is often cached out in terms of deaths prevented or quality-adjusted life years saved (QALYs). QALYs attempt to adjust life-years for different states of health. However, the very method of QALY calculation, which typically involves surveys asking about trade-offs, might have some blind spots. For instance, if a disease state is rare, than most likely the requisite survey data for it has never been collected. The surveys themselves may have blind spots too. Consider these points from Andrés Gómez Emilsson (emphasis mine): “Someone described the experience of having a kidney stone as ‘indistinguishable from being stabbed with a white-hot-glowing knife that's twisted into your insides non-stop for hours'. It's likely that the reason why we do not hear about this is because (1) trauma often leads to suppressed memories, (2) people don't like sharing their most vulnerable moments, and (3) memory is state-dependent (you cannot easily recall the pain of kidney stones .. you've lost a tether/handle/trigger for it, as it is an alien state-space on a wholly different scale of intensity than everyday life).” Andres Gomez Emilsson As Daniel Kahneman describes in his book Thinking Fast and Slow, the remembering self is different than the experiencing self. People have trouble describing and conceptualizing extreme events, either positive or negative. People also don't like thinking about extreme negative events generally, whether they experienced them or others did. I personally sometimes notice my brain flinching away when thinking about kidney stone pain, even though I haven't experienced it myself. In the first part of this post I'll go over the evidence for extreme pain events. Then, I'll focus on kidney stones. The main reason for focusing on kidney stone pain is that over the past two years I've worked off-an-on on automated deep learning based software for detecting and measuring kidney stones in CT scans (see my paper in Medical Physics). So I have some expertise on the subject. Currently I am working with a radiologist at Massachusetts General Hopsital who is an expert on stone disease, Prof. Avinash Kambadakone. Background - suffering focused ethics “In my opinion human suffering makes a direct moral appeal, namely, the appeal for help, while there is no similar call to increase the happiness of a man who is doing well anyway.” “Instead of the greatest happiness for the greatest number, one should demand, more modestly, the least amount of avoidable suffering for all.”— Karl Popper, The Open Society and Its Enemies (1945) The idea that we should focus on eliminating suffering over increasing pleasure is intuitive to many people. See this recent Twitter poll from Robin Hanson: So, I don't think I need to spend much time here convincing people that reducing suffering should take precedent over increasing happiness. Note what I have in mind here is what is called “weakly-negative utilitarianism” which is quite different than pure negative utilitarianism, which focuses only on eliminating suffering. Readers interested in diving further into these topics should check out Lukas Gloor's essay “The Case for Suffering-Focused Ethics”. Background - long-tailed distributions of pleasure and pain “...

Episode 72 The Myth of Average: Time for Change

AiArthritis Voices 360 Podcast

Play Episode Listen Later Apr 3, 2022 73:16

Join Tiffany and Deb, both persons living with AiArthritis diseases, then Tiffany and John O'Brien, CEO of the National Pharmaceuticals Council, as they put the topic of importance of innovation & treatment access in research and public policy 'on the table'. Learn how YOU can join efforts to impact change! Who is 'at the table'? Tiffany Westrich-Robertson, CEO AiArthritis, person living with non-radiographic axial spondyloarthritis Deb Constien, AiArthritis Volunteer, person living with rheumatoid arthritis John O'Brien, President & CEO of the National Pharmaceutical Council (NPC) In this two segment episode, first join AiArthritis team - Tiffany and Deb - as they put a new show topic "on the table" for the first time: the importance of innovation in research and patient access to the treatments that are best for their individual characteristics. In this episode we introduce questions, like "Why does a treatment work for you and not me?" and "What if we don't tick all the boxes (are 'atypical', including having comorbidities) - how can I still access the best treatment for my unique needs?" The bottom line is that AiArthritis diseases are unique to the individual, and as a result, we may be better off using treatments that are not the same ones our health systems tell us we need to use. For change to happen, we need to hear stories from many patients (not just a few), but there are several barriers to achieving this. However, AiArthritis is working on a solution that involves creative, patient-led public policy classes focusing on four topics - two which are discussed today (innovation in research and value assessments). Listen to Deb and Tiffany explain more! In the second segment, Tiffany welcomes John to the table, as his organization - the National Pharmaceutical Council (NPC) - published a few of the reports AiArthritis is referencing in our courses. Together they explore how new research can lead to improved patient care and potential access to the right treatment for individualized need and based on patient-doctor decided management. Finally, they start the conversation about value assessments (a complex and difficult process of assigning value and cost to medications), highlighting both an understood need for healthcare systems to provide fair access to all, while considering patient uniqueness and the future of personalized and precision medicine. Show Notes: Part One: Tiffany and Deb talk about the importance of innovative research and getting more patient voices involved in public policy (both which influence access to therapeutic care). :53: Tiffany introduces show, herself (nr Axial Spondyloarthritis), and co-host Deb introduces herself (Rheumatoid Arthritis and several comorbidities) 2:25: Tiffany explains the topic of the show today, which involves patients who are not typical, including those with many diagnoses or who do not “check the boxes”. 3:35: Today we will focus on the importance of being “average” and “not-so-average”. The time being now for all of us to come forward and speak out in regards to our access to therapies and disease management - through public policy and research efforts. This is especially important for those who don't tick the boxes, or a model, textbook perfect patient. 4:25: Tiffany mentions the second half of the show will feature a guest, John O'Brien, President & CEO of the National Pharmaceutical Council (NPC) who will be discussing their paper, “The Myth of Average”, and the importance of innovation in research and understanding value assessments (or the processes around health systems procedures that help determine which medications we are able to access). This is a problem when we don't all fit a ‘general patient population' when we are thinking about policies that are centered around access for the average - and in a time when we are moving towards a world of precision medicine (treating the individual). How can patients come to the table and make a difference so we don't lose access to options? 5:25: Tiffany and Deb discuss their patient journeys and, specifically, how they would not be considered ‘typical', or classified in the group of general patient population. “We are all unique.” Tiffany further explains how her current diagnosis did not exist a decade ago, so thankfully for research her disease now has a name and treatments that work well for her. 8:45: Deb and Tiffany discuss how different therapies work for different people, which is important to understand. “What works for you may not work for me.” 9:27: Tiffany asks listeners to think about the evolution of our patient journeys and, as a result, the importance of patients/supporters to stay connected with their legislators (people partially responsible for access to healthcare) over time so they can see that our diseases are not static. They often evolve and, in turn, our treatment needs can change. 11:40: Tiffany and Deb discuss the analogy of using cough medicine. Why is “your” cough medicine better? We need to realize drugs in the same classes work differently because they have various differences that, in turn, react differently in each person's body. “This is why AiArthritis is highly invested in precision medicine, or matching the right person to the right treatment at the right time.” We are also invested in personalized medicine, or personal preferences, which also factor in. 13:18: Tiffany states the organization's mission to help others, like them, have a voice - alongside other stakeholders as equals - so, together, we can solve problems that impact education, advocacy (public policy), and research. 13:45: Tiffany explains the role of ethics in healthcare access, referencing a paper she authored in 2015, the Ethics of Step Therapy, in collaboration with two bioethicists and “bestie” Kathleen Arntsen from Lupus and Allied Diseases, Inc., where AiArthritis learned all about the different roles of ethics for both health providers and doctors. She specifically explains the ethical Principle of Justice, which involves the responsibility to ration healthcare for all. “If we are going to see change, we need to meet in the middle, respectful of the agencies who need to consider access for all." 15:50: They discuss show design, in that this is the first time this topic was put ‘on the table'. In 2022, we are starting “360its”, or spin off episodes and segments that will be in a variety of methods and platforms so all people can contribute to this conversation (globally). Then, together, we can all be part of the solution in regards to healthcare access and desire for innovation to continue. Keep in mind, perspectives may vary depending on where you live. For example, in socialist societies, value assessments - which tend to favor general patient populations - may be more accepted. 17:05: They discuss research and ‘atypical' factors into this. Cost savings is often based on citing credible research, which is mostly conducted in general patient populations (or whomever signs up to participate!) 17:50: Patient stories are vital to helping legislators humanize health access issues, but it's always a challenge finding stories - or the stories are from the same people. So AiArthritis spent 2021 figuring out why and, as a result, identified several barriers to participation, including lack of clarity and confidence. As a result, AiArthritis is launching a patient-led creative classroom program in 2022 that will cover four topics, including innovation and value assessments. Goals: 1) Increase number of patients/supporter voices in public policy and story sharing (including demographic contribution), 2) Recruit our own organization ambassadors, 3) Build confidence, 4) Identify the issue in relationship to self and others (patient to patient invitation to learn how to have a voice). 30:10: They discuss how this conversation will 360it and how to get involved in this new program: https://www.aiarthritis.org/advocacy 30:55: Part of the program will involve a program library of resources, including featuring The Myth of Average publication by the National Pharmaceutical Council (NPC)- which is why we are talking to John from the NPC next. 30:23: Tiffany thanks Deb Part Two: Tiffany and John discuss innovation in research and value assessments (measurements used, in some cases, to determine patient access to certain treatments) 31:39: Tiffany welcomes John to the show, who introduces himself and the NPC 32:58: Tiffany explains John is ‘at the table' because AiArthritis, who has developed a new patient-led public policy education program, is referencing several of the NPC's publications for the class library (including the Myth of Average). The piece is in keeping with AiArthritis' core priorities to help foster precision medicine and assess the right treatments for the individual. 35:42 John explains that innovation means the never ending search for cures and new treatments and why this resonates with him. Why does one treatment work for one person and not the other? Uses the example of Tiffany's journey, as she was diagnosed in 2009 before her disease had a name (nr-axial spondyloarthritis). So researchers were finding solutions for people like Tiffany, the Mystery Patient, and that's innovation. “This is all why it is essential that patients and researchers stay connected during the development process.” 39:00: Patients who are atypical, but who are lumped into the general patient population model, may risk being on the wrong treatment and that can have negative consequences. “Putting me on the right treatment that was indicated for my disease made a world of difference. It was like night and day.” 40:07: Tiffany tells us a story about her quality of life challenges prior to access to the right treatment, which involved the inability to get out of bed in the morning. 40:53: Also, people like Deb - who have exhausted all treatments - what do these patients do? Patients like us need to understanding the importance of research and the public policies that can influence our access to the treatments best for our individual needs. Join AiArthritis' efforts at https://www.aiarthritis.org/advocacy 42:15: John talks about the importance of the patient-doctor decision for treatments, which can be sidetracked if the healthcare system does not cover that therapy. Researchers ask, “Why do different individuals have different responses to the same treatments?” Furthermore, if the treatment was designed for the general patient population, how will this impact a patient's access to their doctor recommended therapy? The Myth of Average report highlights that age, genetics, ethnicity, behaviors, biology, and other individualized factors are not always considered when rationing treatments on a population level. “Patients should have access to the treatment that their doctor believes is clinically relevant for them.” 48:00: Tiffany talks in detail about AiArthritis' Ethic of Step Therapy investigation (2015) - which has relevant takeaways regardless of health system (location): patient-centeredness, fairness, clinical relevance. They revisit the Principle of Justice (from Part One with Deb) The ah-ha moment that led to the conclusion of the paper was a result of Tiffany's inability to be included in clinical trials because she was an atypical patient. She realized that clinical trials include patients who meet a general patient population criteria - the same research that insurance companies cite to justify their cost savings. However, in cases like Tiffany - where the patient is atypical - then the decision should default to the doctor, who is ethically obligated to treat to the individual characteristics of the patient. “Drop mic.” 50:44: This ethics paper led to the research work - and public policy work - we do at AiArthritis, in part, because a lot of money goes into this research using the general patient population, yet only 40-60% will respond once that drug goes to market (because the rest of us are atypical). “We need more treatments” for the rest of us. Shout out to Autoimmune Association and the Let My Doctor's Decide Initiative, which AiArthritis serves as an advisor. 52:02: John tells a story about how patients/supporters often understand the mechanisms of their disease and the different treatments researchers as well as the company themselves. “So not just at this table, but we have so much to learn from each other - especially when it comes to what the condition is like in the real world.” 53:25: John talks about health technology assessments (value assessments), what they are - including some access barriers they may cause (due to lack of transparency, caregiver burden, and drug measurement vs personalized experiences). Provides an example of employer based insurance (USA) if an employee is unable to work due to inability to access the best treatment for them. 55:42: John defines health technology assessments (HTAs), including the history of them and further discusses Quality Adjusted Life Years (QALYs) - which in his opinion can be discriminatory for people living with disabilities. 1:00:08: There is a need to cut costs and have a well distributed system of rationing spending. Even on some HTA websites in their own explanation of how these work they state the process is not designed for the individual but rather for the population. However, in a world that's moving towards precision medicine, and considering the patients who have exhausted all treatments, how do we ensure value assessments can be fair and considerate of the new science? They further discuss the QALY. No one is telling employers who are choosing health plans (USA) that if their employee isn't able to work because they don't have access to the right treatment that it could end up costing them more in time off, hospitalizations, and overall less work production. “This is the risk of using QALY's when trying to establish population based coverage decisions.” Tiffany confirms that it's difficult to work if you have brain fog, pain, or fatigue. 1:03:35: Tiffany reminds the audience that now in 2022 we are spinning off from each episode - any bullet point, any topic segment - and continuing the conversation in a variety of ways so we can include all of you in the conversation. Called #360its. Patients, supporters, legislators, researchers, pharma, etc - we need you all at the table! “The people who I always learned the most from are the patients.” Patients need to share their experiences. 1:06:00: Tiffany mentions a good 360it would be to discuss the continued need to use shared decision making in healthcare (patient-doctor decisions), because that is also relevant in regards to accessing the treatment that may be innovative and new (if that is the right treatment for us). Personalized and precision medicine consideration. 1:07:20: The research the NPC does is to help patients gain access to medications and foster innovation. “What If Groundbreaking Medicines Never Existed?” paper, which outlines where we would be today without drug innovation. Tiffany suggests that people read this, then let's do a 360it to discuss. 1:09:15: Tiffany reiterates the reason John is here is because the work the NPC does is in line with our AiArthritis work to fight for precision medicine (access to the right treatments at the right time for the individual person's needs). As such, we are including a few of their reports in our resource library in our new patient-led public policy education program. Learn more and sign up at https://www.aiarthritis.org/advocacy. 1:10:10: Tiffany thanks John for pulling up a seat at the table. 1:10:31: Tiffany closes out the show. If you like the show and listen on your favorite podcast station, please subscribe and give us a rating. Find all the episodes at our website at www.aiarthritis.org/talkshow Also, if you want to help us continue offering this program, please consider a donation, because your support makes this show possible! Find us on all social media channels at @IFAiArthritis where you can message us to have a seat at the table or email us at podcast@aiarthritis.org. _____________________________________________________________________________________________________________ Patient Voices and All Other Stakeholders - Join our AiArthritis Voices Program and Connect to Opportunities to Have Your Voice Counted If you are a patient, a parent of a juvenile patient, or any other stakeholder (doctor, nurse, researcher, industry representative, or other health services person) - are you ready to join the conversation? It's your turn to pull up a seat. Join our new AiArthritis Voices program, where people living with AiArthritis diseases and other stakeholders who we need 'at the table' to solve problems that impact education, advocacy, and research sign up to have a voice in our initiatives. By signing up, you'll get notified of opportunities to be more involved with this show - including submitting post-episode comments and gaining insider information on future show topics. Patients and all other stakeholders are encouraged to join so we can match you with opportunities to pull up a seat and TOGETHER - as equals - solve the problems of today and tomorrow. JOIN TODAY! AiArthritis Voices 360 is produced by the International Foundation for Autoimmune and Autoinflammatory Arthritis. Visit us on the web at www.aiarthritis.org/talkshow. Find us on Twitter, Instagram, TikTok, or Facebook (@IFAiArthritis) or email us (podcast@aiarthritis.org). Be sure to check out our top-rated show on Feedspot!

Sue Peschin - “CMS is in an untenable position” on Alzheimer's disease

Vital Health Podcast

Play Episode Listen Later Mar 10, 2022 27:08

When the Centers for Medicare and Medicaid Services (CMS) published its draft determination requiring further clinical trials in Alzheimer's disease treatments, Sue Peschin was one of the first to state her opposition to the policy. Sue is the President and CEO at the Alliance for Aging Research, the US' leading non-profit organization dedicated to improving healthy aging for all. In addition, Sue has also had senior roles at the Alzheimer's Foundation of America and has been critical of the many efforts to implement one-size-fits-all cost controls under Medicare. In this Vital Health Podcast, Sue digs into the broader implications of Vital Transformation's research and analysis of the CMS guidance, which was commissioned and funded by Biogen, and its potential impacts on her Alliance members. We also discuss the many potential unintended consequences of the proposal, including risks to the accelerated approval pathway, and to the development of new treatments in areas of high unmet needs such as neurological disorders. Sue highlights that CMS' guidance positions them as a de facto health technology assessment agency and spells out the implications for Medicare program participants. Additionally, we discuss existing case law in America which has previously decided that using QALY (quality adjusted life years) for reimbursement is age discriminatory, further bringing into question the viability of CMS' Alzheimer's disease guidance. See omnystudio.com/listener for privacy information.

america ceo president foundation position alzheimer's disease alliance medicare centers cms biogen medicaid services cms aging research qaly

S2, Ep 5- UnQALYfied: Putting a Price on Patients Life

Healthcare Matters

Play Episode Listen Later Mar 9, 2022 9:33

In the United States, data analytics are used to determine the price of drugs. But many patients don’t fit into neatly organized statistics, especially ones who have multiple diseases and, as a result, are denied therapies. In this week’s episode, we explore the method used in the U.S. to set drug prices, called the Cost per QALY or cost-effectiveness. "Data in health care has never been meant to be static. We need to provide even greater scrutiny concerning these models and how they’re applied and make coverage decisions for life-saving biopharmaceuticals,” says Dr. Robert Popovian, Chief Science Policy Officer at the Global Healthy Living Foundation. Among the highlights in this episode: 1:42: Listener review 2:15: Organizations that have global influence over patient care and access 3:30: How do these organizations define value in the marketplace? 4:07: A chronic patient doesn’t always benefit from this analytic approach to pricing drugs 4:35: “If the patient doesn’t fit the profile, they’re denied the therapy,” says Robert 5:00: Are these analytical tools misleading? 5:25: What happens if it ends up on a list in the U.S.? 6:18: From year to year, medications on these lists change 7:25: Robert shares his final thoughts Contact our hosts: Dr. Robert Popovian, Chief Science Policy Officer at GHLF, rpopovian@ghlf.org Conner Mertens, Patient Advocate and Community Outreach Manager at GHLF, cmertens@ghlf.org We want to hear what you think. Send your comments, or a video or audio clip of yourself to Healthcarematters@GHLF.org. Listen to all episodes of Healthcare Matters on our website or on your favorite podcast channel.See omnystudio.com/listener for privacy information.

united states data cost putting price patients organizations patient advocate community outreach manager qaly global healthy living foundation ghlf robert popovian

Health and happiness research topics—Part 1: Background on QALYs and DALYs by Derek

The Nonlinear Library: EA Forum Top Posts

Play Episode Listen Later Dec 11, 2021 127:25

welcome to the nonlinear library, where we use text-to-speech software to convert the best writing from the rationalist and ea communities into audio. This is: Health and happiness research topics—Part 1: Background on QALYs and DALYs, published by Derek on the effective altruism forum. Sequence contents Background on QALYs and DALYs The HALY+: Improving preference-based health metrics The sHALY: Developing subjective wellbeing-based health metrics The WELBY (i): Measuring states worse than dead The WELBY (ii): Establishing cardinality The WELBY (iii): Capturing spillover effects The WELBY (iv): Other measurement challenges Applications in effective altruism Applications outside effective altruism Conclusions Sequence summary Note: As many of the posts have not yet been completed, I may edit this summary to reflect the final content. This series of posts describes some of the metrics commonly used to evaluate health interventions and estimate the burden of disease, explains some problems with them, presents some alternatives, and suggests some potentially fruitful areas for further research.[1] It is primarily aimed at members of the effective altruism (EA) community who may wish to carry out one of the projects. Many of the topics would be suitable for student dissertations (especially in health economics, public health, psychology, and perhaps philosophy), but some of the most promising ideas would require major financial investment. Parts of the sequence—particularly the first and last posts—may also be worth reading for EAs with a general interest in evaluation methodology, global health, mental health, social care, and related fields. I begin by looking at health-adjusted life-years (HALYs), particularly the quality-adjusted life-year (QALY) and the disability-adjusted life-year (DALY). By combining length of life and level of health in one metric, these enable direct comparison across a wide variety of health conditions, making them popular both for evaluating healthcare programmes and for quantifying the burden of diseases, injuries, and risk factors in a population. I've also heard EAs using these concepts informally as a generic unit of value. However, HALYs have a number of major shortcomings in their current form. In particular, they: neglect non-health consequences of health interventions rely on poorly-informed judgements of the general public fail to acknowledge extreme suffering (and happiness) are difficult to interpret, capturing some but not all spillover effects are of little use in prioritising across sectors or cause areas This can lead to inefficient allocation of resources, in healthcare and beyond. Broadly, three alternative measures[2] could be developed in order to address these limitations: The HALY+: a tweaked version of the original QALY or DALY that captures some non-health outcomes and/or relies on more informed preferences. The sHALY: a “subjective wellbeing-based HALY” that retains the health-focused descriptive system but assigns weights to health states using experienced wellbeing rather than preferences. The WELBY: a wellbeing-adjusted life-year that can, in principle, capture the benefits of all kinds of intervention. A variation, the pWELBY, uses preferences to assign weights to each level of wellbeing. After introducing these metrics, this sequence of posts considers the additional research required to create them, and potential applications both within and outside EA. The importance, tractability, and neglectedness of each major project is briefly considered, though I do not attempt a formal priority ranking.[3] For individual researchers, my extremely tentative view is that work to establish the “dead point” (below which are states worse than dead) and lower bound on wellbeing scales is likely to have the greatest payoff—but, as with careers in general, the best choice of project is likely to depend heavily on personal fit. For well-funded research teams, including some large EA orga...

health measuring ea capturing daly eas welby qaly happiness research qalys

Pushing Against the QALY Criticism in Drug Pricing

A Health Podyssey

Play Episode Listen Later Sep 7, 2021 30:11

The quality-adjusted life-year (QALY) combines the expected effects on longevity and the expected effects on quality-of-life into a single standard measure. QALYs are often used as part of cost effectiveness analysis, particularly when analyzing the effectiveness of drugs.QALY as a measurement has received a lot of criticism. It's been criticized in concept or in the specifics of how it's defined or used. This criticism often forms the basis for opposition to price negotiations or any limitations on access to a particular drug. This kind of criticism can make it difficult to reach a consensus on processes that might yield negotiated or regulated drug prices.Leah Rand, a postdoctoral fellow at Harvard Medical School and Brigham and Women's Hospital, joins A Health Podyssey to talk about QALY, its criticism, and how to respond to that criticism.Rand and coauthor Aaron Kesselheim published in the September 2021 issue of Health Affairs a systematic literature review of critiques of QALYs and their relevance to drug health technology assessments. They identify three main categories of ethical and practical critiques of QALYS, including methodological concerns, criticisms of neutrality, and potential discrimination. Rand and Kesselheim conclude that understanding and addressing criticism of the QALY is essential for the move to value-based pricing.Listen as Health Affairs Editor-in-Chief Alan Weil interviews Leah Rand on the pros and cons of the Quality Adjusted Life Year measurement in health policy.Subscribe: RSS | Apple Podcasts | Spotify | Stitcher | Google Podcasts

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Author Interview: How Should Willingness-to-Pay Values of Quality-Adjusted Life-Years Be Updated and According to Whom?

AMA Journal of Ethics

Play Episode Listen Later Aug 1, 2021 20:54

Dr Paul T. Menzel joins Ethics Talk to discuss his article in the August 2021 issue of the Journal: How Should Willingness-to-Pay Values of Quality-Adjusted Life-Years Be Updated and According to Whom?

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HubWonk: Valuing Life-Saving Drugs: What is the Price of Life and Who Decides? (#59)

The Ricochet Audio Network Superfeed

Play Episode Listen Later Jun 8, 2021

Hubwonk host Joe Selvaggi talks with Pioneer Institute visiting fellow Dr. Bill Smith about Quality Adjusted Life Years (QALY) standards, and the ways in which so-called objective cost-containing strategies use expert opinion to determine the value of a life and thereby disadvantage the elderly, disabled, and those with less common vulnerabilities to disease. Guest: William […]

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Terveystaloustiede, Rokotteet, Suomen tulevaisuus - Kirsi Kautiainen #39

The Mikko Kemppe Podcast

Play Episode Listen Later May 9, 2021 113:06

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Don’t Stifle Medical Innovation!