Podcasts about orphan drug act

In this episode, Dr Katherine Bowen and Dr Julie Warner delve into the world of regulatory designations, providing clarity on what these designations offer, the products that qualify, and the pros and cons of each. They discuss the history of regulatory designations, starting with the Orphan Drug Act of 1983, and explore various designations such as Orphan Drug Designation (ODD), Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Regenerative Medicines Advanced Therapy Designation (RMAT). Tune in to gain valuable insights from their experience and advice and learn how to navigate the regulatory landscape effectively.

This Day in Legal History: James McCord ArrestedOn June 17, 1972, James McCord, security director for President Richard Nixon's re-election committee, and four Cuban-Americans were arrested for breaking into the Democratic National Committee Headquarters at the Watergate complex in Washington, D.C. This event marked the beginning of the Watergate scandal, a major political scandal in the United States that ultimately led to President Nixon's resignation. The burglars were caught wiretapping phones and stealing documents, intending to gather information to sabotage Nixon's political opponents. As investigations unfolded, it was revealed that the break-in was part of a broader campaign of political espionage and sabotage conducted by the Nixon administration. The scandal exposed widespread abuse of power, including illegal wiretapping, break-ins, and attempts to cover up these activities. Journalists Bob Woodward and Carl Bernstein of The Washington Post played a crucial role in uncovering the details of the scandal, leading to increased public scrutiny and pressure on the administration.The investigation led to the indictment of several Nixon administration officials and the creation of the Senate Watergate Committee. The most significant outcome was the discovery of the existence of secret tapes of conversations in the Oval Office, which revealed President Nixon's involvement in the cover-up. Faced with the likelihood of impeachment, Nixon became the first U.S. president to resign from office on August 8, 1974. The Watergate scandal had a lasting impact on American politics, leading to increased transparency and reforms aimed at preventing such abuses of power in the future.The rare disease community is advocating for changes to the Inflation Reduction Act's drug price-setting scheme, which is causing drugmakers and investors to reconsider developing drugs for small patient populations. Companies like Pfizer, Alnylam Pharmaceuticals, Eli Lilly, and Protagonist Therapeutics are altering their research strategies due to concerns over recouping costs under the Medicare Drug Price Negotiation Program. This program exempts orphan drugs with a single FDA-approved indication from price negotiations, but those with multiple indications might not qualify for the exclusion, even if they are not yet approved for additional conditions.Opponents are seeking legislative and judicial changes to amend the Inflation Reduction Act, as companies and investors shift their focus away from rare disease and small-molecule drugs. Jamie Sullivan of the EveryLife Foundation for Rare Diseases emphasized the importance of achieving technical fixes to support innovation. Recent legislative efforts aim to protect the development of these drugs, and companies like AstraZeneca are challenging the provisions in court. The Inflation Reduction Act has made the capital environment more challenging for rare disease drugs, according to John Stanford of the Incubate Coalition.Patient groups and some manufacturers argue that the exemption reduces incentives provided by the Orphan Drug Act, which has historically promoted the research and development of rare disease medicines. These drugs often launch with a single indication but can later be approved for additional uses. Concerns are rising that the current policies may hinder further research into rare diseases. Despite industry concerns, some groups argue that drug companies still have substantial resources for robust R&D and can profit from expanding indications for orphan drugs. A CMS spokesperson stated that the negotiation program aligns with the law and won't harm long-term innovation.US Drug Negotiations Plan Shifts Focus for Rare Disease ProgramsAlex Jones' personal bankruptcy has been converted to a Chapter 7 liquidation, meaning a trustee will now manage how he pays the $1.5 billion in defamation judgments against him. Jones was unable to reach an agreement with the families of the Sandy Hook Elementary School shooting victims, whom he defamed by claiming the 2012 massacre was a hoax. US Bankruptcy Judge Christopher Lopez ruled that Jones' case does not qualify for exceptions to prevent its conversion to Chapter 7, despite Jones' objections.During the same hearing, Judge Lopez dismissed the bankruptcy case of Infowars' parent company, Free Speech Systems LLC, allowing the Sandy Hook families to pursue their claims in state court. The judge clarified that there was never a request to shut down Infowars itself. Lopez expressed the difficulty of the case, acknowledging its connection to the tragic 2012 shooting.Jones' defamation debts were deemed non-dischargeable in bankruptcy due to the intentional and malicious nature of his actions. Following state court verdicts, Free Speech Systems filed for Chapter 11 bankruptcy in July 2022, and Jones filed for personal bankruptcy in December 2022. Efforts to reach a consensual bankruptcy plan failed, prompting the recent conversion to Chapter 7.Jones and the Sandy Hook families had recently agreed on the need to convert his case to Chapter 7, but the Free Speech Systems case remained contentious. Ultimately, the court decided that dismissing the Free Speech case was in the best interest of creditors. Despite potential liquidation, Jones may start a new broadcast, according to attorneys for the Sandy Hook families. This case is being managed under Alexander E. Jones, Bankr. S.D. Tex., No. 22-33553, as of June 14, 2024.Alex Jones Loses Financial Control as Trustee Takes Over (4)Alex Jones' assets to be liquidated as his company exits bankruptcy | ReutersAt a star-studded fundraiser in Los Angeles on June 15, President Joe Biden criticized the U.S. Supreme Court as being "out of kilter," emphasizing that it has never been as out of step as it is today. The event, which featured former President Barack Obama and Hollywood celebrities like George Clooney and Julia Roberts, raised over $30 million for Biden's campaign. Biden highlighted the court's conservative leanings, particularly criticizing Justice Clarence Thomas's remarks on reconsidering issues like in vitro fertilization and contraception. He also warned that if Donald Trump wins the 2024 election, he could appoint two more justices, which Biden described as alarming.The fundraiser was framed by a video montage contrasting Biden's record with that of Trump, drawing cheers from the audience. Biden noted that the Supreme Court, now dominated by conservatives appointed by Trump, has made decisions restricting affirmative action, gay rights, gun control, and environmental regulations. Obama reiterated the importance of elections in determining the court's power, linking the current situation to Trump's 2016 victory.The event marked the largest Democratic fundraiser in history, surpassing a previous record set in New York City. Other celebrities present included Jack Black, Jason Bateman, and Kathryn Hahn. Despite Biden's low approval ratings and concerns about his age, the fundraiser aimed to showcase the campaign's strength and momentum. Biden and Trump are currently tied in national polls, with Trump leading in battleground states. Both candidates have raised significant funds in California, demonstrating the state's financial influence on their campaigns.Biden slams Supreme Court at $30 million fundraiser with Obama, Clooney, Julia Roberts | Reuters This is a public episode. If you'd like to discuss this with other subscribers or get access to bonus episodes, visit www.minimumcomp.com/subscribe

Race Oncology Ltd (ASX: RAC) CEO Daniel Tillett sits down with Proactive's Jonathan Jackson after the US Food and Drug Administration (FDA) extended Orphan Drug Designation (ODD) to its proprietary formulation of bisantrene, RC220. Initially, the ODD was granted to Update Pharma Inc. in 2014 for the RC110 formulation of bisantrene for the treatment of acute myeloid leukemia (AML). The designation was transferred to Race in 2017 and has been maintained through annual reporting of clinical and non-clinical activities to the FDA. The ODD program, established under the Orphan Drug Act of 1983, aims to promote the development of treatments for rare diseases affecting fewer than 200,000 people in the USA. Benefits include seven-year US marketing exclusivity, a 25% federal tax credit for clinical research expenses, waiver of Prescription Drug User Fee Act (PDUFA) fees (valued at US$4 million in 2024) and eligibility for research grants and additional regulatory guidance from the FDA. Tillett, expressed gratitude for the clinical team's efforts in securing the ODD extension for RC220 bisantrene. Chief medical officer Dr Michelle Rashford notes that the designation enables closer collaboration with the FDA on all RC220 bisantrene clinical programs as Race progresses towards opening an FDA Investigational New Drug (IND) application in 2025. #ProactiveInvestors #RaceOncology #ASX #Bisantrene, #RC220, #OrphanDrugDesignation, #FDAApproval, #AcuteMyeloidLeukemia, #RareDiseases, #DrugDevelopment, #ClinicalResearch, #PharmaNews, #OrphanDrugAct, #USFDA, #MedicalResearch, #CancerTreatment, #HealthcareInnovation, #Pharmaceuticals, #ResearchGrants, #RegulatoryApproval, #DrugExclusivity, #TaxCredits #invest #investing #investment #investor #stockmarket #stocks #stock #stockmarketnews

This Day in Legal History: Impeachment Hearings Against NixonOn May 9, 1974, a significant event unfolded in the political and legal history of the United States as the House Judiciary Committee, chaired by Representative Peter Rodino (D-NJ), initiated impeachment hearings against President Richard Nixon. This marked a crucial point in the Watergate scandal, an episode that had gripped the nation for two years following the break-in at the Democratic National Committee headquarters at the Watergate complex in June 1972. The scandal involved allegations of political espionage and sabotage conducted on behalf of Nixon's reelection campaign, a cover-up by high-level White House officials, and subsequent revelations of misconduct.The decision to open hearings was based on accumulating evidence from various investigations, including testimonies provided to the Senate Watergate Committee and the revelations from the Watergate tapes. As chairman, Peter Rodino played a pivotal role in overseeing a fair and meticulous review of the allegations against Nixon, guiding the committee through extensive debates on legal and constitutional grounds for impeachment.The committee's hearings were comprehensive, involving detailed examination of the evidence and intense deliberations on the constitutional criteria for impeachment. The proceedings were conducted with a level of transparency, broadcasted live on national television, allowing the public to witness the meticulous process of democratic oversight.On July 30, 1974, after several months of hearings, the committee voted to impeach Nixon on three counts: obstruction of justice, abuse of power, and contempt of Congress. These charges were centered around Nixon's involvement in the cover-up of the Watergate break-in and other misuse of presidential power.The impeachment process was a watershed moment in American legal history, emphasizing the principle that no one, not even the President of the United States, is above the law. It showcased the pivotal role of legal frameworks and congressional oversight in upholding democratic principles and integrity within the executive branch.Peter Rodino, whose leadership was instrumental during this challenging time, was later recognized for his fairness and dedication to upholding justice. He passed away in 2005 at the age of 95, remembered as a key figure in one of the most challenging periods in American political history. This day in 1974 remains a defining moment, highlighting the robustness of legal and democratic processes in the face of political crisis.Jazz Pharmaceuticals is set to challenge the FDA in a court case regarding the approval of a rival narcolepsy medication, Lumryz, by Avadel CNS Pharmaceuticals. Jazz argues that the FDA's approval of Lumryz violated the Orphan Drug Act because it disregarded the "clinical superiority" clause. Jazz contends that the FDA should not have approved Lumryz as it is considered the same drug as Jazz's Xywav, which has orphan drug exclusivity until 2027. The FDA, however, maintains that Lumryz is not the same because it offers clinical benefits that Xywav does not, such as not requiring a second dose during the night. The case hinges on the interpretation of the term "same drug" and whether the FDA's definition of a clinically superior drug is permissible under the Orphan Drug Act. This lawsuit could potentially reshape how the FDA interprets and applies rules concerning drug exclusivity and could have broader implications for how agencies interpret ambiguous statutory language, following the principles established by the Chevron doctrine.By way of very brief background, the Orphan Drug Act of 1983 provides seven years of market exclusivity for drugs designed to treat rare diseases, preventing the FDA from approving similar drugs for the same condition from other manufacturers unless certain exceptions apply. This law aims to encourage the development of treatments for diseases affecting small patient populations, and thus having a small number of potential sales, by offering incentives like exclusivity to pharmaceutical companies. In the case involving Jazz Pharmaceuticals and the FDA, the dispute centers on whether the FDA can approve a drug claimed to be "clinically superior" despite existing exclusivity agreements under this law.Jazz Pharma to Fight FDA in Court Over Rival Sleep Drug ApprovalProminent law firms Morgan & Morgan PA, Seeger Weiss LLP, Wagstaff & Cartmell LLP, and Motley Rice have been appointed as lead counsel for plaintiffs in litigation concerning side effect warnings for the diabetes drug Ozempic and related medications. The decision was made by Judge Gene E.K. Pratter of the US District Court for the Eastern District of Pennsylvania. This litigation involves multiple lawsuits against pharmaceutical companies including Novo Nordisk A/S, the producer of Ozempic, and Eli Lilly & Co., which manufactures Mounjaro and Trulicity. These companies are accused of underplaying serious gastrointestinal side effects associated with drugs classified as glucagon-like peptide-1 receptor agonists. Ozempic, known for its blood sugar regulation and unintended weight loss effects, has seen a spike in demand leading to the development of the weight loss drug Wegovy. The firms representing the defendants are DLA Piper for Novo Nordisk, and Welsh & Recker PC, Kirkland & Ellis LLP, and Redgrave LLP for Eli Lilly. The case continues to unfold in Pennsylvania.Ozempic and Wegovy, popular diabetes and weight-loss medications, have been linked to severe gastrointestinal side effects, including gastroparesis (stomach paralysis) and cyclic vomiting syndrome (CVS). Gastroparesis causes the stomach muscles to slow down, severely delaying the digestion process, which can result in food remaining undigested in the stomach for prolonged periods. This condition can lead to symptoms such as nausea, vomiting, and abdominal pain. Cyclic vomiting syndrome is characterized by intense, recurrent episodes of vomiting, which can last several hours or days, accompanied by severe abdominal pain and nausea. These side effects significantly impact patients' quality of life, and concerns have been raised about the adequacy of warnings provided by the drugs' manufacturer, Novo Nordisk. Ozempic Plaintiff Attorneys Led by Morgan & Morgan, Seeger WeissRudolph Giuliani, the former mayor of New York, has been accused again by Georgia poll workers Ruby Freeman and Wandrea' Arshaye “Shaye” Moss of making false statements. Despite a previous $148 million defamation judgment against him for similar allegations, Giuliani reportedly continued to claim that these poll workers manipulated the 2020 presidential election results. This accusation came during an April livestream where he addressed a crowd in Tulsa, Oklahoma. Freeman and Moss, who recently won their defamation suit against Giuliani, responded by filing an objection to his motion for an appeal, which he had requested after declaring bankruptcy. They argue that Giuliani's actions are not only a flawed litigation tactic but also a disregard for legal consequences. Meanwhile, a committee of unsecured creditors also opposed Giuliani's motion, highlighting that his legal maneuvers delay resolving the financial claims against him, affecting the creditors adversely. The dispute underscores ongoing legal battles involving Giuliani, who faces significant financial liabilities primarily due to the defamation award.Giuliani Accused of New False Remarks About Georgia Poll WorkersStephen Miller's legal group, America First Legal (AFL), has initiated a lawsuit against IBM subsidiary Red Hat Inc., alleging that the company's diversity initiatives resulted in discriminatory practices against a former White male employee, Allan Kingsley Wood. Filed in the US District Court for the District of Idaho, the lawsuit contends that Red Hat's diversity, equity, and inclusion (DEI) program, which sets hiring goals for women and minorities, led to Wood's wrongful termination. Wood, who was a senior director at Red Hat, reportedly disagreed with the DEI policies based on his religious, personal, and political beliefs, advocating for hiring based on merit. The lawsuit also claims that Red Hat's DEI goals effectively acted as "quotas," aiming to significantly alter the company's workforce demographics by 2028. Shortly after expressing his views, Wood was dismissed from his position, along with 21 other employees, predominantly White males, under the guise of role elimination. Wood is now seeking compensation and an injunction against Red Hat's DEI program. The case highlights ongoing tensions and legal challenges related to corporate diversity efforts following the US Supreme Court's June 2023 decision on affirmative action in college admissions.Stephen Miller Group Sues IBM's Red Hat for White Male Bias (1) Get full access to Minimum Competence - Daily Legal News Podcast at www.minimumcomp.com/subscribe

In this episode, Ayesha talked about Rare Disease Day 2024, a global movement held on the last day of February each year that focuses on raising awareness about rare diseases including their impact on patients' lives. This year, Rare Disease Day falls on February 29, the rarest day in a leap year, making it even more special.Ayesha spoke about how the Rare Disease Day campaign helps increase awareness about rare diseases, promotes research and development, supports advocacy for policies supporting patients and their families and brings together a global rare disease community.Advancements in rare disease research are leading to innovative new treatments for various rare conditions, including genetic disorders, which make up just over 70 percent of all rare diseases. Hear more about new rare disease drugs approved by the FDA in the past couple of years. Also hear about some of the latest trends in rare disease research, including innovative trial designs, patient-centric approaches, the use of cutting-edge technologies like AI and international collaborations.Read the full articles here:Rare Disease Day 2024: Highlighting Statistics and Patient StoriesNew Rare Disease Drugs and Research AdvancementsFor more life science and medical device content, visit the Xtalks Vitals homepage.Follow Us on Social MediaTwitter: @Xtalks Instagram: @Xtalks Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Pink Sheet reporters and editors discuss US FDA advisory committee members' concerns about two proposed RSV vaccines, Lilly's plan to lower the cost of its insulin products, proposed changes to the Orphan Drug Act and the departure of the influential leader of the FDA's Office of Neuroscience.

“Our rare disease community is looking to solve for many different types of policy barriers because we have a very diverse patient community,” says Annie Kennedy, who was drawn to the rare disease issue due to some personal experience early in her life.  After spending many years as a patient advocate -- including being with patients during provider visits -- she has developed a keen understanding of where the healthcare system can be improved to do justice to rare disease patients and families. That insight informs her work as chief of Policy, Advocacy and Patient Engagement at the EveryLife Foundation where she helps provide patient communities with tools and resources they can use to make their voice heard in the policy arena. In fact, next week, EveryLife is holding its annual Rare Disease Week on Capitol Hill during which members of the rare disease community will meet with lawmakers in Washington to provide meaningful perspectives and encourage their support. “There are more than thirty million Americans living with rare diseases, so this is a real public health priority,” she tells Raise the Line host Michael Carrese. One resource EveryLife has brought to the table is a study on the total cost of living with a rare disease, not just the costs of medical care, which is helping to inform policy discussions. You'll also learn about some key steps pharma companies, insurers and providers can take to support the rare disease community and where the field is heading in the next few years.  Mentioned in this episode: https://everylifefoundation.org/

New manufacturing systems for CAR Ts aren't just about speed, Executive Director for Biopharma Intelligence Lauren Martz said on the latest BioCentury This Week podcast. Martz and colleagues discuss the safety, efficacy and access benefits from rapid manufacturing platforms, and how these systems are set to change the way CAR T cells are delivered to patients. BioCentury's editors also analyze the state of incentives for orphan drug development as the Orphan Drug Act turns 40, how the FTC's non-compete proposal could have unanticipated effects for biotech, and what solutions former FDA commissioners are proposing to battle misinformation about science.

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/new-books-network

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/medicine

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/science

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/science-technology-and-society

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices Support our show by becoming a premium member! https://newbooksnetwork.supportingcast.fm/technology

Advances in medicine have made possible better treatments for widespread, familiar human illnesses like cancer, diabetes, and heart disease. Yet there are thousands of much less common diseases, most of genetic origin, each classed as rare because it afflicts only a small number of people. These patient groups were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these orphaned diseases collectively caused misery and expense, often far greater than did more common ailments, for tens of millions of individuals and their families. Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic triggered additional activism, compounded when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. And the third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen employing then-new genetic engineering instead of conventional approaches to pharmaceutical development. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry. Jim Geraghty has been a passionate participant in the orphan drug revolution since its inception--a leader in the field as a strategy consultant, biotechnology executive, and venture entrepreneur. Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology (Cold Springs Harbor Lab Press, 2022) is in part a history, with eyewitness accounts of advances as they occurred and portraits of the pioneering scientists and physicians, tireless activists, and visionary business leaders who made the revolution happen. And it tells deeply personal stories of patients and parents willing to risk new, untried therapies. But Geraghty also uses his exceptional experience and vantage point to look forward to the immense promise of the newest technologies like gene therapy and gene editing for the treatment of patients today and tomorrow. He concludes with thoughtful consideration of important questions. Why do drugs to treat orphan diseases cost so much? How can we ensure they are affordable? How can their effectiveness be responsibly assessed? And how can access to them be expanded internationally? This book graphically and poignantly illustrates how far an important healthcare revolution has come and reminds us that if not nurtured, it could end before its immense promise has been fulfilled. Learn more about your ad choices. Visit megaphone.fm/adchoices

James Geraghty has had an up-close view of the rise of the rare disease drug industry as an entrepreneur, investor, and executive. Now he's added the additional title of “author” with his new book “Inside the Orphan Drug Revolution.” Geraghty looks back through the past 40 years of his career starting with the passage of the Orphan Drug Act in 1983. We spoke to Geraghty about the catalysts that gave rise to the orphan drug industry, his concerns about the changing rare disease policy landscape, and why he believes its essential for companies to take a patient-centric approach to drug development.

This week, both of our storytellers are navigating rare disease diagnoses and the feelings of fear, uncertainty, and loneliness that can often come along with them. This episode was produced in partnership with the Chan Zuckerberg Initiative's Rare As One Project, which brings together rare disease patient advocates from all over the world, uniting them in their quest for cures and working to lift up their efforts by offering new tools, grants programs, and capacity-building support and training. (For more stories like these, you can also check out the previous episode The Story Collider produced with Rare As One in 2019, as well as our Rare Disease playlist.) Part 1: After her child is finally diagnosed with Hermansky-Pudlak Syndome, Donna Appell set off on a mission to make sure other parents have the information she didn't. Part 2: Feeling unmoored after she's diagnosed with LFS, Jenn Perry attends a patient conference that changes her life. Donna Appell is the mother of two children and her oldest child has Hermansky-Pudlak Syndrome (HPS). Feeling desperate in her attempts to find help, she founded The HPS Network in 1992. Ms. Appell was appointed to the American Thoracic Society's (ATS) Public Advisory Roundtable and has received The ATS Public Service Award and the “Presidential Commendation”. For her work in Puerto Rico, she received the inaugural recognition from the ATS, “Innovations in Health Equality Award”. She was employed for 22 years as a RN in a Critical Care Open Heart ICU. In 2013, Appell and her daughter were chosen as one of 30 Heroes to celebrate the 30th Anniversary of the Orphan Drug Act by the Office of Orphan Product Development at the FDA and the National Organization for Rare Disorders (NORD). In 2019, Appell was honored to be awarded a Rare Impact Award from NORD. Jenn Perry is the President of the Li-Fraumeni Syndrome Association. She is a wife and mom of 2 girls ages 28 and 18. As a LFS patient myself Jenn is relentless in the supporting the LFS community in multiple ways. Jenn loves her horse, and competition partner, Maximus. In addition to riding, she has worked as a business consultant in the QSR industry, and she currently co-owns a Gymnastic & sports facility. Gymnastic was her first love, and she enjoy judging competitive gym at all levels. It is her honor to have the opportunity to speak in front of everyone today, as bringing awareness to this syndrome is so needed, in order to find the cure. Learn more about your ad choices. Visit podcastchoices.com/adchoices

Dr. Bill Smith, a visiting fellow in the life sciences at the Boston-based think tank, Pioneer Institute, and former drug industry veteran, joins the State of Health to talk about the evolving market dynamics in the drug industry starting with the Orphan Drug Act and the industry's rush into therapeutic development for rare diseases. In this episode, Gunnar presses Dr. Smith on the sustainability of high list prices and burden for the insurance industry. Dr. Smith also talks about rising coinsurance rates as a barrier to patient access. The show concludes with Dr. Smith's hopes for the future of drug development and evolving market dynamic. Learn more about your ad choices. Visit podcastchoices.com/adchoices

Host Joe Selvaggi talks with Dr. Martin Makary about how to reduce healthcare prices. Dr. Martin Makary is a surgical oncologist and chief of the Johns Hopkins Islet Transplant Center. He is a clinical lead for the Johns Hopkins Sibley Innovation Hub and serves as Executive Director of Improving Wisely, a Robert Wood Johnson Foundation project to lower health care costs in the U.S. by creating measures of appropriateness in health care. Dr. Makary's research focuses on the creation and evaluation of new health care innovations. He is the creator of the Surgery Checklist, publishing its first description and later served on the W.H.O. Safe Surgery Saves Lives committee. He led the W.H.O. workgroup to create global measures of surgical quality. Dr. Makary has published over 200 scientific articles, including the first description of “frailty” impacting surgical outcomes, the original studies on safety culture measurement in hospitals, and an evaluation of the Orphan Drug Act. He is a leading voice for physicians, writing in The Wall Street Journal, and is the author of The New York Times best-selling book Unaccountable about patient safety and physician-led transparency efforts in health care. Dr. Makary is the founder of the Johns Hopkins Center For Surgical Outcomes Research and Clinical Trials and is the recipient of numerous grants to evaluate the effectiveness of new surgical technology and new interventions in health care. He serves jointly as a professor of surgery at the Johns Hopkins University School of Medicine and a professor of health policy & management at the Johns Hopkins Bloomberg School of Public Health. He has pioneered new pancreas operations at Johns Hopkins, including the laparoscopic Whipple procedure. He performed the first series of laparoscopic islet auto-transplantation and other laparoscopic operations in field of surgery. Dr. Makary is among the few highest-volume laparoscopic pancreas surgeons in the United States. He is the recipient of the National Pancreas Foundation Nobility in Science Award. Dr. Makary is a graduate of Bucknell, Thomas Jefferson and Harvard University. He completed his surgical training at Georgetown University and his fellowship at Johns Hopkins Hospital.

Wow! It’s episode 300. That’s a milestone. Because of you, we’ve grown to be one of the largest podcasts for health care executives—so, thank you to every one of you who has recommended the show to your friends and colleagues, which is really the highest compliment. Thanks also to all the listeners of this show who have written reviews, LinkedIn posts, and sent emails. The team over here at Relentless Health Value really appreciates your kind words. They’re super motivating. The emails we love to get are the ones where one of you talks about a success story, like an example where you’ve taken something you heard and made it actionable—how you helped patients get better care to lower cost or how you were able to collaborate with fellow stakeholders in a meaningful way. That’s really why we’re here and why it’s so motivating to hear stories like this, which brings me to a really important point. We’re in this together. All of you health care decision maker/stakeholder types out there, you who can directly effect change, it’s really you who deserve the biggest round of applause, if I do say so. We appreciate the opportunity to kick off the activity or the decision making, but it’s you all who pick up the ball and run with it. And for that, we—as both professionals and patients—thank you. Moving on to today’s episode 300, my guest in this health care podcast, Bruce Rector, MD, is an expert on drug affordability; and he has extensively studied how to make sure we get the right drugs developed by considering innovation incentives among other things. He’s done a lot of work with Doctors for America and the Center for American Progress. He also teaches medical students, pharmaceutical policy, and has worked with drug companies on drug development promotion. So, I felt like that was a pretty rounded perspective of the issues that I wanted to get into here. Let me tell you why I started to think about this. Any one of those stories where somebody dies of an infection that was resistant to antibiotics, they’re always ghastly tales that seem so unnecessary. And every time I hear one of them, I wonder why pharma companies aren’t in the antibiotic business. Clearly, there’s a need. Well, it turns out antibiotics are a great case study of what happens when drug companies don’t have the incentive to develop drugs that are a huge need to society—which brings me to the big hairy challenge I’m talking with Bruce Rector about in this podcast. How do you ensure that pharmaceutical manufacturers are fairly incented and compensated to develop the drugs that are of the most value to society? Orphan drugs, by the way, are a great example of what happens when incentives are put in place to develop drugs. At last count, half the drugs developed in the past decades have been for rare diseases—because of the 1983 Orphan Drug Act that made it quite profitable to develop for rare diseases. So, in this health care podcast, we dig in to two—arguably three—categories of incentives that are typically offered or available to pharma companies in this country today and which are, frankly, used in that Orphan Drug Act. The first two are push incentives, and then there’s pull incentives. Push incentives are when the government, generally, offers incentives to reduce industries’ costs during the R&D (Research & Development) stages, like they give grants or tax credits for clinical research—things like this. Pull incentives, on the other hand, are ways to guarantee demand after the drug is developed or to help the pharma company make more money on the drug, for example, by extending patent exclusivity—like if you, Pharma, develop this drug, we’ll promise to buy millions of doses right up front and/or we’ll bar any generics for two extra years so you get the two extra years of revenue. (You might be thinking about what’s going on with COVID right now. Just sayin’.) So, we have push incentives, we’ve got pull incentives, and then this last one, which is more of a market condition than really anything paid up front or deliberately engineered on the back end. It’s that drugs aren’t like new desk chairs or some other product that, if the price goes too high, your employer just doesn’t buy it. If someone is suffering from a deadly disease and there’s one drug for it with no competition, there’s nothing and nobody in the US marketplace that really has the power to hinder the pharma company from basically charging whatever they want for it. Dr. Vincent Rajkumar talks about this in EP296 if you want to go back and listen to that one for more info. But wait … there’s more I talk with Dr. Rector about in this health care podcast. He brings up two different ways to contemplate paying for drugs. First is the fire extinguisher model, which is really applicable to antibiotics—and we talk about a couple of things I had never thought about relative to antibiotics. And then secondly, we have the subscription model—definitely food for thought for any of you innovative health plan types or policy makers out there. You can learn more by following Dr. Rector on Twitter and LinkedIn. Bruce Rector, MD, is physician whose work spans many important areas of the health care landscape: biopharmaceutical policy advisor, health policy lecturer, life science company consultant, and physician advocate. He focuses on policies to ensure that the right drugs get developed to meet society’s needs and that they are value based and equitably priced. Dr. Rector coauthored an article on value-based pricing, “Grounding Value-Based Drug Pricing in Population Health,” which is published in Clinical Pharmacology & Therapeutics. 05:58 What’s the issue with innovation in the pharmaceutical space? 06:47 “The problem … everyone talks about is antibiotics.” 07:38 What are pharmaceutical companies launching to drive value instead of antibiotics? 08:21 What are orphan drugs? And why is development incentivized for those drugs? 11:56 What are the differences between push incentives and pull incentives? 14:37 “The pharma company is all about how much money [the drug] can make once it hits the market.” 16:28 “These contracts, they know once they hit the market, there’s billions just waiting for them.” 17:17 What are the biggest pull and push incentives in Pharma? 17:40 What are the push and pull incentives with antibiotics? 24:39 What’s the fire extinguisher theory in Pharma? You can learn more by following Dr. Rector on Twitter and LinkedIn. @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma What’s the issue with #innovation in the #pharmaceutical space? @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma “The problem … everyone talks about is #antibiotics.” @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma What are pharmaceutical companies launching to drive value instead of antibiotics? @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma What are #orphandrugs? And why is development incentivized for those drugs? @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma What are the differences between #pushincentives and #pullincentives? @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma “The pharma company is all about how much money [the drug] can make once it hits the market.” @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma “These contracts, they know once they hit the market, there’s billions just waiting for them.” @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma What are the biggest pull and push incentives in Pharma? @BERector discusses #drugdevelopment and affordability in this week’s #healthcarepodcast. #healthcare #podcast #digitalhealth #pharma

The National Organization of Rare Disorders has been helping patients, caregivers, organizations, researchers, and Clinicians for almost 40 years. Introduction by Tom Rhoads (00:02): There are over 300 million people living with one or more of over 6,000 identified, rare diseases around the world. A disease defined as rare in Europe affects fewer than one in 2000 people. Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient suffering from the same disease. Hi, I'm Tom Rhodes, CEO of Spencer Health Solutions. We are honored to have a special guest from the rare disease community on the "People Always, Patients Sometimes" podcast. Pamela Gavin is a Chief Strategy Officer for the National Organization for Rare Disorders. NORD has been educating, supporting, advocating, and building a community for the rare disease community for over 38 years. I know you'll enjoy learning more about NORD on the "People Always, Patients Sometimes" podcast. Janet Kennedy (00:56): Hi, my name is Janet Kennedy and I am your host for "People Always, Patients Sometimes," a production of Spencer Health Solutions. We have invited Pamela Gavin, Chief Strategy Officer of NORD, the National Organization for Rare Disorders to join our podcast. Patients with rare disorders often are overlooked by clinical research and drug developers, or have histories of misdiagnoses. Now that Coronavirus is impacting the globe and awareness of the fragility of persons with rare diseases is triggering teachable moments for all of us. Let's dive in! Welcome to "People Always, Patients Sometimes," Pam!   Pamela Gavin (01:33): Thank you, Janet. Great to speak with you today. Janet Kennedy (01:36): I am so fascinated by your organization. I had no idea that literally there were hundreds and hundreds of rare diseases that you all are tracking and cataloging through your organization. Pamela Gavin (01:49): Yes, it's an amazing, exciting time within the field of rare diseases. We have over 7,000 known, identified rare diseases in the world today. And so NORD as patient advocacy organization supports all of them and the work that we do. Janet Kennedy (02:08): Can you tell me a little bit more about NORD itself and how it's structured? Is this an organization that provides information to people or are you proactive in helping resolve and learn and educate people about the diseases? Pamela Gavin (02:24): So we do some of both of those things and an organization established by patients and caregivers and advocates over 37 years ago, for some of the very things that you mentioned in starting your podcast introduction. And that is to advocate for improvements in it, interest in job development, device development, medical care, and services for people with rare conditions because very little was being done in the field. It was very hard to make it work economically for companies and researchers to study rare diseases because the patient populations were so small, but people were suffering tremendously. Pamela Gavin (03:04): So the community, the patient advocates got together and pushed for legislation that ultimately led to the Orphan Drug Act being passed in 1983. And that legislation established financial incentives for those to do research and study and develop products for people with rare conditions, the majority of which even today still live with a disease for which there are no FDA approved treatments. So we advocate for change. We advocate for people living with rare conditions to have access to proper diagnosis, treatment, and care so that they can live their very best life. We advocate for an environment that promotes research and innovation and fairness and equity across the entire community. As part of that advocacy work to promote access to that which is necessary for people to live their best life. We also do a lot of education to collaborate with organizations across the rare disease spectrum. Many of which are members of NORD, other advocacy organizations to educate people, clinicians about rare diseases. We also educate advocates so that they could advocate on their own behalf. Janet Kennedy (04:23): I'm thinking back to when you were founded 37 years ago, and there's no internet there. So I can't imagine how hard it must have been for patients with a rare disease to find other patients with rare disease. It must have been like living in a cave to feel so alone and to feel like they're so unique that nobody cared about them. Pamela Gavin (04:48): They could live their whole lives without meeting somebody with the same condition. You're absolutely right. Janet, the isolation is just an incredible challenge for people to live with such a burden. I can speak personally from my own family's experience, having grown up part of my professional career without the internet, certainly the beginning of it. And now knowing not only that we have the internet, but all the amazing tools to connect people together, to communicate with one another, to bridge people across many, many boundaries across the world. So there still is isolation today, but it wasn't seemingly hard to connect to people. I can remember the first time we saw a newspaper article where there was a story about somebody with a rare genetic condition that was similar to what my nephew had. And it was as if that piece of paper that photograph trying to connect to that community. It was so impactful and so meaningful. Now we don't have to fax things over a fax machine or try to find a phone number by looking in a yellow pages or white pages or calling institutions and calling and calling and calling until you found someone who may know somebody, a family, you sit at your computer or on your cell phone and start searching. Janet Kennedy (06:10): And sometimes you're looking across States across the country, across the globe to find other people with similar conditions. Pamela Gavin (06:18): Absolutely, absolutely. And we work with organizations like ours across the globe. It's a privilege and an honor to represent the rare disease patient and caregiver community. As we try to unite ourselves across the globe. Janet Kennedy (06:35): Let me ask you a question about your organization. It is made up of other organizations that support a very specific rare disease for instance, but do you also support individuals for whom there is no support organization? Pamela Gavin (06:52): That's exactly right. NORD represents all rare diseases that are known. So we work in collaboration with organizations that exist for those diseases that are known. And then if there is no organizational representation, NORD tries its best to fill that gap and representing those communities as well. Janet Kennedy (07:12): Before we move on to discussion of patients and clinical trials, I'd love to know a little bit more about your history and how did you end up at NORD? Pamela Gavin (07:21): It certainly was not a planned professional path. I started my career working in healthcare. I at first wanted to be a clinician. I wanted to be a physician and ended up finding myself, getting involved in healthcare information systems, technology really fascinated by the opportunity to leverage technology, to improve healthcare delivery, healthcare decision making, and the experience for patients so that they had better outcomes. And I was intrigued by the impact that technology could have disruptive in a positive way to empower clinicians, to make better decisions, to have better information, better tools, equipped to do their job better. And so that's really where I started to focus. I ultimately found myself evolving into an advocacy role because of personal experiences. I had members of my family to be involved in medical errors. So I got involved in advocacy for improvements in patient safety and clinical care safety. Pamela Gavin (08:32): And then that led to getting involved in rare diseases. My brother's first son, my nephew, Trevor was born with metachromatic leukodystrophy. Although we didn't know it right away, they took some time for it to be diagnosed. And this is right when the internet was really beginning to be used predominantly for academic and for business to business purposes. So to your point about isolation, I saw that firsthand. We're a pretty close family. And so we all supported my brother and my sister in law through this journey. But one of the first organizations that they were introduced to once they finally got this devastating diagnosis, the disease for which there was no cure and was going to take my nephew over very over a period of time was the national organization for rare disorders. And I remember what it meant to be able to talk to somebody that knew something about what was taking place in our lives. Pamela Gavin (09:31): And NORD connected my brother and sister law in particular to medical information and to clinicians and researchers that were working in the field. So even though there was no treatment or cure and the outcome was going to be inevitably devastating to know that there were people working on, it provided some comfort. So that's how I was first introduced to NORD many, many years later, an opportunity to join the organization came about where I was really marrying my work in healthcare information technology and data and supporting improvements in clinical outcomes. Marrying that with my rare disease experience, I feel very privileged to have this opportunity to work in this space, representing the community and helping to make a difference for those who today live with a rare disease or love or care for someone with a rare condition in the US. Janet Kennedy (10:35): I think that's such an important thing is to have passion for the work you do. And in your case, it's well, really a job. You can never leave, but it sounds like it's a job that also sustains you. Pamela Gavin (10:49): Thank you for describing it that way. I think you are spot on. It is one I could never leave. If I were to ever be working someplace other than NORD, I'd still be passionately connected to the community and concerned about its wellbeing and progress. I could meet anyone with a rare disease or connected to a rare disease, and it doesn't have to be metachromatic leukodystrophy. And I feel connected to that person or persons, but I do feel lucky. I feel very lucky to be able to do this work. Janet Kennedy (11:19): Tell me a little bit about your role as the strategy leader for the organization. What does that mean? And one of my side questions is - you're supposed to be involved in strategy, which should be three, five, 10-year plans. And suddenly COVID-19 comes around. Did your strategy get blown out of the water? And you're having to rethink a lot of things? Pamela Gavin (11:39): So as Chief strategy officer at NORD, I have the privilege to work with members of the community, our board of directors, our scientific and medical advisory committee, our other committees, as well as our staff, other stakeholders that we collaborate with to as you said, look to the future to ensure that we are planning for and evolving and changing to meet the needs of the community in the future from the patient and caregiver perspective. Clearly COVID-19 has had a dramatic impact on the entire community and, and the organizations that operate in this space, including NORD. We are still looking out for the future, but we certainly had to deliver a lot of immediate resources to address the needs of the community, where we could, where it made sense for us to do so around education resources, to help people financially get over hurdles that were completely unexpected. I certainly don't have to tell you that this is a virus of epic proportions, not really anything we could prepare for. Pamela Gavin (12:52): And so we had to stop some of our earlier work in the beginning of 2020 on strategic planning and divert some of those resources to rapid response programs for our member organizations to help raise money to seed money. So they could stay afloat webinars where we brought clinical scientific experts, psychologists, psychiatrists, geneticists, telemedicine, experts, you name it, a variety of people into our community. Through webinars. We did some surveys early on to try to understand what people were thinking of, where they were being impacted. And then some of our existing programs where we help patients logistically get to clinical trials. Some of those were impacted early on. So we definitely put a pause on some of our and immediate quarter, one quarter, two work to ensure that we could pivot quickly to meet the needs of the community because they were hurting. Janet Kennedy (13:53): Based on what we've all seen in social media, it seems as though there are people who really don't understand the terms, high-risk immunocompromised, people who are at risk in regard to reacting to COVID and the persistence of some people to not understand the importance of wearing a mask and implications for this particular population is really rather astounding to me. Has NORD been challenged to help educate the folks who are not really aware of how large this community is and how serious the implications are of not wearing a mask during COVID? Pamela Gavin (14:35): We certainly, you know, work to promote the guidelines that have been put out by the government and promoting, for example, the CDC guidelines and wearing a mask. And we have been promoting our member organizations who have doing that, especially those diseases for which patients are immunocompromised, but it's been a challenge not uniformly across the country, but it's definitely been a challenge nor has had staff working remotely before. COVID-19 clearly all our staff are working remotely. Now we have offices in the Connecticut area, New York area. So we were early on involved in the first epicenter. So we saw the impact and participated in adopting the guidelines. So we were able to promote those best practices by virtue of the fact that they were supported by the federal government, as well as the local state governments, where we actually had offices. So we saw it firsthand. We have an office in the Boston area, not too far from where you probably heard about the Biogen conference. Pamela Gavin (15:47): And so we were very sensitive to what was happening early on in the country around COVID-19 and some of the early epicenters. So I think we were tuned to it from the very beginning and we've promoted the use of masks. You know, we have staff with rare conditions. We have loved ones with that are immunocompromised, some of which are rare and some of which are not rare. So we promote from within and from personal knowledge and experience, but it's concerning to the entire community that this is not something that is necessarily has been adopted across the country as early on, as we would have liked. Janet Kennedy (16:25): Let's pivot for a second. I'd like to talk to you about NORDs involvement in clinical trials. Do you actually have any kind of funding for programs or are you more about facilitating the conversations that could start clinical trial discovery? Pamela Gavin (16:42): Much of our work tends to be early on in that we raise money for researchers who are looking for seed grants to get them from one phase to the other potentially, or get some of their work over a hump. Our seed grants are about $50,000. So not nearly enough to operate large, expensive clinical trials. We also provide resources and tools and support to help patient organizations who want to establish natural history studies. And those may lead to clinical trials or may help inform the design of clinical trials. So we're very much involved in the peripheral activities or the early supporting activities that may lead to clinical trials. But with clinical trials themselves often conducted by industry stakeholders. We will work with them and CROs to help operationalize trials by working on the logistics side. So to help reduce the burden to patients and families, we will help them with their travel arrangements. We will help them with their costs associated with their participation in the child. And we'll also work to help promote trials and inform people of trials to help with trial recruitment. We're doing a lot in that ecosystem if you will, but we don't conduct the clinical trials ourselves. Janet Kennedy (18:09): I would imagine it'd be very difficult for you to pick one rare disease to focus on. Pamela Gavin (18:16): Yes, they care about them all. That would be a hard decision that's like saying I love one child more than the other, picking a favorite child. Janet Kennedy (18:30): Within your organization do you have the opportunity for patients to actually talk to each other? Do you have a platform for them to engage anonymously or otherwise with people of a similar disorder? Pamela Gavin (18:43): We did have a platform called rare connect that we worked with our colleagues in Europe. Over time, that platform, it was hard for us to participate and continue to support it financially and the different languages and folks tended to gravitate to the more commercial robust platforms like Facebook. So a lot of the direct communication amongst patients are with platforms like that. We'll connect people in other ways to augment that we have meant that by bringing people together around perhaps their educational needs, where we have a patient and caregiver annual forum, and this year it's virtual, it's called living rare living stronger forum, where we bring patients and caregivers, family members together, and they could be a various backgrounds, different diseases represented. Pamela Gavin (19:41): Some of them may be strong policy advocates. Some of them may be, have different areas of interest, but we bring them together. We also bring together clinicians that are providing educational opportunities. They bring their experiences and really trying to provide resources so that people can feel stronger, more empowered to live a better life, and have more successful opportunities to engage in the community, share their experiences, develop friendships, relationships with people. And we were doing this by bringing people physically together and we rotate around the country a different place each year. So we were supposed to be in Cleveland this past year, this past may and said, we're going to do it virtually in a couple of weeks in July. And we're going to go back to Cleveland next year. And we end our event with an annual award ceremony where we, and this has been part of NORD's history since I can't remember over 25 years, where we bring researchers, academics, patients, patient advocates, organizations, members of industry, government, federal, state policymakers, together to celebrate the successes of the previous year. So we will do it virtually this year. And we'll go back to Cleveland and the Rock and Roll Hall of Fame next year. Janet Kennedy (21:12): Oh, well, rock on. That's all I can say. Well, Pam, I can't tell you how important I think your work is and how proud I am of the whole organization. The amount of services and activities, information that you all have is really quite astounding. And let me ask you this. If somebody wanted to be more engaged, whether they are an individual person or they represent an organization, is there a place for them at NORD? Pamela Gavin (21:43): Absolutely. At both of those levels, there is, there's always a place there's so much opportunity, so much to do so much positive energy and opportunity to bridge gaps that exist in the community. And we'll find something for everyone to get involved in. So they shouldn't hesitate to reach out to us. Our website has forums where people can submit questions, comments, requests, or they could call our main number, which will route you to the right department program or project within the NORD. Janet Kennedy (22:15): That's excellent. All right. We'll be sure to have the links to the website and your social media properties in the show notes. So I encourage everybody to please check that out so you can find the links to both Pamela as well as all the organization social media platforms, Pam, I thank you so much for being here for "People Always, Patients Sometimes." It was a great pleasure to talk with you. Pamela Gavin (22:40): It's been my pleasure. Thank you, Janet. Safe and healthy. Connect with Pamela and NORD LinkedIn Website Twitter NORDpod (NORD's own podcast hosted by Matthew Zachary Facebook Instagram

In 1983, Congress passed the Orphan Drug Act which incentivized the development of treatments for rare diseases. Since passing, the legislation has helped to create hundreds of new treatments for rare diseases... but it may have also had some side effects. According to Dr. Peter Bach, a pulmonologist and intensive care physician at Memorial Sloan Kettering Cancer Center, the push towards finding cures for rare diseases has been so strong that drug companies are paying little attention to more common illnesses, including some of the leading causes of death in the United States, like heart disease, cancer, and diabetes.

Ira Pastor, ideaXme longevity and aging ambassador and founder of Bioquark, interviews Dr. David Fajgenbaum, Assistant Professor of Medicine in the Division of Translational Medicine & Human Genetics at University of Pennsylvania, Associate Director, Patient Impact of the Penn Orphan Disease Center, Founding Director of the Castleman Disease Center, and Co-Founder & Executive Director of the Castleman Disease Collaborative Network (CDCN). Ira Pastor Comments: A rare disease is defined as any disease that affects a small percentage of the population. In the United States, the Rare Diseases Act of 2002 defines rare disease strictly according to prevalence, specifically "any disease or condition that affects fewer than 200,000 people in the United States", or about 1 in 1,500 people. This definition is essentially the same as that of the Orphan Drug Act of 1983, a federal law that was written to encourage research into rare diseases and possible cures. In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan, or about 1 in 2,500 people. The European Commission on Public Health defines it meaning fewer than 1 in 2,000 people, but contains some cut-offs for diseases that are not chronically debilitating or inadequately treated. The US organization Global Genes has estimated that more than 300 million people worldwide are living with one of the approximately 7,000 diseases they define as "rare" per the United States definition. Chronic genetic diseases are commonly classified as rare, and among the 7000 conditions mentioned above, rare diseases may result from bacterial or viral infections, allergies, chromosome disorders, degenerative and proliferative causes, and may effect any body organ. Dr. David Fajgenbaum Today's guest, who I'm honored to have join us to further delve into this topic, not just from the perspective of a clinician and investigator, but also as a patient himself, is Dr. David Fajgenbaum, MD. Dr. Fajgenbaum earned his MD from the Perelman School of Medicine at the University of Pennsylvania, and is an Assistant Professor of Medicine in the Division of Translational Medicine & Human Genetics at Penn, Associate Director, Patient Impact of the Penn Orphan Disease Center, Founding Director of the Castleman Disease Center at Penn, and Co-Founder & Executive Director of the Castleman Disease Collaborative Network (CDCN). Dr. Fajgenbaum leads the Castleman Research Program at Penn as principal investigator of 18 translational research studies, including an international natural history study and the first-ever NIH funded research of idiopathic multicentric Castleman disease (iMCD). His published research has changed the way iMCD is researched and treated and as a patient himself, is in his longest remission ever thanks to a precision treatment that he identified. He is also the author of Chasing My Cure: A Doctor's Race to Turn Hope Into Action Dr. Fajgenbaum co-founded the Castleman Disease Collaborative Network in 2012 to accelerate research and treatments for Castleman disease through a 'Collaborative Network Approach,' a business-inspired approach to biomedical research, which has become a blueprint for advancing rare disease research. Dr. Fajgenbaum is also the Co-Founder of the National Students of AMF Support Network (Actively Moving Forward), a non-profit organization dedicated to supporting grieving college students due to a loved one's diagnosis or death (or their own diagnosis) of a terminal illness. He co-authored "We Get It: Voices of Grieving College Students and Young Adults," a book for grieving college students and those who wish to support them. Dr. Fajgenbaum's work has been highlighted by the New York Times, Science, Today Show, Reader's Digest, Forbes Magazine, Perelman School of Medicine, World Affairs Council of Philadelphia, College of Physicians of Philadelphia, Everylife Foundation, Global Genes, and the University of Colorado. Dr. Fajgenbaum also received his MBA from Penn, at The Wharton School, where he was awarded the Joseph Wharton Award, Core Value Leadership Award, Kissick Scholarship, Wharton Business Plan Competition Social Impact Prize, Eilers Health Care Management Award, Mandel Fellowship, and Commencement Speaker., where he was a 21st Century Gamble Scholar. On this show we will hear about: Dr. Fajgebaum's background and life before his diagnosis with Castleman's disease, and his interest in becoming a doctor. The events surrounding his diagnosis with Castleman's disease in 2010. His discovery in 2012 of his own personal therapeutic regimen that helped put him into remission. His development of the Castleman Disease Collaborative Network. A discussion of drug "re-purposing" in the area of rare diseases. Finally, a discussion of his future research visions in the rare disease space.   visit www.radioideaxme.com for all of the links. ideaXme is a global podcast, creator series and mentor programme. Mission Move the human story forward!™ ideaXme Ltd.

We begin this episode with a discussion of two recent clinical trials in lymphoma: CALGB 50303 and REMoDL-B, respectively published in the Journal of Clinical Oncology and The Lancet Oncology. We include a primer on the history of lymphoma and the development of R-CHOP. We follow that with an in-depth interview with Dr. Ameet Sarpatwari of the Harvard Medical School on Risk Evaluation and Mitigation Strategies (REMS), the Orphan Drug Act, and, broadly, the purpose of the US FDA. CALGB 50303: doi.org/10.1200/JCO.18.01994 REMoDL-B: doi.org/10.1016/S1470-2045(18)30935-5 Dr. Sarpatwari's online course: https://www.edx.org/course/the-fda-and-prescription-drugs-current-controversies-in-context Back us on Patreon! www.patreon.com/plenarysession

https://accadandkoka.com/wp-content/uploads/2019/02/Chandra-pic-e1549144948426.jpeg ()Amitabh Chandra, PhD Some drugs cost more than diamonds though their health benefit seems marginal.  Others cost less than a package of M&Ms though they are demonstrably life-saving.  Some drug prices have spiked to 3-fold, 4-fold, or even 10-fold from what they were just a few years ago.  Drug pricing seems to be an irrational and incomprehensible aspect of our economy. To help us gain clarity on the matter, we have as our guest Amitabh Chandra, the Malcolm Wiener Professor of Public Policy at the Harvard Kennedy School of Government and the Henry and Allison McCance Professor of Business Administration and the Harvard Business School.  He is among the best decorated healthcare economists alive, having received the highest awards in the field.  He is an elected member of the National Academy of Medicine, a member of the Congressional Budget Office panel of health advisors, and a research associate at the National Bureau of Economic Research. Professor Chandra has published seminal papers in the most highly ranked journals of economics and medicine.  His research has been featured in The New York Times, The Washington Post, CNN, Newsweek, and on National Public Radio and has testified to the US Senate on matters of healthcare policy. GUEST: Amitabh Chandra, PhD. https://twitter.com/amitabhchandra2 (Twitter) and professional pages at https://www.hbs.edu/faculty/Pages/profile.aspx?facId=868461 (HBS) and https://www.hks.harvard.edu/faculty/amitabh-chandra (HKS). LINKS: Bagley N, Chandra A, Garthwaite C, and Stern A.  https://catalyst.nejm.org/time-reform-orphan-drug-act/ (It’s Time to Reform the Orphan Drug Act.) (NEJM Catalyst, Dec 19, 2018) Chandra A and Gartwaith C.  https://www.nejm.org/doi/full/10.1056/NEJMp1705035 (The Economics of Indication-Based Pricing). (NEJM Perspective, 2017) WATCH ON YOUTUBE: This episode is not available on YouTube Support this podcast

In this week's episode, KGI President Sheldon Schuster chatted with Peter Saltonstall, the President and CEO of the National Organization for Rare Disorders – also known as NORD. Peter joined NORD in 2008 after having served for more than 30 years as a senior executive in both for-profit and not-for-profit healthcare environments. Under his leadership, NORD has maintained the integrity of the Orphan Drug Act while forging new relationships between the patient community and the Executive branch, Congress, HHS, FDA, NIH, Social Security Administration and CMS; as well as with drug and device companies, and with the medical, academic, and investment communities. His efforts to build collaborations stems from his view that advances for the rare disease patient can be achieved best through joint efforts. Today he continues to be one of the country's lead voices on rare disease issues to industry, FDA, Congress, and the Government.

Orphan drugs, therapeutics that target small patient populations, have become an enticing area for Big Pharma as companies are attracted to the premium prices, lower development costs, and faster path to market. A new EvaluatePharma report on the Orphan Drug market forecasts a compounded annual sales growth rate of more than 11 percent through 2022 with orphan drug accounting more than 20 percent of total worldwide prescription sales by then. We spoke to Jon Gardner, U.S. editor for EP Vantage, about the growth of orphan drugs, what's driving the sector, and whether the way the industry is taking advantage of the Orphan Drug Act will cause policymakers to push back.

As pharmaceutical companies, spurred by the Orphan Drug Act, have delved deeper into the development of drugs to treat rare diseases, they have forged closer ties with patient advocates. While these relationships are driven by mutual interest, tensions sometimes arise because of divergent needs. We spoke to Heather Gartman, regional managing director of InVentiv Health, about the firms recent white paper “The New Partnership Paradigm.” Gartman discussed how patient advocates can more effectively influence pharmaceutical companies, some of the mistake pharmaceutical companies make, and how the growing sophistication of patient advocates is changing the balance of power in these relationships.

The Launch of NORD Founder, Abbey Meyers' memoir "Orphan Drugs: A Global Crusade"Mark speaks with Abbey Meyers, founder and past President of the National Organization for Rare Disorders (NORD), about the launch of her new book Orphan Drugs: A Global Crusade. Ms. Meyers opens up about the struggles she had in getting a proper diagnosis for her son, who was eventually diagnosed with Tourette’s syndrome, followed by additional struggles to get treatment option that was safe and effective. The talks about her unwavering efforts that ultimately supported the enactment of the groundbreaking “Orphan Drug Act of 1983”Among her many accomplishments, Ms. Meyers served as the consumer representative on the National Commission on Orphan Diseases (1986-89), the NIH Human Gene Therapy Subcommittee, the NIH Recombinant DNA Advisory Committee (RAC), the FDA Biological Response Modifiers Committee, and the HHS National Human Research Protections Advisory Committee. She was also an Honorary President of the European Organization for Rare Disorders (EURORDIS) and currently hold the honorary title of President Emeritus of NORD.Ms. Meyers asserts to Mark, "One day, I wish everyone dealing with the pain and misery of the 7,000 rare disorders will have access to a treatment that will alleviate their symptoms.”

The Launch of NORD Founder, Abbey Meyers' memoir "Orphan Drugs: A Global Crusade" Mark speaks with Abbey Meyers, founder and past President of the National Organization for Rare Disorders (NORD), about the launch of her new book Orphan Drugs: A Global Crusade. Ms. Meyers opens up about the struggles she had in getting a proper diagnosis for her son, who was eventually diagnosed with Tourette’s syndrome, followed by additional struggles to get treatment option that was safe and effective. The talks about her unwavering efforts that ultimately supported the enactment of the groundbreaking “Orphan Drug Act of 1983” Among her many accomplishments, Ms. Meyers served as the consumer representative on the National Commission on Orphan Diseases (1986-89), the NIH Human Gene Therapy Subcommittee, the NIH Recombinant DNA Advisory Committee (RAC), the FDA Biological Response Modifiers Committee, and the HHS National Human Research Protections Advisory Committee. She was also an Honorary President of the European Organization for Rare Disorders (EURORDIS) and currently hold the honorary title of President Emeritus of NORD. Ms. Meyers asserts to Mark, "One day, I wish everyone dealing with the pain and misery of the 7,000 rare disorders will have access to a treatment that will alleviate their symptoms.”

The Launch of NORD Founder, Abbey Meyers' memoir "Orphan Drugs: A Global Crusade" Mark speaks with Abbey Meyers, founder and past President of the National Organization for Rare Disorders (NORD), about the launch of her new book Orphan Drugs: A Global Crusade. Ms. Meyers opens up about the struggles she had in getting a proper diagnosis for her son, who was eventually diagnosed with Tourette’s syndrome, followed by additional struggles to get treatment option that was safe and effective. The talks about her unwavering efforts that ultimately supported the enactment of the groundbreaking “Orphan Drug Act of 1983” Among her many accomplishments, Ms. Meyers served as the consumer representative on the National Commission on Orphan Diseases (1986-89), the NIH Human Gene Therapy Subcommittee, the NIH Recombinant DNA Advisory Committee (RAC), the FDA Biological Response Modifiers Committee, and the HHS National Human Research Protections Advisory Committee. She was also an Honorary President of the European Organization for Rare Disorders (EURORDIS) and currently hold the honorary title of President Emeritus of NORD. Ms. Meyers asserts to Mark, "One day, I wish everyone dealing with the pain and misery of the 7,000 rare disorders will have access to a treatment that will alleviate their symptoms.”

The Launch of NORD Founder, Abbey Meyers' memoir "Orphan Drugs: A Global Crusade"Mark speaks with Abbey Meyers, founder and past President of the National Organization for Rare Disorders (NORD), about the launch of her new book Orphan Drugs: A Global Crusade. Ms. Meyers opens up about the struggles she had in getting a proper diagnosis for her son, who was eventually diagnosed with Tourette’s syndrome, followed by additional struggles to get treatment option that was safe and effective. The talks about her unwavering efforts that ultimately supported the enactment of the groundbreaking “Orphan Drug Act of 1983”Among her many accomplishments, Ms. Meyers served as the consumer representative on the National Commission on Orphan Diseases (1986-89), the NIH Human Gene Therapy Subcommittee, the NIH Recombinant DNA Advisory Committee (RAC), the FDA Biological Response Modifiers Committee, and the HHS National Human Research Protections Advisory Committee. She was also an Honorary President of the European Organization for Rare Disorders (EURORDIS) and currently hold the honorary title of President Emeritus of NORD.Ms. Meyers asserts to Mark, "One day, I wish everyone dealing with the pain and misery of the 7,000 rare disorders will have access to a treatment that will alleviate their symptoms.”

The Orphan Drug Act has provided critical incentives that have helped fuel the development of scores of drugs to treat rare diseases. But health experts at Johns Hopkins Medicine are calling for reform of the act to stop potential abuses by drugmakers they say have gotten huge subsidies and tax breaks for drugs that have been used far more broadly than the law intended to reward. We spoke to Martin Makary, professor of surgery at Johns Hopkins and a coauthor of a recent commentary in the American Journal of Clinical Oncology, about the Oprhan Drug Act, the unintended role it is playing in the growing controversy over drug prices, and why he thinks reform that protects the original intention of the act is needed.

The U.S. Food and Drug Administration approved more than 230 new drugs to treat rare diseases in the past decade and there are currently more than 450 orphan drugs in development, according to a new report (http://onphr.ma/1EsGJw6) from the Pharmaceutical Research and Manufacturers of America. Though developing drugs for rare diseases presents many challenges, policy changes and scientific breakthroughs have helped changed the landscape. We spoke to Gretta Stone, Deputy Vice President of Policy and Research at PhRMA, about the report, how the Orphan Drug Act encouraged investment in R&D for rare diseases, and what fuels her optimism for drug development efforts to combat this group of diseases in the years ahead. Also, we check in with Run4Rare's Noah Coughlan as he continues his 3,100 mile run across the country to raise awareness for rare diseases.

Host: Bruce Japsen Guest: Jeffrey Aronin, MBA Not all drug companies are in pursuit of billion-dollar blockbuster drugs taken by millions of Americans. In fact, there are drugs treating rare conditions that wouldn't be sold on the market if not for a landmark piece of legislation known as the Orphan Drug Act, now 25 years old. Jeffrey Aronin, president and CEO of Ovation Pharmaceuticals Inc. tells the Chicago Tribune's Bruce Japsen about the silver anniversary of the Orphan Drug Act, its benefits and its future potential.