Podcasts about pdufa

Poolbeg Pharma CEO Jeremy Skillington joined Steve Darling from Proactive to announce a significant milestone in the company's clinical development strategy: the U.S. Food and Drug Administration has granted Orphan Drug Designation to POLB 001, Poolbeg's innovative oral therapy aimed at preventing Cytokine Release Syndrome caused by T-cell engager bispecific antibody immunotherapies. POLB 001, an oral small molecule inhibitor of the IL-1 receptor, is designed to modulate the inflammatory response without suppressing the underlying anti-cancer immune activation. The FDA's Orphan Drug Designation recognizes the potential of POLB 001 to address a rare but serious medical condition, affecting fewer than 200,000 individuals annually in the United States. This designation confers a range of developmental and commercial incentives including seven years of U.S. market exclusivity post-approval, waiver of certain FDA fees, such as the Prescription Drug User Fee Act or PDUFA application fees and others. The company plans to initiate a Phase 2a clinical trial in the second half of 2025, marking the next step in evaluating POLB 001's efficacy and safety in a controlled setting. Poolbeg anticipates completing interim analysis in the first half of 2026, with topline data expected in the second half of 2026. With the Orphan Drug Designation in hand, Poolbeg is now well-positioned to advance discussions with potential partners and stakeholders as it progresses toward its clinical milestones. #proactiveinvestors #poolbegpharma #aim #polb #OrphanDrug #POLB001 #FDAApproval #CytokineReleaseSyndrome #CancerTherapy #BiotechNews #Immunotherapy #ClinicalTrials #PharmaInvesting

President Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump's warning of potential tariffs if companies don't reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing.    Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first nominee, Dave Weldon, days before his senate hearing was expected to begin. If confirmed, Monarez would be the first CDC director since 1953 to not have a medical degree; she holds a Ph.D. in microbiology and immunology from the University of Wisconsin.   In weight loss news, Novo Nordisk is paying China-based United Laboratories $200 million upfront to license a triple agonist of the GLP-1, GIP and glucagon receptors that could one day compete with Eli Lilly's retatrutide. And BioSpace examines the next great challenge for GLP-1s: oral formulation manufacturing.    Two more therapeutic spaces in focus last week are Duchenne muscular dystrophy and spinal muscular atrophy, where companies including Dyne Therapeutics, REGENXBIO and Novartis presented new data on their respective candidates. And the Duchenne community continued to react to news of the death of a patient taking Sarepta's approved gene therapy Elevidys.  In cardiovascular news, Alnylam won a much-anticipated approval for Amvuttra as the first RNAi silencer for transthyretin amyloid cardiomyopathy, setting up a three-way race with Pfizer's tafamidis—marketed as Vyndaqel and Vyndama—and BridgeBio's Attruby. Next up is Milestone Therapeutics' CARDAMYST in paroxysmal supraventricular tachycardia, which has a PDUFA date of March 27.   Finally, the saga of Cassava Sciences' Alzheimer's hopeful simufilam is over, as the company announced it has ended development of the controversial candidate.   

A patient with Duchenne muscular dystrophy taking Sarepta's gene therapy Elevidys has died of acute liver failure, possibly related to a recent viral infection. Sarepta, which said it will update Elevidys' label to reflect the new safety signal, saw its shares drop 22% on the news but analysts still seem positive on the drug, as treatment options for Duchenne remain limited.Meanwhile, both AstraZeneca and Taiho Pharmaceuticals announced acquisitions worth up to $1 billion or more in two sizzling therapeutic spaces, cell therapy and antibody-drug conjugates, respectively.Despite canceling a vaccine advisory committee late last month, the FDA on Thursday selected flu strains to be targeted in the upcoming 2025-2026 flu season. And at another federal agency, the Centers for Disease Control and Prevention, employees will have to wait a bit longer to see who will take the helm under Donald Trump, as the president's nominee, Dave Weldon, was pulled hours before he was set to appear before a Senate committee on Thursday. Like HHS Secretary Robert F. Kennedy Jr., Weldon has expressed anti-vaccine views in the past, particularly his continued suggestion of the link between vaccines and autism. Guggenheim Partners called the move to revoke Weldon's nomination “a positive sign for reigning in vaccine criticism.”In the weight loss arena, BioSpace takes deep dives into the tendency for biopharma to develop fast-followers, or me-too drugs—following a pattern seen with PD-1 checkpoint inhibitors after the approvals of Merck's Keytruda and Bristol Myers Squibb's Opdivo. One key difference between these two markets, however, is that when it comes to GLP-1s for weight loss, patients are not staying on these medicines. Drug developers are trying several approaches to improve treatment persistence, including titration, combinations and even secondary drugs that address side effects. They're also making other moves to differentiate themselves, including focusing on overall health outcomes—in areas like cardiovascular, sleep apnea and kidney disease.Following on BioSpace's coverage of the major patent cliffs that many Big Pharma companies are facing in coming years, we also take a look back at some of the companies that have already weathered such loss of exclusivity. It's rarely a straightforward story of sales crashing off patent, as companies take various tacks to extend their blockbuster sales.Finally, the cardiovascular space is expecting some movement this week. First, Alnylam is anticipating a decision on its RNAi silencer Amvuttra in ATTR-CM. An approval—which is widely expected—would make three companies on the market in this rapidly expanding space after Pfizer's tafamidis was approved in May of 2019, and BridgeBio's Attruby got the greenlight in November last year. And second, Milestone Pharmaceutical has a PDUFA coming up for etripamil in paroxysmal supraventricular tachycardia.

Nicholas Capman of The FDA Group welcomes Ashley Preston, SVP of Global Regulatory Affairs & Quality Assurance at BlossomHill Therapeutics, to discuss the critical elements of successful regulatory agency meetings. With over 20 years of experience in regulatory affairs and quality assurance, Ashley shares expertise on preparing for and executing effective interactions with FDA and EMA.While the FDA and EMA are both considered first-tier regulatory agencies, they handle meetings differently. FDA offers various meeting types with structured timelines, while EMA takes a more committee-based approach through the CHMP.FDA meetings include several key types, from pre-IND consultations to end-of-phase meetings. The end of phase two meeting, where sponsors agree on pivotal trial designs, and the pre-NDA meeting are considered required interactions. Recent initiatives like Project Optimus have made end-of-phase 1 meetings increasingly critical, particularly in oncology. While FDA meetings are covered by PDUFA fees, EMA scientific advice can cost 70,000-80,000 euros unless the product has orphan designation.Meeting preparation requires careful strategy and timing. Preston emphasizes the importance of having sufficient data to support proposed development plans, noting that companies sometimes seek meetings too early, leading to delays or unfavorable feedback. Teams must craft focused questions and present just enough data to make persuasive arguments without overwhelming reviewers.Effective negotiation during meetings requires understanding agency perspectives and finding a middle ground when disagreements arise. Preston advocates for a partnership approach, recognizing that regulators share the ultimate goal of bringing safe, effective medicines to patients. Teams should prepare for various scenarios and be ready to suggest alternative approaches that address agency concerns while maintaining development efficiency.Documentation has evolved in the post-COVID era, with the FDA often drafting minutes during meetings. This real-time approach allows sponsors to ensure critical decisions and agreements are accurately captured. Follow-up mechanisms, from informal clarifications to formal Type D meetings, provide opportunities to address any remaining questions or challenges that emerge during implementation.Preston recommends that companies prepare for their first agency meetings by viewing these interactions as opportunities to build relationships and understand agency expectations. While virtual meetings have become common and efficient, maintaining professional, science-based discussions remains crucial regardless of format. Companies working with both the FDA and the EMA should consider how to harmonize feedback, potentially using sequential meetings to incorporate initial FDA input into EMA discussions.———The FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our regulatory affairs expertise spans the entire drug development cycle, from pre-IND strategy through approval. For project or resource needs, visit ⁠thefdagroup.com⁠.

U.S. political maneuverings are keeping biopharma on its toes. Tuesday, Robert F. Kennedy, Jr. passed a major hurdle as the Senate Finance Committee voted to advance his nomination for HHS secretary to a floor vote of the entire Senate. If confirmed, RFK Jr. could have an impact on myriad regulatory issues, including PDUFA negotiations. In other political news, Donald Trump announced his promised tariffs on Canada, Mexico and China—the last of which could have particular implications for biopharma companies looking to China for promising new drug candidates. Both were subjects of conversation as leading pharma companies—including Pfizer, Merck and Regeneron—reported their Q4 and full-year 2024 earnings. Notably, Pfizer is eyeing deals between $10 billion and $15 billion, while Merck took a 12% stock hit on Gardasil challenges in China and Regeneron issued its first-ever dividends. On the regulatory front, the FDA greenlit Vertex's Jounavx last week as the first new drug for acute pain in more than two decades. Journavx leads a non-opioid pain space that is seeing significant momentum this year, with Tris Pharma, Algiax Pharamceuticals and SiteOne Therapeutics all announcing positive data in the past month. Meanwhile, in obesity news, the U.K.'s pharmacies regulator rolled out stricter guidelines for online pharmacies selling medicines including Novo Nordisk's Wegovy and Eli Lilly's Mounjaro, and an obesity-focused deal inked with Novo last year wasn't enough to keep Omega Therapeutics afloat as the Massachusetts-based biotech revealed it is heading toward bankruptcy. Finally, a new CEO will be taking the reins at Takeda in 2026—but the selection of Julie Kim is an exception in an industry still struggling for gender parity at the top leadership ranks.

Happy New Year! BioSpace released our NextGen: Class of 2025 this week, highlighting 25 biopharma startups to watch this year. The companies on this list are not afraid of a challenge, wading into some of biopharma's most competitive therapeutic spaces. As analysts predict an uptick in M&A in '25, could some of them be potential targets? One NextGen 2025 company that could already be fielding calls is Metsera, which reported promising Phase II data for its investigational subcutaneous GLP-1 therapy on Tuesday. The GLP-1 space continues to garner significant attention, with both Eli Lilly and Novo Nordisk seeking to protect their drugs against competition from compounders. This strategy makes a lot of sense as the sizzling class continues to expand into heart disease, sleep apnea and metabolic dysfunction-associated steatohepatitis, among other indications. On the regulatory side, the FDA approved 55 novel medicines in 2024—including a few inaugural company approvals—and is looking ahead to several key PDUFA dates in Q1, 2025. Meanwhile, the agency released three new guidance documents seeking to provide clarity around confirmatory trials supporting accelerated approvals, the use of AI in drug development and the use of tissue biopsies in clinical trials. Finally, BioSpace senior editor Annalee Armstrong heads to the J.P. Morgan Healthcare Conference next week as biopharma seeks an M&A spark to improve the currently a gloomy sentiment. Stay with us for all the biggest news coming out of JPM.

Last week, Gilead withdrew Trodelvy in bladder cancer after the antibody-drug conjugate failed to meet the primary endpoint in a confirmatory study. This follows Pfizer's recent withdrawal of another therapy that had earned FDA accelerated approval, Oxbryta for sickle cell disease. With few other options available to patients, BioSpace took a look at 5 sickle-cell candidates currently in clinical trials.   Following a disappointing Alzheimer's readout, the company's third neuro stumble in six months, Sage Therapeutics will lay off over 165 employees—about 33% of its workforce. The company is reporting Q3 earnings on Oct. 29.  On a more positive note, Vertex reported full Phase III data this week for its non-opioid pain treatment, su-zetri-gine. If approved, suzetrigine, which has a PDUFA date of Jan. 30, 2025, would be the first new class of acute pain medicine in more than two decades. And on Monday, a company that never leaves the news, Novo Nordisk, announced positive results from a cardiovascular study with its oral version of semaglutide, Rybelsus. In other Novo news, scrutiny around Novo Holdings' acquisition of Catalent is heating up with a coalition of unions, consumer groups and public interest organizations last week expressing their concerns about the buyout. This prompted BioSpace to unpack the unique structure of the collection of organizations that is Novo. Finally, Sanofi is having a busy month, securing the sale of its healthcare unit Opella and separately paying approximately $326 million to obtain a 16% stake in European radiopharma biotech Orano Med. Radiopharma is skyrocketing in popularity, and some companies are even trying to marry it with another hot therapeutic spaces: antibody-drug conjugates. Could radiolabeled ADCs overcome some of the side effects of radiation treatments, speed up treatment times and enable lower doses than traditional therapies?

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.On August 19, 2024, the FDA approved the first at-home syphilis test by NowDiagnostics, providing quick results with just a drop of blood. Dupont acquired Donatelle Plastics for $313 million, while Boston Scientific resubmitted its merger filing for Silk Road Medical. Edwards invested in Genesis Medtech and Biden pledged $150 million for medical technology to enhance cancer surgery. The biotech industry is active, with companies gearing up for the DSCSA deadline and utilizing multiple trial technologies. In the biopharma industry, Bristol Myers' schizophrenia drug is nearing approval, a partial hold was lifted on a Biontech-partnered ADC study, Novo Nordisk plans to build a new plant, and Medicare drug price cuts are impacting pharma profits. Patient-centric commercialization strategies are on the rise, along with concerns about Medicare price cuts affecting drug research and patent protection measures.Bristol Myers Squibb's KarXT faces a PDUFA date for potential approval as a new schizophrenia treatment. AbbVie's Emraclidine also shows promise as a gut-friendly option for schizophrenia patients. Market strategies beyond FDA approval are being explored to ensure successful drug launch and adoption in the competitive landscape.The FDA has cleared Biontech-Medilink's Phase I ADC cancer trial after a partial hold, while Liquidia missed full approval for a pulmonary hypertension drug. Bavarian Nordic is ramping up vaccine production in response to a global health emergency. Developers remain optimistic about psychedelic therapies despite setbacks like Lykos' rejection. Other updates include advancements in various therapeutic spaces and industry shifts such as layoffs.Texas hospitals secured a victory in a lawsuit on low-income payment formulas. Employer healthcare costs are expected to rise due to inflation and specialty drugs. Healthcare bankruptcies have slowed down but financial challenges persist. Payers are leveraging digital technology to improve outcomes and prepare for regulatory changes like the DSCSA deadline. Healthcare organizations are focusing on patient-centric care, combating burnout, and adapting to trends like telehealth and value-based care.In conclusion, the Pharma and Biotech industry is evolving rapidly with advancements in technology, regulatory changes, and shifting market dynamics shaping its future trajectory. Stay tuned for more updates on the latest developments in this dynamic sector.

Visit learnAMAstyle.com for free downloads in writing in medicine and the life sciences.    The FDA has approved blinatumomab (Blincyto) for treating adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase. This bispecific T-cell engager (BiTE®) therapy targets CD19 surface antigens on B cells, allowing T cells to recognize and eliminate malignant B cells. The approval was based on the Phase 3 E1910 trial showing improved overall survival with blinatumomab plus chemotherapy compared to chemotherapy alone.   The FDA has approved roflumilast cream 0.15% (Zorvye) for treating mild to moderate atopic dermatitis (AD) in patients aged 6 years and older. This steroid-free, once-daily topical treatment is a selective, highly potent phosphodiesterase 4 (PDE4) inhibitor designed for long-term disease control. The approval was based on phase 3 studies showing significant improvements in disease clearance, itch reduction, and overall skin condition compared to the vehicle group.   The FDA has approved a new 6mg single-dose prefilled syringe for faricimab-svoa (Vabysmo), facilitating easier administration for neovascular (wet) age-related macular degeneration (AMD), diabetic macular edema (DME), and macular edema following retinal vein occlusion (RVO). Faricimab-svoa is a VEGF and Ang-2 inhibitor, now available in a ready-to-use format. This approval provides an alternative to the existing single-dose vial and is granted to Genentech.   The FDA is set to decide on OX124, a high-dose naloxone nasal rescue medication designed to reverse opioid overdoses, with the PDUFA date set for July 15, 2024. Developed by Orexo, OX124 offers rapid absorption, high bioavailability, and enhanced stability, addressing the critical need for potent rescue medications due to the high rate of synthetic opioid overdoses. If approved, OX124's launch is anticipated later in 2024.   The FDA accepted an NDA for vonoprazan (Voquezna) tablets for treating heartburn associated with Non-Erosive gastroesophageal reflux disease (GERD) in adults, with the PDUFA date set for July 19, 2024. Vonoprazan is a potassium-competitive acid blocker (PCAB) offering an alternative to traditional proton pump inhibitors (PPIs). Non-Erosive GERD, affecting 38 million U.S. adults, is characterized by reflux-related symptoms without esophageal mucosal erosions, impacting quality of life with symptoms like heartburn and chest pain.  

Visit NascentMC.com for medical writing assistance. Visit learnAMAstyle.com for information on AI in Medical Writing and Editing - **mRNA Anti-EBV Cancer Vaccine**: The novel mRNA therapeutic cancer vaccine WGc-043 has been approved for clinical trials by the FDA, targeting Epstein-Barr virus (EBV) related cancers such as nasopharyngeal carcinoma and natural killer T-cell lymphoma. It stimulates the immune system to respond against EBV and associated malignancies, showing superior efficacy and safety in preliminary trials. A phase 1 clinical trial is currently focusing on patients who have failed second-line therapies, aiming to determine the optimal dose and evaluate safety and efficacy metrics. - **BTX-9341 for Breast Cancer**: The FDA has approved the investigational new drug application for BTX-9341, a novel cyclin-dependent kinase (CDK) 4/6 bifunctional degrader, intended for hormone receptor-positive, HER2-negative breast cancer resistant to existing CDK4/6 inhibitors. This drug targets and degrades CDK4/6 proteins, crucial for cancer cell cycle regulation, aiming to overcome resistance to current treatments. A phase 1 trial will assess its safety, biological activity, and efficacy both as monotherapy and combined with fulvestrant. - **Nivolumab Hyaluronidase Formulation**: The FDA has accepted a Biologics License Application for a subcutaneous formulation of nivolumab co-formulated with recombinant human hyaluronidase, enhancing convenience by reducing administration time compared to the intravenous version. This application includes all previously approved solid tumor indications for nivolumab, with a PDUFA date set for February 28, 2025. The subcutaneous version aims to provide faster and easier administration, supported by data from the Phase 3 CheckMate-67T study. - **ColoSense Colorectal Cancer Screening Test**: The FDA has approved ColoSense, a noninvasive colorectal cancer screening test using multi-target stool RNA for adults aged 45 and older at average risk. ColoSense, distinct from traditional FOBT tests, uses RNA biomarkers to detect CRC with high sensitivity and has shown promising results in clinical trials, detecting 93% of CRC cases and 45% of advanced adenomas in average-risk individuals. This new test offers a significant improvement in sensitivity and specificity over existing methods. For the complete shownotes visit nascentmc.com/podcast

·       Nascentmc.com for medical writing assistance for your company.Visit nascentmc.com/podcast for full show notes Cilta-cel for Myeloma: The FDA approved ciltacabtagene autoleucel (Carvykti; cilta-cel) for adults with relapsed or refractory multiple myeloma who have tried at least one prior therapy including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. This CAR T-cell therapy, initially approved in 2022, was confirmed effective in the phase 3 CARTITUDE-4 study, showing significant reduction in disease progression or death risk by 59% compared to standard care. Enhertu for HER2-positive Solid Tumors: Fam-trastuzumab deruxtecan-nxki (Enhertu) received FDA approval for treating unresectable or metastatic HER2-positive solid tumors in adults who have had previous systemic treatment and lack satisfactory alternative options. This therapy, a conjugate of an anti-HER2 antibody and a cytotoxic drug, was first approved in 2019 and targets HER2-expressing cancer cells to potentially minimize damage to normal tissues. Fanapt for Bipolar: Iloperidone (Fanapt) has been approved for the acute treatment of manic or mixed episodes in adults with bipolar I disorder. Previously approved for schizophrenia, iloperidone targets neurotransmitters like dopamine and serotonin. It demonstrated efficacy in a pivotal trial, showing significant improvement on the Young Mania Rating Scale. Zevtera for Multiple Bacterial Infections: Ceftobiprole medocaril sodium (Zevtera) was approved for treating adults with Staphylococcus aureus bloodstream infections, right-sided infective endocarditis, and acute bacterial skin and skin structure infections. Also approved for pediatric community-acquired bacterial pneumonia, ceftobiprole is a broad-spectrum cephalosporin that combats various bacteria including MRSA. TriClip for Tricuspid Regurgitation: The FDA approved the TriClip™ transcatheter edge-to-edge repair system for treating tricuspid regurgitation. This minimally invasive option clips the tricuspid valve leaflets to improve blood flow and prevent the need for surgery. The TRILUMINATE Pivotal trial showed significant improvements in TR severity and quality of life with a good safety profile. Revumenib for Acute Leukemia: The FDA granted priority review to revumenib (SNDX-5613) for treating adult and pediatric patients with relapsed or refractory acute leukemia with KMT2A rearrangements. As a new therapeutic agent, revumenib inhibits the menin-MLL protein interaction crucial in leukemic transformation. Early trial results show promising remission rates, with a PDUFA action date scheduled for September 26, 2024.  

Visit nascentmc.com/podcast for full show notes Visit learnamastyle.com for the free course on ChatGPT4 for medical writers and editors.  - The FDA has approved amivantamab (Rybrevant) in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test. This approval converts the May 2021 accelerated approval of amivantamab to full approval. - The FDA approved the AGENT Drug-Coated Balloon (DCB) for treating in-stent restenosis in coronary artery disease patients, introducing the first drug-coated coronary balloon in the US. It uses a paclitaxel-coated catheter to deliver medication directly to the vessel wall, offering an alternative to traditional treatments and aiming to reduce recurrence risks. The approval was based on the AGENT IDE trial, demonstrating its effectiveness over uncoated balloon angioplasty in reducing lesion failure, with no significant adverse events reported. - The FDA approved an oral suspension form of ibrutinib (Imbruvica) for several conditions, including chronic lymphocytic leukemia and chronic graft versus host disease, addressing the needs of patients who have difficulty swallowing pills. This is the first oral suspension formulation of a Bruton's tyrosine kinase inhibitor, originally approved in 2013 for mantle cell lymphoma. The approval, granted to Johnson & Johnson and Pharmacyclics, aims to simplify treatment for patients challenged with pill-swallowing. - Epcoritamab-bysp (Epkinly) received FDA approval for treating adult patients with relapsed or refractory follicular lymphoma after at least two prior therapies, marking it as the first subcutaneously administered bispecific antibody for this condition. It works by targeting both CD3 on T cells and CD20 on B cells to induce cell death. Based on the EPCORE NHL-1 trial results, this approval extends its use beyond diffuse large B-cell lymphoma, with AbbVie and Genmab sharing commercial responsibilities.  - The FDA granted priority review to the New Drug Application for govorestat (AT-007), a treatment for classic galactosemia, potentially the first of its kind if approved. Govorestat, an aldose reductase inhibitor, aims to reduce harmful galactitol levels, based on phase 3 study results among pediatric patients. Applied Therapeutics announced a PDUFA target action date of August 28, 2024, highlighting the urgent need for treatments in this area. - Obeticholic acid (Ocaliva) received FDA consideration for an expanded application to treat primary biliary cholangitis, building on its 2016 accelerated approval. It's designed for patients with or without cirrhosis, showing promise in post-marketing studies to confirm its clinical benefits. The review includes data from various studies and real-world evidence, with a PDUFA target date of October 15, 2024, aiming to address the needs of this autoimmune liver disease population.  - The FDA approved Biktarvy for HIV patients with suppressed viral loads who exhibit M184V/I resistance, offering a new treatment option for those with specific resistance mutations. Biktarvy combines three therapies in a single tablet, based on successful 48-week study data. Manufactured by Gilead Sciences, this approval expands treatment possibilities for patients facing resistance challenges. - The FDA rejected Minerva Neurosciences' New Drug Application for roluperidone as a treatment for schizophrenia's negative symptoms, citing insufficient evidence from a single study and lack of comprehensive data. Despite showing promise in targeting specific brain receptors, the FDA's concerns highlight the need for more extensive research and data to confirm its effectiveness and safety. Minerva plans to engage with the FDA to address these issues.

This conversation is part of a March, 2023 interview between co-hosts Louise Campbell and Roger Green and Jeff McIntyre of Global Liver Institute, one of the guests on the S5 E2 panel. This particular conversation focuses largely on Jeff's insights around the imminent anticipated drug approvals of obeticholic acid (which was withdrawn several months later) and resmetirom (whose PDUFA date is next month). Jeff's analysis includes mixed levels of enthusiasm around a draft report from ICER which includes pricing estimates for these drugs. The contention lies in the draft's reference to NASH as a non-progressive disease - a position which the panelists readily refute. Jeff's positive note is that the report carefully considers patient perspective and he was able to participate as an expert reviewer.The conversation shifts to speculations around the future standard for noninvasive testing and biomarkers. Jeff emphasizes the “need for a noninvasive that can be scaled with the least amount of burden to primary care providers and the patient populations that need them.” Roger Green points to the various guidelines' referral to FIB-4 as such a test. This summons quote of Quentin Anstee's repeated admonition that “we not let the perfect be the enemy of the good.” For whatever shortcomings of FIB-4, if it can be done now and it's two generations of successive approximation of what we've had before.As the session winds down, Louise Campbell asserts further comments around the ICER draft and NASH as a progressive disease. She recollects the podcast's recent conversation with Tim Jobson, founder of Predictive Health Intelligence, (S4 E5) which discusses developing a simple tool to identify patients at risk of liver disease by analyzing the results of blood tests given throughout their lives.

Leading patient advocates Jeff McIntyre (Global Liver Institute), Milan Mishkovikj (European Liver Patients Association), Tony Villiotti (NASH kNOWledge) and Wayne Eskridge (Fatty Liver Foundation) discuss their expectations for life after the resmetirom PDUFA data and, with it, the potential for the first MASH drug approval, with Louise Campbell and Roger Green. The conversation starts with Roger noting a funny coincidence; the March 14 PDUFA data for resmetirom is Pi Day, which means the first MASH drug approval might come on the math geek's favorite day of the year. He asks panelists to describe their moods as they look forward to the event. All five other panelists Advocates describe their "guarded optimism" regarding whether resmetirom will be approved. Wayne Eskridge also notes the potential for confusion and disappointment because not every patient who anticipates receiving the new drug will do so. Louise Campbell and Milan Mishkovikj describe how US approval might affect their respective countries and regions. Going last, Jeff McIntyre describes the extensive planning GLI is doing to prepare for a PDUFA decision that might raise as many questions as it answers, particularly around coding, reimbursement and definition of appropriate patients. When he has finished discussing the questions we might all face arouind the initial FDA ruling on resmetirom, he goes on to describe a possible approval path for countries outside the US.

00:00:00 Surf's Up: Season 5 Episode 2 Co-hosts Louise Campbell and Roger Green sit with four leading MASLD patient advocates (Tony Villiotti of NASH kNOWledge, Wayne Eskridge of the Fatty Liver Foundation, Jeff McIntyre of the Global Liver Institute, and  Milan Mishkevikj of the European Liver Patients Association) to discuss their plans and actions for MASH patients in 2024. The discussion covers the advocates' hopes and concerns for the year and the programs and efforts each considers top priority.  00:04:09 Meet Milan MishkovikjNorth Macedonian patient advocate  Mishkovikj, a key leader in the ELPA, discusses how he became involved in liver patient advocacy and shares one fact about himself that might surprise you.  00:11:25 Groundbreaker Panelists share one piece of good personal or professional news from the previous week. 00:15:52 Anticipating Pi DayEvery year, Pi Day is March 14. This year, that date has extra significance: the PDUFA Date for resmetirom approval at FDA. Advocates describe their "guarded optimism" regarding whether resmetirom will be approved. Wayne notes the potential for confusion and disappointment because not every patient who anticipates receiving the new drug will do so. Louise and Milan describe how US approval might affect their respective countries and regions. Jeff describes the extensive planning GLI is doing to prepare for a PDUFA decision that might raise as many questions as it answers, particularly around coding, reimbursement and definition of appropriate patients. He also describes a possible approval path for countries outside the US. 00:26:42 Post-Approval Planning As Jeff puts it, "We have to be planning for the second half while we're playing the first half on this," with essential questions about the implications of labeling on the topics he mentioned earlier. Roger asks what role patients might play in access decisions, to learn that the advocates have largely been "blocked out" of the process. GLI anticipates driving conversations with health systems around policies and access. The other US advocates do not anticipate doing so. Milan describes how ELPA will work with EMA, the European Parliament and other relevant groups. Louise adds private insurers and governmental payers to the critical post-launch lobbying and discussion targets.00:38:43 Where Does the Patient Fit? Roger asks where patients fit in the decision paradigms and how that might change over time. Louise shares her view that while patients might consume the medications, the real customer is either the prescriber or payer. Tony describes payers as "the last frontier" in learning how patients feel. Jeff returns to a range of "here and now" access issues GLI can address now. Milan and Roger discuss perspectives on the interplay of regulators, payers, providers and patients.00:49:13 Advocates' Key Programs Each advocate describes one key program they will run in 2024. Wayne describes FLF's State of NASH Care in America survey. Tony describes NASH kNOWledge's children's education programs. Milan describes continuing pilot programs ELPA started in 2023 to education the European Parliament. Jeff discusses GLI's Advanced Advocacy Academy, which "grew roots" worldwide in 2023. At different points earlier in the discussion, each advocate commended the support they get from GLI.00:56:30 Closing QuestionRoger asks what will tell each advocate whether they've been successful when they look back on 2024, 12 months from the recording date. Answers are powerful and vary by organization. 01:02:55 Business Report News on audience metrics, the upcoming Question of the Week, next week's nomenclature discussion and this week's Vault conversation.

This conversation between Scott Friedman, Laurent Castera, Louise Campbell and Roger Green covers the four panelists' general impressions of TLM2023, both the good and bad of it. Scott starts with a simple statement: “It was good to be at an AASLD that felt like an AASLD. Specifically, Scott is talking about the energy and excitement associated with the meeting. He also approves of Boston as a meeting location. Scott's meeting worst was the meeting app. The group elaborates on why they all agree. Laurent Castera agrees with Scott: bad app; excellent meeting with a great deal of colleague interaction. As a virtual attendee, Louise felt her own unique pain with the app. In addition, only 20% of sessions were live, and many were Zoom, which meant ‘Zoom in the US.' She describes this as the most disappointing meeting experience, and she notes that some people paid $1,200 for it. I make two other points: the patient tracks were broad in coverage and highly well-attended, and Saturday night's Diwali celebration. Scott, Laurent and I agree that the meeting was genuinely crowded and hot…but worthwhile. My final comment: tremendous excitement and anticipation about March 14, resmetirom's PDUFA date at the FDA.  

Drs. M. Ali Khan, Ajay Kuriyan, and Sarah Read join host Jay Sridhar to discuss recent developments in intravitreal injectable drugs, including 8-mg aflibercept not initially approved by the FDA, the upcoming PDUFA date for on-label bevacizumab, and approaching injections for dry age-related macular degeneration (AMD). For all episodes or to claim CME credit for selected episodes, visit www.aao.org/podcasts.

On this week's episode of Biotech Hangout, guest Matthew Gline (CEO of Roivant) joins hosts Brad Loncar, Tim Opler and Brian Skorney to discuss Biogen's heavy news week, including their acquisition of Reata for $6.5 billion, their decision to cut 1,000 jobs by 2025 and their quiet stance regarding their upcoming PDUFA date to treat PPD and MDD in partnership with Sage. The hosts also cover other layoff and leadership changes, including the resignation of FibroGen's CEO and layoffs from Infinity and Mersana. The group also touches on the $2.8 billion hypertension deal between Roche and Alnylam and the impact this deal could have on patients. Additionally, they discuss Gilead being accused of slow-walking the development of a new version of an HIV therapy to extend their patent protection, British billionaire Joe Lewis being charged with insider trading and the impact that poor data readouts had on Stoke Therapeutics', Gilead's and Kodiak's shares. *This episode aired on July 28, 2023*

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episodes at nascentmc.com/podcast Here is information on the latest US FDA approvals, the week of July 24 –  July 28, 2023. Just to summarize up front, we have RiVive receiving other the counter approval for opioid overdose; Balfaxar for urgent reversal of warfarin (VKA) therapy; Xdemvy for a common eyelid condition, Demodex blepharitis. PDUFA dates this week: Remestemcel-L for acute GVHD and zuranolone for major depressive disorder and postpartum depression.  Here are the highlights: ·       RiVive (naloxone hydrochloride nasal spray) has been approved by the FDA for over-the-counter use to treat known or suspected opioid overdose, rapidly reversing opioid effects and restoring normal respiration. This approval marks the second nonprescription naloxone product, the other being Narcan. ·       Balfaxar® (prothrombin complex concentrate, human-lans) has received FDA approval for urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy in adult patients requiring urgent surgery or invasive procedures. Balfaxar® replenishes deficient clotting factors caused by warfarin therapy and was supported by the LEX-209 clinical trial, demonstrating hemostatic efficacy and non-inferiority to a comparator, Kcentra®. The medication includes a boxed warning for thromboembolic event risk. ·       XdemvyTM (lotilaner ophthalmic solution 0.25%) has been approved by the FDA as the first treatment for Demodex blepharitis, targeting inflammation caused by Demodex mites. Administered as an eye drop, Xdemvy eradicates the mites over six weeks, providing relief for patients with eyelid inflammation and discomfort. The treatment was well-tolerated, with common adverse reactions being eye stinging and burning in about 10% of patients. ·       Upcoming PDUFAs Remestemcel-L, being developed for acute graft versus host disease (aGVHD), has a PDUFA date of August 2. Derived from mesenchymal stromal cells, it may treat this life-threatening complication of bone marrow transplants. Currently, there are no approved treatments for steroid-refractory aGVHD in children under 12, making Remestemcel-L a potential solution. Zuranolone (SAGE-217) has a PDUFA date of August 5 for major depressive disorder and postpartum depression. This potential treatment, a positive allosteric modulator of GABA-A receptors, aims to rebalance dysregulated neuronal networks to help reset brain function in people with depression.  Intro and outro music Garden Of Love by Pk jazz Collective  

If FDA approves Sarepta's gene therapy for Duchenne muscular dystrophy, patients will soon face the difficult choice between taking this treatment or waiting for a better option among the “unprecedented” amount of innovation ahead in DMD, as taking both may not be an option, BioCentury Executive Director Lauren Martz said on the latest BioCentury This Week podcast. Martz and colleagues assess the landscape for DMD gene therapies ahead of the May 29 PDUFA date for delandistrogene moxeparvovec.Also on the show, BioCentury Washington Editor Steve Usdin discusses takeaways from his conversation with FDA's Peter Marks; three tasks for Monica Bertagnolli to prioritize at NIH; and what the debt ceiling means for the life sciences. And on the heels of Bio€quity Europe, Editor in Chief Simone Fishburn delivers her insights from BioCentury's European conference.This week's podcast is sponsored by Cancer Research Horizons.

The outcome of the recent midterm elections in the USA surprised many by returning a Democrat-led Senate, preventing Republicans from gaining control of the next Congress.Analysts, having expected a stronger showing for Republicans, put some of the blame on former president Donald Trump, whose endorsements and active involvement in campaigning were said to frighten off moderates.In this week's episode of The Pharma Letter Podcast, we are joined by Stephanie Kennan, senior VP for federal public affairs at McGuireWoods Consulting, for a discussion on the possible implications.We'll consider the likely course of future life sciences legislation, in particular the additional provisions which were omitted from the pared back, so-called “skinny” user fee reauthorization in September.We'll also take a quick look ahead to 2024. As Florida's Ron DeSantis basks in the triumph of his gubernatorial campaign, a run at the presidency now looks inevitable, and bookmakers are putting him above Mr Trump as favorite to win power - albeit with a long way to go until polling day.We'll discuss what a DeSantis White House might look like for the life sciences industry.

On the heels of Congress' reauthorization of the Prescription Drug User Fee Authorization (PDUFA) Act, BridgeBio's Chief Regulatory Affairs Officer, Adora Ndu, explains the Commitment Letter, the programs that may be rolled out under PDUFA VII, why right-sizing CBER will help the cell and gene sector going forward as well as how sponsor companies can prepare for 2023 from a regulatory perspective.

Stellar data for Vertex Pharmaceuticals and CRISPR Therapeutics could set a high bar for the next generation of gene editing players to beat in β thalassemia and sickle cell disease, indications that had previously been considered as the best disease to show proof-of-concept for the modality, said Executive Director of Biopharma Intelligence Lauren Martz on the latest edition of BioCentury This Week. The BioCentury podcast team also reviews Associate Editor Stephen Hansen's analysis of how the trend of private equity firms acquiring or partnering life sciences VCs will impact the financing environment, and Washington Editor Steve Usdin's take on the one big difference between the PDUFA user fee bills making their way through the House and Senate. Senior Editor and Head of Discovery & Preclinical Development Karen Tkach Tuzman also reviews the latest highlights from BioCentury's Distillery

ACTION ALERT! Let's not try to fix what isn't broken. In this podcast, Betsy and Ramona Billingslea of Betsy's Health Foods discuss the latest threat to our supplements, S. 4090 and Senator Durbin's rider added to the 400-page Prescription Drug User Fee Act (PDUFA) that must be reauthorized this September 2022. We discuss the particulars of these legislative actions, why Dietary Supplement Health Education Act (DSHEA) works and doesn't need fixed, and the grass roots effort in the early 1990s that helped bring DSHEA into existence. DSHEA defines supplements as food, which is why we can sell them and why we are always adding the disclaimer that supplements don't cure, prevent or treat any illness. DSHEA provides the FDA with the oversight to monitor supplement manufacturing, approve certain structure/function and limited health claims, and more. The proof of DSHEA's efficacy is in action. Supplements have by far the lowest Adverse Event Reporting. Our AERs are less than a percent of all AERs reported (including reports from actual drugs). Your own faith in the supplements products you take, as well as your experience in their efficacy also prove that DSHEA effectively monitors the supplement industry. Though S. 4090 and the policy added to PDUFA may seem harmless or even sound like a decent idea, they actually make way for the FDA to overreach, deciding that your supplements are drugs just because a drug company may have investigated the possibility of using the supplement as a drug. . . 40 years ago. We hope you find this podcast an informative introduction into the current controversy concerning supplements. We want the power of choice to be where it belongs, in your hands. We want you to know that coming together, we can defeat this bad policy. Keep posted through our email and on our website. Our home page includes a section titled ACTION ALERT--SAVE OUR SUPPLEMENTS! We're working to keep that section as up-to-date as possible so you have the tools you need to act. Thank you for your support all these years. We thank you in advance for joining us in this fight to yet again save our supplements. Sincerely, Betsy and Ramona

Straight from Benzinga newsdesk, hosts Brent Slava and Steve Krause bring you the market news and stocks to watch.Steve and Brent focus on:DATS - other metaverse stocks - FB RBLX MTTR WIMI VRAR IMMR KOPN BHATAKBA - what is a PDUFA date? Prescription Drug User Fee ActCRTD - other NFT stocks - NKE DKNG CSCW PLBY WWE HOFV DLPN TKAT TAOP FNKO how do we find these correlations (ie metaverse, NFT stocks)?Today's 5 Stock Ideas:AMC Entertainment (AMC) - A play on further investment in meme stocks. A Monday morning article from Reuters highlighted commentary from AMC CEO Adam Aron "I'd like to think there will be more third-party external M&A announcements going forward where AMC can reach for the stars and intriguing investments that have potentially attractive returns." Shares of AMC's recent investment, Hycroft Mining (HYMC), traded up more than 30% Monday morning on the comment.lululmeon (LULU) - Will report Q4 earnings Tuesday after market close.DatChat (DATS) - A play on the metaverse. DatChat shares traded up more than 30% Monday morning following news from the company it will launch a metaverse advertising and non-fungible token monetization platform.Akebia Therapeutics (AKBA) - A play on an upcoming FDA date. An FDA panel will rule on Tuesday on the company's treatment candidate for anemia related to chronic kidney disease.Creatd (CRTD) - A play on non-fungible tokens. The company launched the beta version of its NFT gallery Friday, named "OG Gallery."Hosts:Steve Krause Reach out to Steve at stevekrause@benzinga.comSr. Reporter Benzinga NewsdeskBrent Slava Reach out to Brent at brent@benzinga.comSr. Reporter, Head of Benzinga Newsdeskpro.benzinga.comFree 2-week trial, no credit card requiredUse coupon code YOUTUBE20 to get 20% offDisclaimer: All of the information, material, and/or content contained in this program is for informational purposes only. Investing in stocks, options, and futures is risky and not suitable for all investors. Please consult your own independent financial adviser before making any investment decisionsAdvertising Inquiries: https://redcircle.com/brandsPrivacy & Opt-Out: https://redcircle.com/privacy

As Rare Disease Month comes to a close, we take a look at a major piece of legislation that impacts the FDA, and consequently, patients with rare diseases. With the Prescription Drug User Fee Act (PDUFA) up for renewal in Congress, much is at stake for the rare disease community. This act expedites the drug reviewal process for new treatments, including those for patients with rare and unmet medical needs. Without it, drug reviews would slow down dramatically. And speaking of the FDA, the agency finally has a new Commissioner, Dr. Robert Califf. How will he shape the agency? To give listeners an inside look, Terry and Bob speak with past FDA Associate Commissioner Peter Pitts, who explains the importance of PDUFA and what Dr. Califf will bring to the patient community. And to conclude rare disease month, Kate Pecora speaks with patient advocate Yolanda Bermudez. Hear her story as she discusses how her rare disease changed her cancer journey. Hosts: Terry Wilcox, Executive Director, Patients RisingDr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public InterestKate Pecora, Field CorrespondentGuests: Peter Pitts, Co-founder and President of the Center for Medicine in the Public Interest Yolanda Bermudez, Patient Advocate Annika Wojtowicz, Patient Correspondent Jearlean Taylor, Patient Correspondent Links:Prescription Drug User Fee Amendments | FDAPDUFA Renewal and the FDA: What Do Patients Need to Know?Twitter | PatientsRiseNow Health Subcommittee Hearing on PDUFARobert M. Califf M.D | FDA CommissionerPeter Pitts | CMPIThe Affordable Insulin Now Act Creating a promising pathway for faster access to new drugs — and a Califf confirmationRaphael G. WarnockNeed help?The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.orgHave a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent?Drop us a line: podcast@patientsrising.orgThe views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising.

Congress is set to start its once-every-five-years review of the law that authorizes user fees to finance the hiring of personnel to speed the FDA review of drugs. The periodic renewals of “PDUFA” also give lawmakers a chance to make other changes to the agency at the hub of the pandemic.Meanwhile, the FDA could also find itself at the center of the abortion debate and a controversial new medication to treat Alzheimer's disease.Anna Edney of Bloomberg News, Joanne Kenen of the Johns Hopkins Bloomberg School of Public Health and Politico, and Sarah Karlin-Smith of the Pink Sheet join KHN's Julie Rovner to discuss these issues and more.Plus, for extra credit, the panelists recommend their favorite health policy stories of the week they think you should read, too.Julie Rovner: KHN's “Ready for Another Pandemic Malady? It's Called ‘Decision Fatigue,'” by Jenny Gold.Joanne Kenen: The New Yorker's “What Happened After the Chicken-Pox Vaccine,” by Jessica Winter.Sarah Karlin-Smith: Stat's “Despite Biden's Big Promises and a Far Better Understanding of the Virus, Covid-19 Is Still Raging Through the Nation's Prisons,” by Nicholas Florko.Anna Edney: Reuters' “Special Report: Inside J&J's Secret Plan to Cap Litigation Payouts to Cancer Victims,” by Mike Spector and Dan Levine.Click here for a transcript of the episode. See acast.com/privacy for privacy and opt-out information.

Weathering the bear market. Oncology chief's change in thinking on PD-1 therapies. Plus BioCentury's 2021 innovative start-ups.

If you find this podcast useful please give it a rating and review on iTunes by clicking here Joe Wiley of Amryt Pharma discusses their strong operational and financial results in Q3 Joe Wiley CEO of Amryt Pharma #AMYT discusses their strong Q3, which saw 14.6% YoY revenue growth to $56.5M & the 7th consecutive quarter of positive EBITDA generation. Q3 2021 Highlights - Chiasma, Inc. acquisition completed on August 5, 2021 and integration proceeding well - Oleogel-S101 target PDUFA date set by the FDA for November 30, 2021 - Oleogel-S10 MAA accepted by EMA and CHMP opinion expected in Q4 2021 - Commercial launch plans well advanced for Oleogel-S10 launch, if approved - FY 2021 revenue guidance increased in September to $220M - $225M, representing 20-23% growth over 2020 - 14.6% YoY revenue growth in Q3 2021 to $56.5M (Q3 2020: $49.3M) - Excluding the impact of sporadic LATAM ordering and Mycapssa®, revenues grew 26.3% YoY and 5.7% QoQ - 21.5% increase in metreleptin revenues YoY to $36.3M in Q3 2021 (Q3 2020: $29.9M). Excluding the impact of sporadic LATAM ordering, metreleptin revenue growth was 51.6% YoY. - US accounted for 51.7% of global metreleptin revenues and EMEA accounted for 43.0% in Q3 2021 - EMEA metreleptin revenues grew 143.2% YoY in Q3 - Mycapssa® delivered $1.45M in Q3 (from August 5 when Chiasma was acquired) - Mycapssa® revenues in Q3 were impacted by pre-ordering in Q2; normalised ordering patterns have returned in September with Mycapssa® delivering $1.14M in the month - The integration of Chiasma is significantly advanced and we continue to extract cost synergies - $21.4M operating loss before finance expense for Q3 2021 (Q3 2020: $3.6M operating loss). Excluding non-cash items, share based compensation expenses and Chiasma restructuring and acquisition costs, this resulted in EBITDA3 - Cash of $123.2M at September 30, 2021 (June 30, 2021: $142.9M) post repayment of outstanding Chiasma revenue interest financing debt and transaction related costs in the period. About Amryt Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets. Amryt's commercial business comprises three orphan disease products – metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); and lomitapide (Juxtapid®/ Lojuxta®). Myalept®/Myalepta® (metreleptin) is approved in the US (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name Myalepta®) as an adjunct to diet for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. For additional information, please follow this link. Mycapssa® (oral octreotide) is approved in the US for long-term maintenance therapy in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only oral somatostatin analogapproved by the FDA. Mycapssa® has also been submitted to the EMA for regulatory approval. For additional information, please follow this link. Juxtapid®/Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Colombia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel and Brazil (under the trade name Lojuxta®). For additional information, please follow this link. Amryt's lead development candidate, Oleogel-S10 (Filsuvez®) is a potential treatment for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez® has been selected as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB but has been submitted to the FDA for approval and in June 2021, Amryt received confirmation from the FDA that its NDA for Oleogel-S10 had been accepted and granted priority review. The FDA also set a target PDUFA date of November 30, 2021. In Europe, a MAA for Oleogel-S10 was accepted for assessment by the EMA in March 2021. Amryt's pre-clinical gene therapy candidate, AP103, offers a potential treatment for patients with Dystrophic EB, and the polymer-based delivery platform has the potential to be developed for the treatment of other genetic disorders. Amryt also intends to develop oral medications that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information on Amryt, including products, please visit www.amrytpharma.com.

Richard Hullihen, CEO of Polarean Imaging #POLX says he's happy with the company's progress just ahead of their FDA approval date on 5th October and describes their technology as unique. About Polarean The Company and its wholly owned subsidiary, Polarean, Inc. (together the "Group") are revenue-generating, investigational drug-device combination companies operating in the high-resolution medical imaging research space. The Group develops equipment that enables existing MRI systems to achieve an improved level of pulmonary function imaging and specialises in the use of hyperpolarised Xenon gas (129Xe) as an imaging agent to visualise ventilation. 129Xe gas is currently being studied for visualisation of gas exchange regionally in the smallest airways of the lungs, across the alveolar tissue barrier, and into the pulmonary bloodstream. In October 2020, the Group submitted a New Drug Application ("NDA") to the FDA for hyperpolarised 129Xe used to evaluate pulmonary function and to visualise the lung using MRI. In December 2020, the Group received confirmation of acceptance of its NDA by the FDA, with a target PDUFA action date of 5 October 2021. The Group operates in an area of significant unmet medical need and the Group's technology provides a novel investigational diagnostic approach, offering a non-invasive and radiation-free functional imaging platform. The annual burden of pulmonary disease in the US is estimated to be over US$150 billion.

Vadim Alexandre, Head of Healthcare at SP Angel discusses Polarean Imaging #POLX the chance of FDA approval & commercialisation opportunities. About Polarean The Company and its wholly owned subsidiary, Polarean, Inc. (together the "Group") are revenue-generating, investigational drug-device combination companies operating in the high-resolution medical imaging research space. The Group develops equipment that enables existing MRI systems to achieve an improved level of pulmonary function imaging and specialises in the use of hyperpolarised Xenon gas (129Xe) as an imaging agent to visualise ventilation. 129Xe gas is currently being studied for visualisation of gas exchange regionally in the smallest airways of the lungs, across the alveolar tissue barrier, and into the pulmonary bloodstream. In October 2020, the Group submitted a New Drug Application ("NDA") to the FDA for hyperpolarised 129Xe used to evaluate pulmonary function and to visualise the lung using MRI. In December 2020, the Group received confirmation of acceptance of its NDA by the FDA, with a target PDUFA action date of 5 October 2021. The Group operates in an area of significant unmet medical need and the Group's technology provides a novel investigational diagnostic approach, offering a non-invasive and radiation-free functional imaging platform. The annual burden of pulmonary disease in the US is estimated to be over US$150 billion.

Pink Sheet reporter explains the increased pressure on the FDA now that a coronavirus vaccine booster plan is in place, the surprising results of a survey of agency advisory committee members, and the recalculation of FY 2022 PDUFA fees.

Harry's guest this week is Jeff Elton, CEO of a Boston-based startup called Concert AI that's working to bring more "real-world data" and "real-world evidence" into the process of drug development. What's real-world data? It's everything about patients' health that's not included in the narrow outcomes measured by randomized, controlled clinical trials. By collecting, organizing, and analyzing it, Elton argues, pharmaceutical makers can it design better clinical trials, get drugs approved faster, and—after approval—learn who's really benefiting from a new medicine, and how. Concert AI, which has offices in Boston, Philadelphia, Memphis, New York, and Bangalore, specializes in providing “research-grade real-world data” and AI-based analytical services to companies developing cancer drugs. Before joining Concert AI, Elton was managing director of strategy and global lead of predictive health intelligence at Accenture, and before that he was a senior vice president of strategy and global chief operating officer at the Novartis Institutes of BioMedical Research. He's the co-author with Anne O'Riordan of a 2016 book from Wiley called Healthcare Disrupted: Next Generation Business Models and Strategies.Please rate and review MoneyBall Medicine on Apple Podcasts! Here's how to do that from an iPhone, iPad, or iPod touch:• Launch the “Podcasts” app on your device. If you can't find this app, swipe all the way to the left on your home screen until you're on the Search page. Tap the search field at the top and type in “Podcasts.” Apple's Podcasts app should show up in the search results.• Tap the Podcasts app icon, and after it opens, tap the Search field at the top, or the little magnifying glass icon in the lower right corner.• Type MoneyBall Medicine into the search field and press the Search button.• In the search results, click on the MoneyBall Medicine logo.• On the next page, scroll down until you see the Ratings & Reviews section. Below that, you'll see five purple stars.• Tap the stars to rate the show.• Scroll down a little farther. You'll see a purple link saying “Write a Review.”• On the next screen, you'll see the stars again. You can tap them to leave a rating if you haven't already.• In the Title field, type a summary for your review.• In the Review field, type your review.• When you're finished, click Send.• That's it, you're done. Thanks!Full TranscriptHarry Glorikian: I'm Harry Glorikian, and this is MoneyBall Medicine, the interview podcast where we meet researchers, entrepreneurs, and physicians who are using the power of data to improve patient health and make healthcare delivery more efficient. You can think of each episode as a new chapter in the never-ending audio version of my 2017 book, “MoneyBall Medicine: Thriving in the New Data-Driven Healthcare Market.” If you like the show, please do us a favor and leave a rating and review at Apple Podcasts.Harry Glorikian: In the world of drug development, there's a tendency to think that the only data that matter are the data that get collected from patients during randomized controlled clinical trials. That's the type of study that drug companies use as the gold standard to test the safety and effectiveness of new drugs and that the FDA uses to make drug approval decisions. But it's just not true. Way before clinical trials begin, there's a ton of genomic or proteomic or chemical data that can go into identifying new drug candidates, as we've learned from many of our previous guests on the show. And today my old friend Jeff Elton is here to tell us about another important kind of data that get collected before, during, and even after clinical trials that can have a huge impact on how drugs are used.It's called real-world data, and it basically means everything about a patient's health that isn't included in the narrow parameters and outcomes measured by clinical trials.Jeff is the CEO of a startup here in Boston called Concert AI that specializes in organizing and analyzing this real-world data. And his argument is that when you pay attention to real-world data, it can help you to design better clinical studies. It can help support the core clinical data that drug companies submit to the FDA when they're applying for approval. And after approval, it can help show who's really benefiting from a new medicine, and how. Jeff has been thinking about the importance of real-world data for a long time, at least since 2016, when he leading predictive health intelligence at Accenture and he published a book called Healthcare Disrupted. The book argued that real-world data from wearable devices, the Internet of Things, electronic medical record systems, and other sources could be combined with advanced analytics to change how and where healthcare is delivered. In our interview, I asked Jeff to explain how Concert AI is helping patients and how the predictions he made in the book are playing out today.Harry Glorikian: Hey, Jeff, welcome to the show. Jeff Elton: Thank you Harry. Pleasure to be here. Harry Glorikian: Yeah, it's been a long time since we've actually seen each other. I mean I feel like it was just yesterday. We were you know, interacting. Arshad was there and we were talking about all sorts of stuff. It's actually been quite a few years and, and, and you have now transitioned to a few different places and, and right now you're running something called Concert AI. And so, I mean, let's just start with what is Concert AI, for everybody who's listening. Jeff Elton: Yeah. So Concert AI is a real-world evidence company. We'll spend a little bit of time breaking that down. We are very focused on oncology, hematology, urological cancers. So we kind of tend to stay very much in that space.And within the real-world evidence area, we really focus on bringing together high credibility research grade data. This usually means clinical data. Genomic data can include medical images combined with technologies that aid gaining insights out of those particular data and that kind of align with our own various use cases.A use case could be designing a clinical study, it could be supporting a regulatory submission. It could be gaining insight, post-approval, about who's benefiting, who's not benefiting. And you know, our whole mission in life is accelerating needed new medicines and actually improving the effectiveness of current medicines out there.Harry Glorikian: So who's like, I don't know, the user, the beneficiary, in a sense, of this.Jeff Elton: So, you know, we like to think we have a very heavily clinical workforce. You know, we always put the patient first. So I'm actually gonna say that a lot of the reason why we're doing things is that we have the benefit to be stewards, combined with provider entities, of focusing on questions that matter for patient outcomes.So the first beneficiary is patients. I think the second beneficiary are biomedical innovators. We're trying to kind of support those innovations. We're trying to understand how to go into the clinic. We're trying to understand how to design those clinical trials to have them be more effective. We're trying to understand how to show that relative to the current standard of care, they offer a range of incremental therapeutic benefit. A lot of medicines become improved once they're actually already approved. And so we actually spend time doing a lot of post-approval research that actually begins to improve the outcomes by beginning to kind of refine the treatment approaches.And then the clinical communities we work very closely [with]. We're a very close working partner with American Society of Clinical Oncology and their canceling program. We're in a 10-year relationship with them that allows us to do work in truly high need areas. We did a COVID-19 registry jointly with ASCO that worked off of some of the data we brought together because it you know, COVID-19 uniquely hit cancer and particularly hematological malignancy patients.We do work with them in health disparities, making sure that racial, ethnic, and economic groups can be the beneficiaries of new medicines and are appropriately part of doing clinical trials, clinical studies. And then we work directly with provider communities who oftentimes are seeing the value of the work we're doing and making sure that for research purposes, we have appropriate access to data, information to conduct that research.Harry Glorikian: Yeah. I want to get into, you know, I think we're going to, I'm going to hit on some of that later, but I just want to make sure everybody's sort of on a level playing field with some of these wonky terms we use. How do you define real-world data and real-world evidence. I mean, I know what the FDA defines it as. I'm just curious. Jeff Elton: Yeah. So yeah. And FDA does have some, they have some publications really there that came out at the end of 2018 that actually began to lay out a framework around that, which I would encourage folks to reference. It's actually a very well-written document.So real-world data is sort of what it sounds like. It's the data. Right. And You know, if you were a clinician, if you were sitting in a clinical care environment, you probably wouldn't be using the word real-world data because those are the data generated through your treatment of the patient. So clinicians sometimes actually kind of pause for a moment to say, what's real-world? It's the things I'm doing. And in fact, you know, real-world data would be structured data in a structured field. It's a lab value that may have come in from the laboratory information system or a drop down menu. Did they smoke or not? Which can be a fixed field in an EMR. All the way over to physician notes, to appended molecular diagnostic reports, to imaging interpretation reports.So all those are forms of data. Now, evidence is a little bit about also what it would sound like. Data are not evidence. You have to actually, and in fact, to generate evidence, I want to have to trust the data. I have to believe those data are an accurate reflection of the source systems they came from. I have to believe they're representative or appropriate for the question that I'm actually trying to address. And then I have to make sure that the methodologies I'm using to analyze something, either comparing the effectiveness of two drugs relative to each other, actually then when I look at that analysis, I'm willing to either make a regulatory decision or a guideline modification.And the intent of evidence is either to support a regulatory decision or something that can inform practice of medicine or nature of treatment. So there's a bar, right, that one has to achieve to actually become evidence. But I think evidence is the right goal by what we're trying to do.Harry Glorikian: So you know, in the past, I mean, because I've, worked with companies like Evidation Health and so forth right there, some of this data was in paper form, right. Not in electronic form. So, what holes in the current system of, say, drug development would better real-world data or real world evidence help fill or, or drive forward.Jeff Elton: Yeah, that's a super good question. And, you know, Harry, you were kind of going back to your, I mean, you were one of the primary, leading individuals around that when the days of personalized and individualized and precision medicine, and even some of molecular medicine kind of came around. In fact, that's probably where you are my first point of interaction.And I come back to that concept because when you, when you're looking at data—and again, not all data are kind of created equal here—when I think about setting up and designing a clinical study, so now I'm with an experimental therapeutic or I'm thinking about moving it in. If it worked in one solid tumor and I suspect that same molecular pathway or kind of disease mechanism may be at work in another one. And so I want to kind of think about doing a pan tumor strategy or something of that nature. When I actually, when I, if I can bring together molecular diagnostic information, aspects of the individual patients, but do it at scale and understand the homogeneity, the heterogeneity and the different characteristics in there, I can design my trials differently and I can make my trials more precise. And the more precise the trials are, the higher the likelihood that I'm going to get meaningful outcomes. The outcomes here that are meaningful is what actually helps medicines progress. It's actually getting those questions to be as narrow and as precise and as declarative in their outcomes as possible.And so a lot of these data can actually be used to help guide that study design. Now, if I also have very rare cancers or very rare diseases—so this would apply even outside of oncology, although most of our work is oncology related—even if I'm outside of that, if I'm in very rare, oftentimes finding, you know, putting a patient on a  standard of care therapy as a control oftentimes may not be in the patient's best interests. And so this notion of either a single arm or having an external control or having a real-world evidence support package, as part of that, may be part of what can occur between the sponsor and actually the FDA, et cetera, for kind of moving that through.But, you know, this has to be done individually around the individual program and the program and the characteristics have to kind of merit that, but these are big deals. So we feel that these are forms of data that can complement what would have been traditional legacy approaches to give more confidence in the decisions being made in the evaluation, the ones actually coming, too.Harry Glorikian: Yeah, I can hardly wait. I mean, maybe it's a dream, but I can hardly wait until we get rid of first-line and second-line and we just say, okay, look, here's a battery of assays or whatever. This is what you should be taking. No more first line or second line. I mean, these are sort of in my mind, I mean, almost arcane concepts from, because we didn't have the tools in the past and now we're starting to move in that direction.Jeff Elton: Yeah. So, Harry, just to, maybe to build on that a little bit. So if you look at some of our publications and things that we presented at this last ASCO, there's work one can do when you look at different features of patient response, et cetera. We're a company, but we also have a very strong data science backbone to what we do. And AI and ML applications. There are features that sometimes you can predict metastatic status. You can predict rate of response. You can predict progression. Now the very fact that I can make that statement kind of indicates that as you started thinking about the paradigm in the future, particularly when I start doing it liquid tumor, biopsies and surveillance mechanisms where I can see response much more rapidly in less invasive ways, you are going to start even over the course of this next five years, I think some of these will start to start influencing practice patterns in some very positive ways for patients, Harry.Harry Glorikian: From your lips to his or her ears. It needs to move faster. But, but it's interesting, right? I feel like you've been on this path for quite some time, like, I want to say since you're at least since your book in 2016, if not before. Jeff Elton: Yeah. So, you know yeah, you and I, in fact, you and I interacted first, I think we were kind of in the hallways, first interaction of what had been the Necco candy factory on Massachusetts Avenue in the Novartis building, where I was working in the Novartis Institute for Biomedical Research at the time.And Even prior to that, I think I did my first work back in the days of Millennium Pharmaceutical when it was still a standalone company, doing work in precision medicine and personalized medicine all the way through. And obviously Novartis's strategy was looking at pathway biology and actually using that as the basis of actually understanding where in a pathway system one could actually target and actually understanding that it is a system, it's got redundancy both in a bad, in a positive way. How do we use it to progress new medicines? So there's been an aspect of this that's always been kind of a little bit hard. I think I kind of made a decision to kind of pivot much more to a large scale data-centric, insight-technology-centric approach, and actually at scale, bring some of that back to the biomedical innovators. But yeah, it's been a progression over time and some of this it's a field that I feel, you know, strong passion around and will stay committed to for the duration of whatever my professional career looks like.Harry Glorikian: So can you give us maybe an example? I mean, I know some of it may be confidential. How does the data that you're providing, say, improve maybe drug safety or effectiveness? Jeff Elton: So you know, we're doing a project right now that that's safety related and I'll kind of try to keep it such that it I'm not betraying anybody's confidence. Eventually this will be in a publication, but it's not at the point yet. We're looking at a subpopulation that had severe adverse events, cardiac adverse events in the population. And originally the hypothesis was, it was a relatively homogeneous group. And we brought together some of our deepest clinical data, which means we have many different features of intermediate measures of disease, recurrence, progression, response, adverse events, severe adverse events. And we also brought some of our data science and AI solutions to it. And one of the major insights that came out of that is actually it wasn't a single homogeneous group. One group was characterized by having a series of co-morbidities that then linked to this significant adverse event and the other were purely immunological based.And so therefore actually in both cases, they're screenable, they're predictable. They're surveillable. And monitorable. And so therefore, but the actions would be very different if you didn't know what the two groups are. So in this particular case, we could discriminate that now. Well, we'll take that into more classical biostatistical analysis and do some confirmatory work on that, but that has significant implications on how you're going to kind of screen a patient survey of patients, look for whether or not they exhibit that area, and how you would kind of handle it, manage that. That would improve the outcome significantly of that subpopulation.So that's one example. In other areas, some of our data was actually being used as part of a regulatory submission. It was a very, very rare population in lung cancer. And it was unclear exactly how nonresponsive they were to the full range of current standard of care. And we were actually illustrating that there was almost a complete non-response to all current medicines that were actually used against this particular molecular target because of a sub mutation. And that actually was part of the regulatory submission. And that program both actually got breakthrough designation status, and that actually supported that and actually got an approval ahead of the PDUFA date. So when you start pulling some of these pieces together, they work to again, provide more confidence and interpretation and more confidence in decision-making. And in this particular case, certainly accelerated medicines being available to patients. Harry Glorikian: Oh yeah. Yeah. Drive value for patients and drive value for the people that are using the, the capability to get the product through. So, you know, we're talking about data, data, data. At some point, you've got to turn this into a product or a service of some sort or, or some, or maybe a SaaS as, as, as you guys might look at it, but you've got something called, you know, Eureka Health, right, in your product lineup. Can you give us an idea of what that is? I think it's a cloud-based SaaS product. You call it research-ready real-world data. So I'm just curious how that works. Jeff Elton: Yeah. So we do think.. So if you think about what we're trying to do, we're trying to allow a level of scale and a level of precision and depth on demand in the hands of individual researchers, from translational scientists, folks in clinical development, post-approval medical value and access. Kind of in that domain. And so each of those have different use cases. Each of those have different kind of demands that they'll place on data and technology for kind of doing that.We're trying to move away from the world of bespokeness, because by nature of bespokeness, the question has its own orientation. The data is just unique to the question and that utility later is very low and, you know, in a way, what we'd rather do, what have we learned about what actually kind of create utility out of data, and let's make sure that we're covering the use cases of interest, but let's do it at very large scale. And that scale itself and the data we even represent at that very large scale is in itself representative and actually has significance whether it's on a prevalence basis of sub cohorts of disease or not. Now, the reason why I'm spending so much time developing that is when you put that in the hands of the right people, you're avoiding bias, but you're also giving utility at the same time and so you're actually improving their ability to conduct rapid question interrogation, but also structure really good research questions and have the discipline if I have a good research methods right around that. So we do structure those as products.And so, so actually one of the things we think of is, the work that we do in non-small cell lung cancer is an extremely large data set. It also has high depth on the molecular basis of non-small cell lung cancer. And it's created in a way that actually allows you to make those questions from translational through post-approval medical and doing that.Eureka is the technical environment. It is a cloud environment we are working in, and it actually allows you to do on-the-fly actually insights. So, outcome curves, which are called Kaplan-Meier and a few other measures. I can compare groups. I can compare cohorts. I can ask questions. It's actually exceptionally fast.And so this ability to navigate through a series of questions, its ability to make comparisons of alternative groups of patients on different classes of questions and finally get down to the patient cohort of interest that you may want to move into in the next phase, your research is done a lot faster. Now we took that, and now we're integrating more AI and ML into that. So we now have created probably what's one of the leading solutions for doing clinical study design. So we can optimize different features of that study design. We can actually release lab values. We can change parameters. There's a level of kind of fitness, ECOG scoring. We can actually modify that and show what the changes would be in the addressable patient population, and actually optimize that study design all the way down to the base activity level. And we're basically creating a digital object that's rooted on huge amounts of data. Underneath the 4.5 million records runs inside that particular area.There is no other solution in oncology, hematology that gets anywhere to that depth of information that can reflect, with different optimization, to the endpoint and even reflect statistical power. Now we're integrating in work around health disparities. How do you assure that if it's a disease like multiple myeloma, which may disproportionately affect black Americans, that I'm actually getting adequate representation of the groups that in fact, actually may be afflicted by the disease and actually assure the design of the study itself assures their representativeness actually in that work?Harry Glorikian: This dataset, what are some of the features of it? What is it? What sort of information does it have in it that you would be pulling from? Because my brain is like going on all sorts of levels that you would pull from, and some of it is incredibly messy.Jeff Elton: Yeah. So you are absolutely right. And so there have been expressions in the field of people who do work in real-world data that the real world's messy you know, fields may be empty. Do you know, as an empty field, because nothing got put there where's the empty field, because in that electronic medical record environment empty means it was not true of the state of the patient. That may sound like a nuanced thing, but sometimes empty actually is a value and sometimes empty is empty. And so you start getting into some things like that, which you start thinking about, like, those are pretty nuanced questions, but they all have to do with, if you don't know which it is, you don't know how to treat and move the data through.So back to your question here a little bit. What we actually, the sources of where we bring data from are portions of a clinical record. So, you know, we work under businesses, the work we do is either research- or quality-of-care-focused. And so, you know, we work actually, whether it's with the American Society of Clinical Oncology and et cetera, appropriately under all HIPAA guidelines and rules for how you interact with data around doing that. So I'll put that as a caveat because methods and how you do that security and everything else is super, super important. We have a clinical workforce. These are all credentialed people. Most of them have active clinical credentials. Most of them were in the clinic 10 to 15 years and even still interact on it. So a lot of my people feel they're still in clinical care. It's just happens to be a digital representation pf the individuals that are in there. And we're seeing, whether it's features of notes, depth of the molecular diagnostic information, radiologically acquired images that may show how the tumor progressed, regressed, et cetera, that's in there, any other, the medications, prior treatment history, comorbidities that may confound, actually, response. So all those different features are brought together, but if you don't bring it together consistently, we have tens of thousands of lines of business rules, concepts, and models that we try to publish around about how you bring a concept forward.So if you want to bring a concept forward, want to do it consistently, we come out of 10 different electronic medical record environments, and we're, we're actually interacting with the work of 1,100 medical oncologists and hematologists, et cetera. You have a lot of heterogeneity. Handle that heterogeneity with a clinical informatics team into a set of rules as it's coming forward so that everything comes to the point that you can have confidence in that, you know, in that particular analysis and that presentation.So there's something called abstraction, which is a term applied to unstructured data—and unstructured just means a machine can't read it on the fly. And so we're actually interacting with that, which could have a PDF document or something else. And from that, we use the business rules to then develop something that now is machine-readable, but actually has a definition behind it that one can trust, that one can, that kind of comes from some published basis about why did you create that variable? So I could measure outcomes of interest progression-free survival, adverse events, severe, whatever the feature of interests can. Help me answer the question we try to kind of bring through. So we're usually creating about 120 unique variables that never would have been  machine-readable, in addition to the hundred, that probably were machine-readable when we bring that together. Harry Glorikian: So you're using a rule-based AI system, maybe not just a straight natural language processing system, to parse the words.Jeff Elton: Yeah. So natural language processing gets a little tricky. We do. We have, actually, excellent natural language processing. We'll sometimes use that for pre-processing, but you have to be careful with natural language processing. If it has context sensitivity, and if you're parsing for sets of reliable terms, it can actually be relatively accurate. If I'm doing something like a laboratory report that's so discreet, so finite, and it's so finite with how many alternatives you have with the same concept, it works really well. When you start getting into things that are much more nuanced, you actually start to have a combination of technology with the expert humans to actually have confidence in the ultimate outcome.Now we do have some very sophisticated AI models. Like I'll give you an example. When you're looking at a medical record, usually metastatic status has just done a point of first but diagnosis in cancer care. So if the patient actually progressed and they made through there that they don't update the electronic medical record because they want to maintain what the starting point was when therapy was administered.But a biomedical researcher wants to know it at a point in time. So we have models that can literally read the record and bring back that status at any point in the time of disease progression. Now, would that work up to the grade of, say, for regulatory submission? No, but for a rapid analysis to pull back your question of interest and have it done in minutes, as opposed to weeks or months it works exceptionally well.Harry Glorikian: Understood. Understood. So now you and I both know that clinical trials, you know, are available only to a certain portion of the population really participate for  a whole bunch of reasons. And then if you go down to sort of, you know, equality or, or across, you know, the socioeconomic scale, it, it gets even, it gets pretty thin, right? You guys, I, I think you've been pushing around inequality and cancer care and you have this program called ERACE which I think stands for Engaging Research to Achieve Clinical Care Equality. So help me out here. What is that? Jeff Elton: So we are, as an organization we're super privileged to have a very, very diverse workforce. And you know, men, women all forms of background races, ethnicities, and we really value that. And we've tried very hard to build that in our scientific committee. And I think when the public discourse around kind of equity, diversity, inclusiveness came forward, and you know, as you know, Harry, this has been a unprecedented period of time for just about anything, any of us. I mean, COVID-19 and social issues. You know, things of that nature. It's, it's really been a very, very unprecedented time in terms of how we work and how we interact and the questions.Our organization and our scientists actually came forward to me and said, you know Jeff, we have a tremendous amount of data. We have partners like American Society of Clinical Oncology and some of the leading biopharmaceutical researchers in the world. And we've got technology, et cetera. We want relevance. We really want what to make contributions back and we believe that actually, we can do some research that no one else can do. And we can actually begin to deliver insights that no one has the capability to do. Would you kind of support us in doing that? And so we put together the ERACE program and it actually was named by a couple of our internal scientists.And the program actually now is being collaboratively done. We've done a couple of webinars, with you know, some of our partners and that's included, you know, folks from, whether it's AstraZeneca, Janssen, and BMS, et cetera. It's become something around, how can we rethink how research takes place and actually assure its representativeness for all groups, but particularly in specific diseases. It impacts different groups differently. And so can we make sure it reflects that? Would we be generating the evidence so that they can in fact be appropriate beneficiaries earlier? And a lot of this came from when we looked at aspects of diagnostic activity we could say that, you know, black American women have a higher incidence of triple negative breast cancer and a few other diseases. When we look at patterns of diagnosis and activity, unfortunately, the evidence that we even have is not substantially in the practice of what we're actually seeing sometimes when we begin reviewing our data. And so we began confederating through our own work. We now have actually set up research funding. So we actually now will fund researchers who come in the academic community. If they come up with research proposals that have to do with, you know, health related disparities, whether it's economically based, or if it's racial, ethnically based. Those questions. We've got an external review board on those proposals. We'll provide them data technology and financial support to get that research done. We're doing it with our own group and we're doing it collaboratively with our own kind of biopharma sponsor partners kind of as well. So for us right now, it's about confederating an ecosystem, it's about building it into the fabric about how research questions are framed, research is conducted, clinical trials are conducted, and then actually those insights put into clinical practice for the benefit of all those groups. And so, you know, it's even changing where we get our data from now. So it's, it's like an integral part of how of everything we do. Harry Glorikian: So you saw, I don't want to say an immediate benefit, fooking at it this way or bringing this on, but I mean, you must have seen within a short period of time, the benefit of, of, I don't want to say broadening the lens, but I can't think of a better way to frame it. Jeff Elton: We were surprised how quickly, whether it was academic groups or others, rallied around some of the concepts and the notions. And we were surprised how quickly we were able to make progress in some of our own research questions. And we were pleased and astonished, only in the best ways, that we saw industry and biomedical research, the whole biomedical community, attempting to integrate into their research and the questions that they asked actually different ways of approaching that.And in fact, it's probably one of the most heartening areas. You couldn't have legislated this as quickly as I believe leading industry biomedical innovators decided it was time to kind of change portions of the research model. And you made a, Harry, you made a statement earlier on that. It's not just about kind of us analyzing data. Sometimes bow you find that to broaden actual, say, clinical trial participation, I actually have to go to sites that historically didn't conduct clinical trials. I may need to have investigators that are trusted, because some of the populations we may want to interact with don't trust clinical research and have a long history about why they didn't trust clinical research.So you're changing a social paradigm. You're changing research locations and capacity and capability for that research. So we're now moving research capacity out into community settings in specific communities with this idea that we actually, we actually need to bring the infrastructure to the people and not assume again, that people want to kind of go to where the research historically was conducted because that wasn't working before, you know? Harry Glorikian: At some point, you turn the crank enough, you start to influence, you should be able to influence, you know, standard of care and all that stuff, because if you're missing data in different places, you've got to make sure that we fill these holes. Otherwise we're never going to be able to diagnose and then treat appropriately.Jeff Elton: Generate the evidence that supports actually doing that and do it on an accelerated basis, but also that it gets confidence for those decisions. Absolutely. That's part of our goal. Harry Glorikian: Yeah. So I want to jump back in time here and sort of go back to your your Healthcare Disrupted book. You know, I feel like, you know, we're on the same page because I think the message was, you know, pharma, devices, diagnostics, healthcare, they need to rethink their business model to respond to this digital transformation, you know, which is obviously something in my own heart. I've been sort of banging that drum for quite some time.In particular, you argued in the book that real-world data from EMRs, wearables, the Internet of Things could be combined to change how and where healthcare is delivered. Is there a way in which like Concert AI's mission reflects the message of your book? Can I make that leap?Jeff Elton: I appreciate the way you asked the question and I think if you said our principles and perspectives about that, we need to kind of focus on value and outcomes, and then we're going to be bringing insights, digital cloud, and a variety of other tools to underpin how we work and operate. Absolutely.And in fact, I think, you know, positively. I had a lot of engagement and did a lot of interviews, even as we were putting the book together, which took place over a couple of months ago, it was probably, you've done your own books. Whatever you think it's going to be, it's a lot longer. So I'll leave it at that. I have recovered from the process now, but I think we had a lot of engagement, whether it was with medical community, biopharma, leadership, community, et cetera. And I think that alignment is some of the alignment we have with our partners today. It's actually around some of the same principles.What I couldn't have predicted, in fact, I was a couple of years ago and this probably would have been towards the tail end of 2019, I was already starting to think about, okay, I've recovered from the first writing. How did I do? And what would I say now? And at the time I was beginning to say certain things seem to be taking shape slightly more slowly than I originally forecast, but then COVID-19 happened. And all of a sudden certain things that we kind of had thought about and kind of had put there actually accelerated. And in fact, I think, you know, out of adversity, you'd like to say we bring sources of strength we didn't know we would kind of be beneficiaries of. But out of that, you could argue this concept of say a decentralized trial activity.So we have, let me pick up, you know, I'm one company, but let me pick a parallel company that I have respect for, say, Medable as an example, and Michelle [Longmire] leads that company, it does a very nice job, but that's the idea. Everything could be done remotely. I can actually do a device cloud around the individual. I can do a data collection and run RCT-grade trial activity. Now that doesn't work super well in oncology, hematology, et cetera, where I'm, you know, I'm doing chemo infusion and I have to do very close surveillance, but that concept is an accelerated version and got broader adoption and actually was part of some of the COVID-19 kind of clinical studies and capability. And it's not going to revert back. So actually what happens is you find it has a level of efficiency, a level of effectiveness and a level of inclusiveness that wasn't available before, when it had to do facilities-based only. Now we ourselves now we're asked to accelerate, we bring technologies and integrate them into provider settings for doing retrospective analysis. But actually during that period, not only did we bring our clinical study design tools and use AI and ML for doing that, which led to, we've supported the restart of many oncology studies now, and actually the redesign of studies to be able to move into different settings that they never were in before.And actually now we're beginning to use some of our same approaches for running prospective studies, but from clinically only derived data sources. It's a very different paradigm about how you conduct clinical research. So when you think about this, there are unpredictable shocks, you know, which, you know, some of may have called Black Swan events or whatever you may ascribe to it, that actually are now consistent with everything we did. But actually accelerating it and in a weird way back on trajectory, if you will. But I think, yes, everything we're doing was informed by a lot of that seminal work and research and foundation about what worked in health system and didn't how are people being beneficiaries or not? How do we need to change how we do discovery translational clinical development? And we're very committed to doing that. Harry Glorikian: Yeah. I mean, it's interesting cause you almost answer my next two questions. I'm really hoping it doesn't slide backwards. That's one of my biggest fears is, you know, people like to revert back to what they were used to.Jeff Elton: But you know, maybe to encourage you and me. So one of the things, if you take a, let's take a look at a teleconsult. So during COVID-19, HHS opened up and allowed as a coded event, doing a digital teleconsult for kind of digital medicine, telemedicine, and that was put into place on an emergency basis by HHS. And then before the outgoing HHS had that, it's now made permanent. And it's now part of the code that actually will continue to actually be a reimbursable event for clinicians. That was actually super important during COVID-19. What's not that well known is, not only did that allow people to be seen, but hospital systems were really financially distressed because most of their work was informed by kind of, you know, elective procedures and things of that nature. And that couldn't take place. But the teleconsult became a very important part of their even having economic viability, which you can't underestimate the importance of that during a pandemic. Right. So now that's part of how we're going to work. My personal view is, now that people are using digitally screening tools, they have decentralized trials, some of the solutions that we're putting into place, AI-based, bringing RWE as part of a regulatory submission, I don't see anything going back. And the work we're doing is if we can start putting 30 to 50% time and cost improvements and add more evidence around a decision, more robustly than we did before, that's not going backwards at all.Harry Glorikian: Good. That's that makes me. I'm hoping that we're all right, because we've been saying this and beating this drum for quite some time.It's interesting, right? Because I don't think I've gotten over the whole writing thing because I've got a new book coming out in the fall. So you know, I, I couldn't help myself. I hope, you know, we. We're able to give the listeners sort of a view of where this whole world is changing, how data's changing it.I mean, I've had the pleasure of talking to people about digital twins and that sort of data. And I believe that this, we're gonna be able to make predictions, as you say off this data almost proactively. It's interesting because I do talk to some people who are in the field that look at me strange when I say that, but after working with different forms of data in different places for so long, I can see how you can look at things predictively and sort of, you know, decide what's, you know, see what's going to happen almost before it happens for the most part, if you have a big enough data set. Jeff Elton: So we do a lot of prediction thing in the AI and ML world. And we predict, you can actually be relatively accurate on who's going to adhere and not adhere. You can begin to look at the biological response to being placed on a new therapy and understand whether that response is kind of in a direction that, that patient's going to remain on that therapy, or you need to discontinue to be placed on a new therapy.And you're right. And in fact, some of these features…well, the question, we use it from generating insights to design and hopefully improve outcomes, et cetera. That's a rapid process. I mean, I've seen things in the last three years in setting up Concert AI that would have taken me a decade to have seen in previous methods. But we're still not as fast and as effective as we can be.And the very fact that I can in my digital laboratory, if you will, create AI/ML to predict whether that patient is going to be discontinued or continue on to that course of therapy. Some of that needs to be brought into confidence tools that can start to inform parts of practice as well. They're not ready for that. They have to ascend to that. But when you look at these, some of these, whether it's coming in as software, as a medical device, sets and solutions to augment, are going to add a huge, huge amount of utility. And you're finding a lot of interest, even biomedical innovators are looking for predictive tools, too, complement their medicines.And you know, we're doing a couple of things that would be definitely considered in a more confidential area around doing that right now. And I have to tell you I've been so pleased and it's just for me, it's so, so catalyzing of our energy to be brought into this, to see people willing to reshape the paradigm about how they do things that actually will reshape how medicine's delivered and care provided too. Harry Glorikian: Oh yeah. I mean, look, ideally, right, I think every physician wants to give the patient the optimal therapy. Not pick the wrong one and have to redo it again. But, but I think a lot of these tools are also gonna lend themselves to adjudication.Jeff Elton: Absolutely. Harry Glorikian: Right? And that is a huge paradigm shift for everybody to wrap their head around. And I think we're going to get pushback from some people, but I can't see how you don't end up there at some point. You can see where it's going. You know, what's going to work, here's the drug. And if it doesn't work, here's the data to show [why] it didn't work.Jeff Elton: Well, and actually and Harry, to your point, right now you're thinking about how payers authorized the treatment that's proposed by our clinician for super expensive medicines. Right? But if I'm an oncology, I can tell you right now that claims data as a single data source can't tell you much about whether that patient responds, whether they're being treated according to NCCN ASCO guidelines or not. So you're wondering what's the basis of that. Whereas I can actually look at the data and I can understand how that patient presents and I can see what's actually the intended treatment. And you can immediately say that perfectly makes sense, given how everything's matched up and I can continue to kind of say what that response is it consistent with what I would have hoped for placed in that patient on that specific treatment. So to your point, this is going to change all sorts of things.Harry Glorikian: I love it when it changes on that level, it just makes me all happy inside. So, Jeff, it was great catching up with you. I hope when this pandemic is open, we can get together in person and you know, have a beer. Maybe we'll even bring Arshad because I think he's been working in this whole data area with a number of companies for a while now. Jeff Elton: Yeah. Would love it.Harry Glorikian: Excellent. Jeff Elton: All right. Harry Glorikian: Thank you.Jeff Elton: Thank you too.Harry Glorikian: That's it for this week's show. You can find past episodes of MoneyBall Medicine at my website, glorikian.com, under the tab “Podcast.” And you can follow me on Twitter at hglorikian.  Thanks for listening, and we'll be back soon with our next interview. 

Dr. Jennifer Gudeman, Vice President, Medical and Clinical Affairs at Avadel Pharmaceuticals discusses new clinical data from the pivotal phase 3 REST-ON clinical trial that was presented at the SLEEP 2021 Annual Meeting. The investigational therapy, FT218, for the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy is currently under review at the FDA with a PDUFA target date of October 15, 2021. She also talks about the current treatment landscape and the unmet need among patients for a differentiated treatment option. #AvadelPharmaceuticals #SLEEP2021 Dr. Jennifer Gudeman was appointed Vice President, Medical and Clinical Affairs in December 2020. In 2017, Dr. Gudeman received the Healthcare Businesswomen's “Rising Star” award. During her career, she has led or contributed to six commercial product launches and three clinical development programs, as well as led interactions with medical societies and patient advocacy organizations to help ensure that commercial medications fulfill their clinically proven therapeutic benefits to patients and providers. Dr Gudeman has published numerous peer-reviewed papers and provided domestic and international presentations on partnership with industry for drug development in high-risk pregnancies. Prior to joining Avadel, Dr. Gudeman was Vice President, Medical Affairs at AMAG Pharmaceuticals, overseeing a team of medical science liaisons and scientific communications. Prior to her time at AMAG Pharmaceuticals, she was Director of Medical Affairs at Lumara Health. Additionally, she began her industry career at Mallinckrodt Pharmaceuticals. Dr. Gudeman graduated summa cum laude with a bachelor's degree in pharmacy and magna cum laude with a doctorate in pharmacy from St. Louis College of Pharmacy in 1999 and 2000, respectively.

Michael Weiss explains TG Therapeutics cancer drugs with expected PDUFA dates. He says they have a multiple sclerosis drug with a target approval for mid-year 2022. He says their R&D expense has come down and they have $600 million in cash as of the end of the year.

Listen NowWith the election of Donald Trump Congressional Republicans are poised to repeal the Affordable Care Act (ACA) within the first 100 days of the 115th Congress via the budget reconciliation process.  (At some future date they are pledging to draft ACA replacement language or legislation.)  The expectation is repeal would have an effective date of 2018 or 2019, or after the mid-term Congressional elections.  However, many if not most health policy experts agree the simple act of repeal would cause such instability state insurance marketplaces would collapse long before repeal would, legally, go into effect.  Congressional Republican leadership is also promising to move major Medicare, via premium support and major Medicaid legislation, via block grants or per capita caps, though the upcoming Congress.       During this 23 minute conversation Chris Jennings discusses whether, and moreover how if at all, Congressional Republicans can repeal the ACA with an out-year effective date without fatally damaging state insurance marketplaces, the likely consequences of a simple repeal (without replace), when and how will Republicans will replace the ACA and whether that effort would garner any Democratic interest or support, what substantively explains Republican opposition to the current law and what might Republican leadership do to reform the Medicaid program and its likely effects.Chris Jennings is currently Founder and President of Jennings Policy Strategies, a DC-based policy firm where he consults with foundations, purchasers and other aligned stakeholders on policies to ensure higher quality, more affordable health care for all Americans.  Previously, Mr. Jennings served as Deputy Assistant for Health Policy to President Obama and for eight years as White House Health Care Adviser to President Clinton.   Prior still he served for a decade in the US Senate for Senators Glenn, Pryor and Melcher where he worked on Medicaid CHIP, HIPAA, PDUFA, major Medicare reforms in the 1997 Balanced Budget Act and related work concerning long term care, prescription drug coverage, rural healthcare and other related issues.  Chris has been a campaign adviser to six Democratic presidential campaigns and is a frequent contributor on health reform issues to the New England Journal of Medicine and numerous other scholarly journals, periodicals and newspapers.    This is a public episode. If you would like to discuss this with other subscribers or get access to bonus episodes, visit www.thehealthcarepolicypodcast.com