Heidi Wang

Latest on MDMA rejection; Novo vs. Lilly earnings; Recursion, Exscientia to merge; Q&A with Genentech CEO; and more https://lnkd.in/gRuf3XRp Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up [...] #LosAngeles #OrangeCountyCA #Biotech #Lifescience #News

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And another chapter in the #drugpricing debate for #genetherapies, touching on many hot-button #healthpolicy issues from #acceleratedapproval for gene therapies and the reliability of #surrogate #endpoints to #alternativepaymentmodels for #genetherapies and their #cost, #value, and benefit-risk-tradeoffs. This is certainly not a new debate, but one that is increasingly gaining attention given the growing number of cell and #genetherapies on the market and the robust pipeline. It does raise (at least) two issues for me: First, it is easy to forget the real-life impact of #accelerated approval on the #patients and families. On average, #accelerated approval shaves 4.5 years off the #drugdevelopment timeline by allowing FDA's approval decisions to be based on surrogate or intermediate clinical endpoints, rather than having to wait for traditional (validated) clinical endpoints. For neurodegenerative diseases in particular, it can take years or decades for the clinical endpoints to occur. Getting these products into the hands of clinicians earlier is huge - especially for #genetherapies with narrowly defined age limits. ELEVIDYS, the product at issue in this paper, is currently only approved for 4 and 5 year olds. How many children would have aged out of the chance for a potential cure (or something close to it) without accelerated approval? Second, nobody questions that one-time payments of $3.2 million are substantial costs for any #healthinsurance #payer. It has to be seen in the context of the direct and indirect costs averted over time, although health insurance portability means that in many cases the payer that realizes the cost is probably not the one that realizes the subsequent savings. Moreover, exactly how large the subsequent savings really are remains somewhat unclear given remaining questions around long-term safety and durability of effect. That makes #innovativepaymentmodels particularly important, allowing for some of the risks to be shared across insurance providers and with manufacturers. But the reality is that current data systems in the U.S.'s fragmented healthcare ecosystem and are woefully inadequate to generate the type of evidence needed to support meaningful and broadly applicable #outcomesbased #arrangements. I hope the policy debate around the price of current #genetherapies will evolve to one that provides tangible policy solutions and that truly places the patient at the center. What we need to move forward is centralization, standardization, and data sharing, led by the Centers for Medicare & Medicaid Services, to enable meaningful outcomes-based arrangements that simultaneously mitigate some of the economic risks associated with #genetherapies and provide the evidence to support accelerated approval through robust confirmatory evidence, so that this critical pathway will remain available to the life-altering #orphandrugs that need it. https://lnkd.in/euzyxV_a

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COTA and the FDA will advance #cancerresearch using #realworlddata, focusing on #regulatory decision-making and improving treatment evaluation and availability. Read at The Evidence Base® #heor #healtheconomics #outcomesresearch #rwe #rwd #realworldevidence #pharma #biopharma #medicalaffairs #regulation #regulatoryaffairs #healthdata

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Glad to see more commitment from Bristol Myers Squibb Bristol Myers Squibb Announces 10-Year Strategy to Reach More Patients in Low- and Middle-Income Countries PRINCETON, NJ / ACCESSWIRE / May 22, 2024 / Bristol Myers Squibb (NYSE:BMY) today announced ASPIRE (Accessibility, Sustainability, Patient-centric, Impact, Responsibility and Equity), a 10-year strategy to advance access to our innovative treatments and help patients in low- and middle-income countries (LMICs) gain access to potentially life-saving medicines. This strategy supports the company's commitment to reach more than 200,000 patients in LMICs by 2033 with its innovative treatments. "Access to potentially life-saving innovative medicines is very limited in some parts of the world, leaving patients with few treatment options for serious diseases like cancer," said Christopher Boerner, Ph.D., board chair and chief executive officer, Bristol Myers Squibb. "Bristol Myers Squibb believes patients who can benefit from our medicines should have access to them. Our ASPIRE strategy is an important step toward accelerating and expanding patient access to these much-needed treatments." As part of ASPIRE, Bristol Myers Squibb (BMS) is developing tailored strategies to increase affordability and availability of its medicines in LMICs. Our work to help ensure patients who need our potentially life-saving medicines can access them begins by ensuring that 100 percent of our marketed products are supported by access plans. Today we have defined access pathways for every country in which our medicines may be available to patients. In low- and middle-income countries, to help improve the affordability of our medicines, we have introduced local brands of many of our innovative medicines to address affordability issues, expand access, and help reduce the time lag between the availability of our medicines in higher-income countries and lower-income countries. In 2023, BMS filed a total of 11 local brands of which five received regulatory approvals. BMS local brands are making an impact in countries like Thailand, which has the highest prevalence of beta thalassemia, by providing treatment options like Rojusna™ to help combat this widespread disease. BMS is also making its medicines available where we don't have a commercial presence through the Direct Import and the Direct-to-Institution pathways. Through the Direct Import pathway, BMS is providing access to 12 medicines in more than 80 LMICs, using tiered pricing commensurate with affordability. The Direct-to-Institution (DTI) pathway is a scalable and sustainable channel that provides safe, broad, timelier access to BMS' innovative medicines to multiple patients through collaboration with local institutions. The company's initial DTI efforts are in five LMICs in East Africa and Pakistan and West Africa https://lnkd.in/gR42JzGj

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🔬 Biopharma Layoffs: Navigating Challenges in a Shifting Landscape Recent trends in biopharma layoffs have left many scientists reeling. Here are key takeaways from a recent STAT article: 67% of life science layoffs in H1 2024 were at large biopharma companies (>$1B annual sales), up from 47% in 2021-2023. Job postings in biotech dropped from 21,366 to 12,300 between early 2021 and 2024. Scientists face unique challenges in job searches, including maintaining confidentiality about previous work. Visa constraints add pressure for international workers, limiting job search time. Networking remains crucial: Some successful companies are hiring primarily through personal connections. The industry sees this as part of a cyclical pattern, influenced by patent expirations and acquisitions. Despite these challenges, opportunities still exist. Adaptability, networking, and a broader job search approach are key for scientists navigating this evolving landscape. What strategies have you found effective in weathering industry changes? Share your thoughts below! #BiopharmaJobs #CareerTransition #LifeSciences #Networking

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FDA’s Endorsement: The Marvel of MRD Minimal Residual Disease (MRD) “Elevating MRD to the Forefront” and Revolutionizing Cancer Treatment Approaches The FDA’s Oncologic Drugs Advisory Committee’s recent meeting in April 2024 has cast a spotlight on Minimal Residual Disease (MRD), underscoring its critical role in the advancement of cancer therapies. Here’s how the FDA’s recognition is reshaping the future: 1. Multiple Myeloma: The FDA’s committee has officially recognized MRD as a valid endpoint for accelerated drug approvals. This pivotal decision by the FDA is a testament to MRD’s potential in changing the treatment paradigm for multiple myeloma. 2. Acute Lymphocytic Leukemia (ALL): MRD serves as a vital prognostic indicator, and the FDA’s stance influences treatment choices and regulatory approvals of new medications. It’s a significant step forward, with the FDA’s backing enhancing the credibility of MRD in clinical settings. 3. Chronic Lymphocytic Leukemia (CLL): The MRD status is integral to treatment planning, and the FDA’s consideration is crucial during the approval process of new treatments. The FDA’s involvement ensures that MRD remains a key factor in CLL therapy strategies. 4. Acute Myeloid Leukemia (AML): MRD is being considered by the FDA as a potential surrogate endpoint in trials, which could expedite the approval of innovative AML therapies. The FDA’s support could pave the way for quicker access to new treatments. These examples underscore the vital role of MRD as a clinical endpoint across various cancer types. It’s not just a measure; it’s a movement towards enhancing patient care and fast-tracking the introduction of innovative treatments. The FDA’s Oncologic Drugs Advisory Committee’s discussions and decisions highlight the indispensable role of MRD, now backed by the FDA’s authoritative support. It’s a monumental stride in enhancing patient care and introducing cutting-edge treatments more swiftly, all through the FDA’s visionary lens. For more details, please contact, sales@oliveridleyadvisory.com

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