Neil McFarlane’s Post

Today, Denali joins top experts in science, advocacy, drug development, and policy at the Reagan-Udall Foundation for the FDA workshop, Qualifying Biomarkers to Support Rare Disease Regulatory Pathways. Together, we will discuss the urgent need to accelerate delivery of promising therapies to families living with rare neurodegenerative disorders. Learn more here: https://lnkd.in/ebQuRN44

Despite significant changes in treatment options over the past decade, MM patients still face inevitable relapse. Confirmative trials typically last 5+ years, focusing on PFS (primary endpoint) and OS (key 2nd endpoint). With MRD as an early endpoint, new treatments could reach patients sooner. MRD testing is well-established for MM and correlates directly with disease burden. We eagerly await detailed FDA guidance, especially regarding single-arm studies and another early endpoint, objective response rates. Additionally, FDA expanded approval for two CAR T-cell therapies as earlier treatment options for Relapsed/Refractory MM. Despite concerns from CBER about early deaths, the ODAC committee views these risks as manageable. Personally, I remain skeptical about the overall survival benefit of BMS/Celgene therapy due to high crossover rates. Overall, this April brings new hope for MM patients and oncology professionals.

Drug combination therapy & Drug repositioning .. the direction where Science is heading

Happy to be presenting tomorrow at the Rare Drug Development Symposium hosted by Global Genes and Orphan Disease Center. This amazing panel will share insights into the different pathways open for rare disease therapeutics, why the involvement of patients is essential to establishing meaningful outcome measures in these pathways, and what patient advocates can do to influence clinical trial design. #PatientLeadership #advocacy #JansensDisease #ClinicalTrials