EmPartners

Yesterday (April 28th) the FDA Commissioner Dr. Marty Makary announced the launch of a new pilot program for real-time clinical trials. This program is being conducted with Amgen (for Tarlatamab in SCLC) and AstraZeneca (for Acalabrutinib in MCL) as initial test subjects. While both pilot trials are in oncology indications, the announcement from FDA suggests the goal is for this protocol to be available for other high priority indications, e.g., pandemic-level infectious diseases.   The goal is a valid one: to reduce the overall amount of time it takes for potentially revolutionary new medicines to reach patients and provide benefit. Dr. Makary boldly claims 45% of the drug development timeline is “dead time”, wasted on unnecessary bureaucracy & paperwork. The real-time concept revolves around safety and efficacy datapoints being received by FDA reviewers in real-time, rather than waiting for database lock and being curated by the sponsor company. However, there are several significant technical considerations and concerns that surround such an endeavour. 👉 Does removing the database lock step mean that individual datapoints will be harder to interpret in isolation? 👉Will this place additional pressure on study sites to liaise directly with FDA, rather than with the trial sponsor? 👉Could sharing real-time data lead to unintentional unblinding of carefully controlled trials? 👉How will data from other trials & sources be folded into the review process of a real-time trial? Currently there are several other uncertainties around how this would be implemented. For example, whether sponsors would need to apply for real-time trial designation, and if this protocol is intended to be available for any drug development, or only for use in emergency situations such as a future pandemic or a high risk cancer type.    From a drug development and competitive intelligence perspective this initiative means approval timelines could be accelerated, while becoming harder to decipher due to the review process becoming more opaque. Broad adoption of this real-time review protocol would mean launch timings for competitors become much less predictable, creating a more uncertain competitive environment. How realistic do you think this real-time review process will be to implement broadly? And what could be the impact on US drug development?   #FDA #drugdevelopment #Amgen #AstraZeneca #regulatoryprocess #pharma #biotech https://lnkd.in/e5qzcxYj

🧠 New Blog Alert: Call for Action to Unlock Neuroprotection by Targeting the Blood–Brain Barrier (BBB). For decades, the blood–brain barrier has been viewed as an obstacle to drug delivery - but what if it could be the key to neuroprotection itself? In our latest blog, Aniv Brukner and Bailey Bojalad explore how innovative therapeutic strategies can shift the paradigm: rather than bypassing the BBB, we can harness it to clear toxic species, restore vascular function, and protect neurons in neurodegenerative diseases. Read the full piece here 👉 https://lnkd.in/efjbTVa2 #Neuroscience #Neuroprotection #BloodBrainBarrier #BBB #Neurodegeneration #AlzheimersDisease #ParkinsonsDisease #CNSResearch #DrugDiscovery #TranslationalScience #Biotech #PharmaInnovation #BrainHealth #epilepsy #stroke #MS #multiple sclerosis #TraumaticBrainInjury #TBI #HeadTrauma #Axoltis #Vaccinex #mtpharma #OTR3 #SciNeuro #Ovidrx #Neurelis #TheriniBio #CavaHealthcare #Neuvasq #LysTherapeutics #Athira #Accure #Nyrada #Sanofi

💉mRNA vaccines changed the game during COVID-19, but what happens to mRNA therapeutics now the federal funding is being pulled back?    In our white paper, Consultant Stephanos Nicolaou discusses the impact of the #HHS funding cuts, the rationale, and where #mRNA innovation might go next.   Read the full article at https://lnkd.in/es37mNt3    #Pharma #mRNA #LifeSciences #HHS #VaccineInnovation #ScienceAndPolicy #BARDA

The journey of ELEVIDYS, the first FDA-approved gene therapy for Duchenne Muscular Dystrophy, has been both groundbreaking and turbulent. Once celebrated as a beacon of hope, it now faces heightened scrutiny following safety concerns and regulatory setbacks. What does this mean for patients, clinicians, and the future of gene therapy in DMD? In this piece, we explore the evolving story of ELEVIDYS—its promise, challenges, and the broader implications for rare disease treatments. #ELEVIDYS #Sarepta #DMDResearch #MuscularDystrophy #DuchenneMuscularDystrophy #RareDisease #PatientCare #GeneTherapy #Biotech #LifeSciences #PharmaInnovation #ClinicalResearch #FDAApproval Authored by: Isobel Blacksell, PhD, EmPartners