CER Daily Newsfeed

Mixed Signals Cloud the Orphan Drug Market

(3/25, Kelly Bilodeau, PharmaVoice) comments “...[W]hen the FDA rejected Biohaven’s troriluzole for spinocerebellar ataxia, it raised concerns about trial design including the use of real-world evidence, even though the agency had previously provided input on the study design. ‘The FDA issued a complete response letter despite Study 206-RWE being reviewed by FDA and achieving statistical significance in the study’s prespecified primary and secondary outcome efficacy endpoints,’ Biohaven said in a press release. ‘FDA cited issues that can be inherent to real-world evidence and external -control studies including potential bias, design flaws, lack of pre-specification and unme--asured confounding factors.’” Full

New Real-World Data Platform Launched to Support Alzheimer’s Research

(3/25, Joanne Walker, The Evidence Base) reports “...A new large-scale research initiative, the Multimorbidity Three-City Alzheimer’s Disease EHR (M3AD) Study and Real-World Data Metaplatform, has been launched to support advances in Alzheimer’s disease and related dementias (AD/ADRD) research using real-world clinical data. The initiative, supported through a grant from the National Institute on Aging, is led by researchers at Columbia University Mailman School of Public Health, working with collaborators from the Vagelos College of Physicians and Surgeons, the School of Nursing, the University of Miami, and the University of Chicago.” Full

From Real-World Data To Real-World Impact: Building The Evidence Capability Pharma Actually Needs

(3/25, Hayden B. Bosworth, Ph.D., Steven C. Grambow, Ph.D., and Drew Narayan, MBA, Clinical Leader) comments “...The consequences of underdeveloped RWE capability are significant. Weak evidence strategies increase regulatory burden and downstream study costs, undermine payer positioning, fragment launch execution, and compress revenue generation by limiting pricing power and slowing uptake. In an environment where comparative effectiveness, value-based contracting, and outcomes-based reimbursement are expanding, the ability to produce defensible, decision-aligned RWE is increasingly tied to revenue durability and competitive differentiation.” Full

UK: Better Data for Better Decisions

(3/25, Sophie Cooper, NICE Blog) comments “...The new value set will not replace the overall system NICE uses to assess treatments. It simply provides better data to feed into that system. NICE will still be asking for data collected using the EQ-5D questionnaire and will still be calculating cost effectiveness in the same way – this just makes those calculations more accurate and reflective of what matters to people today.” Full