Cole Werble
Latest From Cole Werble
US Medicare To Offer Guidance On Real World Evidence To Support Coverage
The US Medicare agency is encouraging use of observational trials to fulfill Coverage With Evidence Development requirements, but details will await an upcoming guidance on “fit for purpose” studies.
Gene Editing And Cell Therapy: US FDA Still Wants Careful Characterization
The US FDA does not seem ready to depart from its safety-focused emphasis on product characterization as cell therapies become more complex, despite calls for approaches to allow human studies that let different edits “compete” and “find the winners inside the human tumor.”
US Price Negotiation Hearing Lays The Groundwork For Future Improvements
The pharma industry is hoping that litigation might end Medicare price negotiation before it begins. But a House hearing also starts the process of developing legislative improvements that might be viable in years to come – starting with trying to restore the lifespan of small molecules.
As Sickle Cell Gene Therapies Move To Market, Health Inequities Could Help Pricing Debate
Positive tandem ICER reviews for bluebird bio’s and Vertex/CRISPR’s gene therapies show the impact that concerns about addressing racial inequities could create a favorable context for products as they enter pricing and coverage discussions.
The Evolving Door? What Having A ‘Big Data’ Commissioner Might Mean For US FDA
Robert Califf’s nomination to return as FDA commissioner will raise protests about a ‘revolving door’ pathway between industry and agency. But, in this case, it may be more apt to talk about how Califf’s time outside FDA led to evolving views on the role of big data in drug regulation.
AAV Vector Consortia Address Quality Assays, Endpoints
Two outside groups aim to help CBER on gene therapy questions from ‘N of one’ to markers for hemophilia.