Merck’s Quality-Of-Life Argument For Ridaforolimus Draws ODAC Tongue-Lashing
The pivotal study failed to capture patient-reported outcomes data on drug-related side effects, and use of observational data to support the view that the mTOR inhibitor has a positive risk/benefit profile is “crazy,” committee members said.
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Oncology Experience With PROs
There has been some success in getting PRO data into oncology labeling, but also notable failures.
Accelerated Approval Could Spur Development Of Patient-Reported Outcomes Tools
A subtle takeaway from the Oncologic Drugs Advisory Committee’s March 20 review of GlaxoSmithKline’s Votrient (pazopanib) for treatment of advanced soft tissue sarcoma was that accelerated approval, with a post-marketing requirement to confirm clinical benefit in a difficult-to-treat population, could incentivize sponsors to develop instruments for assessing symptom improvement.
Intercept’s Ocaliva: ‘Dangling’ PBC Indication At Risk As Near-Term NASH Approval Looks Unlikely
Accelerated approval for primary biliary cholangitis in 2016 came with three postmarketing requirements, but studies were terminated early; firm working toward sNDA submission in 2023 for regular approval, but US FDA says reports already are overdue and it expects to take PBC indication back to an adcomm.