How BIO and PPMD plan to create a blueprint for patient preference studies
Facing calls from its members and patient groups for a framework on how patient preference studies for drugs should be designed and executed, BIO enlisted Parent Project Muscular Dystrophy to help create a set of general guidelines and recommendations that stakeholders can use to design and execute disease-specific patient preference studies.
Patient preference research is grounded in the notion that patients have distinct preferences about the trade-offs and risks inherent in medical decisions, which are frequently different from the choices physicians would make.
The research methods are well-established and have been applied in healthcare settings for 25 years, but their use to inform medical product development and regulation is quite new.
Only one medical product has been approved based on data from patient preference studies. The Maestro Rechargeable System from EnteroMedics Inc. missed the primary endpoint in a trial to treat obesity. But FDA's Center for Devices and Radiological Health (CDRH) was able to