Medical product sponsors may choose to include patient perspective input (PPI) in their FDA regulatory strategy to augment clinical data, argue for alternative clinical trial designs, or support indications in specific patient populations. FDA has published a number of guidance documents to provide industry with expectations for including PPI data in regulatory submissions.
From an industry perspective, guidance from CDRH regarding patient preference and that from CDER and CBER regarding patient experience are complementary. In fact, the basic tenants of observational research promoted in CDER/CBER’s guidance could apply to studies conducted to obtain patient preference or experience data. Similarly, CDRH’s guidance for patient treatment preference can be applied across therapeutic areas and diseases.
However, there remain differences in guidance across FDA centers. For example, CDRH PPI guidance is focused on “patient preference.” In contrast, CDER and CBER are primarily focused on patient experience and clinical outcome assessments (COAs). Though FDA has hosted cross-center workshops, there is no apparent coordinated effort to harmonize guidance to provide industry with consistent recommendations about how to conduct PPI activities. Without coordinated patient-centered policy from FDA, there is the potential for confusion and a lack of consistency within industry and patient communities about how PPI data may be obtained and applied within the regulatory process.
A coordinated FDA policy framework that recognizes the potential for different types of PPI data to be used in any medical product regulatory submission is warranted. In addition, there is a need for FDA to clarify how patient-centered policies across the different FDA centers apply to medical product sponsors. Creating consistent, coherent, and transparent patient-centered policy will encourage sponsors to seek out patient input more often and with greater certainty of the value these data may have to the regulatory clearance and approval of their products.