Symposium Title: Preference Evidence for Regulatory Decisions

Excerpt

Interest in regulatory use of patient-preference information has been growing in recent years [1‐3]. Several initiatives have sought to help guide the collection, analysis, and interpretation of such information [4‐6]. These initiatives have taken place in the context of a broader evolution in regulatory science and benefit–risk evaluations [3, 4, 7]. The Center for Devices and Radiological Health (CDRH) at the United States Food and Drug Administration (FDA) recently issued guidance for submitting preference information for regulatory reviews. Health technology assessment agencies in Europe (IQWiG [Institute for Quality and Efficiency in Healthcare] in Germany and NICE [National Institute for Health and Care Excellence] in the UK) have acknowledged the relevance of such information. In the US, recent approval of the 21st Century Cure Act, the Medical Device User Fee Act Amendments (MDUFA) IV, and the Prescription Drug User Fee Act Amendments (PDUFA) VI has also identified the need for more patient engagement in the regulatory process [8]. While there is considerable interest in patient-centered decision-making, it is still unclear how preference data should be integrated into existing regulatory procedures and criteria for new drug or device applications. It is also currently unclear what differences we should expect to see in regulatory outcomes from incorporating preference information. The evolving landscape on using preference information in the regulatory context has raised the need to continue an open dialog among regulatory agencies, industry, academia, and those at the center of this movement, patients. …