Califf: Patient Preferences Too Often Overlooked in Medical Product Development, Approvals

Regulatory NewsRegulatory News | 19 November 2015 |  By 

As patients play a more active role in which regulated products are used in their care, the US Food and Drug Administration (FDA) is working on several programs to ensure patient perspectives have an effect on which of those products are developed and approved, FDA commissioner nominee Robert Califf and other agency officials said in a JAMA Viewpoint published Thursday.

FDA is now participating in a number of different drug and medical device initiatives that directly involve patients throughout a product’s lifecycle, including a tool that helped to incorporate patient preferences into clinical trial designs for obesity treatments, Califf, Nina Hunter and Kathryn O’Callaghan said in the viewpoint.

Medical Devices

Most recently, FDA created the Patient Engagement Advisory Committee (PEAC), which the authors say is an example of the FDA’s commitment to partnering directly with patients.

“By including patient experts from different backgrounds, the PEAC will help ensure that broad, diverse perspectives on the needs and experiences of patients inform the FDA’s deliberations,” the officials say.

FDA is also a member of the Medical Device Innovation Consortium, which is the first US public-private partnership focused on medical device regulatory science.

This consortium catalogs methods for eliciting patient preferences and helps guide researchers looking to incorporate patient perspectives on the benefits and risks of medical devices for regulatory and other purposes.

Back in May, FDA posted a draft guidance that provides “background information, structure for incorporating patient preferences (defined as qualitative or quantitative assessments of the relative desirability or acceptability of attributes that differ among alternative diagnostic or therapeutic strategies) into regulatory decision making, and advice regarding collection and use of patient preference information for manufacturers and other stakeholders to support certain medical device approvals.”

In addition, FDA scientists teamed with behavioral economists to test a method for capturing patient sentiment and translate it into a tool that incorporates patient preferences into clinical trial designs for obesity treatments. The agency later approved a new weight-loss device informed in part by data from this study.

Drugs and Biologics

As FocusPatient-Focused Drug Development (PFDD) Tracker shows, FDA has been meeting publicly with patients suffering from a number of different conditions regularly since the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012.

And although FDA initially planned for 20 PFDD meetings, the agency is now on track to hold 24 meetings by 2017, the officials say.

“Each meeting is summarized in a report that captures participants’ experiences and perspectives in their own voices. Learnings from the past few years are now shaping plans for expanding and advancing work in patient-reported tool development,” they added.

PROMs, Precision Medicine

The officials also point to NIH’s knowledge base of patient reported outcomes established via its Patient-Reported Outcomes Measurement Information System (PROMIS). In addition, patients can offer information to regulators on what level of change in their symptoms or functioning would be meaningful to them.

Following the release of guidance on this topic, “the use of patient-reported outcome measures (PROMs) in regulatory submissions to the CDRH with clinical study protocols has increased from about 20 submissions per year prior to the issuing of the guidance to more than 120 in 2014 alone, indicating substantial interest on the part of industry and clinical researchers in generating patient-centered evidence from studies done for regulatory purposes,” they say. 

Patient involvement in President Barack Obama’s Precision Medicine Initiative will also be crucial as the program seeks to engage up to one million participants to account for individual differences in genes, biology, lifestyles, environments and preferences.

“FDA is committed to integrating patient perspectives into its regulatory evaluations and decision making and also encourages medical product innovators and clinical researchers to collect information on patient perspectives to inform development programs,” the authors conclude. “By advancing the science of eliciting and understanding patient input and incorporating PROMs into product evaluations, these programs can accelerate the development and selection of new products that best meet individual needs.”

Journal of the American Medical Association



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