How FDA is laying the groundwork for a coordinated approach to preference research
The realization that patients have distinct preferences about the trade-offs and risks inherent in medical decisions -- and that their choices are frequently different from those physicians or regulators would make -- has mobilized FDA, Congress and industry to begin to embed patient input into the development and evaluation of medical products.
FDA has concluded that patient preference information is necessary to inform regulatory decision-making, and in some cases may be the only way to enable an approval when benefit-risk trade-offs are uncertain.
But the science of patient preference research is in its infancy in medical product development. There are no standards for study design, conduct and analysis, and few sponsors or regulators have experience interpreting the resulting data.
In many ways, patient preference research today is where patient-reported outcomes (PROs) were almost 10 years ago. And while FDA, companies and academics have made some progress on developing, deploying and interpreting the results from PROs, that progress has been slow and painful as companies and review divisions have felt their way in the dark, one development program at a time.
“Our ultimate goal is to develop a systematic way of eliciting, measuring and incorporating patient preferences into the medical product life cycle.”
In contrast to that approach, FDA is now working to quickly clear a path across all three of its centers for product sponsors to incorporate patient preference studies into product development.
“We recognize that patients are the ones who live with their medical condition and make choices about their personal care. It’s critical that we capture their viewpoints where we can and incorporate them into FDA’s regulatory decision-making,” said Denise Hinton, FDA’s acting chief scientist, at a Dec. 7-8 workshop to discuss preference studies.
“Our ultimate goal is to develop a systematic way of eliciting, measuring and incorporating patient preferences into the medical product life cycle so that we can enable more patient-centric innovation, evaluation and delivery,” said Hinton.
During the workshop, FDA representatives, academics and patient representatives sought to bring clarity to when preference studies are most useful, and which types of studies are suited to answering what types of questions.
Patient advocates at the meeting said preference information should be sought early and often during product development, that studies should account for heterogeneity of patient experiences and that results of preference studies should be shared to accelerate adoption.
Three industry executives who attended the meeting told BioCentury afterward that creating a systematic process across the agency should make it easier for companies to conduct these studies. They’d also like to see harmonization with international regulatory bodies such as EMA.
But all the stakeholders, including companies, FDA and