5:29 PM
Dec 22, 2017
 |  BioCentury  |  Regulation

Prioritizing preferences

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.”

Denise Hinton, FDA

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 patient groups, will need to invest in building capacity to design, execute and evaluate the studies.


The December workshop reflected a strategic decision by FDA’s Office of Regulatory Science and Innovation, which sits within the Office of the Chief Scientist, to ensure consistent integration of patient preference research and evaluation across the Center for Devices and Radiological Health (CDRH), the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER).

To date, CDRH and CBER have been ahead of CDER in the use of patient preference research. The device and biologics centers issued a joint draft guidance in May 2015 that was finalized in August 2016.

The guidance outlines qualities of patient preference studies, recommendations for collecting patient preference data, steps required to get patient preference data included on a device’s label and hypothetical examples that illustrate how patient preference information may inform the centers’ regulatory decision-making.

In September 2015, CDRH granted the first-ever product approval that was based on a patient preference study -- ReShape Lifesciences Inc.’s implantable Maestro Rechargeable (RC) System for obesity.

“This can give you more ways to succeed and provide more ways to bring a useful drug to patients.”

Bennett Levitan, J&J

The device missed the primary endpoint of a 10% improvement in weight loss compared to a control group. But a preference study conducted by CDRH revealed that a group of patients would accept the risks associated with the device for the amount of weight loss it was expected to provide.

The following year, CBER launched its Science of Patient Input initiative to systematically incorporate patient feedback into regulatory decision-making.

CDER has been learning about disease-specific patient preferences in 24 patient-focused drug development meetings it has held since 2013. In April, the center for the first time included patient preference information on a drug label with the approval of Genentech Inc.’s Rituxan Hycela subcutaneous rituximab (see “Preference Pioneers”).

“Our goal is to get everyone on the same page about the fundamental concepts and how it works in regulatory decision-making,” said Anindita Saha, director of external partnerships and expertise at CDRH. Doing so will increase the use of these studies within the agency and among other stakeholders, she added.

Rebecca Noel, global benefit-risk leader at Eli Lilly and Co., said FDA appears to be learning from the difficulty it had getting consensus across review divisions on implementation of its PRO guidance.

Matt Reaney, global head of clinical outcomes at Sanofi, noted that after the PRO guidance was published in 2009, there was a...

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