Convergence: FDA, industry experts lead way on patient experience data

PITTSBURGH — A US Food and Drug Administration (FDA) official and industry experts shared best practices for the collection, use, submission, and communication of patient experience data (PED) during an 8 October session at RAPS Convergence 2025.

Amid growing demand for evidence of a drug’s impact on patients’ quality of life – not merely on surrogate endpoints – industry has been clamoring for more information about how the FDA assesses PED when considering applications for therapeutic products. (RELATED: Stakeholders seek clarity, case studies on patient experience data in applications, Regulatory Focus 17 February 2025)

The 8 October Convergence session featured Robyn Bent, director of the patient-focused drug development program at FDA’s Center for Drug Evaluation and Research, speaking by phone live and remotely, along with industry and patient advocacy representatives. Barbara Brophy, director of science and regulatory advocacy at the Pharmaceutical Research & Manufacturers of America (PhRMA), moderated the panel.

Bent defined PFDD as a systematic approach to help ensure that patients’ experiences, perspectives, needs and priorities are meaningfully incorporated into drug development and evaluation. The agency’s many activities in this area include FDA-led PFDD meetings, a methodological guidance series, a clinical outcomes assessment and related endpoints grant program, and a reflection paper by the International Council for Harmonisation.

“We really do believe that patient input is critically important to medical product development,” Bent said. “But obviously, most of these efforts are useless without industry also ensuring that they're hearing from patients and incorporating those perspectives.”

Patients should be involved across drug development, including helping identify potential targets for therapy and endpoints before moving into clinical trials, contributing outcomes data during a trial to enable benefit-risk assessments, and providing input after commercialization.

“At the end – after all of that is said and done – patient input in that postmarket phase can help us – and by ‘us’ I mean globally us – to better inform patients and providers about medical products and to facilitate more informed decision-making,” Bent said.

Sometimes FDA reviewers find it challenging to understand the type of patient experience data that is submitted and what sponsors would like the agency to do with that information, Bent said. What would help? Bent suggested having early discussions so reviewers know what’s coming and why, clear rationales behind the data and a well-organized dossier that really tells the story.

The agency developed an electronic common technical document conformance guide with instructions for sponsors submitting patient experience data as part of their applications for therapeutics. It wants industry to develop a patient experience data table telling FDA what data is being submitted and where reviewers can find that information as part of the application.

To be effective, early engagement with the agency is a must, said Brett Hauber, patient preference evidence integration lead at Pfizer.

“I think the challenge with the early engagement is not when I'm developing evidence that's actually going to go in the submission, but when we as a company are making decisions – whether that be about a particular endpoint, a particular element of the trial design or even a dose selection – where we may have insight that from patients that helps us make those decisions, but that insight may not be in a form that can be shared as evidence with the agency,” Hauber said.

Evolved patient-focused landscape

The FDA launched its PFDD initiative in 2012 with the passage of the Food and Drug Safety Innovation Act (RELATED: FDA Announces Sixth Patient-Focused Drug Development Meeting, Regulatory Focus 5 November 2013). The landscape has evolved a lot since then and industry, regulators, patients and other stakeholders are really taking this on and thinking about how to strategically work together to advance the field, said Pujita Vaidya, who leads regulatory science and policy in North America at Sanofi.

“Industry routinely engages with patients and patient advocacy groups to collect patient experience data,” she said.

Sanofi has developed a strategic framework internally – referred to as its patient disease strategy – to drive company thinking overall, across early development and onwards through post-marketing, including milestones. The patient voice is incorporated into the company’s overall endgame, she explained.

“From the very beginning (preclinical and Phase 1), we are trying to understand, what is the unmet need? What's the disease burden? How can we characterize some of the impacts of the disease and get those very early patient insights on their experiences, perspectives, needs and priorities when it comes to a specific disease or a treatment,” Vaidya said.

The work doesn’t stop with a regulatory submission. Sanofi then thinks about long-term safety, collecting real-world data, understanding patient adherence, patient satisfaction, long-term tolerability, and cost-effectiveness, Vaidya said.

Meanwhile, patient organizations are partnering with industry, the agency and academic institutions to try and bring the lived experience of their communities into early decision-making – whether it’s informing regulatory processes, helping to shape a trial, or thinking about what outcomes are most important, said Jason Harris, vice president of government relations and advocacy at the National Psoriasis Foundation.

“Patients, caregivers, communities – they tend to define what's a meaningful outcome a little bit differently than what a clinical or research team want,” Harris said. “It’s a different perspective that does need to be included and thought about.”

When a therapeutic is approved, patients want to know what the health benefit is, how it will help their life and how soon it will be available.

“And then there's the whole access and affordability piece that I think is front and center,” Harris added.

Organizations like the National Psoriasis Foundation want to be kept in the loop with information about clinical trial and regulatory developments.

“The communication piece is a huge part of the partnership,” Harris said.

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Convergence: FDA, industry experts lead way on patient experience data

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