How Patient Voices Shape Regulatory Drug Decisions: Eleanor Perfetto, PhD

Patient experience data are critical to patient-focused drug development and the regulatory process. This direct feedback can encompass symptoms, daily challenges, and desired treatment outcomes. The FDA uses these data, which include both informal feedback and a formalized, rigorous methodology, to evaluate new therapies across the drug evaluation process.

Manufacturers, pharmaceutical companies, device companies, and life science companies benefit from knowing this information, explains Eleanor Perfetto, PhD, consultant to patient organizations and pharmaceutical companies and an expert on both patient experience data and drug pricing policy. In this clip, she highlights how and why these data are integral to regulatory decision-making and the drug application approval process.

This transcript was lightly edited for clarity; captions were auto-generated.

Transcript

Can you explain the importance of patient experience data and their role in regulatory decision-making?

Patient experience data are data that come directly from patients, not through other people, but directly from patients, and it's them talking about the experience of having a disease and the impact that it has on their life every day. That can be anything from the symptoms that they experience to the challenges that they experience to their preferences or the outcomes that they prefer most. If a treatment is going to work best, then they would describe what outcomes they're looking for that would tell them that a treatment is working best. It's any aspect of their experiences that are related to the disease and the treatment that come directly from the patient and that are the most important things to them.

Right now, in particular, the data are collected through research projects or through informal mechanisms to try to understand what's going on with patients. The Food and Drug Administration has an initiative that's called the Patient-Focused Drug Development Initiative, and through that patient-focused drug development initiative, called PFDD, the Food and Drug Administration is using patient experience data to help inform regulatory decision-making, and it's a much more formal process. It begins with perhaps informal data collection from patients to understand what words they use when they describe their disease, when they describe their symptoms, when they talk about the experiences they are having, to make sure that you understand that you're communicating with them well, that you understand the terms that they're using, and how they describe things.

That can be done a little bit more informally, but then it's also through a very formal process, where once you understand what those most important things are, how do you collect that data from those patients? There's a very formal process that's rigorous. That FDA guidance guides manufacturers, pharmaceutical companies, device companies, life science companies. It guides them on how to do this properly so that the data that come out of it can be regulatory-grade, and that's more formal, rigorous patient experience data. But what patients tell you can come from that spectrum of informal conversations all the way through to that regulatory-grade information that could actually end up in a new drug application, actually end up in the label for the drug, and that's really what companies would like to see. They'd like to see that they learned from patients what's most important to them, collected information on what's most important to them, and their product affected the things that are most important to patients, so that patients really know that the product was working for them. That's the ideal goal, and that's the way the FDA would like to see it. And they guide industry through that, through the 4 guidance documents that they have for PFDD.