Holly Peay, PhD, on Keeping Patient Preference in Focus

Historically, patient testimony was the main way that patient voices were heard in the regulatory decision making process for drug and therapy approvals. Agencies like the FDA are largely driven by data in their decisions, and as such, patient testimony has generally been supplemental or subordinate to data during the approval process. In the past 10 to 15 years, however, patient preference research has begun to be incorporated into the regulatory approval process in the United States. Patient preference allows for patients’ voices to be heard in a way that can be presented to regulatory bodies as data, and thus more directly help to inform decisions. Patient preference is especially important in rare disease indications where current treatment options are limited in number or efficacy, and/or may come with substantial drawbacks. Furthermore, patient preference research can help to define meaningful end points for clinical trials of novel treatments, including less well-established modalities like gene therapy.

Holly Peay, PhD, senior research scientist at RTI International, gave a talk on the importance of patient preference research at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida. In an interview at the conference, held shortly after Peay presented, Peay spoke about the progress that has taken place in recent years with regard to patient preference research. She noted that novel targeted therapeutics for rare disease often don’t resemble well-established treatment methods, and therefore patient preferences can play a helpful role when they are integrated into regulatory filing as additional data.

Click here to view more coverage of the 2024 MDA Conference.