Holly Peay, PhD, on Patient Preference Research as a Means of Patient Expression

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The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.

This is the second part of an interview with Holly Peay, PhD. For the first part, click here.

“One of the things that we find often in preference research is that people living with conditions think differently than we might expect about what they want out of a treatment, what they hope for out of treatment, and what they will tolerate out of a treatment. I often work with different patient advocacy groups when I do these and often we find things that even advocate leadership who engage with people regular really didn't expect. Doing this kind of study is a really central way of getting patient voices [heard]—and not just the advocates who are at every meeting—but getting a broader group of patients who are able to make their preferences known in a systematic way.”

When it comes to the development of products belonging to relatively novel therapeutic modalities like gene therapy and cell therapy, especially for rare disease indications, it can be difficult to know what types of effect from such products patients would consider meaningful changes to their disease, and what risks they are willing to tolerate to receive this benefit. One way government agencies and academic institutions gather more information about this topic is through patient preference studies, in which members of the patient community for a particular disease are asked to give their own opinion on potential treatment options and outcomes. Notably, patient preference studies require skilled experts to conduct and can get quite expensive depending on the scale of the study and the number of patients involved.

At the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, Holly Peay, PhD, senior research scientist at RTI International, gave a presentation on the important role of patient preference research in the context of rare neuromuscular disease. After her talk, CGTLive® spoke with Peay to learn more. She emphasized that although having a large patient population for these types of studies is beneficial, meaningful insight can still be gathered from very small studies, even those with fewer than 10 participants, which can make carrying out these studies financially feasible and practical for ultrarare disease indications. Peay also highlighted the fact that patient preference studies often reveal surprising findings about what members of the community actually want from treatment, and that they are an important means for patients to have their voices heard.

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

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