Few would argue that clinical trial design and patient enrollment are critical to the successful approval of a new drug. Additionally, costs are significant regardless of success or failure. Why then is the patient voice not integral to the development of trial design concepts? We have conducted marketing research with patients that have informed inclusion of meaningful endpoints, ensured impactful QOL measures, and identified educational and support needs to aid and sustain trial enrollment. As an example, imagine you had an immunological disease with fatigue as a primary symptom. Which of the following would you consider most relevant when deciding to take a medication: a reduction from baseline on the Fatigue Severity Score or a reduction in the number of work days missed? Although the FSS endpoint may be clinically necessary to include, fewer work days missed per month is more meaningful to the potential medication users we interviewed. In the age of patient-centricity and explosion of personalized medicine, I applaud the inclusion of patient insights to inform trial concept design and argue that they are incredibly valuable.
We engage very early, even when we are developing the concept design of early phase trials. That is where we learn a lot about patient needs.”