Rare Disease Patients As Partners: Value Beyond Enrollment

By Nicole Carswell

When developing novel rare disease therapeutics, teams have found that engaging with patients at various points of a clinical development process can be very valuable. Clinical teams that are looking to employ these practices in their own studies often question to what extent they should collaborate with patients. And are there ways to further apply their valuable insights to optimize their study’s success?

Recently, there has been increased emphasis on creating processes and frameworks to encourage sustainable patient partnerships and to establish a paradigm of patient-centered research that begins at the earliest stages and continues throughout all phases of development. This paradigm shifts the focus from engaging patients in ancillary roles, such as assisting with recruiting patients to a trial or enrolling as patients themselves, to empowering them in more active roles where they drive the greater influence of the study development plan.

If we’re being honest, the most fruitful partnerships involve true collaboration—not just information exchange, but mutual learning where both patients and researchers gain deeper insight into each other’s challenges. Learn why teams are putting an increased emphasis on creating processes and frameworks to encourage sustainable patient partnerships and establishing a paradigm of patient-centered research.