Dr. Richard Faris, our Chief Commercial and Clinical Officer, recently submitted a letter on behalf of PANTHERx in response to the FDA’s request for public comment on its Rare Disease Innovation, Science, and Exploration (RISE) workshop series. In the submission, he proposes a workshop exploring how specialty pharmacies can help generate real-world evidence that complements clinical trials and expands our understanding of patients’ experiences with rare disease therapies and how those therapies perform in routine clinical practice.
By working closely with patients, prescribers, manufacturers, and patient advocacy groups, rare pharmacies can help generate high-quality, real-world data that support future research, inform regulatory decisions, and ultimately improve care for people living with rare diseases.