Dr. Richard Faris, our Chief Commercial and Clinical Officer, recently submitted a proposal 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. The proposal highlights the critical role of real-world evidence (RWE) and patient-reported outcomes (PROs) in strengthening the evidence base for FDA review and approval of rare disease therapies, especially when traditional clinical trial frameworks face limitations due to small numbers of patients and other factors.
By leveraging the unique role of specialty pharmacies focused on people living with rare conditions, we can help capture and analyze meaningful data to inform regulatory science, improve patient outcomes, and accelerate access to life-changing treatments.