BSF continues to inform best practices for Patient Focused Drug Development (PFDD) meetings being led by patient advocacy groups. BSF's Executive Director, Emily Milligan, recently spoke at the 7th annual Biopharma Congress cautioning peer organizations that PFDD meetings are "not a one-and-done" and demanded more transparency about ways in which the FDA is incorporating patients' perspectives into the Agency's review protocols. “It wasn’t apparent to us how” the testimony from the PFDD was “being incorporated into internal deliberations within the Agency as well as actual decision-making. I would like to have [...] more transparency around that,” she said.
Transparency and consistency are high priorities for BSF as the FDA continues to deliberate both a way forward for elamipretide, potentially the first-approved drug for Barth syndrome, as well as a viable path ahead for any future potential therapies for our ultra-rare disease. Currently, BSF is in the process of coordinating a meeting with the FDA to discuss possible solutions and to advocate for fair and appropriate regulatory review of therapies for Barth syndrome, now and in the future.
