Drugs

FDA Rare Disease ARC Program Update

The FDA Accelerating Rare disease Cures (ARC) program used its first year to strengthen internal and external partnerships with stakeholders and engaged with outside experts to help find solutions for the challenges in rare disease drug development. That’s the report of CDER associate director for rare diseases Kerry Jo Lee, writing in an online post marking the first year of ARC. She says that in that time, ARC has “emerged as a conduit for empowering rare disease stakeholders (patients, patient advocates, drug developers, and academic researchers) to harness their collective experiences and expertise to drive progress.”

Under ARC, Lee says, CDER launched the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) initiative to better understand the hurdles in bringing rare disease products to market.

Looking to the future, Lee writes that in year two ARC will build on outreach efforts to further empower the community by:

creating and disseminating educational materials through the LEADER 3D initiative;
partnering with the CDER Patient-Focused Drug Development program to develop patient materials;
sharing what has been learned from using novel endpoints for rare disease therapies in the Rare Disease Endpoint Advancement pilot program; and
participating in patient listening sessions with rare disease advocates.

“The program also aims to make strides in the scientific and regulatory aspects of rare disease drug development,” Lee adds. “This includes expanding efforts in translational medicine approaches for individual rare diseases.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome