ARM commends Reps. Upton and DeGette for their continued leadership in driving scientific innovation and patient access. The Cures 2.0 discussion draft released today would help accelerate the development and delivery of life-changing cell and gene therapies. Harnessing real-world evidence, providing educational programming and training for caregivers, and requiring the FDA to provide more clarity around CMC standards for breakthrough and RMAT-designated therapies would help bring the future of medicine to patients in need.
The draft would also require HHS to submit a report to Congress outlining the current state of cell and gene therapy regulation and foreseeable regulatory challenges for FDA in the future. This report would help educate policymakers about how we can more effectively develop durable treatments, and possibly cures, for patients living with serious and sometimes fatal diseases.
However, an improved regulatory framework alone will not enable our healthcare system to deliver 21st Century innovations to patients. ARM looks forward to working with policymakers to remove barriers to the establishment of innovative payment models, including outcomes-based agreements, as part of Cures 2.0 or other legislation.