Tag: Rare Disease

FDM illustration - checklist clipboard with reform icons

Makary’s Year 2 Wishlist: A New FDA Pathway, Lower User Fees, and What Florida Physicians Should Be Asking For

Marty Makary's second-anniversary wishlist for Congress includes a new Plausible Mechanism Pathway, lower PDUFA fees, and a permanent rare-disease voucher program. Florida physicians need to be in the room — here's what to do now.