Comment from The Cure Starts Now Foundation / DIPG/DMG Collaborative
AnonymousOtherAdvocacy
Summary: The commenter, representing a global coalition of patient advocacy groups, acknowledges the benefits of the proposed program but argues it is currently ill-equipped for rare and pediatric cancers. They request further revisions to the guidance to better accommodate these specific patient communities and offer to participate in future efforts.
Expedited Investigational New Drug Pilot Program; Request for Information 2026-12621 (91 FR 37996)
While improvement on FIH guidelines, like the proposed, that establish collaboratives with AMCs, HNs, CROs and regulatory advisors in an attempt to shorten timeless are good for many patients fighting terminal cancers, this new model seems ill-equipped to deal with terminal, pediatric, and rare cancers. Considering the difficulties of a “well-controlled” model or experiment to adapt to the rare cancer community, the lack of end-point data and the preference, though not explicitly-mandated, desire to avoid FIH pediatric trials, this new collaboration will need further revisions in order to be fully applicable to our communities.
As a member of this community and a parent that has lost my daughter to a rare, pediatric brain cancer, I uniquely understand this environment and would welcome involvement in future efforts to better equip our regulatory guidance to foster strategies in the pursuit of the cure. I represent a coalition of over 80 chapters and foundations worldwide and serve as a patient advocate and steering council member on many research initiatives.
Not only would this inspire better care and reinforce innovative invention here at home, but it might also advance therapies for those cancers that many experts believe may provide a homerun cure strategy for cancer.