Haystack Project Releases Report:
“Redefining Rigor: Fit-for-Purpose Trials to Unlock Rare Disease Therapy”
Haystack Project’s white paper was shaped by insights from a Scientific Workshop held on March 12, 2025, with expert clinicians, researchers, statisticians, patients, and industry. Their input informed key recommendations to modernize FDA processes. These findings were shared during the White Paper Launch Webinar to drive awareness and policy change.
Read the White Paper here.
Watch the Webinar Below:
Haystack Project Support Makary’s Push for
Smart FDA Reforms in Rare Disease
In an Inside Health Policy article, the Haystack Project responds to FDA Commissioner Marty Makary’s proposal for faster rare disease drug approvals without randomized trials. While supporting quicker access, Haystack warns that lowering standards could hurt patients by leading to insurance denials. They advocate for alternative evidence pathways that maintain the FDA’s rigorous “substantial evidence” standard and call for Congressional action. Read here.
Haystack Project working with Janet Woodcock on a New Rare Disease FDA Approval Pathway
Wednesday, October 23, 2024: Capitol Hill Event - When Exceptions Need Their Own Rule: A New Rare Disease Approval Pathway
moderated by Janet Woodcock Haystack Project’s mission-focused advisor
Panelists:
Kara Berasi Haystack Project, CEO
Jeanne Ireland Former Senior Advisor to the Commissioner, FDA
David Fox Former Associate Chief Counsel, FDA
Tam Roshan Lal Chief Medical Officer, Uncommon Cure