Haystack Files Petition for Rulemaking for Context-Based Evidence Framework for Rare Diseases
April 9, 2026— Haystack Project announced its submission of a Petition for Rulemaking to the U.S. Food and Drug Administration (FDA), calling for a pragmatic refinement to the FDA’s approach in evaluating rare disease therapies.
The petition calls for a legally binding regulation aligning study design, endpoints, and analytical methods with the scientific/clinical characteristics of each rare disease and studied treatment. It also formalizes the role of disease-specific expertise in informing endpoints and study design.
Read the Press Release here.
Read Haystack Project’s Petition for Rulemaking here.
Read the Inside Health Policy article here.
Read the Pink Sheet article here.
"What is a Petition for Rulemaking? How Could it Improve FDA Processes for Rare Diseases?"
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Haystack Project Releases Report:
“Redefining Rigor: Fit-for-Purpose Trials to Unlock Rare Disease Therapy”
Read the White Paper here.
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. View one-pager here.
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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 “Rare Disease Approvals”
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