The Haystack Project is a 501(c)3 nonprofit that brings together patients organizations representing patients suffering from or caring for patients with extremely rare diseases. Our mission is to educate policymakers and other stakeholders about the need for policies that recognize the unique circumstances of extremely rare conditions and treatments and expand incentives critical to ensuring they can reach patients.

Haystack News

FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue→

FDA Approves First Clobazam Oral Film for Lennox-Gastaut Syndrome→

First Signs of Huntington’s Disease Detectable Via a New Blood Test→

Should FDA Treat Rare Disease Drugs Differently? – Patient advocates, industry call for looser regulation→

FDA approves new treatment for a rare genetic disorder, Fabry disease→

FDA approves first-of-its kind targeted RNA based therapy to treat a rare disease→

Taking New Steps to Meet the Challenges of Rare Diseases→

Consumer Updates – Orphan Drug Act – FDA→

What do women (and men) want? Parents weigh in on genetic testing for rare diseases in children. CDC→

FDA Could Rethink Orphan Drug Incentives – Center for Biosimilars→

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