Haystack Project

A very special thanks to the Haystack Project patient organization members who joined us at our Scientific Workshop yesterday! You are our WHY!

We are so excited to welcome NPHP1 Family Foundation to the Haystack Project family! If you are a member of a rare disease patient organization and want to join us, please reach out to [email protected] or visit our website www.haystackproject.org to learn more about our work!

Observation #3:?NASEM recognized rare disease research is significantly complicated by factors inherent to small populations?-?gaps in knowledge of disease etiology, ethical concerns, disease severity, small sample sizes, & unmet needs. NASEM emphasized?rare requires additional methods of demonstrating substantial evidence of effectiveness?and said “[n]ew?approaches in study design and data analysis?need not require lower regulatory standards.”??Sounds?like what Haystack is working on! Come join us!? https://lnkd.in/ekCjpdpm

Observation#2 re: recent?NASEM?rare disease report: ?FDA subverted Congressional intent by limiting NASEM’s?focus to new chemical entity development. Why leave out ?repurposing?and (the often lacking)?follow-on rare indications??This limitation was not in the statute.?We know all too well incentives are sorely lacking to get?follow-on indications?on?label?in extremely rare diseases,?and?access to approved but repurposed?or off-label uses?often faces statutory prohibitions or insurmountable policies. ? https://lnkd.in/ekCjpdpm

Observation #1: While NASEM confirmed Haystack’s long-held belief of “outsized impact” to rare disease patients from the?inextricable link between FDA processes, pricing and payer decisions,?nothing in the statute directed NASEM to conflate access, pricing, and payer decisions in the study Congress required of them. We oppose efforts to begin cost containment needed across the US health care system with children dying of rare diseases. https://lnkd.in/ekCjpdpm

CEO of Haystack Project, Kara Berasi, provides public comment at the Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub https://lnkd.in/g-Q3TQXN

Finishing up the week by highlighting another article surrounding our Capitol Hill event https://lnkd.in/eFyshN2m

Haystack Project, whose membership comprises rare and ultra-rare disease patient advocacy organizations, also raised concerns about the draft guidance’s language on waivers. ? “Several of Haystack Project’s member organizations expressed concerns with FDA’s stated intentions on DAP waivers and suggested that FDA implement a presumption that studies in populations with fewer than 2,000 patients would qualify for a waiver,” the group’s?comments?state. “The final guidance should also include details and examples illustrating its view on instances for which a DAP would be impracticable.” https://lnkd.in/e9Sug-np

Check this out! Haystack Project is featured as the 'Top Story' on Inside Health Policy today!

Haystack Project in the news!