The United for Medical Research released the latest in their "Why Invest in NIH?" series. "Enabling Patient Stories" is a fact sheet that profiles five individuals who were a part of biomedical breakthroughs. The breakthroughs, which have transformed lives, were made possible by NIH-funded research.

Read and share "Why Invest in NIH?" series to remind policymakers and the public why funding NIH is essential to helping sustain and advance lifesaving biomedical research and innovation. Tag your social media posts with #keepNIHstrong.

Samuel Kirton, NAF President, talks about the importance of advocacy work in rare disease communities and opportunities to get involved. Kirton says, “Advocacy efforts do make a difference. For instance, the National Ataxia Foundation and Friedreich’s Ataxia Research Alliance worked together through two federal budget cycles to add Friedreich’s ataxia to the Peer Reviewed Medical Research Program (PRMRP) for fiscal year 22.”

You can read his full article in the latest edition of Pulmonary Fibrosis News.

Provided by American Brain Coalition and Alliance for a Stronger FDA.

The House Appropriations Committee approved its Fiscal Year 2023 Labor, Health and Human Services, Education, and Related Agencies Appropriations bill. The House committee would provide a total of $47.5 billion for the National Institutes of Health (NIH), an increase of $2.5 billion over the FY 2022 enacted level. The committee bill also proposes $620 million for the BRAIN Initiative, equal to the FY 2022 level of funding. Under the committee’s bill, the Advanced Research Projects Agency for Health (ARPA-H), funded separately from NIH, would receive $2.75 billion in FY23. More detail on these House appropriations bills and their associated reports is available here.

The House Adopts FY23 Appropriations Minibus Including Ag/FDA Funding. Under the bill, BA (budget authority) funding for FDA would reach $3.645 billion, an increase of $341 million (10%). More detail on these bills is available here.

The House passed H.R. 7667 in a 392-28 vote. H.R. 7667 is a bipartisan bill that is an important step forward in reauthorizing the FDA’s medical product user fee programs (PDUFA) and helps strengthen the accelerated approval pathway that protects the intent of the Orphan Drug Act. This passage of amendments is vital for the passage of PDUFA to happen by its expiration. It is now up to the Senate to pass the proposed amendments for PDUFA in H.R. 7667.

The FDA's Center for Drug Evaluation and Research rolled out a new program to expedite and increase the development of new therapies for rare diseases. The Accelerating Rare Disease Cures (ARC) Program will leverage regulatory and scientific engagement to promote the development and approval of these treatments. For further detail, read a recent review here by Rachel Sher, formerly NORD’s VP for Public Policy and Regulatory Affairs.

The National Library of Medicine is recruiting participants for feedback as part of the ClinicalTrials.gov modernization effort.

If you are a patient, patient advocate, family member, or health care provider, the ClinicalTrials.gov modernization team wants to speak with you! Participants will be asked to perform tasks on the Beta website, complete a survey or card sorting activity, or to participate in a virtual interview to help improve the experience for users. The duration may range from 15 to 60 minutes.

Please email catherine.kihara@nih.gov to learn more.

The US Food and Drug Administration (FDA) is requesting your recommendations for patient representatives to serve on its advisory committees or panels. The FDA relies on its many advisory committees to help them make sound decisions based on the best science available.

Patient organizations interested in participating in the selection of an appropriate voting or nonvoting member to represent patient interests on an FDA advisory committee or panel may send a letter or email to ACOMSSubmissions@fda.hhs.gov stating that interest by Monday, August 15, 2022.

Patient representatives may be self-nominated or may be nominated by a patient organization. Patient representative nominations should be submitted electronically through the FDA Advisory Committee Membership Nomination Portal by August 15, 2022.

Attendees will meet with their Members of Congress in-person in Washington, DC on September 14^th. A reception to celebrate medical research will be held on September 13th for all partners and participants.