Washington is currently abuzz with proposals that directly impact research. The Advanced Research Project Agency for Health (ARPA-H) just launched, the Prescription Drug User Fee Act (PDUFA) needs to be reauthorized by September 30, Cures 2.0 is being drafted and on top of all this, FY 23 Appropriations are being negotiated. It is an exciting time to be an advocate for FA research!
One of topics that has come up during these discussions is possible changes to the FDA Accelerated Approval Program. Accelerated approval is an alternative approval process for new drugs that allows drugs to get to market quicker. The FDA established this pathway in 1992 during the AIDS epidemic with a specific target toward potential therapies for “serious conditions that filled an unmet medical need.” While granting patients treatment access at the earliest potential moment, the accelerated approval pathway is not any less rigorous than the traditional new drug approval process.
The main differentiator between accelerated approval and the traditional pathway is the use of a surrogate endpoint. Also called a biomarker, it is a measure thought to predict clinical benefit while not being a measure of clinical benefit. For example, in oncology, tumor shrinkage would be an appropriate surrogate endpoint instead of waiting to observe decreased mortality rates. Surrogate endpoints can more accurately capture real-time progression and improvement.
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The accelerated approval process is composed of 3 parts: application, approval, and post-accelerated approval. A drug company will first submit its application to the FDA for data review about the efficacy, safety, and effectiveness of the drug. If the data shows benefit, the FDA will then approve the drug, and the drug will go to market. Under this approval, the FDA will specify a timeline for required future testing and post-market surveillance. In the post-accelerated approval stage, scientists must conduct additional research to verify the expected outcomes as the drug remains on the market. However, approval may be withdrawn and/or labeled indications of the drug may be changed if trials fail to show clinical benefit or reveal an unjustifiable risk-benefit ratio.
Accelerated approval is different from the FDA’s fast-track designation. Fast-track designation expedites the review of a new drug application and works with other FDA programs like accelerated approval and priority review designation if the relevant eligibility criteria are met. For example, Reata’s Omaveloxolone was granted fast-track designation which allowed for an expedited review of the new drug application and priority review designation which shortens the review time from ten to six months.
FARA will continue to monitor any developments around this program. For more information on accelerated approval, check out the excellent resource created by the EveryLife Foundation.
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On May 25, HHS Secretary Xavier Becerra announced the formal establishment of ARPA-H “as an independent entity within the National Institutes of Health” and the appointment of Adam H. Russell, DPhil, a former DARPA program manager, as Acting Deputy Director. ARPA-H will provide a novel pathway to catalyzing transformative health breakthroughs that cannot readily be accomplished through traditional research or commercial activity.
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Prescription Drug User Fee Act (PDUFA) Released
The House Energy & Commerce Committee released the legislation for the updated user fee agreements on May 4th. The section by section summary and full text is available for your review. Click here for background information on PDUFA courtesy of the EveryLife Foundation.
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Congressional Hearings Held
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The House Energy & Commerce Committee held a hearing on March 17th titled The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight. The discussions included numerous health related bills including ARPA-H, Cures 2.0, PDFUA (Prescription Drug User Fee Act) and the BENEFIT Act to name a few. To view the recording of the hearing, click here.
On May 17, the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies focused on the NIH. To view the recording, click here.
As noted from our partner Research!America, securing a meaningful increase for NIH as well as boosted funding for other key health agencies pivots on the amount of overall funding the subcommittee receives when total FY23 appropriations are allocated across all 12 appropriations subcommittees. FARA joined Research!American and 115 advocacy partners in sending a letter to House and Senate appropriators making the case for a robust Labor-H Subcommittee funding allocation.
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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.
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RDLA is hosting a Rare Disease State Advocacy Day in Boston, Massachusetts on Thursday, June 9th, 2022. This event is an opportunity for Massachusetts advocates to meet with their state legislations for the purpose of educating them on rare disease and help advancing the policy priorities of the rare disease community. Please note this event is open to Massachusetts residents ONLY. Registration is now closed but you can email Rachelle Raudes at: rraudes@everylifefoundation.org to be placed on the waitlist.
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Registration for the EveryLife YARR Leadership Summer Series 2022 is open!
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Gain knowledge and confidence with sessions on timely advocacy topics hosted by and for young adults in the rare disease community.
PDUFA (June 8th)
Accelerated Approval (July 14th)
LinkedIn (August 10th)
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Register now for Rare Across America 2022:
Virtual and In-Person Meetings
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Meet other rare disease advocates.
Share your rare disease story.
Make an impact in your district.
Webinar training on July 14th & 19th
Meeting with Members of Congress:
August 8th - 19th
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Save the Date - United Against Ataxia Hill Day! |
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The fourth annual United Against Ataxia Hill Day will be held virtually on Wednesday, September 21, 2022. Mark your calendars now so that you will not miss this important event!
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Newborn Screening State News
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Iowa Signs RUSP Alignment Bill into Law
On April 21st, the Iowa Governor signed SF 2345 into law. The law implements a two-and-a-half-year timeline in which screening must begin for new conditions added to the federal Recommended Uniform Screening Panel (RUSP) and ensures that the Iowa Department of Health provides an annual status report on newborn screening. Iowa is the second state this year to adopt RUSP alignment legislation and the ninth overall.
Maryland Signs RUSP Alignment Bill into Law
Maryland became the third state to adopt RUSP alignment legislation this year and the tenth overall.
These laws are a result of increased advocacy efforts. To learn more about the legislation, see what your state is screening for, and learn how to support newborn screening, visit rarescreening.org.
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BIG NEWS FROM NORD ON
RARE DISEASE ADVISORY COUNCILS (RDACs)
The past two weeks have been an exciting time for Project RDAC. Legislation was signed into law in Maine and Georgia enacting RDACs and additional RDAC legislation passed through Connecticut and Colorado's state legislatures and await the Governor’s signature!
None of this incredible progress would have been possible without the advocates and grassroots volunteers who reached out to their lawmakers to move these important bills forward. For more information on NORD’s Project RDAC, click here.
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The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals with, or affiliated with, a rare disease to tell their story through art. Now in year 12, the Rare Artist Annual Contest is focused on providing a national platform for artists to advocate through visual artwork and powerful artist statements. Rare Artist finalists participate in individualized artist-to-advocate coaching to strengthen their advocacy voice. Rare Artist awardees are presented with checks and invited to speak during Rare Disease Week on Capitol Hill in Washington, D.C. Their artwork is showcased throughout the year at various patient and biopharmaceutical conferences, displayed at the Rare Hub, social media, and website posts. |
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Rare Artist Contest 2022
Submissions Open June 1st - 13th
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