Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813)
On May 25, 2021, the bipartisan, bicameral Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813) was introduced. FARA actively supported this bill because it had several provisions that would help expedite ataxia research: 1) it established a Health and Human Services (HHS) Public-Private Partnership for Rare Neurodegenerative Diseases between the NIH, the FDA, and eligible stakeholders with a connection to the patient population(s) to advance the understanding of rare neurodegenerative diseases and foster the effective development and evaluation of treatments; 2) it commissions the publication of an FDA Action Plan on rare neurodegenerative diseases; and 3) it created a FDA grant program to fund research and therapy development for life-threatening or severely debilitating rare neurodegenerative diseases. The ACT for ALS was passed by the House and Senate in December and signed into law by President Biden on December 23, 2021 (Public Law No: 118-79). FARA will monitor the implementation of relevant provisions this year.
COVID-19 Effects on Research
FARA will continue to address issues arising from COVID-19 that directly impact FA research. Current issues include supplemental funding, access to vaccinations, telemedicine, and application of lessons learned from the pandemic that could expedite drug development.
Info for COVID-19 and Friedreich’s Ataxia letter.
Newborn Screening Saves Lives Reauthorization Act (S. 350/H.R. 482)
The Newborn Screening Saves Lives Reauthorization Act, H.R. 482/S. 350 was introduced in the House on January 25, 2021 by Representative Lucille Roybal-Allard (CA) and in the Senate on February 19, 2021 by Senators Maggie Hassan (NH) and Roger Wicker (MS). This Act would continue critical federal programs that aid states to improve and expand their newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening. Authorizations for these programs expired in 2019.
The BENEFIT Act (S.526/H.R.1092)
The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT Act) will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, Patient-Focused Drug Development (PFDD), and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the risk-benefit assessment. This action will send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.
Click here to learn more.
PRV Program
The Rare Pediatric Disease Priority Review Voucher (PRV) Program encourages pharmaceutical companies to invest in treatments for rare pediatric diseases by offering a valuable incentive: a voucher that can be used to speed up FDA review of a future drug application or sold to another company providing crucial funds to continue rare disease research. This flexibility is often what makes it possible for high‑risk, complex drug development efforts to move forward—especially when patient populations are small and traditional market incentives are limited. As a result, the program has helped bring more than 40 new rare disease treatments to market, including Skyclarys, the first and only approved treatment for FA.
