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Rare Disease Week 2024, taking place February 25–28, is an important annual reminder of the need to find cures and improve therapies for patients. Throughout the week, patient advocates will be on Capitol Hill advocating for a range of legislative priorities to help advance the needs of patients and improve outcomes.
A disease is defined as “rare” in the United States when it affects fewer than 200,000 people. Within a U.S. context, more than 30 million Americans—1 in 10—live with one or more rare diseases.
At the moment, the national economic burden of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019. This pales in comparison, however, to the struggles of patients who have an average wait time of 6.3 years after symptoms present before ultimately receiving a confirmed diagnosis (typically after multiple incorrect diagnoses). Furthermore, 93–95% of the more than 10,000 known rare diseases have no U.S. Food and Drug Administration (FDA)-approved therapies, resulting in great uncertainty and anxiety for patients with rare diseases.
Below, we look at some of the priority issues for the Biotechnology Innovation Organization (BIO) and patient advocates, and what we’re watching during Rare Disease Week 2024.
BIO supports the ORPHAN Cures Act
This year, Rare Disease Week is a crucially important opportunity to advance support for the Optimizing Research Progress Hope and New Cures Act (ORPHAN Cures Act). BIO strongly supports this bipartisan legislation which will greatly benefit rare disease patients.
The Inflation Reduction Act (IRA) exempts orphan drugs for rare diseases from price controls—but only if the drugs are approved for a single indication. The ORPHAN Cures Act would make an essential improvement to the law. While the IRA would exempt drugs developed for a single rare disease indication from price controls, drugs that are approved for two or more indications could still be subjected to the government’s price-setting structure.
The ORPHAN Cures Act would change the IRA’s incentive structure to encourage follow-on development, meaning development for more than one indication. This legislation was introduced in the House in September, and in the Senate in October. In addition to counting on BIO’s support, this legislation is also backed by the Council of State Bioscience Associations (CSBA), the coalition of state-based BIO affiliate organizations.
Reauthorizing the Rare Pediatric Disease Priority Review Voucher (PRV) Program
The Creating Hope Reauthorization Act (H.R. 7384), bipartisan legislation, would reauthorize the PRV program for four years. This incentive program has no cost to taxpayers and helps promote innovation in treatments to address rare diseases that disproportionately impact children.
The Creating Hope Act would expand the PRV program to include drugs that treat rare pediatric diseases. The program was last reauthorized in 2020 for a four-year period. Without congressional action, companies will have to receive a Rare Pediatric designation by September 30, 2024 to be eligible. After September 30, 2026, the FDA will no longer be able to award Rare Pediatric PRVs.
The Rare Pediatric Disease PRV program is essential for developing drugs for rare pediatric diseases. This process is challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis, and more. Furthermore, about 70% of rare diseases are exclusively pediatric onset.
BIO strongly supports the passage of this legislation, a priority shared with the EveryLife Foundation for Rare Diseases.
Additional issues to watch during Rare Disease Week
In addition to the PRV program, the EveryLife Foundation for Rare Diseases is advocating for a few more issues on Capitol Hill. These include:
- Accelerating Kids’ Access to Care Act, aiming to streamline access to out-of-state specialized medical care for children with rare diseases covered by Medicaid. This bipartisan effort addresses bureaucratic hurdles that delay crucial treatments by simplifying the process for providers to serve Medicaid children across state lines.
- The Safe Step Act, which seeks to reform medication step-therapy protocols. The bipartisan bill proposes an exception process for cases where the treatment is contraindicated, ineffective, likely to cause adverse reactions, expected to decrease daily function, or when the patient is stable on current medication. It aims to ensure timely access to necessary treatments, improving health outcomes, and reducing long-term healthcare costs.
