Recognizing the 40th Anniversary of the Orphan Drug Act, the Rare Disease Company Coalition calls on policymakers to renew commitment to advancing innovation for rare disease patients

Rare Disease Company Coalition

Recognizing the 40th Anniversary of the Orphan Drug Act, the Rare Disease Company Coalition calls on policymakers to renew commitment to advancing innovation for rare disease patients

WASHINGTON, DC – As 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), the Rare Disease Company Coalition (RDCC) recognizes the advances made over the past four decades and calls on Congress to renew its support for policies that promote investment and innovation in rare disease treatments.

The ODA has spurred biopharmaceutical companies to pursue treatments that would otherwise be financially unfeasible. ODA policies reduce the risk associated with rare disease research and development (R&D) and incentivize companies to discover new treatments. Since its enactment in 1983, the number of orphan drugs approved by the Food and Drug Administration (FDA) has increased by 1,576% – from just 38 to more than 600 treatments for more than 1,000 rare diseases. Today, RDCC members collectively have over 200 promising rare disease treatments currently in development.

As we recognize the ODA’s role in incentivizing biomedical innovation, we also acknowledge that the vast majority of rare disease patients still lack any FDA-approved treatment options, necessitating urgent policy considerations to bring hope to the 1 in 10 Americans suffering from a rare disease.

“Over the past 40 years, the ODA has established a strong foundation for the biotech community to invest in and develop new medicines for rare diseases,” says Amanda Malakoff, Executive Director, Rare Disease Company Coalition. “We need to build on that strong foundation so we can ensure continued innovation for the 95% of rare diseases that currently have no approved treatments.”

Researchers and developers rely on steady and supportive policy and regulatory environments to reduce the risk of investing in new treatments for small patient populations. While the ODA has supported a stable environment for the past 40 years, recent policy changes threaten ODA-established incentives like the Orphan Drug Tax Credit. New one-size-fits-all policy proposals fail to fully address the nuances of rare disease research and development and would drastically undermine this progress.

“When you look beyond rare diseases that are currently being treated or have the potential for new therapies, you see millions of people who now have hope thanks to the ODA,” said Nevan Charles Elam, JD, Chief Executive Officer & Founder, Rezolute Bio. “While the disease may be rare, the people are not, and without the ODA, the incentive to research and develop new therapies for patients in need would simply not be there.”

Continued progress requires policymakers be active and intentional about enacting and protecting policies that build on 40 years of the ODA. The RDCC calls on policymakers to:

  • Bolster the Orphan Drug Tax Credit to its original 50% of qualified R&D costs by advancing legislation to ensure stability and protect advances made in rare diseases research and development.
  • Support exemptions for rare disease treatments in the Inflation Reduction Act (IRA) and future drug pricing policies to ensure that future research for new rare indications are not put at risk.
  • Incentivize companies to complete promising clinical trials that were put on pause during the pandemic.
  • Make permanent the priority review vouchers (PRVs) that are set to expire between 2024-2026 and consider new types of incentive modeled on the PRV.

“In recognizing the 40th anniversary of the ODA, it is more important than ever for policymakers, patients, patient advocacy groups, providers, researchers, and manufacturers to come together and reaffirm their commitment to the rare disease community by showing steadfast support for upholding and strengthening the ODA,” Malakoff concludes.

To learn more about the Orphan Drug Act and policy priorities from the Rare Disease Company Coalition, please visit

About the Rare Disease Company Coalition (RDCC)

Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases.

Source: RDCC