Celebrating 40 years of the Orphan Drug Act on Rare Disease Day

While the advancements of rare disease treatments are truly remarkable, last year, Congress took a step backwards by including drug price-setting provisions as part of the Inflation Reduction Act (IRA).

Headshot of Abigail Lore
Abigail LoreFebruary 28, 2023

Celebrating 40 years of the Orphan Drug Act on Rare Disease Day.

For over a decade, Rare Disease Day has been celebrated on February 28 to raise awareness and generate change for the more than 30 million patients in the U.S. living with a rare disease. This year’s Rare Disease Day is particularly special because this year also marks the 40th anniversary of the Orphan Drug Act (ODA), allowing us to celebrate all the progress that’s come to patients because of it.

Here are some key things you should know about the ODA:

The ODA has supported the development of hundreds of new treatment options for patients living with rare diseases who previously had no options.

There are unique challenges of researching and developing medicines for diseases with a U.S. population of less than 200,000 people. Recognizing that, Congress passed the ODA in 1983 through bipartisan support. Because previous incentives had been insufficient, the ODA provides important targeted incentives to help protect investments in, and encourage companies to embark on, the necessary but risky research needed for rare disease drug development. Since the passage of the ODA, over 600 orphan drugs have been approved, including 20 new FDA approvals for rare diseases in 2022 alone. In the decade before the ODA was passed, only ten drugs were brought to market to treat rare diseases.

The unique challenges in developing new treatments for patients with rare diseases may deter investment in this area without the incentives of the ODA. Potential challenges include:

  • The complexity of rare diseases and uncertainty about underlying causes
  • Affected populations are small and may be spread out across the country, or world
  • Physicians may be unfamiliar with the disease
  • There may be many subtypes to the same disease, each with their own complexities

These considerations, along with the challenges of recruiting and retaining patients for trials, may impact the length and overall success rates of clinical trials for rare disease treatments. As of 2019, overall development for rare diseases takes nearly four years longer than non-rare disease conditions and the success rate of orphan drugs in clinical trials is estimated to be only six percent.

Despite significant progress in the fight to treat rare diseases, unmet patient needs remain.

Although each rare disease impacts less than 200,000 Americans, their impact on public health is far-reaching. In total, rare diseases affect more than 30 million Americans, but less than 10% of all rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment. The need for new treatments and cures is reflected in the unique hardships and challenges that patients and their families face. Obtaining a diagnosis can be a long arduous process due to lack of physician awareness and access to screening. And once a diagnosis is obtained, often later in disease progression, the prognosis may be life threatening. The burden of rare disease is a driving factor in the commitment of the industry to help these patients and their families.

To help address these needs and improve the quality of life for patients and their families, biopharmaceutical researchers are committed to fighting rare diseases. In fact, today, there are more than 700 medicines in development to treat many of these devastating illnesses.

To meet the unmet needs of rare disease patients, policymakers must prioritize policies fostering innovation and not undermine the success of the ODA.

While the advancements of rare disease treatments are truly remarkable, last year, Congress took a step backwards by including drug price-setting provisions as part of the Inflation Reduction Act (IRA). These provisions undermine the previous bipartisan support for incentivizing rare disease drug development.

In particular, the law inadvertently discourages post-approval R&D, which is common in orphan drug development. For example, post approval R&D on medicines initially approved for use in adults with rheumatoid arthritis (RA) subsequently led to the approval of those medicines in juvenile idiopathic arthritis (JIA), providing children with this disease the ability to slow disease progression and prevent significant disability later in life. JIA is a group of six rare, often severe autoimmune or autoinflammatory diseases which cause the patient’s immune system to mistakenly attack the body’s own tissue. Post-approval research is critical in driving treatment advances for patients with orphan conditions such as JIA.

  • In 2012, a medicine was approved to treat adults with RA. Additional research led to the discovery that adult RA was closely related to JIA, and the medicine was subsequently approved for patients two years and older who have a particular form of JIA impacting the joints (polyarticular JIA).
  • In 2013, a different medicine was approved for adults with RA. In 2020 the medicine received two additional approvals for use in children and adolescents with 2 other forms of JIA that previously had no disease-modifying treatment options—including polyarticular JIA and another form (psoriatic).

R&D to develop treatments to address unmet needs in rare diseases is highly risky and takes significant time and investment. While the IRA does provide a specific exemption from price setting for medicines with a single orphan designation, the exemption is far too narrow and undermines the ODA incentives for developing new treatments for rare diseases. This forces companies to make hard choices when it comes to R&D investment. In a recent survey of PhRMA member companies on the impacts of the IRA, a majority reported that the price setting timelines in the law would cause them to reconsider investing in medicines for small patient populations and areas of high scientific uncertainty such as rare diseases.

Despite this new decision paradigm for rare disease R&D by the price setting provisions in the IRA, the biopharmaceutical industry is committed to fighting rare diseases just as they have for the last 40 years. On behalf of the scientists and researchers who have dedicated their life to rare disease patients and their families, I'm glad we get to celebrate and reflect on the progress we’ve made.

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