Reflecting on 42 Years of the Orphan Drug Act (ODA) and Its Impact on the Rare Disease Community
The 42nd anniversary of the Orphan Drug Act (ODA) in January offers a moment to reflect on its substantial impact for patients with rare diseases and their families. Signed in 1983 by Ronald Reagan, the ODA addressed the lack of treatments available for rare conditions, motivating pharmaceutical companies to develop new drugs to treat these diseases. Until then, the medical, scientific, and pharmaceutical communities had focused on researching and developing drugs that could be distributed to more people, since the development of drugs is costly and smaller rare disease communities are often too small to cover those costs by purchasing the drugs.
History of the ODA
In the 1980s, an FDA task force and a constituent of U.S. Rep. Henry Waxman of California brought the “orphan drug problem” to the attention of lawmakers, prompting Waxman to lead a bipartisan effort to address this public health concern. Waxman drafted the Orphan Drug Act to create financial incentives through tax breaks, grants, and seven-year market exclusivity to encourage the scientific and medical communities to research, develop, and administer treatment for those with rare diseases.
While leading hearings for the act in 1982, Waxman inspired his fellow lawmakers:
“For the rest of us who believe that people with rare diseases suffer no less and are no less deserving because they are so few, this situation is intolerable. It must be changed.”
The rare disease community and advocacy groups also worked to garner support for the signing of the ODA. Thanks to community, Maurice and Jack Klugman, two big names in Hollywood at the time, joined the movement. The Klugman brothers starred and produced a popular television show, Quincy, M.E., which followed a county medical examiner, and the Klugman brothers wrote several episodes that helped shine a light on the experiences of people with rare diseases. Jack Klugman later went on to testify in front of Congress in favor of the ODA. The awareness created by the rare disease community, advocacy groups, and the Klugman brothers was pivotal in the ODA being signed into law.
Successes of the ODA
Before the ODA, only 38 drugs had been approved for rare diseases; today, there are over 650, treating more than 1,000 conditions. This surge in the number of FDA-approved drugs has provided hope for millions of people affected by rare diseases. The act’s financial incentives for the medical and scientific communities—including tax breaks, grants, and seven-year market exclusivity—have been key to promoting pharmaceutical investment.
In addition to improving access to treatments, the ODA and its creation have also helped bring national attention to the rare disease cause. During its path to being signed, the ODA garnered attention from the FDA. This focus not only expedited regulatory processes but also inspired collaborations between policymakers, researchers, and patient advocacy groups to address the unique challenges of rare diseases. In fact, since the passage of the ODA, “more than 7,000 rare diseases have been identified and over 1,100 orphan indications for treatments have obtained FDA approval.” As more diseases are identified, awareness of rare diseases and the collective power of the rare disease community has grown.
For the LGDA community specifically, the ODA has been critical in improving the treatment available to patients with CLAs. Since its passing, several drugs including Sirolimus (Rapamycin), Trametinib, Zometa, Propranolol, Interferon-Alpha, and VEGF Inhibitors (e.g., Bevacizumab) were created. These drugs are not FDA-approved for CLAs but they are FDA-approved for other indications and are being used to treat patients with CLAs. Additionally, in 2022, Vijoice(r) (alpelisib) was approved by the FDA for the treatment of PIK3CA-Related Overgrowth Spectrum (PROS). It covers many rare conditions caused by the PIK3CA mutation including complex lymphatic anomalies. However, not all CLAs are affiliated with the PIK3CA mutation.
Remaining Gaps and Challenges
Despite the ODA's successes, around 95% of rare diseases still lack an FDA-approved treatment, leaving many patient communities underserved. A significant portion of approved drugs target a small subset of diseases, such as certain rare cancers, leaving many other rare conditions without attention or funding. Recent policy changes, like reductions in the Orphan Drug Tax Credit and potential drug pricing restrictions, could diminish incentives for new drug research. Advocates emphasize the importance of maintaining financial incentives for drug developers.
Looking Forward
The rare disease community is advocating for updates to the ODA, including restoring the Orphan Drug Tax Credit and re-evaluating market exclusivity rules. Legislative proposals, like H.R.7640, aim to strengthen the act to address these modern challenges. Recommendations include targeted support for diseases with limited or no treatment options and better data-sharing frameworks to accelerate research and improve patient outcomes. The future of the ODA could prioritize greater patient input in the drug development process, recognizing the unique needs of each rare disease community.
While the ODA has undoubtedly transformed the treatment landscape for rare diseases, continued advocacy, and policy evolution are essential to address the remaining gaps. By working together as a community, we can continue to push for life-changing policies for our patient community.
