When it comes to cancer, not all diagnoses are created equal.
While some cancers, like breast or lung cancer, benefit from extensive research, abundant clinical trials, and a robust treatment arsenal, others remain in the shadows. Rare cancers, including mesothelioma, sarcomas and pediatric cancers, often lack sufficient treatment options due to limited funding, small patient populations, and minimal commercial incentives for drug development.
That’s where the FDA’s Orphan Drug Designation (ODD) steps in to help.
What Is Orphan Drug Designation?
The Orphan Drug Designation is a special status granted by the U.S. Food and Drug Administration (FDA) to drugs and biologics intended to treat, diagnose, or prevent rare diseases or conditions. For a disease to qualify as “rare,” it must affect fewer than 200,000 people in the United States.
Mesothelioma, for example, is a rare cancer that starts in the linings of the lungs, abdominal cavity or heart. The disease is diagnosed in just 2,500 people in the U.S. each year. Treatment is limited to surgery, chemotherapy, immunotherapy and radiation – and often is not especially effective for patients.
In the context of cancer, many aggressive or less common types – such as mesothelioma, anaplastic thyroid cancer, or certain pediatric brain tumors – fall into this category. ODD is not an approval of the drug itself, but rather a way to incentivize developers to bring promising new therapies to the table.
What Makes ODD Important?
Orphan Drug Designation plays a vital role in advancing cancer care by addressing the critical gap in treatment development for rare and hard-to-treat malignancies. Many cancers affect relatively small patient populations and are often overlooked in mainstream drug development due to limited commercial incentives.
By offering benefits like market exclusivity, tax credits and regulatory support, ODD encourages pharmaceutical companies to invest in innovative therapies that might otherwise be deemed “financially unviable.” This framework not only accelerates the availability of life-saving treatments for underserved cancer patients but also supports a more equitable and inclusive approach to oncology research – ensuring that even the rarest cancers are not left behind.
Orphan Drugs and Mesothelioma Care
If we take a look at mesothelioma, a rare and aggressive cancer primarily caused by asbestos exposure, it has historically lacked treatment innovation. However, the ODD program has helped change that landscape.
One example is nivolumab (Opdivo), an immune checkpoint inhibitor that received orphan designation for malignant pleural mesothelioma. The designation supported faster development and eventual approval from the FDA in combination with ipilimumab (Yervoy) for unresectable cases, improving survival outcomes for many patients.
Similarly, novel CAR T-cell therapies and targeted inhibitors for rare molecular subtypes (like ALK, ROS1, or BAP1 gene mutations) are now being explored under the ODD umbrella – offering new hope.
Mesothelioma actually got headlines recently for an oncolytic virus receiving ODD for the cancer. Oncolytic viruses use engineered forms of viruses to infect and kill cancer cells – and alert the immune system to the tumor’s presence.
Ethical and Practical Challenges
Though effective, the ODD program isn’t perfect. Critics point out that some companies may exploit the designation by segmenting common diseases into rare subgroups to qualify. Others argue that market exclusivity can drive up costs after FDA approval. Yet, the consensus remains: for genuinely rare and devastating cancers, ODD is a lifeline.
Looking Ahead
The future of cancer care depends not just on treating the common diseases but also championing treatment for the rare conditions. As we move toward personalized oncology, more rare types of cancer may qualify for orphan status – opening the door to targeted, life-saving therapies.
Orphan Drug Designation is more than a label. It’s a commitment to innovation where it’s needed most.
Sources & Author
- Miller KD, Nogueira L, Devasia T, et al. Cancer treatment and survivorship statistics, 2022. CA Cancer J Clin. 2022;72(5):409-436. doi:10.3322/caac.21731.
- Baas P, Scherpereel A, Nowak AK, et al. First-line nivolumab plus ipilimumab in unresectable malignant pleural mesothelioma (CheckMate 743): a multicentre, randomised, open-label, phase 3 trial [published correction appears in Lancet. 2021 Feb 20;397(10275):670. doi: 10.1016/S0140-6736(21)00369-X.]. Lancet. 2021;397(10272):375-386. doi:10.1016/S0140-6736(20)32714-8.
- Vokinger KN, Kesselheim AS. Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU. BMJ Open. 2019;9(10):e028634. Published 2019 Oct 10. doi:10.1136/bmjopen-2018-028634.
- Ipe, A. V., Fermaglich, L. J., Kraft, S., Mease, C., Le, T., & Miller, K. L. (2024). The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022. Expert Opinion on Orphan Drugs, 12(1), 12–18. https://doi.org/10.1080/21678707.2024.2333245.
- Fermaglich LJ, Miller KL. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act. Orphanet J Rare Dis. 2023;18(1):163. Published 2023 Jun 23. doi:10.1186/s13023-023-02790-7.
Sources & Author
