Orphan Drug Exclusivity and Catalyst: A Possible Catalyst for Gaps in Treatment Within Subpopulations of Rare Diseases and Conditions

Orphan Drug Exclusivity and Catalyst: A Possible Catalyst for Gaps in Treatment Within Subpopulations of Rare Diseases and Conditions

Journal article

1 online resource

Rare diseases are often left undiagnosed or are misdiagnosed, and people with rare diseases, thus, are often left looking at their physicians with hope for something more. And when they cannot find something more from that physician, they may need to cross multiple state lines to speak with an expert who can provide proper treatment for their rare disease, being one of the few with experience in treating and studying that disease. Considering the dearth of knowledge regarding rare diseases, and how there is yet a further need for understanding and treatment, it is important to balance encouragement of innovation under the Orphan Drug Act (“ODA”) through providing exclusivity while also ensuring that the entire population with a rare disease or condition has access to apt research and development (“R&D”), and treatment. In this sense, pharmaceutical companies must be incentivized to treat rare diseases, especially because they typically might not recoup costs to invent and produce a drug for a specific use for a rare disease. Particularly, this considered, Congress “[r]ecogniz[ed] that pharmaceutical manufacturers could ‘reasonably expect ... to incur a financial loss' for drugs that treat only a small number of patients, [and] concluded that ‘it [wa]s in the public interest to provide ... incentives' .... As such, Congress included an orphan drug exclusivity (“ODE”) provision in the ODA which prevents FDA from approving “the same drug for the same [rare] disease or condition” as an approved orphan drug or biological product until seven years have expired from the date the orphan drug was approved or licensed for the designated orphan disease or condition.

Crossref

AIPLA Quarterly Journal, Fall 2023, Volume 51, Issue 4, p. 517.

[New York, NY] : Thomson Reuters, 2024.

English

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