A first-in-class medication is a pharmaceutical that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's

Center for Drug Evaluation and Research The Center for Drug Evaluation and Research (CDER, pronounced "see'-der") is a division of the U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmetic Act. Some biological products are also leg ...

tracks first-in-class medications and reports on them annually, first-in-class is not considered a regulatory category. Although many first-in-class medications qualify as breakthrough therapies, Regenerative Medicine Advanced Therapies and/or

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

s, first-in-class status itself has no regulatory effect.

Examples

Controversy

Safety

By definition, a first-in-class drug does not have the safety evidence from analogous products that not-first-in-class drugs would have. However, a study investigating recalls and warnings in relation to first-in-class drugs approved between 1997 and 2012 by

Health Canada Health Canada (HC; french: Santé Canada, SC)Health Canada is the applied title under the Federal Identity Program; the legal title is Department of Health (). is the Structure of the Canadian federal government#Departments, with subsidiary unit ...

has found that first-in-class drugs actually have a more favourable benefit-to-harm ratio.

Economics

First-in-class drugs are often seen as commercially more attractive as they may tap into a market segment that has hitherto been underserved, but this may be illusory. In fact, most blockbuster drugs (drugs with annual sales revenues exceeding ) were not first-in-class drugs. The economic potential of a first-in-class drug, which is typically priced higher than later drugs in the same class, has been largely declining due to efforts by

health insurers Health insurance or medical insurance (also known as medical aid in South Africa) is a type of insurance that covers the whole or a part of the risk of a person incurring medical expenses. As with other types of insurance, risk is shared among ma ...

to restrict what

specialty drugs Specialty drugs or specialty pharmaceuticals are a recent designation of pharmaceuticals that are classified as high-cost, high complexity and/or high touch. Specialty drugs are often biologics—"drugs derived from living cells" that are injectab ...

are covered and prevent incumbency advantages.

Costs

A lower number of available therapeutic options correlates with higher prices. In addition, many first-in-class medications are

and

s, which means that manufacturers have to recoup development costs from a smaller market. This raises ethical questions about the sustainability of the high prices on these costs.{{Cite journal, last=Greene, first=Jan, date=January 2017, title=EpiPen Controversy Reveals Complexity Behind Drug Price Tags, url=https://www.annemergmed.com/article/S0196-0644(16)31329-4/fulltext, journal=Annals of Emergency Medicine, language=English, volume=69, issue=1, pages=A16–A19, doi=10.1016/j.annemergmed.2016.10.025, issn=0196-0644, doi-access=free

References

Pharmaceuticals policy Food and Drug Administration Pharmaceutical regulation in the United States