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 l ...
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 o ...
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 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
Blockbuster or Block Buster may refer to:
*Blockbuster (entertainment) a term coined for an extremely successful movie, from which most other uses are derived.
Corporations
* Blockbuster (retailer), a defunct video and game rental chain
** Blo ...
(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 injecta ...
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
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 injecta ...
and
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 o ...
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