The Origin of First-in-Class Drugs: Innovation Versus Clinical Benefit.

First-in-class (FIC) designation became a hallmark of innovation, however, even at the marketing authorization stage, little is known about the clinical benefits these products deliver. We identified the provenance of the FIC drugs that entered the French market from 2008 to 2018 and matched these medicines to the clinical benefit grading by Haute Autorité de Santé (HAS) and Prescrire. Analyses were performed using descriptive statistics to present our findings by drug origin and therapeutic area and to establish the degree of concordance between HAS and Prescrire. Of the 135 FIC drugs identified, 71.1% (n = 96) originated from the industry, 16.3% (n = 22) from academia, and 12.6% (n = 17) from joint partnerships. Three therapeutic areas accounted for most FIC medications: antineoplastic (25.9%, N = 35), anti-infective (14.1%, N = 19), and metabolic (11.1%, N = 15) agents. HAS and Prescrire agreed on 60.74% of clinical benefit gradings. According to HAS, only 5% of all FIC drugs had substantial added benefit, and only 3%, according to Prescrire. HAS and Prescrire graded 45.9% and 68.2%, respectively, of FIC drugs as no clinical benefit and 48.9% and 28.9%, respectively, as some clinical benefit. FIC-designated drugs are primarily of industry (> 70%) rather than academic origin. We found that 55% of FIC medicines that entered the French market over the 10-year period deliver no additional clinical benefit. Whereas FIC medicines may represent important scientific advancements in drug development, in > 50% of cases, the new mode of action does not translate into additional clinical benefits for patients.
(© 2023 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.)