Improving the drug development process: More not less randomized trials
A partir de données de la Food and Drug Administration entre 2000 et 2012, ces deux études analysent, pour l'une, les caractéristiques des essais cliniques ayant servi de base à une autorisation de mise sur le marché d'une nouvelle molécule et, pour l'autre, les raisons scientifiques et réglementaires liées à un retard ou un refus d'autorisation
Drug development is often a lengthy and expensive process. Extensive preclinical testing via in vitro and animal experimentation aims to select drugs most likely to work in humans. Under the current system, only about half of the drugs succeed in moving from phase 1 (dose-finding) to phase 2 (safety and efficacy). For drugs that enter phase 2, less than 1 in 3 succeed; for those entering phase 3 (pivotal efficacy), that number decreases to less than 1 in 2. Less than 20% of drugs entering phase 1 testing successfully reach the end of the 3-phase evaluation. The percentage can vary from one specialty area to another, and it can be less than 5% to 10% for oncologic and neurologic diseases.
JAMA , commentaire, 2013