Numerous pharmaceuticals are approved and marketed based on the premise that they favorably change disease state-biomarkers. However, recent data has shown that altering the biomarkers indicative of disease does not necessarily translate to ameliorated health. In an effort to improve the quality of drugs on the market, FDA has intervened by requiring companies to run outcome studies before granting drug approval. FDA has placed particular emphasis on chronic disease pharmaceuticals, which are typically intended for long-term use by patients. To address FDA’s guidelines and the payers’ concerns about the drugs, companies are often conducting large clinical trials, termed ‘Mega-Trials’ that aim to demonstrate the benefits over time of licensed drugs compared to generic or already established therapy drugs.
In a recent article on Forbes.com, Dr.John LaMattina, former president of Pfizer R&D and senior partner at PureTech Ventures, describes the conundrum discussed above using various marketed drugs to exemplify the issue. Dr. LaMattina also comments on an article in the Journal of the American Medical Association (JAMA), entitled ‘Mega-Trials for Blockbusters’ by Dr. John Ioannidis. In the JAMA article, Dr. Ioannidis calls for an increase in large clinical trials. While Dr.LaMattina agrees that large clinical trials are necessary, he critiques Dr. Ioannidis’ recommendations and proposals for carrying out such trials, citing cost as a major issue. To read Dr. LaMattina’s full commentary on Forbes.com, click here.
What do you think about the practicality of ‘Mega-Trials?’ Do the trials improve the quality of drugs on the market, or ultimately hinder the efficiency of the pipeline?