LONDON, Aug 16 (APM) - Fewer than half of the drugs granted accelerated approval by the U.S. Food and Drug Administration from 2009-2013 had their efficacy confirmed in post-approval trials three years after approval, according to a new report.
A study published in JAMA on Tuesday looked at 22 drugs granted accelerated approval in 24 indications over the five year time period to analyse the impact of this process, which allows for the early approval of promising investigational medicines before the usual amount of data has been collected to confirm their effectiveness and safety.
As a consequence, companies have to collect further data in post approval studies in order for the drug to remain on the market.
The researchers from the London School of Economics and Brigham and Women's Hospital in Boston, Massachusetts, found that of the required post approval confirmatory studies, half of the total of 38 were completed a minimum of three years after approval, and of these, most showed some benefit but relied on surrogate measures rather than clinical outcomes.
Eleven more follow-up studies were underway and said to be on schedule, six others were delayed by more than 12 months, and two had been discontinued.
Just 42% of drug indications granted accelerated approval from 2009-2013 had efficacy confirmed in post approval trials a minimum of three years after approval.
Speaking to Thomson Reuters, one of the study's authors Dr Huseyin Naci, was critical of the lack in rigour for the continued evaluation of these products.
He said: "[W]e have found numerous situations in which required confirmatory studies with rigorous designs and outcomes are not pursued or are not completed in a timely fashion, and in these cases, we are concerned that regulators appear to accept data that would not otherwise meet FDA standards."
Naci added: "Our findings suggest that expediency in drug development and approval can be successful but that drugs approved via the shorter route to market are rarely subject to tests even in the post-approval period that use established and clinically meaningful outcomes."
Questions have also been raised about early access schemes for new drugs in Europe, with the European Medicines Agency (EMA)'s Dr Hans-Georg Eichler telling APM in June that extreme adverse events from drugs approved under early approval programmes, such as the PRIME scheme, could shake public confidence (APMMA 53397).
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