Faster drug approval: challenges for safety.
More rapid drug premarketing procedures pose a challenge for regulatory agencies in terms of innovation and improving real-world safety and effectiveness Areas covered: This review considers the blockbuster drugs used over the previous fifteen years with adverse reactions after marketing, the elements and time span of risk identification and the measures implemented or considered, based on the existing literature and reports from the agencies Expert opinion: Risk prediction is founded on several factors: randomization, sample size, a well-established endpoint for safety, use of a comparator rather than placebo and a longer Phase-III period, in which a serious illness may be identified by early signs of alteration in the primary parenchyma with the latest biochemical, instrumental and imaging techniques. In comparative non-inferiority evaluations, increased safety should be preferred, with the exception of drugs that may be useful in serious or life-threatening diseases for which there are few or no effective existing therapies. A period of restricted use may be required to test and dispense new drugs, as well as to implement specific methods for the early detection of adverse events. It is important not to regard a new medicine axiomatically as the best treatment before it comes into wide use.
a Institute of Pharmacology , Catholic University School of Medicine , Roma , Italy.
Paolo
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Preziosi
D017277:Drug Approval / Q000379:methods*; D064420:Drug-Related Side Effects and Adverse Reactions / Q000517:prevention & control; D023381:Endpoint Determination; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D012107:Research Design; D018570:Risk Assessment / Q000379:methods*; D013997:Time Factors
Adverse drug reactions; GAIN; accelerated approval program; breakthrough drugs; clinical trial evaluation; drug regulatory measures; early warning of adverse events; expanded access program; named patient programs; priority review