Faster drug approval: challenges for safety

Abstract

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.

Identification of cellular targets involved in cardiac failure caused by PKI in oncology: an approach combining pharmacovigilance and pharmacodynamics

“…A safety assessment is essential before the full approval of a new drug; however, clinical trials are mainly designed with the objective of efficacy, with safety often considered a secondary objective. During the last decade, rapid innovation has led to a shortened duration of drug development, from 10 years before 2010 to 6 years or less today . This trend has led to a reduced duration of exposure to new drugs and a decreased number of exposed subjects in clinical trials.…”