Opinion in 2013

Regulatory agencies have been criticized both for being overly tolerant of risks or being excessively risk-averse, but the potential for adverse effects on public health owing to the absence of new drugs because of regulatory risk-aversion is less apparent. Here, Eichler and colleagues discuss the consequences of regulatory risk-aversion and suggest what might be done to best align acceptance of risk and uncertainty by regulators with the interests of public health.

Here, the authors discuss issues relating to the co-development of targeted cancer therapies and companion diagnostics that were not covered in depth in the draft guidance released by the US Food and Drug Administration in 2011. They propose potential strategies that will be useful to mitigate challenges and to help guide the future co-development of drugs and diagnostics.

There is substantial debate over the best approach for developing transformative drugs and how this might be supported. To help inform this debate by improving the understanding of the characteristics of transformative drug innovation, the authors surveyed a US-based group of ∼180 expert physicians in 15 medical specialities, who identified the drugs that they considered to be the most transformative in their fields over the past 25 years, as well as key factors affecting their opinions.

The therapeutic outcome of a drug or procedure is influenced by the placebo response in both drug development and clinical practice. Enck and colleagues examine how the placebo response can be utilized in these settings to ensure that the most desirable outcome is attained.

Pharmacogenetics is an increasingly important tool for drug development, which has been reflected in recent guidelines from regulatory agencies on the application of pharmacogenetics in studies of investigational drugs. In this article, authors from Europe, the United States and Japan overview the guidelines from the regulatory agencies in each region and discuss the common themes and differences.