The evidence base for new drugs

Marketing campaigns cast new drugs as “must haves.” In reality, the public evidence base for new drugs often leaves more questions than answers.1 2 While marketing authorization indicates that regulators judged the risk-benefit profile to be favorable, product labels rarely list and quantify these benefits and harms. Publications of premarketing trials might fill in some gaps, but readers beware: not all trials are published and those that are may be inaccurately or incompletely reported.3 4

Data transparency in clinical trials has emerged as a way to tackle the reporting biases that affect literature, enabling independent scrutiny of trials.5 But it is not enough. Even with open data we rarely know how new drugs compare with existing options or whether they improve patient centered outcomes. An analysis published in this issue (doi:10.1136/bmj.h796) suggests that new legislation in Germany may provide another piece of the puzzle.6

The German act on the reform of the market for medicinal products (AMNOG) was introduced in 2011 to inform drug pricing for all new drugs. To control costs, sponsors must submit a standardized dossier including evidence of the drug’s added benefit over already available drugs. This dossier is then reviewed by scientists, usually at the Institute for Quality and Efficiency in Health Care (IQWiG), who produce an assessment report. Their report, plus some (but not all) of the documents …