Rethinking the appraisal and approval of drugs for type 2 diabetesBMJ 2015; 351 doi: https://doi.org/10.1136/bmj.h5260 (Published 09 October 2015) Cite this as: BMJ 2015;351:h5260
- Huseyin Naci, assistant professor of health policy 1,
- Richard Lehman, senior research fellow2,
- Olivier J Wouters, researcher 1,
- Ben Goldacre, senior clinical research fellow3,
- John S Yudkin, emeritus professor of medicine4
- 1LSE Health, Department of Social Policy, London School of Economics and Political Science, London, UK
- 2Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK
- 3Centre for Evidence Based Medicine, Nuffield Department of Primary Care Health Sciences, University of Oxford
- 4Division of Medicine, University College London, London, UK
- Correspondence to: Huseyin Naci
The aim of drug regulation is to ensure that only effective and safe treatments reach patients. Ideally, regulatory decisions are based on good quality data from large trials measuring real world, patient centred outcomes. Licensing agencies, however, routinely approve treatments on the basis of small placebo controlled trials evaluating short term, surrogate endpoints in selected populations. Consequently, medicines are commonly prescribed without good quality data on their long term benefits and harms.1 2 Current licensing standards are inadequate to predict the real world therapeutic value of new medications.3
This is particularly problematic for preventive treatments given to large populations, which should be subject to a high standard of proof of benefit and absence of appreciable harm. These drugs present interesting challenges because, firstly, the real world benefits often take many years to arise and, secondly, those benefits are often modest, although clinically important. Trials should quantify the benefits and harms for the various populations that will use these drugs, ideally using prespecified subgroups of sufficient size. This would provide clinicians and patients with dependable knowledge for shared decision making. In this article we use the example of drugs for type 2 diabetes to highlight the shortcomings of the evidence standards for licensing, reimbursing, and adopting new preventive drugs and suggest some solutions.
Time to curb regulatory enthusiasm for “timely market access”
Blood glucose lowering is the only benchmark used by regulatory agencies to grant market approval to antidiabetic drugs (box 1). There are more than 30 antidiabetic drugs on the market, and for most we have insufficient evidence from randomised trials about their long term clinical benefits or harms.
Box 1: Link between glucose control and diabetic complications
Is there a strong link between blood glucose and diabetes complications?
Complications of diabetes show a correlation with blood …