October 5, 2017

expert reaction to evidence for cancer drugs approved by the EMA

The BMJ has published a study investigating the effectiveness of cancer drugs approved by the European Medicines Agency (EMA).

Prof. Carl Heneghan, Professor of Evidence-Based Medicine & Director of the Centre for Evidence-Based Medicine, University of Oxford, said:

“This systematic evaluation of 48 cancer drugs approved in Europe shows disappointingly that the benefits of many new drugs are small regarding survival. It is hard to understand why half the drugs were approved in the first place if they provide no clinically meaningful benefit. Beyond survival, what matters is the gain in quality of life for any new treatment offered, however, the large majority (90%) of new drugs provided no improvement in this important outcome.

“Selective reporting might have prevented full access to the data and therefore led to inconsistencies in the results. This remains a problem in that some studies might go unpublished, or when published they omit essential detail about the methods, the intervention or the outcomes.

“What this analysis does show is that the evaluation of new cancer needs to be more rigorous in its approach: only approving those drugs that make a difference to patient care and are backed by robust evidence and importantly provide clinically meaningful differences.”

Prof. Winette van der Graaf, Professor of Personalised Oncology, The Institute of Cancer Research, London, and Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, said:

“In recent years regulators such as the European Medicines Agency have started to show more flexibility in their approval of cancer drugs – we absolutely applaud them for this. If we want to speed up access for patients to the latest, most innovative new treatments, it’s essential that regulatory bodies and indeed NICE are willing to look at data other than just overall survival from large phase III randomised trials.

“It takes a long time and costs a lot of money to take treatments through phase III trials and demonstrate an overall survival benefit. Showing more flexibility, such as by evaluating data from smaller, smarter trials where drugs are targeted at those patients who are most likely to benefit, is essential if we are to get new drugs to patients quickly and at a cost that is affordable for the NHS.

“In my area of research of rare cancers, the level of evidence called for here is very hard to obtain, meaning that these patients would find it extremely difficult to gain access to new treatments.

“But the authors are right in saying that there is room for improvement in the design of clinical studies. Ideally, studies should try also measuring early markers of treatment failure, so that health authorities can make well-balanced decisions. We also need to look at different types of clinical studies, including the evaluation of the impact on quality of life, and to continue to collect data after approval in a clinical setting.”

* ‘Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13’ by Davis et al. published in The BMJ on Thursday 5th October.  

Declared interests

Prof. Carl Heneghan: “Carl Heneghan is a founder of AllTrials, which call for calls for all past and present clinical trials to be registered and their full methods and summary results reported.”

Prof. Winette van der Graaf: No conflicts of interest.