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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

BMJ 2017; 359 doi: https://doi.org/10.1136/bmj.j4530 (Published 04 October 2017) Cite this as: BMJ 2017;359:j4530

Rapid Response:

Re: 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

“We demand rigidly defined areas of doubt and uncertainty!” This quote was lifted from Douglas Adams’ Hitchhiker’s Guide to the Galaxy and is perceived by most to be absurd (those who thought it was extracted from NICE’s Guide to the methods of technology appraisal, go to the bottom of the class).

But you might be forgiven for expressing exactly that sentiment after reading this article. The researchers found that most oncology drugs entering the market between 2009 and 2013 did so without clear evidence of a marked improvement in the quality or quantity of patients’ lives.

However, in order to approve a treatment for use, the European Medicines Agency must conclude that the benefits of introducing a new treatment outweigh the risks. So, how can we be sure that only cancer treatments that are clinically and cost effective become available in the NHS? How do we balance the demand for early access to innovative treatments with the concern that public funds could be wasted? It’s a tough job but one that NICE relishes.

In 2016, there were some important changes to the way we appraised cancer medicines, and NICE and NHS England starting working together much more closely. The Cancer Drugs Fund was reformed to become a managed access fund for promising treatments with significant clinical uncertainty. This means that before a medicine is made available via the Cancer Drugs Fund, a managed access agreement is drawn up. It specifies the data that will be collected to address the uncertainty. The price the NHS pays during the managed access period reflects the level of uncertainty in the evidence. Afterwards, NICE reviews its guidance in light of the new evidence to decide if the treatment can be made routinely available in the NHS.

Perhaps in the context of managed access, ‘rigidly defined areas of doubt and uncertainty’ isn’t such an absurd concept after all!

Competing interests: No competing interests

09 October 2017
Linda J Landells
Associate director - Technology Appraisals (Cancer Drugs Fund) - National Institute for Health and Care Excellence
National Institute for Health and Care Excellence, Level 1A, City Tower, Piccadilly Plaza, Manchester M1 4BT