EFPIA response to BMJ article on efficacy of cancer medicines

Today, 2 out of 3 people diagnosed with cancer survive at least 5 years.  Between 1991 and 2011 deaths from cancer have fallen by 21% across the EU5 (UK, France, Germany, Spain and Italy).  This progress has been driven by rapidly advancing science, better diagnostics and innovative therapies.  The impact for patients has been longer survival and better quality of life.

Against this backdrop, EFPIA notes the publication in the BMJ of a study on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13.  The study questioned the clinical benefit of a number of new cancer medicines.

The study predominantly focused on clinical trials, rather than on real world data on actual patient outcomes.  The gathering together of overall survival data on new cancer medicines, generated in clinical settings, continues to represent a significant challenge both in terms of complexity and time. To help determine how medicines will function in a real world setting surrogate endpoints (often laboratory tests indicative of an expected improvement), are often employed as clinical endpoints in more complex diseases such as cancer.  As an EMA spokesperson, quoted in Politico today expressed it, “Restricting approvals of cancer medicines only to situations where there is indisputable evidence of improvement in overall survival or quality of life will not improve the outlook for cancer patients in the EU. On the contrary, such an approach may deprive patients of early access to effective medicines for patients in urgent need.”

New therapies in Europe undergo rigorous cost-effectiveness assessments and are only reimbursed if they meet the cost-effectiveness criteria against existing standards of care.  In cancer, the value of many treatments has tended to increase over time through impact on survival, or use in earlier lines of therapy and stages of a disease. For example, seven out of 10 cancer medicines approved by the EMA between 2003 and 2005 had additional approved value expansions following their initial indication, including for additional types of cancer.[1]

Studies in cancer are very disease-specific, survivability varies according to tumour type.  Indeed, a relatively small survival benefit could in fact be very significant in the context of a disease where patients may otherwise have had a very poor prognosis.  Clinicians need treatment options to individualise care to the patient.

Looking to the future, with the rapid evolution of cancer treatments and standards of care, it will become increasingly critical to assess the value and performance of new medicines in real world settings and work together to find solutions such as the greater involvement of patients in clinical trial design and the use of patient reported outcomes. As such, EFPIA calls for stakeholders to work together to optimise our ability to assess value with the tools already at our disposal and to also form a consistent approach for the collection and meaningful use of Real World Data.


[1] Rejon-Parilla JC, et al. The expanding footprint of oncology treatments. Office of Health Economics; May, 2014.