Response to BMJ article on efficacy of cancer medicines

The European Federation of Pharmaceutical Industries and Associations (EFPIA) has issued a response to the recently published study in the British Medical Journal (BMJ) on the efficacy of cancer medicines, noting the importance of real-world data.

According to a statement from the EFPIA between 1991 and 2011 the rates of death from cancer have fallen by 21% across the five EU countries — UK, Germany, France, Spain and Italy. With two out of three cancer patients surviving at least five years now.

However, the paper published in the BMJ, which questioned the clinical benefit of many approved cancer therapies, was more focused on the clinical trial data rather than the real-world data on actual patient outcomes.

Gathering overall safety data on new cancer medications is not only challenges from a time perspective but is also complex in nature. To determine real-world outcomes surrogate endpoints are needed. These are often predictions of how medicines are expected to improve patient survival and quality of life, which can be oftentimes less clear cut in complex diseases such as cancer, explained EFPIA.

“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,” noted EFPIA. “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.”

Cancer studies are very disease-specific and as such survivability is variable depending on the type of tumour. The EFPIA stressed that in essence a relatively small survival benefit could be significant for patients who would otherwise have a poor prognosis, so, clinicians need treatment options to individualise care to the patient.

EFPIA concluded: “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.”