Who judges the benefit of cancer drugs?


In a study published in the British Medical Journal (BMJ) last month, a team led from Kings College London explored the use of 48 cancer drugs across a 4-year period, and found that in only 10% of the uses did the drugs improve quality of life. Overall, 57% of uses showed no benefits for either survival or quality of life. Where survival benefits were shown, the team said these were clinically meaningless in almost half of the cases.

Responding to the study, Carl Heneghan, professor of evidence-based medicine at University of Oxford, called for a more rigorous approach to evaluating cancer drugs. “It is hard to understand why half the drugs were approved in the first place if they provide no clinically meaningful benefit,” he said.

But what does ‘rigorous’ mean? According to Winette van der Graaf, professor of personalised oncology at the Institute of Cancer Research, it should include making decisions based on smaller studies looking at benefits other than overall survival, including speed to access to treatment. Francesco Pignatti, Head of oncology, haematology and diagnostics at the European Medicines Agency (who approved the drugs in the study for use as treatments) adds that data based largely on clinical trials using only clinical endpoints to judge success miss out smaller but incremental benefits. He highlighted the importance of having “a systematic evaluation of patients’ [own] preferences”.

And here lies the rub. Who is joining up the dots, taking real-world evidence from patients, across years of their experience and combining it with data from clinical trials? To a degree, the UK’s Cancer Drugs Fund is attempting this, examining each drug on an individual basis with bespoke data collection rather than a ‘one size fits all’ approach.

But how the different data sources are weighted and evaluated involves an element of subjectivity, perhaps seen to some as an arbitrary assessment. And even when patient preferences are included, to what extent are they open-sourced vs. gathered using pre-set lists derived from earlier studies?

As a qualitative healthcare researcher, I would argue that the bare minimum for a holistic assessment of patient-centred cancer drug benefit would include a meta-analysis of primary qualitative findings, updated annually and presented alongside clinical data.

My own experience of over 40 patient projects since 2002 is that patient-defined outcomes are consistently undervalued as a contributor to a holistic measurement of ‘benefit’. Whilst the shrinking of a tumour can be measured precisely, it is harder to capture the value of treatment and support (pharmacology can do both) which helps someone cope with the exhaustion or nausea from chemotherapy. Such support can make the difference to be able to hold down a job, maintain a sense of self or self-confidence in social life, but its lack of a uniform endpoint across a patient population renders its benefit hard to show ‘scientifically’.

The cost and toxicity of cancer treatment are reasons enough to demonstrate why oncology drugs need the most stringent of benefit assessment before regulatory approval and subsequent patient use. Yet, such judgement should have at its core that ‘benefit’ starts and ends with the patient, and as such, attempts to include patient-derived insight into benefit scoring should be as exhaustive as clinical data.

As I prepare to start a UK study this month with people living with a form of blood cancer, I hope my research will both do justice to, and contribute to the lived experience that these patients describe. Their journey through one of the most feared of human illnesses may not be conveyed to me with scientific precision. But their real-world candour and emotion can most certainly determine what works and what doesn’t for them as individuals, and that surely has a rightful place in the evidence base of healthcare.

Photo by jennifrog – http://flic.kr/p/5ENxea