Most drugs paid for by £1.27bn Cancer Drugs Fund had no “meaningful benefit”BMJ 2017; 357 doi: https://doi.org/10.1136/bmj.j2097 (Published 28 April 2017) Cite this as: BMJ 2017;357:j2097
The Cancer Drugs Fund (CDF) has not “delivered meaningful value” to patients with cancer and may have exposed them to “toxic side effects of drugs,” an analysis has found.1
The CDF was established in 2010 in England to provide “patients with faster access to the most promising new cancer treatments” and to ensure “value for money for taxpayers.” It funded drugs that were not available through the NHS because the drugs had not been appraised, were in the process of being appraised, or had been appraised but not recommended by the National Institute for Health and Care Excellence (NICE). The fund was overhauled last year.
Ajay Aggarwal, academic clinical oncologist at the London School of Hygiene and Tropical Medicine and a lead author of the analysis, said, “From 2010 when it started to 2016 when it closed, the Cancer Drugs Fund cost the UK taxpayer a total of £1.27bn [€1.51bn; $1.64bn], the equivalent of one year’s total spend on all cancer drugs in the NHS.
“The majority of cancer medicines funded through the CDF were found wanting with respect to what patients, clinicians, and NICE would count as clinically meaningful benefit. In addition, no data on the outcome of patients who used drugs accessed through the fund were collected.”
The researchers looked at 29 drugs that had been approved for use through the CDF in January 2015 for 47 specific cancer indications and analysed them according to six criteria including validated clinical benefit scales, clinical trials, and observational data assessing effectiveness.
Reporting their findings in the Annals of Oncology,1 they said that the “majority of CDF-approved indications have been based on studies that reported minimal to no benefit in survival.” The authors used this outcome, as it is “an unequivocal benefit and desired.”
Of the indications analysed, they found that only 38% (18/47) were supported by clinical trials showing a statistically significant overall survival benefit—the median of which was 3.2 months (range: 1.4 months (aflibercept in metastatic bowel cancer) to 15.7 months (pertuzumab in first line metastatic HER2 positive breast cancer)).
Of the remaining 29 indications in the CDF, 36% (17/47) were approved for use despite “no statistically significant overall survival benefit” in the index clinical trials, and a further 26% (12/47) were approved without overall survival data being available.
Even when validated scores of meaningful clinical benefit were used, only 18% (9/47) met this criterion.
The authors also queried why so many “clearly ineffective drugs were prescribed in the first place” given that the CDF’s intended role was to “empower clinicians” to use drugs that “extend or improve life.”
Richard Sullivan, professor of cancer and global health and director of the Institute of Cancer Policy at King’s College, London and an author of the analysis, highlighted “political failure across the board.” This included politicians, health professionals, and patient groups who have lobbied for access to cancer drugs despite NICE not deeming them to be cost effective.
Sullivan also raised concern about the design of some of the trials, suggesting that several did not ask the right question and showed a lack of scrutiny over trial design. He added that cancer care was about more than drugs.
“A ringfenced drugs fund was created despite a lack of evidence that prioritising drug expenditure would improve outcomes for cancer patients over and above greater investment in the whole cancer management pathway, which includes screening, diagnostics, radiotherapy, surgery, and palliative care,” he said. “We recommend that other countries that are considering similar ringfenced drug access funds for high cost cancer drugs should adopt a more rational approach to funding high cost health technologies.
“Our findings underline the importance of reimbursement decisions for all drugs, procedures, and interventions in cancer care being made through appropriate health technology appraisal processes. Only in this way will decisions be made on the best available evidence so as to maximise the value for cancer patients and society as a whole.”
A revamped CDF was launched in July last year. Under new rules the fund would pay for drugs for a limited period in the hope that real life experience will prove their worth and enable them to meet the cost effectiveness threshold for NHS use. Drugs that failed were excluded from the fund.2
Responding to the analysis, Paul Workman, chief executive of the Institute of Cancer Research in London, said, “The old Cancer Drugs Fund was always just a sticking plaster, and we welcomed its overhaul because it was too expensive, unsustainable, and provided little certainty to patients and their doctors. The new, more evidence based system, where NICE appraises all cancer drugs, should address some of the issues highlighted in this study.
“But, while we support the rigorous drug evaluation that NICE carries out, it’s essential that the new system continues to offer fast access to the most innovative and exciting cancer drugs. We need NICE to reform the way it evaluates drugs to place greater emphasis on how innovative they are, to ensure patients are not denied the most promising treatments purely because of their cost.”