Countries struggling to broaden access to expensive new cancer drugs are being strongly warned to put cost-effectiveness first and not give in to political pressure.
A team of researchers studied the impact of the NHS Cancer Drugs Fund (CDF) that was introduced in the UK in 2010 following media criticism that cancer patients were being denied access to the latest treatments. By 2016 it had spent £1.3 billion (€1.5 billion) on cancer drugs that had not yet been approved or had been rejected by the National Institute for Health and Care Excellence (NICE) because they did not meet cost-effectiveness thresholds.
Only 38% of the drugs paid for from the CDF have reported statistically significant overall survival, with an overall median survival of 3.1 months. Less than half (48%) of the drug indications met ASCO criteria when assessed according to clinical benefit scales, and only 18% met ESMO criteria.
Four drugs – bevacizumab, cetuximab, everolimus and lapatinib—were approved for a total of 18 separate indications but 13 of these indications were subsequently delisted by the CDF due to insufficient evidence of clinical benefit.
In their report, researchers from the London School of Hygiene and Tropical Medicine, King’s College London, Colombia University and the University of Bristol conclude “The CDF was created following intense public and political pressure to provide access no matter what the cost or the evidence for their benefit.
“There was no stated estimation of the ‘number of lives that could be saved’, nor, more realistically, of the number of lives that may be extended. This was a debate played out in the media, limiting the role of NICE as the final arbiter for deciding what constitutes optimal value for society.”
After six years and £1.3 billion of spending, the CDF has been rolled back into the system and NICE is once more responsible for health technology appraisal.
Some patients have praised the CDF for giving them hope and access to drugs for orphan indications or rare conditions that NICE would ordinarily not appraise. However, researchers note that reported benefits for cancer patients could have been outweighed by opportunity costs because funding was not available elsewhere in the NHS.
Many of the drugs were approved by CDF on the basis of progression free survival, but the report says this may not equate to a clinical benefit because of serious toxicities that may arise.
Moreover, the CDF failed to monitor patient outcomes and collect the data to further assess these treatments. The report says: “We would argue from this analysis that the CDF has not provided meaningful value to cancer patients and wider society because the supporting data has been wanting.”
“We would suggest other countries considering a patient access scheme for drugs awaiting formal health technology appraisal use value frameworks that determine the likely benefit from reimbursement as part of the appraisal process. If drugs are made available pending an appraisal process, this should be accompanied by rigorous collection of outcome data through coverage with evidence development schemes.”
Such schemes should not replace assessment of overall cost-effectiveness, “to ascertain the societal benefit gained from drug reimbursements relative to other health technologies across the disease spectrum.”
One of the authors of this report is Richard Sullivan, Professor of Cancer Policy & Global Health at Kings College London and Director of the Kings Institute of Cancer Policy and a regular contributor to Cancer World.
He told the BBC (Today Programme 28 April 2017) that the CDF “was essentially a free for all” which prioritised some cancer drugs with severe toxicities over surgery and radiotherapy and over other health challenges. “I think you should ask the original committee and the politicians who set this up why they did not rigorously review what was happening. We are doing this report not for the UK but for other countries who are thinking of this sort of patient access scheme to say to them you must rigorously follow up the outcomes for patients.”
Baroness Delyth Morgan chief executive of Breast Cancer Now told the BBC that many patients were alive today because they had access to the fund. However, she said it was a sticking plaster which had failed to resolve the question of how to deal with innovations. “Patients with life threatening cancer are in the middle of a big debate about how we deal with this issue.”
Aggarwal, T. Fojo, C. Chamberlain, C. Davis & R. Sullivan: Do patient access schemes for high-cost cancer drugs deliver value to society?—lessons from the NHS Cancer Drugs Fund. Review in Annals of Oncology https://doi.org/10.1093/annonc/mdx110
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