Over the last decade, the rapid development of immuno-oncology (I-O) therapies has transformed the cancer treatment landscape. However, a thorough analysis of the rationale behind I-O reimbursement is yet to be provided. We evaluated the I-O treatment appraisal process by the National Institute for Health and Care Excellence (NICE) in the UK, to quantify their success rate and highlight the main reimbursement barriers.

We used the Cancer Research Institute classification to identify I-O therapies published between 2011-2020 from the NICE website (excluding terminated appraisals). We then extracted approval determinants, including all NICE critiques of I-O treatment clinical and cost-effectiveness evidence, from Final Appraisal Documents.

Of the 75 I-O appraisals identified, 62.6% (47/75) were recommended for reimbursement by the National Health Service, 22.7% (17/75) within the Cancer Drugs Fund (CDF), and 14.7% (11/75) were not recommended. Most NHS-recommended treatments (59.6%, 28/47) demonstrated significantly improved overall survival (OS) during clinical trials, as opposed to 35.3% (6/17) of CDF-recommended treatments, for which OS uncertainties were frequent. The majority of non-recommended treatment also improved OS (63.6%, 7/11); the main barrier to their reimbursement was their incremental cost-effectiveness ratio (ICER) exceeding the NICE accepted threshold. Notably, due to modest clinical effectiveness only 27.3% (3/11) of non-recommended treatments met NICE's end-of-life criteria (for which the accepted ICER threshold is substantially higher than treatments that do not meet these criteria) as opposed to 53.2% (25/47) of recommended treatments. Additional barriers to recommendation were OS data immaturity, leading to uncertainties in long-term survival extrapolations (3/11), and lack of survival benefit (3/11). Trial treatment regimen and choice of efficacy outcomes were also frequently criticised by NICE.

Most I-O treatments receive positive recommendations by NICE. However, manufacturers should beware that high ICERs, not meeting end-of-life criteria and long-term survival benefit uncertainties can undermine I-O approval.