The Cancer Drugs Fund (CDF) was set up in 2011 in England to enable patients to access oncology therapies that are not routinely publicly funded. In April 2016, the CDF became a temporary reimbursement fund under the remit of the National Institute for Health and Care Excellence (NICE) with the aim of collecting observational data to inform subsequent technology appraisals. This study aims to evaluate how the reformed CDF has been utilized in the 18 months since this reform.

NICE Final Appraisal Determinations for Single Technology Appraisals of oncology drugs from (29 July 2016 to 24 November 2017) were identified and key data extracted.

Seventy-four oncology drug:indication appraisals were identified, 54 (73 percent) were recommended/optimized, 10 (14 percent) were not recommended and 10 drug:indication pairings (14 percent: osimertinib, brentuximab vedotin, pembrolizumab, olaratumab, obinutuzumab, venetoclax, nivolumab [3 indications], and ibrutinib) were referred to the CDF. For most, the greatest uncertainty in their cost-effectiveness analyses related to their survival benefits, intended to primarily be resolved through subsequent clinical trial readouts. However, for venetoclax, ibrutinib and brentuximab, the main areas of uncertainty (relating to comparative survival benefit, pre-progression mortality, and rate of subsequent stem cell transplants, respectively) are expected to be resolved primarily through observational data collected under the CDF.

The newly reformed CDF has been utilized in a minority of cases. Typically, the CDF acts as a temporary access mechanism for treatments that receive market authorization based on early/single-arm trial data until longer-term and/or Phase III data are available. However, venetoclax, brentuximab, and ibrutinib demonstrate how the CDF may address significant areas of uncertainty through the collection of uncontrolled observational data. For venetoclax, with only single-arm supportive clinical trial data, observational data of this intervention and appropriate comparator are to be collected, providing a potential case study of how to appropriately manage reimbursement in the face of significant clinical uncertainty.