OBJECTIVES: In July 2016 the National Health Service in England changed the way in which cancer treatments are appraised and funded (NHS England, 2016) so that all new cancer treatments expected to receive a marketing authorisation are appraised by NICE. We explore the effect of these changes on the appraisal of treatments for rare cancers and the implications for patient access.

METHODS: The outputs from NICE’s health technology appraisal (HTA) programme from March 2000 to January 2018 were reviewed. All cancer topics, those identified as rare cancers (defined as < 6 cases per 100,000 (Rare cancer list, 2018)), the individual final recommendation and publication date were extracted.

RESULTS: Of the 276 individual appraisal recommendations for cancer treatments from the HTA programme, 39 were classified as a rare cancer. Before the new Cancer Drugs Fund (CDF) was launched in July 2016, 9% (n=18/200) of all individual recommendation for cancer treatments were for rare cancers compared with 27% (n=21/76) afterwards. Of the rare cancer topics that were appraised before July 2016, 53% (n=10/18) received a positive recommendation for routine commissioning (within the marketing authorisation or optimised) compared with 90% (n=18/20) after July 2016. NICE has recommended 2 further treatments for rare cancers as options for use within the CDF while further data collection takes place as part of a managed access agreement.

CONCLUSIONS: It was anticipated that changes to the appraisal and funding of cancer drugs would present a considerable challenge for the approval of new therapies for rarer cancers. However, results so far show an increase in NICE’s positive recommendations for rare cancer treatments and subsequently funded by NHS England in routine commissioning. In addition, the revised processes have allowed a route for patients to access promising treatments while further evidence is collected to address the clinical uncertainties.