An exploration of UK real world evidence and comparison with clinical trial data in supporting evaluation of 4 drugs that have exited the United Kingdom’s Cancer Drugs Fund (CDF).

We want to compare real-world data from the Systemic Anti-Cancer Therapies (SACT) database with clinical trial data for certain chemotherapy treatments to see how outcomes differ and explore why these differences might exist. The SACT database includes information on all cancer treatments given across National Health Service (NHS)
England. For a drug to be used in the UK, it needs approval from NICE (National Institute for Clinical Excellence), which evaluates the clinical and cost-effectiveness of new treatments through a process called Health Technology Appraisal (HTA). If NICE is not sure about a drug’s effectiveness, the Cancer Drugs Fund (CDF) allows patients to access it while further assessments are performed.

We have chosen four chemotherapy treatments that NICE has reviewed with varying recommendations:
• Ixazomib with lenalidomide and dexamethasone (TA870, 2023)
• Pembrolizumab (TA766, 2021)
• Ibrutinib (TA795, 2022)
• Nivolumab + Ipilimumab (TA780, 2022)

To carry out this research, we will need pseudonymised patient-level data (data where personal identifiers have been removed to protect privacy) from the clinical trials that were submitted to NICE. We will also request pseudonymised data from NHS England’s routine datasets for patients who received these treatments. The patients will be identified using the SACT dataset, which records chemotherapy use, and the Cancer Registry, which records cancer types. Each patient in NHS England’s databases has a unique pseudonymised ID, allowing for detailed follow-up across various data sources (such as the Radiotherapy Dataset, Hospital Episode Statistics, SACT, Cancer Registry, and Outcomes and Services Dataset).

This study is important because it could impact how future chemotherapy assessments are conducted and show if real-world data can support clinical trial findings for decision-making. We will also conduct additional analysis using updated UK routine data to compare patient outcomes during and after the CDF period, providing comprehensive long-term surveillance. This work may help inform updates to NICE’s guidance for these therapies.