The Increasing Use of Real-World Data in Health Technology Appraisals of Oncology Therapies: A Systematic Review

OBJECTIVES: To conduct a systematic literature review on the use of real world data (RWD) across all UK NICE therapeutic appraisals in oncology.

METHODS: A systematic literature review was undertaken into all active Oncology TAs between financial year 2000/2001 – 2022/2023. TAs were extracted on 23rd August 2023. Dual review was undertaken based on a set of questions and a standardised report form was completed for the review of each TA. After individual reviews were completed, responses to each question were agreed upon by the two reviewers and any disagreements were resolved. Descriptive statistics and figures were generated to report findings of the review. Case studies were created to highlight specific concerns on the use of RWD by the committee or evidence advisory group.

RESULTS: Of 284 Oncology TAs reviewed, 135 used RWD, of which 56 (41%) have used RWD in the last 3 years of the review. Of those using RWD, the most used datasets were Systemic Anti-cancer Therapies (SACT) & Cancer Drugs Fund (CDF) data. Whilst the majority of datasets used RWD for either utility values, population characteristics or survival outcomes, in the last decade matching adjusted indirect comparisons (MAIC) and external control arm (ECA) analyses have been used in 15 and 9 studies respectively. Concerns the review committee had included missing variables such as imaging and laboratory test results in the datasets and generalisability of the data sources used to the UK population.

CONCLUSIONS: RWD is becoming increasingly important in the submission of Oncology TAs in the UK. The recent use of patient level analyses such as ECAs and concerns about missing variables and generalisability to the UK population make detailed UK secondary care datasets of great importance for future TA submissions.