The Acceptance of Using External Data to Inform Survival Extrapolations for Oncology Health Technology Assessments by NICE

OBJECTIVES: Pivotal trial data is often immature at the time of health technology assessments. The National Institute for Health Care and Excellence (NICE) technical support document 21 (TSD21) and real-world evidence framework (RWE) provide guidance and support for the use of external data for survival extrapolations to decrease uncertainty around longterm survival estimations. This study provides an update of a previous review on how external data for survival extrapolation were used in oncology NICE technology appraisals (TAs) in the past year.

METHODS: A review update was conducted on all oncology NICE TAs that were published between May 2023-May 2024. Eligible TAs were subjected to a full-text review if they were indicated for oncology and utilized external data for quantitative survival extrapolation. Terminated TAs were excluded. The following items were extracted: indication, intervention, comparator, extrapolation method, type of external data, NICE recommendation, and critiques. The findings were then compared to those of the original review (2001-mid 2023).

RESULTS: Eight out of 40 oncology TAs met the inclusion criteria. A similar trend was observed compared to previous years: 20% compared to 23-26% in 2021-2023 of the TAs used external data quantitatively to inform survival extrapolations. Two, five, and three TAs used registry data, clinical trial data and key opinion lead, respectively. Three TAs received negative recommendations from NICE on their approach of which two TAs received positive drug recommendations despite the critique. Main concerns of NICE included lack of transparency on the methods, and the comparability and fit of the external data to the pivotal trial data.

CONCLUSIONS: The proportion of oncology TAs using external data to quantitatively inform survival extrapolations showed a similar trend compared to previous years. Despite the guidance provided by TSD21 and the RWE framework, the primary concerns raised by NICE, such as transparency and data reliability, have not been alleviated.