OBJECTIVES: Survey data as a source of real-world evidence (RWE) can be associated with a lower quality of evidence compared with that derived from other sources such as patient registries, but is often a necessity to fill data gaps. The National Institute for Health and Care Excellence (NICE) introduced the RWE framework in 2019 on best practices for planning, conducting and reporting RWE studies (such as surveys and patient registries), including methods to minimise bias and characterise uncertainty. In light of this, our study assesses the impact of survey data in HTA submissions whilst also assessing the suitability of different survey types in addressing specific questions.

METHODS: NICE single technology appraisals (STA) for rare diseases submitted in 2019–2023 were searched using the terms ‘survey’, ‘questionnaire’ and ‘interview’ to identify STAs that incorporated survey data as supporting evidence. Extracted data included types of reported survey outcomes, characteristics of the survey and participants and NICE appraisal decisions and comments.

RESULTS: Of 118 rare disease STAs, 94 used survey data (83 structured questionnaires and 63 interviews) as supporting evidence. Patient and caregiver experiences (82%) and life with the condition (32%) were commonly reported survey topics. Ninety-five percent of STAs received recommendations, among which 75% were recommended for use through patient access schemes, 29% through commercial access agreements, and 11% through the Cancer Drugs Fund. Analysis of the comments from the Evidence Review Group reveals how survey data has contributed to the submissions, allowing the identification of impactful survey types or characteristics.

CONCLUSIONS: Following the introduction of the NICE RWE framework in 2019, most approved rare disease STAs have incorporated survey data, providing valuable insights for future discussions and research on the evolving role of survey data in HTAs.