OBJECTIVES: To pilot assessment of rare disease patient organisations’ (RDPOs) experiences of formal engagement in health technology appraisal (HTA) processes in the UK and to identify the key data sources utilized by RDPOs to support HTA patient group submissions.

METHODS: The survey was conducted amongst UK RDPOs in October and November 2017 using an online survey platform. The questionnaire comprised 34 multiple-choice and open-ended questions to: characterize the RDPO and respective rare disease (RD); assess the RDPO’s prior experience of HTAs; evaluate the level of difficulty reported by RDPOs in providing responses to domain-specific questions in HTA submissions; and identify the data sources RDPOs utilized in these responses.

RESULTS: CONCLUSIONS: More comprehensive assessment of RDPOs’ experiences in other jurisdictions would be beneficial to inform methodological improvements, including RDPO education on HTAs, to more effectively and systematically capture and represent the patient voice in RD HTAs.