OBJECTIVES: To investigate the best-practice approaches to engage patients in drug development and incorporate the patient perspective in health technology assessment (HTA).

METHODS: The National Institute for Health and Care Excellence (NICE) process and patient engagement policies were reviewed to identify proposed approaches to incorporating patient perspective. Additionally, four case studies of rare disease NICE appraisals were analyzed. This was followed by semi-structured interviews with five experts internal and external to the company with experience of patient engagement in the drug development and HTA process. The interviews consisted of open-ended questions on the impact of, timing, and nature of patient involvement. The interviews were recorded and analyzed to identify critical factors for successful patient engagement.

RESULTS: The document analysis highlighted the importance of incorporating the patient perspective in HTA, with most relevance to describing the disease burden and quality of life of patients and their carers. Evidence suggests patient statements were utilized in the NICE process to inform discussions on the validity of the utility estimates in economic modelling.

In the interviews, experts highlighted the value of patient involvement to support not only research design and HTA, but also gaining a broader perspective of patient needs to ensure drugs are developed to address these. The importance of asking open questions to learn from the patient community, particularly in the beginning of work in a particular disease area was also stressed as important. It was however frequently mentioned that cooperation with the patient community should be respectful of the oft limited capacity patient organizations may have.

CONCLUSIONS: Incorporating patient perspectives throughout the drug development and in HTA is essential to ensure treatments are developed to address patient needs and facilitate optimal decisions, in particular in rare diseases where evidence is often scarce.