A Targeted Review of the Challenges and Strategies for Submitting Patient-Generated Evidence to Health Technology Assessment (HTA) Agencies

OBJECTIVES: This study examined the uses of patient experience data (PED) by the patient community, strategies employed when submitting this evidence to HTA agencies, and potential challenges in assessment of PED.

METHODS: Case studies of PED submitted to HTAs were identified through a review of publicly available submissions to various agencies. A targeted literature review (TLR) was also conducted to identify additional case studies and other relevant information. Cases were evaluated by an international workshop involving patients, patient advocacy groups (PAGs), and experts in patient engagement.

RESULTS: 48 cases across 17 diseases containing PED from patient groups were analysed. In these cases, survey PED was the most common (36%), followed by testimonials (27%), and interviews (12%). Moreover, the analysis showed that PED can enhance HTA processes by increasing focus on patient-relevant outcomes (e.g., QoL and patient preferences), identifying subpopulations with unmet needs, and strengthening the consideration of patient perspectives. The TLR demonstrated that effective use of PED in HTA processes will require capacity building support for PAGs to meet HTA and patient needs. Additional challenges identified in the TLR included difficult-to-use online HTA templates, inconsistencies in preferred PED formats, short timeframes to generate robust PED, and lack of transparency in HTA decision-making. The workshop attendees made the following key recommendations: 1) Patient and PAG input should convey a clear narrative supported by both testimonials and quantitative PED; 2) Clarity in how HTA committees evaluate data is necessary for strengthening future PED input; and 3) Additional collaboration with industry sponsors and HTA agencies is needed to enhance the collection of PED during clinical development.

CONCLUSIONS: Although PED improves the quality of HTA decision-making, the ability of PAGs to submit high-quality PED is limited by current HTA practices and could be enhanced if all relevant stakeholders worked together to improve practices, processes, and support for PED.