OBJECTIVES: This study aims to compare the level and process of patient involvement in regulatory and HTA assessments, to analyse convergence and divergence of approaches.

METHODS: A review of articles, published in English between 2015 and 2021, was conducted to analyse how patients are involved by regulatory & HTA agencies in assessment processes and to what extent their inputs are considered in decision making. Other data sources include regulatory and HTA websites.

RESULTS: 146 articles were identified and a total of 75 were included in the analysis. For example, in US (FDA), Europe (EMA), Australia (TGA) & health Canada, patients are committee member whereas patient involvement in regulatory process is optional for e.g., in Brazil (NVISA). Frameworks are developed by some regulators (FDA, EMA, TGA, MHRA, & PMDA) to make patient engagement more efficient and meaningful. Significant variability was identified in the level of patient involvement in HTA decision making ranging from formal processes, e.g., involvement in submissions/consultations and representation in committees (UK, Canada, Germany, Australia) to limited patient involvement (France, Taiwan, New Zealand, Korea). Level of involvement varied within country between regulatory and HTA body, e. g. Canada has established patient involvement in policy-based regulatory decision making and in the HTA agency (CADTH), patients have voting rights. The Japanese Pharmaceutical Agency (PMDA) has established a process of patient involvement, in contrast in HTA activities the patient community is not formally included.

CONCLUSIONS: Though regulators & HTA bodies put strong efforts to embed patient feedback for meaningful decision making, alignment between regulatory and HTA bodies and across regulatory and HTA bodies may pose an opportunity to further strengthen the impact. Ideally, patient engagement efforts are designed to be responsive to the needs of regulatory and HTA bodies, to minimize efforts for the patient community and involved bodies.