Patient Voice and Patient Engagement in Value Assessment Frameworks and HTA Decision-Making for Gene Therapies

OBJECTIVES: There is a growing understanding by industry and health authorities that the patient experience is critical for a comprehensive value assessment. For example, ICER established a Patient Council in July 2023 to amplify the patient voice in value assessment, demonstrating their commitment to soliciting insights from patient groups. Patient engagement is especially important in value assessments of innovative therapies such as gene therapies (GTs), given their unique administration and uncertainties associated. Our research evaluated the use of patient engagement within HTA appraisals of GTs across seven markets over 5 years.

METHODS: We reviewed HTA guidance documents/appraisals across the United States (US), Canada, EU4, and the United Kingdom (UK), including the ICER patient value frameworks, to determine the ways of use and impact of the patient voice on GT access. A search was conducted identifying HTA appraisals for GTs from 2019-2023 in relevant countries. We also reviewed EUnetHTA21 methods for future Joint Clinical Assessments (JCA).

RESULTS: While research is ongoing, preliminary findings indicate the use of patient insights in HTA decision making for GTs is greater in the US, UK and Canada, compared to Spain and Italy. In the majority of cases, the patient perspective was captured via use of Patient Reported Outcomes in clinical studies. However, in several cases, patient insights were used to better define the impact of the disease, impact of the GT on health-related quality of life, and to inform utility gain discussions.

CONCLUSIONS: The patient voice is increasingly utilized to support market access decision-making globally. However, there are differences in how these insights are captured and used. Further research is required to understand the full potential of the patient voice in HTA appraisals. This is also particularly true Europe, with lack of clarity on how patient insights will be collected and used by assessors during JCAs.