Update of Our Review of Real-World Evidence in HTA Decision-Making of Gene Therapies in the US, Canada, UK, EU4, and Japan

Speaker(s)

Dabbous M1, Waththuhewa M2, Olid Gonzalez A3, Raza J4, Dabbous F5, Khemakhem A6, Sullivan N2
1Evidera Value and Access Consulting, Tampa, FL, USA, 2Evidera Value and Access Consulting, Waltham, MA, USA, 3Evidera, part of PPD clinical research business of Thermo Fisher Scientific, New York, NY, USA, 4Evidera Value and Access Consulting, New York City, NY, USA, 5Data Analytics, Real World Evidence, Evidera, IL, USA, 6Evidera Ltd., Paris, France

Presentation Documents

OBJECTIVES: As evidence submitted for gene therapies (GTs) during health technology assessments (HTA) is usually associated with uncertainty, many HTA bodies are increasingly requesting Real-World Evidence (RWE) to address this. Several have also adopted new guidelines/frameworks on the use of RWE. Our aim was to update prior research on the role of RWE in HTA decision-making for GTs.

METHODS: Regulatory websites in US, Canada, UK, EU4, and Japan were reviewed to identify GTs with marketing authorization (MA). HTA websites of the respective countries were reviewed to extract appraisals for these GTs. RWE-relevant data were then extracted for analysis. As Japan had no formal HTA body prior to 2019, its regulatory site was the main source of information.

RESULTS: Twenty-two GTs were found to have MA in the geographical scope. Of these, 19 had been appraised by HTA bodies and 2 were not yet assessed.

The results show greater RWE use in France, Germany, and Italy, by leveraging registries for reimbursement decisions and re-assessments. Most HTA bodies mentioned uncertainty of the evidence translating to real-world practice as the reason for needing RWE.

While our analysis is ongoing, an initial review of the evidence indicates the impact of RWE in driving HTA recommendations is limited when submitted as part of the full evidence package. However, RWE has been used as leverage to accelerate access when pre-planned and used to address areas of uncertainty arising from clinical studies.

CONCLUSIONS: It is expected HTA bodies will increasingly request RWE as more GTs come to the market. However, while the trends seen in our research indicate a growing interest, the EUnetHTA21 methods seem critical of non-randomized evidence. Further analysis is ongoing to understand how EUnetHTA 21 guidelines will be interpreted by assessors at the time of appraising GTs and RWE during JCAs.

Code

RWD143

Topic

Clinical Outcomes, Health Technology Assessment, Study Approaches

Topic Subcategory

Clinical Outcomes Assessment, Decision & Deliberative Processes, Registries

Disease

Rare & Orphan Diseases