OBJECTIVES: Gene therapies (GTx) are a “gamechanger” in many rare diseases where patients previously had limited or no treatment options. Since 2012, 12 single-administration non-oncological GTx have been approved in the US and Western Europe. However, outside those regions, adoption of GTx is often hindered by challenges such as limited financial resources and administrative capacity, and the absence of GTx-specific health technology assessment (HTA) methodologies. This research aimed to assess the availability of single-administration GTx and HTA decision drivers in selected CEE countries.

METHODS: We conducted a targeted review of HTA reports and access conditions of non-oncological single-administration GTx in Poland, Slovakia, Czech Republic, Lithuania, Estonia. For comparison, HTA reports and access conditions in EU4 and the UK were analyzed.

RESULTS: The review identified 11 HTA reports on 6 single-administration GTx (eladocagene exuparvovec, etranacogene dezaparvovec, valoctocogene roxaparvovec, voretigene neparvovec, atidarsagene autotemcel, onasemnogene abeparvovec). Onasemnogene abeparvovec was the only therapy assessed and reimbursed in all countries. All 6 GTx were assessed in Poland, 2 in Czech Republic and 1 in Slovakia, Estonia and Lithuania each. The main drivers of HTA decisions through the Polish “Highly innovative health technologies” pathway were: 1) innovative therapy leading to a significant improvement in the disease course; 2) rare disease; 3) efficacy demonstrated in clinical trials; and 4) positive reimbursement decision in other European countries. Raised concerns were associated with uncertainty around lifetime benefits and high costs. Although 3 therapies received positive HTA decisions in Poland, only 1 was reimbursed by the National Health Fund.

CONCLUSIONS: The availability of GTx in selected CEE countries is limited. Considering there are 88 ongoing GTx phase 3 clinical trials, with 58 expected to be completed within the next 5 years, the decision-makers will likely face even greater challenges to balance patient access and impact on public budgets.