Challenges to economic evaluations of gene and cell therapies.
Medical Review (Med. pregled), 2025, 61(6), 27-32.
T. Vekov1, S. Djambazov2, V. Belcheva3
1 MHAT „Heart and brain“ – Pleven
2 MU – Pleven
3 Trakia University – Stara Zagora
Abstract. Economic assessments of health technology (HTA) involving gene and cell therapies can pose significant challenges. They are mainly driven by the high degree of uncertainty surrounding clinical efficacy and cost-effectiveness, due to the limited or even non-existent evidence of utility value, long-term effects, and costs of treatment versus comparator drug therapies, which hinders the correct calculation of quality-adjusted life years (QALYs) gained for gene and cell therapies. A critical review of the HTAs of gene and cell therapies found that overall the evidence for clinical effectiveness is limited and weak, but this varies considerably across technologies. The main difficulty in the paucity of clinical data is the design of the trials, which are mostly singlearm, unblinded, with small sample sizes and short follow-up times. The indirect comparisons proposed in some of the economic evaluations are based on a number of assumptions and are of low certainty. Adaptation of the conventional decision-making process could improve the HTAs of gene and cell therapies.
Key words: health technology economic evaluations, gene therapies, cell therapies, challengeses
Address for correspondence: Prof. Toni Vekov, PhD, DSc, e-mail: t.vekov.mu.pleven@abv.bg