Economic Evaluation of GENE Therapies: IMPACT of Alternative Payment Models on Uncertainty Around the Icer
Author(s)
Pochopien M1, Clay E1, Skrzeczek A2, Aballea S1, Toumi M3
1Creativ-Ceutical, Paris, 75, France, 2Creativ-Ceutical, Krakow, Poland, 3Aix-Marseille University, Marseille, France
OBJECTIVES Gene therapies (GT) offer the potential for a one-time treatment to produce long-term benefits. One of the concerns of decision-makers when appraising the value of GT is the uncertainty around durability of the effect. Our objective was to assess the impact of different payment models and assumptions on the durability of the effect of GT on the incremental cost-effectiveness ratio (ICER) and associate uncertainty. METHODS A simple model for a progressive genetic disease with onset in infancy was developed with fictitious data. Five health states were included in the model: No disease; Disease, stage I; Disease, stage II; Disease, stage III and Death. GT therapy was assumed to cure patients, and scenarios with a re-emergence of the disease after 5 years or more were considered. Utility values were dependent on the health state, irrespective of treatment. The total price of GT was assumed to be £2,000,000, and it could either be paid entirely at the beginning, or spread over 20 years. Yearly payments were determined so that the total discounted cost of GT was the same in both situations. The discount rate was 3.5%. When the disease re-emerged, yearly payments were stopped. RESULTS The incremental costs and QALYs gained with GT with a lifetime efficacy amounted respectively £1,539,247 and 10.22, resulting in an ICER of £150,568/QALY gained. Depending on the durability of the obtained effect, when a single payment for GT was accounted, the ICER ranged from £150,568 to £2,055,842. The ICER range was from £150,568 to £623,211 when the cost was spread over 20 years. CONCLUSIONS The ICER range in an economic evaluation of GT can be so wide that the analysis does not really inform decision-making. The uncertainty around cost-effectiveness results due to unknown durability of the effect of GT can be substantially reduced by spreading the payment over several years.
Conference/Value in Health Info
2020-09, ISPOR Asia Pacific 2020, Seoul, South Korea
Value in Health Regional, Volume 22S (September 2020)
Code
PBI2
Topic
Economic Evaluation, Health Policy & Regulatory, Medical Technologies, Methodological & Statistical Research
Topic Subcategory
Cost-comparison, Effectiveness, Utility, Benefit Analysis, Implementation Science, Pricing Policy & Schemes
Disease
Genetic, Regenerative and Curative Therapies
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