The potential value of disease-modifying therapy in patients with spinocerebellar ataxia type 1: an early health economic modeling study.
Teije H van ProoijeSanne RuigrokNiels van den BerkmortelRoderick P P W M MaasStan R W WijnWilleke M C van Roon-MomBart P C Van De WarrenburgJanneke P C GruttersPublished in: Journal of neurology (2023)
Our model indicates that the maximum price for a hypothetical therapy to be cost-effective is considerably lower than currently available RNA-based therapies. Most value for money can be gained by slowing progression in the early and moderate stages of SCA1 and by stopping therapy upon entering the severe ataxia stage. To allow for such a strategy, it is crucial to identify individuals in early stages of disease, preferably just before symptom onset.