The Economics of Gene Therapy and of Pharmacogenetics

摘要: This paper focuses on the economic issues arising from two uses of genomics: 1) development gene therapy; 2) and use pharmacogenetics to identify a patient’s genotype before treatment exclude those who will not benefit or may be harmed. We conclude that private-sector investment aimed at developing therapy for monogenic diseases is likely socially suboptimal. Short-term administration regimens yielding long-term therapeutic benefits are meet payer resistance large “one-off” costs because budget constraints or, in competitive systems, concerns savings would accrue future insurers attract high-cost patients. For some diseases, patient numbers too small support commercial without changes orphan drug legislation willingness accept higher cost-effectiveness thresholds. In case pharmacogenetics, we it can often optimal test treatment, particularly if proportion nonresponders high, there potential serious adverse reactions, inexpensive. Genetic testing fragments population could reduce incentives R&D unless prices adjusted reflect expected targeted per patient. Even situations where adjusted, populations make viable. problem with analogous associated require similar remedies society values treatments these diseases.