Respiration; international review of thoracic diseases · 2005
Two full-term pregnancies in a patient with mitochondrial myopathy and chronic ventilatory insufficiency.
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Shafrin Jason, Thahir Suhail, Klimchak Alexa C, Audhya Ivana, Sedita Lauren E, Romley John A
Аннотация
OBJECTIVES: To quantify the magnitude of an ISPOR novel value element, insurance value, as applied to new treatments for a rare, severe disease with pediatric onset: Duchenne muscular dystrophy (DMD).
STUDY DESIGN: Prospective survey of individuals planning to have children in the future.
METHODS: A survey was administered to US adults (aged ≥ 21 years) planning to have a child in the future to elicit willingness to pay (WTP) for insurance coverage for a new hypothetical DMD treatment that improved mortality and morbidity relative to the current standard of care. To identify an indifference point between status quo insurance and insurance with additional cost that would cover the treatment if respondents had a child with DMD, a multiple random staircase design was used. Insurance value-the value individuals receive from a reduction in future health risks-was calculated as the difference between respondent's WTP and what a risk-neutral individual would pay. The risk-neutral value was the product of the (1) probability of having a child with DMD (decision weighted), (2) quality-adjusted life-years (QALYs) gained from the new treatment, and (3) WTP per QALY.
RESULTS: Among 207 respondents, 80.2% (n = 166) were aged 25 to 44 years, and 59.9% (n = 124) were women. WTP for insurance coverage of the hypothetical treatment was $973 annually, whereas the decision-weighted risk-neutral value was $452 per year. Thus, insurance value constituted 53.5% ($520) of value for new DMD treatments.
CONCLUSIONS: Individuals planning to have children in the future are willing to pay more for insurance coverage of novel DMD treatments than is assumed under risk-neutral, QALY-based frameworks.
STUDY DESIGN: Prospective survey of individuals planning to have children in the future.
METHODS: A survey was administered to US adults (aged ≥ 21 years) planning to have a child in the future to elicit willingness to pay (WTP) for insurance coverage for a new hypothetical DMD treatment that improved mortality and morbidity relative to the current standard of care. To identify an indifference point between status quo insurance and insurance with additional cost that would cover the treatment if respondents had a child with DMD, a multiple random staircase design was used. Insurance value-the value individuals receive from a reduction in future health risks-was calculated as the difference between respondent's WTP and what a risk-neutral individual would pay. The risk-neutral value was the product of the (1) probability of having a child with DMD (decision weighted), (2) quality-adjusted life-years (QALYs) gained from the new treatment, and (3) WTP per QALY.
RESULTS: Among 207 respondents, 80.2% (n = 166) were aged 25 to 44 years, and 59.9% (n = 124) were women. WTP for insurance coverage of the hypothetical treatment was $973 annually, whereas the decision-weighted risk-neutral value was $452 per year. Thus, insurance value constituted 53.5% ($520) of value for new DMD treatments.
CONCLUSIONS: Individuals planning to have children in the future are willing to pay more for insurance coverage of novel DMD treatments than is assumed under risk-neutral, QALY-based frameworks.