Jason Shafrin, PhD

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 per year.

Using direct oral anticoagulants as a case study, the authors examined how delayed adoption of novel treatments could impact patient health outcomes and cost.

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear.

Jason Shafrin, PhD, serves as the vice president of Health Economics at PRECISIONheor, and Meena Venkatachalam, MSc, is the senior director of Health Economics at PRECISIONheor.