Drug Costs Not A Primary Driver Of Rare Disease Economic Burden
EveryLife Foundation study finds inpatient and outpatient care, not prescription drugs, are the primary excess medical costs associated with rare diseases.
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Focusing on orphan-first drugs may be best route to address pricing concerns, Health Affairs study suggests after finding that just one-fifth of spending on 15 top-selling partial orphan drugs was for the drugs’ rare disease indications.
The $500m in additional funds would support pandemic-related activities, potentially for additional staff or technology upgrades.
US FDA received 110 designation requests in July, a high mark that reflects the 60 days needed to review such requests ahead of the 30 September statutory expiration; agency is allowing email submission of RPD and orphan drug designation requests during the COVID-19 pandemic and expects to roll out an online portal for orphan designation requests later this year.