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- W4311353103 abstract "Orphan drugs offer significant clinical benefit, however their cost leads to potentially unsustainable budget impact. US Managed Care Organizations (MCOs) and Pharmacy Benefit Managers (PBMs) often provide coverage, regardless of price and limitations of data at launch, while European payers review data more critically before providing coverage. This poses a challenge to manufacturers of how to design clinical trials that ensure their product will be reimbursed across geographies." @default.
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- W4311353103 date "2022-12-01" @default.
- W4311353103 modified "2023-09-27" @default.
- W4311353103 title "HTA184 Investigating How Orphan Drug Manufacturers Can Optimize Clinical Trial Design so Patients across Geographies Can Access Life-Saving Innovations" @default.
- W4311353103 doi "https://doi.org/10.1016/j.jval.2022.09.1642" @default.
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