Assessing the Value of Orphan Drugs Using Conventional Cost-Effectiveness Analysis¶
Nicod, E. (2022). Assessing the Value of Orphan Drugs Using Conventional Cost-Effectiveness Analysis: Is It Fit for Purpose?. Orphanet Journal of Rare Diseases, 17(1).
Cited by¶
1 citation across 1 artifact.
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Primes¶
- Majority-Dominated Aggregate Objective
- Attention and media metrics — aggregate engagement is dominated by median-click contributions; rare-but-important content is starved. Public-health surveillance — aggregate cost-effectiveness measures dominated by common-condition burden divert resources from rare-but-severe events.
This sourceShows conventional cost-per-QALY analysis systematically favors common conditions over rare diseases, diverting resources from rare-but-severe events whose burden is small in aggregate.
Supported in partVerified against the publisher's abstract
Abstract argues conventional cost-per-QALY analysis is ill-suited to rare diseases and misaligns reimbursement, supporting the direction but not the resource-diversion mechanism.
“This "extra-welfarist" approach has produced pricing and reimbursement systems that are not well- aligned with the unique considerations of orphan drugs.”
- Attention and media metrics — aggregate engagement is dominated by median-click contributions; rare-but-important content is starved. Public-health surveillance — aggregate cost-effectiveness measures dominated by common-condition burden divert resources from rare-but-severe events.
Verification¶
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