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Updated: May 15, 2026
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
What is wrong with orphan drug policies?
1Faculté des sciences de l'administration, Université Laval, Québec, QC, Canada. Andre.cote@mng.ulaval.ca
Summary
Orphan drug policies create business opportunities but often result in high drug prices, limiting patient access. This raises concerns about equitable healthcare access and drug cost-effectiveness.
Area of Science:
- Health Economics
- Pharmaceutical Policy
- Drug Accessibility
Background:
- Orphan drug policies aim to incentivize rare disease drug development.
- These policies have raised concerns regarding patient access and reimbursement.
Purpose of the Study:
- To evaluate the real-world accessibility of drugs under orphan drug policies.
- To identify unforeseen consequences of these policies on patients and manufacturers.
Main Methods:
- Analysis of orphan drug policy effects on drug accessibility.
- Identification of causes for inaccessibility of orphan drugs.
- Examination of implications for healthcare equity and payer decisions.
Main Results:
- Orphan drug policies present unique business opportunities for manufacturers.
- High pricing of orphan-approved drugs often leads to inaccessibility for patients.
- Off-label use contributes to accessibility challenges.
Conclusions:
- Orphan drug policies may inadvertently create barriers to equitable drug access.
- The high cost of orphan drugs challenges payer decisions regarding value for money.
- Revisiting policy incentives is crucial for improving patient access to rare disease treatments.
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