Citation Tools
PDF
Other content recommended for you
- FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross sectional analysis
- Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review
- Time to inclusion in clinical guidance documents for non-oncological orphan drugs and biologics with expedited FDA designations: a retrospective survival analysis
- Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
- Trends in utilization of FDA expedited drug development and approval programs, 1987-2014: cohort study
- From blockbuster to “nichebuster”: how a flawed legislation helped create a new profit model for the drug industry
- Affordability and availability of off-patent drugs in the United States—the case for importing from abroad: observational study
- Characteristics of efficacy evidence supporting approval of supplemental indications for prescription drugs in United States, 2005-14: systematic review
- Should rare diseases get special treatment?
- Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs