Article info
Commentary
Double bad luck: Should rare diseases get special treatment?
- Correspondence to Adam Hutchings, Dolon Limited, London, UK; adam.hutchings{at}dolon.com
Citation
Double bad luck: Should rare diseases get special treatment?
Publication history
- Received January 3, 2022
- Accepted January 3, 2022
- First published January 21, 2022.
Online issue publication
January 21, 2022
Request permissions
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.
Copyright information
© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.
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