What makes an orphan drug?

  title={What makes an orphan drug?},
  author={Nigel Hawkes and Deborah Cohen},
  journal={BMJ : British Medical Journal},
Nigel Hawkes and Deborah Cohen investigate claims that drug companies are making easy profits by licensing existing treatments for rare diseases 
Regulation is flawed
  • C. Counsell
  • Political Science
    BMJ : British Medical Journal
  • 2010
Something is fundamentally wrong with a drug regulatory system that makes it increasingly difficult to do independent pragmatic trials of widely used but unproven drugs yet licenses a ridiculously
Orphan drugs in oncology
The number of designations will “continue toincrease exponentially” within the next months and years and consequently will the number of successful marketing authorisations increase.
Rare diseases and orphan drugs.
This paper illustrates mainly the European initiatives and will discuss the problematic and controversial aspects surrounding orphan drugs, as well as activities and measures adopted in Italy.
Effectiveness, safety and costs of orphan drugs: an evidence-based review
Evaluating the clinical effectiveness of orphan drugs that have been granted marketing licenses in Europe, determining the annual costs of each drug, compare the costs of branded orphan drugs against their generic equivalents, and explore any relationships between orphan drug disease prevalence and annual costs suggest there is inconsistency in the quality of evidence.
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
The present system should be changed to find better ways of fostering the development of effective and sustainable treatments for patients with orphan diseases, and more stringent criteria to assess OMPs’ efficacy and cost/effectiveness would improve the clinical value and the affordability of products allowed onto the market.
One small step for the NHS, but one giant leap for its principles?
This paper documents how the British health service has moved away from this principle during the past 30 years, as very minor initial changes in specific policy areas have led eventually to major changes towards private funding.
Contratos de riesgo compartido, ¿con medicamentos huérfanos?
If thoroughly managed, RSA may reasonably contribute to value-based pricing of OD, improve their pharmacovigilance, knowledge about their comparative effectiveness, and to reducing uncertainty and its consequences on patients, industry, payers and clinicians.
Application of orphan drug designation to cancer treatments (2008–2017): a comprehensive and comparative analysis of the USA and EU
This study shows that the FDA approves more cancer drugs with orphan designations compared with the EMA, especially for subgroups of more prevalent cancers.
Too early to question effectiveness of Dutch system
The study by Evers et al is the first to show a higher mortality among births that started in primary care compared with secondary care.1 We have concerns about the methods used. Firstly, although
Analysis of Health Technology Assessments of Orphan Drugs in Ireland from 2012 to 2017
The number of submissions to the NCPE for orphan drugs has increased in recent years and the rapid review and HTA process in Ireland plays a role in supporting the reimbursement decision-making process for orphan Drugs in a similar manner to the process established for non-orphan drugs.


Incentives for drug development--the curious case of colchicine.
Drs. Aaron Kesselheim and Daniel Solomon write that it came as a surprise to many patients and physicians that the FDA not only approved the new version of colchicine but also granted the
Orphan drug development is not taking off.
A large number of patients and a fragmented market means it is an area of little commercial interest for pharmaceutical companies, and the high price of orphan drugs tends to put them out of reach for the National Health Service.
Pricing of orphan drugs