Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress
Clinical Collections
Advances in Alzheimer’s

Keynote webinar | Spotlight on medication adherence

LIVE: Thursday 27th June 2024, 18:00-19:30 (CEST)
Join our expert panel to discover why you need to understand the drivers of non-adherence in your patients, and how you can optimize medication adherence in your clinics to drastically improve patient outcomes.
ADVANCED SEARCH
Log in
Top
Applied Health Economics and Health Policy
Published in: Applied Health Economics and Health Policy 5/2010

01-09-2010 | Current Opinion

Issues surrounding orphan disease and orphan drug policies in Europe

Authors: Alain Denis, Lut Mergaert, Christel Fostier, Irina Cleemput, Dr Steven Simoens

Published in: Applied Health Economics and Health Policy | Issue 5/2010

Login to get access

Abstract

An orphan disease is a disease with a very low prevalence. Although there are 5000–7000 orphan diseases, only 50 orphan drugs (i.e. drugs developed to treat orphan diseases) were marketed in the EU by the end of 2008. In 2000, the EU implemented policies specifically designed to stimulate the development of orphan drugs. While decisions on orphan designation and the marketing authorization of orphan drugs are made at the EU level, decisions on drug reimbursement are made at the member state level. The specific features of orphan diseases and orphan drugs make them a high-priority issue for policy makers.
The aim of this article is to identify and discuss several issues surrounding orphan disease and drug policies in Europe.
The present system of orphan designation allows for drugs for non-orphan diseases to be designated as orphan drugs. The economic factors underlying orphan designation can be questioned in some cases, as a low prevalence of a certain indication does not equal a low return on investment for the drug across its indications. High-quality evidence about the clinical added value of orphan drugs is rarely available at the time of marketing authorization, due to the low number of patients. A balance must be struck between ethical and economic concerns. To this effect, there is a need to initiate a societal dialogue on this issue, to clarify what society wants and accepts in terms of ethical and economic consequences. The growing budgetary impact of orphan drugs puts pressure on drug expenditure. Indications can be extended for an orphan drug and the total prevalence across indications is not considered. Finally, cooperation needs to be fostered in the EU, particularly through a standardized approach to the creation and use of registries.
These issues require further attention from researchers, policy makers, health professionals, patients, pharmaceutical companies and other stakeholders with a view to optimizing orphan disease and drug policies in Europe.
Footnotes
1
Orphan designation refers to the award of orphan status to a drug, while marketing authorization refers to the approval to market the drug.[1]
 
Literature
1.
2.
European Medicines Agency. Annual report of the European Medicines Agency 2007. London: European Medicines Agency, 2008
3.
Denis A, Simoens S, Fostier C, et al. Health technology assessment policy governing orphan diseases and orphan medicines. Brussels: Belgian Health Care Knowledge Centre, 2009
4.
Yin W. R&D policy, agency costs and innovation in personalized medicine. J Health Econ 2009; 28: 950–62PubMedCrossRef
5.
European Medicines Agency. COMP report to the Commission in relation to article 10 of regulation 141 /2000 on orphan medicinal products. London: European Medicines Agency, 2007
6.
Dear JW, Lilitkarntakul P, Webb DJ. Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. Br J Clin Pharmacol 2006; 62: 264–71PubMedCrossRef
7.
8.
Sheridan C. EU to review rare disease drugs market exclusivity [letter]. Nat Biotechnol 2004; 22(9): 1061PubMedCrossRef
9.
European Medicines Agency. Committee for Medicinal Products for Human Use post-authorisation summary of positive opinion for Revatio. London: European Medicines Agency, 2009
10.
11.
12.
13.
Cleemput I, Neyt M, Thiry N, et al. Cost-effectiveness thresholds in health care. Brussels: Belgian Healthcare Knowledge Centre, 2008
14.
Drummond M, Evans B, LeLorier J, et al. Evidence and values: requirements for public reimbursement of drugs for rare diseases — a case study in oncology. Can J Clin Pharmacol 2009; 16: e273–81PubMed
15.
Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics 2005; 31: 164–8PubMedCrossRef
16.
Littlejohns P, Garner S, Doyle N, et al. 10 years of NICE: still growing and still controversial. Lancet Oncol 2009; 10: 417–24PubMedCrossRef
17.
Connock M, Juarez-Garcia A, Frew E, et al. A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry’s disease and mucopolysaccharidosis type 1. Health Technol Assess 2006; 10: iii–113
18.
Heemstra HE, de Vrueh RL, van WS, et al. Orphan drug development across Europe: bottlenecks and opportunities. Drug Discov Today 2008; 13: 670–6PubMedCrossRef
19.
Schlander M, Beck M. Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI. Curr Med Res Opin 2009; 25: 1285–93PubMedCrossRef
20.
Cook JP, Vernon JA, Manning R. Pharmaceutical risk-sharing agreements. Pharmacoeconomics 2008; 26(7): 551–6PubMedCrossRef
21.
Orofino J, Soto J, Casado MA, et al. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy 2010; 8(5): 301–15PubMedCrossRef
22.
23.
24.
Owen A, Sprinks J, Meehan A, et al. A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry. J Med Econ 2008; 11: 235–43PubMed
25.
Metadata
Title
Issues surrounding orphan disease and orphan drug policies in Europe
Authors
Alain Denis
Lut Mergaert
Christel Fostier
Irina Cleemput
Dr Steven Simoens
Publication date
01-09-2010
Publisher
Springer International Publishing
Published in
Applied Health Economics and Health Policy / Issue 5/2010
Print ISSN: 1175-5652
Electronic ISSN: 1179-1896
DOI
https://doi.org/10.2165/11536990-000000000-00000

Other articles of this Issue 5/2010

Applied Health Economics and Health Policy 5/2010 Go to the issue

Original Research Article

Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007

Original Research Article

Burden and cost of hospital admissions for vaccine-preventable paediatric pneumococcal disease and non-typable Haemophilus influenzae otitis media in New Zealand

Editorial

Economic efficiency of alcohol policy

Original Research Article

Cost effectiveness of long-acting risperidone in Sweden

Original Research Article

Cost of illness and drivers of cost in atrial fibrillation in Sweden and Germany