Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

5,000 rare diseases need drugs, but Europe only approves a handful each year

06.03.2006


Only seven per cent of drug applications for treating people with rare diseases were approved in Europe between 2000 and 2004, despite the fact that there are currently more than 5,000 conditions needing medication.



Yet during the same period, more than 79 per cent of the other drug applications submitted to the European Agency for the Evaluation of Medicinal Products (EMEA) were approved, according to research published in the latest British Journal of Clinical Pharmacology.

“It’s difficult to find a balance between the urgent need for drugs for patients with rare diseases and guaranteeing their quality, efficacy, safety and, where necessary, making comparisons with existing drugs” says co-author Professor Silvio Garattini from the Mario Negri Institute for Pharmacological Research in Milan, Italy.


“The lack of reliable methods for evaluating ‘orphan drugs’ in a small number of people probably explains the poor quality of the applications.

“However, it is clear that less stringent criteria are acceptable for orphan drugs than for drugs for more common diseases, particularly in view of the small number of patients.”

Between August 2000, when new legislation came into force, and December 2004, EMEA’s Committee on Orphan Medical Products reviewed 255 possible drugs for rare diseases that affect less than five people in 10,000.

Only 18 orphan drugs were approved on the basis of epidemiological data, medical plausibility and potential benefit.

During the same period the EMEA received 193 marketing authorisation applications for non orphan drugs and 153 of these were approved.

“However, ten of the 18 orphan drugs approved were authorised under exceptional circumstances which means that the dosier was not complete and the Committee required additional studies in order to maintain the marketing authorisation” says Professor Garattini.

Rare diseases covered by the approved drugs included two rare forms of leukaemia, Fabry disease, which affects the body’s ability to break down lipids and Wilson’s disease, in which copper build-up can damage vital organs.

“In the last 20 years international efforts have been made to encourage companies to develop orphan drugs by providing incentives like tax credits and research aids, simplifying marketing authorisation procedures and extending market exclusivity” adds Dr Jeffrey Aronson, Chair of the journal’s editorial board. “Only the last of these incentives is available in Europe.

“The experience in Europe also contrasts sharply with the USA, where there are more incentives - 1,100 drugs and biological products were designated orphan products there between 1983 and 2002 and 231 were approved.”

Dr Aronson, Reader in Clinical Pharmacology at Oxford University, also points out that the UK National Institute for Health and Clinical Excellence (NICE) does not generally approve drugs for National Health Service use unless their costs are below £30,000 per Quality Adjusted Life Year. Orphan drugs are likely to cost more than that.

“This study suggests that we need more incentives in Europe to develop orphan drugs and to develop them cost-effectively, so as not to compromise our ability to manage other diseases” he adds.

“The tension between equity and affordability is unbearable and pulls in both directions – those with rare diseases deserve to be treated but those with common diseases should not be expected to subsidise them.”

Annette Whibley | alfa
Further information:
http://www.blackwellpublishing.com/bjcp

More articles from Health and Medicine:

nachricht Team discovers how bacteria exploit a chink in the body's armor
20.01.2017 | University of Illinois at Urbana-Champaign

nachricht Rabies viruses reveal wiring in transparent brains
19.01.2017 | Rheinische Friedrich-Wilhelms-Universität Bonn

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Traffic jam in empty space

New success for Konstanz physicists in studying the quantum vacuum

An important step towards a completely new experimental access to quantum physics has been made at University of Konstanz. The team of scientists headed by...

Im Focus: How gut bacteria can make us ill

HZI researchers decipher infection mechanisms of Yersinia and immune responses of the host

Yersiniae cause severe intestinal infections. Studies using Yersinia pseudotuberculosis as a model organism aim to elucidate the infection mechanisms of these...

Im Focus: Interfacial Superconductivity: Magnetic and superconducting order revealed simultaneously

Researchers from the University of Hamburg in Germany, in collaboration with colleagues from the University of Aarhus in Denmark, have synthesized a new superconducting material by growing a few layers of an antiferromagnetic transition-metal chalcogenide on a bismuth-based topological insulator, both being non-superconducting materials.

While superconductivity and magnetism are generally believed to be mutually exclusive, surprisingly, in this new material, superconducting correlations...

Im Focus: Studying fundamental particles in materials

Laser-driving of semimetals allows creating novel quasiparticle states within condensed matter systems and switching between different states on ultrafast time scales

Studying properties of fundamental particles in condensed matter systems is a promising approach to quantum field theory. Quasiparticles offer the opportunity...

Im Focus: Designing Architecture with Solar Building Envelopes

Among the general public, solar thermal energy is currently associated with dark blue, rectangular collectors on building roofs. Technologies are needed for aesthetically high quality architecture which offer the architect more room for manoeuvre when it comes to low- and plus-energy buildings. With the “ArKol” project, researchers at Fraunhofer ISE together with partners are currently developing two façade collectors for solar thermal energy generation, which permit a high degree of design flexibility: a strip collector for opaque façade sections and a solar thermal blind for transparent sections. The current state of the two developments will be presented at the BAU 2017 trade fair.

As part of the “ArKol – development of architecturally highly integrated façade collectors with heat pipes” project, Fraunhofer ISE together with its partners...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Sustainable Water use in Agriculture in Eastern Europe and Central Asia

19.01.2017 | Event News

12V, 48V, high-voltage – trends in E/E automotive architecture

10.01.2017 | Event News

2nd Conference on Non-Textual Information on 10 and 11 May 2017 in Hannover

09.01.2017 | Event News

 
Latest News

Helmholtz International Fellow Award for Sarah Amalia Teichmann

20.01.2017 | Awards Funding

An innovative high-performance material: biofibers made from green lacewing silk

20.01.2017 | Materials Sciences

Ion treatments for cardiac arrhythmia — Non-invasive alternative to catheter-based surgery

20.01.2017 | Life Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>