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 New malaria analysis method reveals disease severity in minutes
14.08.2017 | University of British Columbia

nachricht New type of blood cells work as indicators of autoimmunity
14.08.2017 | Instituto de Medicina Molecular

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: Fizzy soda water could be key to clean manufacture of flat wonder material: Graphene

Whether you call it effervescent, fizzy, or sparkling, carbonated water is making a comeback as a beverage. Aside from quenching thirst, researchers at the University of Illinois at Urbana-Champaign have discovered a new use for these "bubbly" concoctions that will have major impact on the manufacturer of the world's thinnest, flattest, and one most useful materials -- graphene.

As graphene's popularity grows as an advanced "wonder" material, the speed and quality at which it can be manufactured will be paramount. With that in mind,...

Im Focus: Exotic quantum states made from light: Physicists create optical “wells” for a super-photon

Physicists at the University of Bonn have managed to create optical hollows and more complex patterns into which the light of a Bose-Einstein condensate flows. The creation of such highly low-loss structures for light is a prerequisite for complex light circuits, such as for quantum information processing for a new generation of computers. The researchers are now presenting their results in the journal Nature Photonics.

Light particles (photons) occur as tiny, indivisible portions. Many thousands of these light portions can be merged to form a single super-photon if they are...

Im Focus: Circular RNA linked to brain function

For the first time, scientists have shown that circular RNA is linked to brain function. When a RNA molecule called Cdr1as was deleted from the genome of mice, the animals had problems filtering out unnecessary information – like patients suffering from neuropsychiatric disorders.

While hundreds of circular RNAs (circRNAs) are abundant in mammalian brains, one big question has remained unanswered: What are they actually good for? In the...

Im Focus: RAVAN CubeSat measures Earth's outgoing energy

An experimental small satellite has successfully collected and delivered data on a key measurement for predicting changes in Earth's climate.

The Radiometer Assessment using Vertically Aligned Nanotubes (RAVAN) CubeSat was launched into low-Earth orbit on Nov. 11, 2016, in order to test new...

Im Focus: Scientists shine new light on the “other high temperature superconductor”

A study led by scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg presents evidence of the coexistence of superconductivity and “charge-density-waves” in compounds of the poorly-studied family of bismuthates. This observation opens up new perspectives for a deeper understanding of the phenomenon of high-temperature superconductivity, a topic which is at the core of condensed matter research since more than 30 years. The paper by Nicoletti et al has been published in the PNAS.

Since the beginning of the 20th century, superconductivity had been observed in some metals at temperatures only a few degrees above the absolute zero (minus...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Call for Papers – ICNFT 2018, 5th International Conference on New Forming Technology

16.08.2017 | Event News

Sustainability is the business model of tomorrow

04.08.2017 | Event News

Clash of Realities 2017: Registration now open. International Conference at TH Köln

26.07.2017 | Event News

 
Latest News

A Map of the Cell’s Power Station

18.08.2017 | Life Sciences

Engineering team images tiny quasicrystals as they form

18.08.2017 | Physics and Astronomy

Researchers printed graphene-like materials with inkjet

18.08.2017 | Materials Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>