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


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:

More articles from Health and Medicine:

nachricht Scientists develop tiny tooth-mounted sensors that can track what you eat
22.03.2018 | Tufts University

nachricht NIH scientists describe potential antibody treatment for multidrug-resistant K. pneumoniae
14.03.2018 | NIH/National Institute of Allergy and Infectious Diseases

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: Researchers Discover New Anti-Cancer Protein

An international team of researchers has discovered a new anti-cancer protein. The protein, called LHPP, prevents the uncontrolled proliferation of cancer cells in the liver. The researchers led by Prof. Michael N. Hall from the Biozentrum, University of Basel, report in “Nature” that LHPP can also serve as a biomarker for the diagnosis and prognosis of liver cancer.

The incidence of liver cancer, also known as hepatocellular carcinoma, is steadily increasing. In the last twenty years, the number of cases has almost doubled...

Im Focus: Researchers at Fraunhofer monitor re-entry of Chinese space station Tiangong-1

In just a few weeks from now, the Chinese space station Tiangong-1 will re-enter the Earth's atmosphere where it will to a large extent burn up. It is possible that some debris will reach the Earth's surface. Tiangong-1 is orbiting the Earth uncontrolled at a speed of approx. 29,000 km/h.Currently the prognosis relating to the time of impact currently lies within a window of several days. The scientists at Fraunhofer FHR have already been monitoring Tiangong-1 for a number of weeks with their TIRA system, one of the most powerful space observation radars in the world, with a view to supporting the German Space Situational Awareness Center and the ESA with their re-entry forecasts.

Following the loss of radio contact with Tiangong-1 in 2016 and due to the low orbital height, it is now inevitable that the Chinese space station will...

Im Focus: Alliance „OLED Licht Forum“ – Key partner for OLED lighting solutions

Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP, provider of research and development services for OLED lighting solutions, announces the founding of the “OLED Licht Forum” and presents latest OLED design and lighting solutions during light+building, from March 18th – 23rd, 2018 in Frankfurt a.M./Germany, at booth no. F91 in Hall 4.0.

They are united in their passion for OLED (organic light emitting diodes) lighting with all of its unique facets and application possibilities. Thus experts in...

Im Focus: Mars' oceans formed early, possibly aided by massive volcanic eruptions

Oceans formed before Tharsis and evolved together, shaping climate history of Mars

A new scenario seeking to explain how Mars' putative oceans came and went over the last 4 billion years implies that the oceans formed several hundred million...

Im Focus: Tiny implants for cells are functional in vivo

For the first time, an interdisciplinary team from the University of Basel has succeeded in integrating artificial organelles into the cells of live zebrafish embryos. This innovative approach using artificial organelles as cellular implants offers new potential in treating a range of diseases, as the authors report in an article published in Nature Communications.

In the cells of higher organisms, organelles such as the nucleus or mitochondria perform a range of complex functions necessary for life. In the networks of...

All Focus news of the innovation-report >>>



Industry & Economy
Event News

Virtual reality conference comes to Reutlingen

19.03.2018 | Event News

Ultrafast Wireless and Chip Design at the DATE Conference in Dresden

16.03.2018 | Event News

International Tinnitus Conference of the Tinnitus Research Initiative in Regensburg

13.03.2018 | Event News

Latest News

Modular safety concept increases flexibility in plant conversion

22.03.2018 | Trade Fair News

New interactive map shows climate change everywhere in world

22.03.2018 | Earth Sciences

New technologies and computing power to help strengthen population data

22.03.2018 | Earth Sciences

Science & Research
Overview of more VideoLinks >>>