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 Advanced analysis of brain structure shape may track progression to Alzheimer's disease
26.10.2016 | Massachusetts General Hospital

nachricht Indian roadside refuse fires produce toxic rainbow
26.10.2016 | Duke University

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: Etching Microstructures with Lasers

Ultrafast lasers have introduced new possibilities in engraving ultrafine structures, and scientists are now also investigating how to use them to etch microstructures into thin glass. There are possible applications in analytics (lab on a chip) and especially in electronics and the consumer sector, where great interest has been shown.

This new method was born of a surprising phenomenon: irradiating glass in a particular way with an ultrafast laser has the effect of making the glass up to a...

Im Focus: Light-driven atomic rotations excite magnetic waves

Terahertz excitation of selected crystal vibrations leads to an effective magnetic field that drives coherent spin motion

Controlling functional properties by light is one of the grand goals in modern condensed matter physics and materials science. A new study now demonstrates how...

Im Focus: New 3-D wiring technique brings scalable quantum computers closer to reality

Researchers from the Institute for Quantum Computing (IQC) at the University of Waterloo led the development of a new extensible wiring technique capable of controlling superconducting quantum bits, representing a significant step towards to the realization of a scalable quantum computer.

"The quantum socket is a wiring method that uses three-dimensional wires based on spring-loaded pins to address individual qubits," said Jeremy Béjanin, a PhD...

Im Focus: Scientists develop a semiconductor nanocomposite material that moves in response to light

In a paper in Scientific Reports, a research team at Worcester Polytechnic Institute describes a novel light-activated phenomenon that could become the basis for applications as diverse as microscopic robotic grippers and more efficient solar cells.

A research team at Worcester Polytechnic Institute (WPI) has developed a revolutionary, light-activated semiconductor nanocomposite material that can be used...

Im Focus: Diamonds aren't forever: Sandia, Harvard team create first quantum computer bridge

By forcefully embedding two silicon atoms in a diamond matrix, Sandia researchers have demonstrated for the first time on a single chip all the components needed to create a quantum bridge to link quantum computers together.

"People have already built small quantum computers," says Sandia researcher Ryan Camacho. "Maybe the first useful one won't be a single giant quantum computer...

All Focus news of the innovation-report >>>



Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

Agricultural Trade Developments and Potentials in Central Asia and the South Caucasus

14.10.2016 | Event News

World Health Summit – Day Three: A Call to Action

12.10.2016 | Event News

Latest News

How nanoscience will improve our health and lives in the coming years

27.10.2016 | Materials Sciences

OU-led team discovers rare, newborn tri-star system using ALMA

27.10.2016 | Physics and Astronomy

'Neighbor maps' reveal the genome's 3-D shape

27.10.2016 | Life Sciences

More VideoLinks >>>