US Better for Rare Disease Drug Applications; Europe Needs to Do More

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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

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 '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

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. "

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Notes to editors

Orphan drug development is progressing too slowly. Joppi, Bertele and

Garattini, Negri Institute for Pharmacological Research, Milan,

Italy. British Journal of Clinical Pharmacology. Volume 61.3, pages

355 to 360 (March 2006).

A Quality Adjusted Life Year is calculated by estimating the total

number of life years gained from treatment and weighting each year

with a quality of life score to reflect the quality of life in that

year.

The National Institute for Health and Clinical Excellence is part of

the National Health Service (NHS). Its role includes promoting better

use of resources in the NHS by focusing them on treatments which

achieve the greatest health gain.

The British Journal of Clinical Pharmacology is published monthly on

behalf of the British Pharmacological Society by Blackwell

Publishing. It contains papers and reports on all aspects of drug

action in humans: invited review articles, original papers, short

communications and correspondence. The Journal, which was first

published in 1974, enjoys a wide readership, bridging the gap between

the medical profession, clinical research and the pharmaceutical

industry. www.blackwellpublishing.com/bjcp