The Pharmaceutical Journal
Vol 280 No 7490 p216-218
23 February 2008

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Do orphan medicines benefit patients?

More and more governments are providing incentives to companies to develop treatments for rare diseases. However, although many of these initiatives have been a success, the treatments do not always reach those who need them.
Jackie Holding reports

SUMMARY

Some diseases are so rare that no pharmaceutical company would consider it financially viable to develop a treatment or cure without special support or incentives. An estimated 30 million EU citizens and 20 to 25 million US citizens have one of these 6,000 “orphan” diseases so although each disease affects a tiny number of people, collectively they represent a significant health problem.

Although some of these conditions are untreatable, others are not and where governments have acted to provide incentives to industry research and development of orphan medicines have been stimulated, albeit with varying degrees of success.

Incentive schemes have contributed to the development of a variety of new treatments, but these — especially in the UK — do not always reach the patient. Access may be barred or slow if the local health funding body refuses to pay what is often a premium for medicines to treat rare diseases.

Many countries have their own definition of orphan disease. In the US, for example, the term refers to conditions with a prevalence of seven cases per 10,000 population whereas in Japan the definition is narrower, at 2.5 cases per 10,000 population. The EU definition is a prevalence of five cases or fewer per 10,000 population.

In the US, the incentive scheme introduced under the 1983 Orphan Drugs Act helped increase the number of medicines and biologic products that came to market exponentially: 282 have come on stream in the past 24 years, compared with 10 approved by the Food and Drug Administration in the eight to 10 years preceding the Act.

Under the Act all designated orphan products are eligible for a federal tax credit up to 50 per cent of the clinical research expenditure. Orphan products are exempt from the application fee for FDA approval and the first product authorised for a specific indication gets a seven-year marketing exclusivity period. Congress assigns about $20m for FDA for orphan product grants.

The considerable success of US efforts to provide incentives for companies to develop orphan medicinal products have led other countries to follow suit and similar schemes were introduced in Japan in 1995, in Singapore in 1997 and in Australia in 1998.

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