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“The only thing necessary for these diseases to the triumph is for good people and governments to do nothing.”




Orphan drugs need foster parents



European pharmaceutical companies need to guarantee commercial returns from the new drugs they develop - so tough luck if you suffer from a rare illness or live in a developing country. But experience in the USA, Japan and Australia shows 'orphan' drugs can be profitable.

I t costs an estimated 250 million to develop a new drug. If pharmaceutical companies cannot sell cures to millions of people, they stand no chance of recouping huge research costs. So it's hardly surprising they concentrate on potential best-sellers: drugs for heart disease, ulcers, diabetes, Alzheimer's disease, the common cold. Drugs for unprofitable diseases, known as 'orphan' drugs, are generally abandoned at birth.

Unprofitable diseases include those that are rare and those in developing countries with no money to buy expensive medications. Producing affordable therapies for developing countries is difficult, but for rare-disease sufferers there is more hope. By changing legal constraints which affect profitability, we may persuade pharmaceutical companies that rare diseases are worth targeting. This approach seems to work elsewhere, and now the European Parliament is tackling the issue.


Low incidence doesn't mean few sufferers

The label 'rare' is usually taken to mean that a disease affects fewer than 7.5 people per 10,000. This includes well-known scourges such as multiple sclerosis, which affects some 500,000 people in Europe. The World Health Organisation (WHO) estimates that there are around 5,000 rare diseases, affecting 20 million people.

Rare diseases can be difficult to diagnose because doctors tend to be unfamiliar with them. They are also hard to cure: 50-80% are genetic in origin and require high-tech remedies such as gene therapy rather than traditional organic chemistry. But, argues the Parliament's STOA Panel, most of the investment needed for gene therapy will be in the development of basic techniques which serve as a platform for other treatments. Effectively, basic R&D costs for orphan drugs are much the same as for any other drugs. To make orphan drugs commercially attractive, something else will have to change.

Lowering the barriers

In the USA and other countries with positive experiences in orphan drug development, that something is the regulatory framework. The US Orphan Drug Act of 1983 and its amendments give the developer an exclusive seven-year marketing period to recoup development costs. Orphan drugs also benefit from fast-track registration and a 50% tax credit on clinical trials in the USA.

It seems to work. In 14 years, 890 substances have been granted orphan drug status in the USA and 173 have received marketing authorisation. Ninety-two organisations have launched one or more orphan drugs. As a result, an estimated 6.5 million American patients stand to benefit.

Orphan drugs in Australia have an exclusive five-year marketing period, exemption from the normal fees imposed by the Therapeutic Goods Administration (TGA), and their own registration procedure. In Japan, special status granted to orphan drugs in 1993 has encouraged the launch of 35 new formulae.


Progress in Europe

France, Spain, Sweden, Denmark, the UK and Germany have all considered the status of orphan drugs, but European harmonisation and commercial encouragement are lacking. The EU now proposes official regulatory status for orphan drugs, with full or partial exemption from registration fees, an exclusive marketing period of ten years, and help with clinical trials.

Tax breaks for R&D are a possibility too. In the USA and Japan, around 40% of the organisations that have licensed orphan drugs are small or medium-sized pharmaceutical companies. This is remarkable in a competitive climate which is forcing even the largest firms to merge to offset the huge costs of drug development. Orphan drugs represent a good opportunity for small firms, and this, says the European Commission, will help Europe remain competitive in the world biotech stakes.

Under the Fifth Framework Programme, research on rare diseases will remain a priority.