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Drug Companies Accused Of Exploiting Rules on Treatments for Rare Diseases

From Guardian (UK) (November 17, 2010)

Drug companies are today accused of making exorbitant profits from the NHS by exploiting arrangements designed to encourage them to develop new drugs for rare diseases.

Twenty consultants and a patients’ group are publishing an open letter to the prime minister, calling for an inquiry. They tell David Cameron that, far from inventing new drugs, companies are in effect repackaging them to get a licence, enabling them to hike the price hugely.

Legislation was brought in by the EU to encourage companies to devise and seek licenced for new drugs for what are called "orphan" diseases - those for which there is not a huge market because they are relatively rare.

But the letter’s signatories say the change in the rules has had unintended consequences. They cite a drug which has been used for the last 20 years to treat two rare muscle diseases. Although it did not have a licence for that use, doctors could prescribe it - and did - on their own authority. It used to cost around pounds 800 to pounds 1,000 per patient per year.

But the manufacturer, BioMarin, sought a licence to supply throughout Europe what it says is a more stable and reliable version of the drug, which it calls Firdapse. It charges pounds 40,000 to pounds 70,000 per patient per year - up to a 70-fold increase.

The doctors say there was no need for a new version. "This high cost means firstly that some funders [primary care trusts] have refused to pay for the drug . . . It also means that, where it is funded, no additional funding source has been identified, which must mean that patients in other areas are being deprived of NHS funding. The cost to the NHS is likely to be above pounds 10m a year," they say in their letter, published on the British Medical Journal website today.

Other examples include a drug for chronic myeloid leukaemia called hydroxycarbamide. Doctors also used it for sickle cell disease, a condition for which it was not licensed. A year’s treatment with 500g capsules cost pounds 160. A version that has been licensed now costs pounds 14,900 a year. "In the present economic situation, it seems vital to ensure that systems are in place to prevent excessive commercial profits being made at the expense of patients and public spending," say the signatories.

They conclude: "Legislation on orphan drugs, far from encouraging the development of new treatments for orphan diseases, is severely limiting the availability of existing treatments. We believe that the Medicines and Healthcare Products Regulatory Agency and Department of Health should not just state the rules but should act now . . . to instigate change."


Posted: November 2010