The price you pay

In 2000, the European Union (EU) legislated to encourage the pharmaceutical industry to develop ‘orphan’ drugs (i.e. drugs for rare diseases).1 Incentives include up to 10-year market exclusivity, licensing fee reductions, and initiatives supporting research and development.1 Without incentives, such drugs would be unlikely to attract industry investment. However, sometimes there has been a high resulting price to pay.

The EU legislation is based on the principle of equity: “Patients suffering from rare conditions should be entitled to the same quality of treatment as other patients.”1 To date, over 60 orphan-designated medicines have been licensed in the EU, potentially benefiting over 2.6million patients.2 Many of these medicines are new chemical entities. However, older drugs (unlicensed, or licensed for a different indication) have also been approved as orphan drugs, and then sold at a much higher price than the previously available versions.3 The latest such product is ▼amifampridine 10mg, licensed in May this year for Lambert Eaton myasthenic syndrome (LEMS). An unlicensed version has been used for many years in the UK for this disorder, at an annual cost of around £1,000 per patient. The comparative cost with the licensed product ranges from £11,000 to £44,000 per year. Of note, no published randomised controlled trials are available on the safety and efficacy of the licensed formulation or how it compares with the unlicensed version (for which there are two small trials).

There are around 150 patients with LEMS in the UK. If supplies of unlicensed amifampridine run out, the additional annual cost to the NHS will be in the range of £1.5–6.5million. While the EU support for orphan medicines is laudable, we believe that price regulation should be part of the deal.

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