High costs of orphan medicines


Igho Onakpoya Research Fellow

Do the high costs of orphan medicines (medicines used to treat rare diseases) provide good value for money?
This is a controversial issue which has been debated over the past few years.

In an attempt to answer this question, we assessed the data on effectiveness, safety and costs of orphan medicines approved for use in Europe.

We searched the European Medicines Agency (EMA) evidence to identify orphan medicines which have been approved over the past 20 years. We then searched other scientific databases to evaluate the evidence regarding the benefits and harms of the approved medicines. We also explored the factors which influence the high costs of the medicines, and we compared the prices of branded orphan drugs with their generic or unlicensed versions.

We identified 74 orphan medicines approved by the EMA up until April 2014.
Of this number, 54 (73%) had the moderate quality of evidence, while 16 (22%) had low evidence, and 4 (5%) had very low evidence.  Approximately 85% of the medicines showed evidence of some benefit for their approved indications, but 86.5% of them also showed possibility of causing severe harms. The annual treatments costs per patient for the medicines ranged from £726 to £378000 depending on the type. When compared with their unbranded or unlicensed versions, the branded orphan medicines were 1.4 to 82000 times more expensive.

Interestingly, we were unable to determine how the annual costs of the drugs were arrived at. In addition, some of these orphan medicines are re-packaged versions of medications which had previously been available for use.

So is it rational to keep paying huge amounts for orphan medicines?

Related links

Evidence of Value of Orphan Drugs Inconsistent


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