EMA orphan approval hike reflects biology advances and pharma economics says expert

By Gareth Macdonald

- Last updated on GMT

Related tags Small molecule drugs

Orphan approvals up in Europe in 2014
Orphan approvals up in Europe in 2014
The record number of orphan drugs approved in Europe last year reflects the pharma industry’s need to refill pipelines and scientific advances says Genetic Alliance UK

According to European Medicines Agency (EMA), 17 of the 82 drugs approved in 2014 were for rare diseases with PTC Therapeutics’ muscular dystrophy pill Translarna and Chiesi’s stem cell eye disease medicine Holodar being the highlights.

The 2014 figures fit with a general uptick in orphan approvals over the past decade according to Alastair Kent OBE, Director of Genetic Alliance UK, who told in-Pharmatechnologist.com the growth reflects converging economic and scientific developments.

The pharmaceutical industry realises it has picked all the ‘low hanging fruit’ small molecule drugs for large populations and is shifting its focus to products for smaller patient populations to refill pipelines.

In addition, our understanding of biology has reached a point where even for common diseases we can identify subpopulations more likely to benefit from treatments than other groups​,” Kent said, adding that “under these circumstances it is not surprising more drugmakers are looking at rare disease​.”

This idea is supported by EMA data which shows that 327 applications for orphan drug designation were filed by the pharmaceutical industry in 2014​, up from 201 in 2013 and 197 the year before that.   

Orphan drug costs

Another reason for renewed industry interest is the realisation that, despite significant criticism, manufacturers are able to charge high prices for rare disease treatments.

For example, a year’s supply of BioMarin’s drug Vimizim – which was approved in the US last February for the rare disease Morquio A syndrome – costs $380,000.

Similarly, Genzyme’s mucopolysaccharidosis treatment Aldurazyme can cost $500,000 and Alexion’s paroxysmal nocturnal hemoglobinuria drug Soliris will set you back $440,000 for a year’s supply.

Kent acknowledged that such revenue potential was a driver for industry interest and said his organisation “has no problem with high prices, if they are fair.”

Industry needs to be transparent. Firms setting prices should not ask ‘what can the market bear?’ but what is fair value based on the difficulty of developing and manufacturing the product​.”


Aside from economics and biology, the European regulatory landscape is slowly become more conducive to firms developing drugs for rare diseases according to Kent, who said while the current system is not fit for purpose “things are moving in the right direction​.”

National competent authorities and the EMA are thinking about how to create an improved framework for orphan drugs,” ​Kent said, citing adaptive licensing and early access models as the key developments.

He contrasted this with regulatory initiatives like the £200m UK Cancer Drug Fund (CDF) – which was established in 2011 to pay for cancer drugs that are not routinely available on the National Health Service (NHS) – which he said was “not sustainable.​”

You can’t have a system that prioritises some diseases – be they rare or common – and expect equality in terms of regulatory reviews​.”

Controversial drug fund

In related news, reports this week suggest that the NHS in England may remove some drugs from the list of those accessible through the CDF, with products made by Roche, Sanofi and Eisai likely to be affected.

Kent’s comments echo those of Richard Sullivan, director of the Institute of Cancer Policy, London, who told the Guardian​ “the Cancer Drugs Fund was a cheap political fix. Worse, it is unethical.

You cannot give priority to cancer over all other serious illnesses, including coronary ailments and dementia. These types of patients are just as deserving of expensive medicines as are cancer patients.”

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