Orphan Drug Approvals on the Up



Orphan drugs are those drugs that treat neglected and rare diseases. Historically, they have been left on the shelf, predominately due to the fact that the countries with the highest pick-up for these medications tend to be financially poor. But latest research is showing that the tide may be turning, as more orphan drugs than ever before are being given FDA approval.

The research was conducted by The Tufts Centre for the Study of Drug Development in the US. It showed that there was an average of 5 drugs being approved each year for orphan diseases, which is an increase of over 50% compared to 10 years ago.

The big three are certainly hogging the limelight though- malaria, TB and paediatric indications for HIV are responsible for most of this good news, although there are currently drugs in the pipeline for rarer diseases such as Buruli ulcer, Dengue fever and trachoma.

An inescapable fact of orphan drugs however is their cost. Pharmaceutical companies usually spend a long time researching them and trialing them before they obtain those elusive approvals, which means their finished price tends to be very high. To get these drugs out to the countries that often need them most relies heavily on government subsidies and negotiations with the drug developers.

There can be immense benefits to orphan drug research though- it is down to such research that sufferers of cystic fibrosis can survive into their 50s nowadays and the research into homozygous familial hypercholesterolemia has led to the development of statins, cholesterol-lowering drugs taken by many over-50’s.

To read more about the research and about orphan drugs, please click here:




Image provided courtesy of http://beltina.org


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