The Orphan’s Drug Market – Part 1

February 07, 2013 | By Márcio Barra

A few days ago, I grabbed hold of an issue of Hospital Pharmacy Europe, the May/June 2012 issue I had lying around at home. Looking at the cover, the featured article on orphan drugs picked my interest and I ended up reading the admittedly short article.

As I read, some question came to mind. Namely, if the increased focus on orphan therapies is a sustainable market. And so I was prompted to write this article.

Please enjoy this three part article on orphan drugs. If you like it, please share with your colleagues.


Orphan Drugs

The most distinguishing feature of a drug with an orphan designation, versus a normal drug is the intended market and target disease. An Orphan drug is for life threatening or chronically debilitating conditions, whose prevalence in the EU is not more than 5 in 10,000. Thus they are handicapped in generating sufficient profits to pay off its development. This threshold changes from a country to another. In the USA its 7.5 per 10,000 and in Japan it’s 4 per 10,000. (note: some drugs can be for more common diseases but also have an indication for an orphan disease) (1).The other prerequisite is that no other method of treatment for the given condition exists, or, if it already exists, then the orphan drug must be of significant benefit to the patient (2).

Orphan drugs were, until very recently, rare. It was not an attractive market for big pharma. Twenty years ago, it was the age of the blockbuster. Companies had a “go big or go broke” mentality, and drug development was aimed at molecules that had a big pool of patients.

When the US Congress passed the Orphan Drug Act in 1983, about one new treatment a year entered the market with an orphan designation. Similar acts followed in 1991 in Singapore, 1993 in Japan, 1997 in Australia, and finally, 2000 by the European Union, establishing the EMA Committee for Orphan Medicinal Products (COMP) (3). Nowadays, save for some key areas, like neurology, the commercial future for drugs targeting common, everyday diseases is pretty limited, as the market gets more and more saturated with statins and proton pump inhibitors. Facing growing generic competition as their blockbusters fall off patent (EvaluatePharma predicts that $290 billion in sales are at risk from patent expirations between 2012 and 2018 (4)), what was once unnatractive became attractive to pharma Execs.


Today, about 200 orphan drugs per year enter development, according to the FDA. Of the 39 new drugs approved last year by the US Agency, about a third of them were assigned orphan status, including Kalydeco for cystic fibrosis, Elelyso (gaucher disease), Bosulif (CML), Gattex, (short bowel syndrome) and Signifor (Cushing’s disease) (5). This number includes new molecular entities as well as already existing products, but re-tasked to orphan diseases.

The orphan drug market was worth more than $50 billion in 2011, and its rate of growth was bigger in the last ten years than the traditional drug market. Big companies are opening rare disease units to meet the demands of the estimated 25 million people in the US alone that live with some sort of orphan disease. In Europe 6% to 8% of the population has a rare disease (6) (7) (8).

End of part 1. Part 2 will be published tomorrow!

  1. 1. Toumi M. Issues affecting access to orphan drugs in Europe. Hospital Pharmacy Europ. 2012(64).
    2. EMA. Orphan designation. [cited 2013 07-02-2013]; Available from:
    3. Comission E. Orphan medicinal products. [cited 2013 07-02-2013]; Available from:
    4. Palmer E. FDA Approvals of 2012. FiercePharma [cited 2013 07-02-2013]; Available from:
    5. Gaffney A. 30 Years Later, FDA Looks Back on Passage of Orphan Drug Act. Regulatory Focus2013 [cited 2013 07-02-2013]; Available from:
    6. Silverman E. Orphan Drugs: ‘Rare’ Opportunities To Make Money. Forbes2012 [cited 2013 07-02-2013]; Available from:
    7. Reuters T. THE ECONOMIC POWER OF ORPHAN DRUGS. Thomson Reuters, 2012.
    8. EURORDIS. Rare Diseases: understanding this Public Health Priority. EURORDIS, 2005.

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