Orphan Drugs, a New Strategy for Pharma Companies


There are so many terms in the medical field, knowing them is a key step towards a change of career or a healthcare job and will demonstrate to potential employers and clients that you command a working knowledge of the medical field and are someone they can put their confidence in.

One of those terms that gets thrown around is “orphan drug.”  Simply put an orphan drug is one that is developed to treat a specific, usually rare, disease. These rare conditions or disorders are often called “orphan diseases.”  Most rare diseases are genetic, and are present throughout the person’s entire life, even if symptoms do not immediately appear. Examples of rare diseases include cystic fibrosis, Homozygous familial hypercholesterolemia and Wilson’s disease.

Traditionally these diseases were ignored by pharmaceutical companies in favor of drugs that have larger diseases populations, like cancer.  Because the process from the discovery of a new drug to its marketing is long (10 years is the average), the expense of developing is high (several tens of millions) and the results are uncertain (among ten drugs tested, only one may have a therapeutic effect) in the past larger disease populations allowed a greater chance for the recovery of the investment in a new drug. But that is all changing. A combination of factors like patent expirations, competition from generics and government and private foundation support for the creation of orphan drugs are increasing research and development in this arena.

The development of orphan drugs was been financially incentivized through US law via the Orphan Drug Act of 1983. Its success led to the adoption of similar laws in Japan in 1993 and in the European Union in 2000. Thanks to these laws it is easier for companies to gain marketing approval for an orphan drug, and there are other financial incentives, such as extended exclusivity periods.

The National Organization for Rare Disorders (NORD), which was instrumental in establishing the Orphan Drug Act, currently estimates 30 million Americans suffer from 7,000 rare diseases.  Other statistics say as many as one-in-ten Americans suffers from rare disease. New rare diseases are discovered every week and many have no treatments available. NORD says that prior to the 1983 Act, 38 orphan drugs were approved. To date, 468 indication designations covering 373 drugs have been approved.

Prior to NORD, cystic fibrosis patients rarely lived beyond their early teens. However, drugs like Pulmozyme and Tobramycin, both developed with aid from the Orphan Drug Act, revolutionized treatment for cystic fibrosis patients by significantly improving their quality of life and extending their life expectancies. Now, cystic fibrosis patients often survive into their thirties and some into their fifties.

The same incentives that help these drug be developed also means that orphan drugs are some of the highest-priced medicines out there. EvaluatePharma‘s 2013 Orphan Drug Report reveals that orphan drugs are now showing a greater return on investment than products aimed at larger patient pools. Many drugmakers are figuring out that a small patient group can prove extraordinarily lucrative.

According to Fierce Pharma, “the worldwide orphan drug market set to reach $127 billion by 2018, accounting for nearly 16% of total prescription drug sales, it’s becoming the next arena for some of pharma’s biggest breakthroughs and fiercest patent wars.”  Orphan drugs are poised to become a big player in the pharma world.

Remember, the more you read and learn, the more you will be able to present a strong, memorable presence at an interview, or succeed within your chosen medical field.

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