The global orphan drugs market is estimated to have increased at a compound annual growth rare (CAGR) of 9.6% between 2005 and 2011, hitting a market value of $8.63 billion by the end of 2011.
It is forecast, however, that the orphan drugs market will witness near flat sales growth between 2011 and 2017.
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.
In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive.
The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum.
For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question.
Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable, government intervention is often required to motivate a manufacturer to address the need for an orphan drug. Critics of free market enterprise often cite this as a failure of free market economic systems.
Tumbling over the patent cliff, many Big Pharma companies are being forced to find new strategies for sustaining sales growth, with orphan drugs seen as a great opportunity for this.
An estimated 25 million people in the US alone collectively live with some sort of orphan disease. Despite the understanding that specific diseases may afflict relatively small numbers of individuals, this overall growing pool of patients is now becoming an attractive opportunity for the pharmaceutical industry. This reflects the fact that appropriate treatments for unmet medical needs that generate high levels of reimbursement.
The prize for the top selling drug went to Rituximab, an oncology treatment sold by Roche that is also known as Rituxan, which has present day peak sales value of $7 billion and a discounted peak value of $154 billion. In fact, four of the top 10 drugs are oncology treatments, although the No. 2 spot went to the Lucentis eye treatment, which is another Roche drug.
Meanwhile, of the 86 orphan drugs included in a recent study, 25 were blockbusters, which meant that 29% generated annual sales greater than $1 billion.
For more information on the global orphan drugs market, see the latest research: Global Orphan Drugs Market
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