Orphan Drugs: Development Trends and Strategies

 In Articles

The challenges they present for Pharmacy and Pharmaceutical companies: how to achieve the right balance between the need to raise capital for R&D, the profit drive, and ensuring free access to health care for all?

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The spiraling costs of drug development that must be carried out according to stringent regulations, coupled with the need for a “reasonable” return for any pharmaceutical company’s investment, often tends to discourage pharmaceutical innovators from developing products for extremely small patient populations.

Rare diseases in these small patient populations are thus “orphaned” by the pharmaceutical industry; having but a few approved drug treatment options available, they are referred to as “orphan diseases”…

An orphan drug is one that has been developed specifically to treat a rare medical condition – an “orphan disease” – diseases so rare that a physician may observe less than one case a year.

Orphan diseases are so classified if they manifest in patient populations, representing, at maximum, 6–8% of the world’s population. Less than 1 in 2000 is born with an orphan disease. 80% can be traced to genetic origins. The rest are the result of infections (bacterial or viral) and allergies, or are due to degenerative and proliferation causes. Symptoms of some rare diseases may appear at birth or in childhood; however, more than 50% of rare diseases appear during adulthood. Today there are over 5000 rare diseases listed in the world, with five new rare diseases a week described in the medical literature.

Often, orphan drugs are developed by the pharmaceutical industry to respond, for a variety of reasons, to a public health need, i.e., the treatment of orphan diseases. Orphan drugs can also include drugs developed originally for other diseases, but which are found to also be of use in treating orphan diseases.

Because of the obvious disincentives for Pharmaceutical Companies when it comes to developing orphan drugs, academic physician/scientists have tried to fill the vacuum. But there are many obstacles along this path: lack of career opportunities, lack of funding, and the need for multiple areas of expertise.

There are, however, positive developments in the area of the development of orphan drugs as well, including the formation of Orphan Drug and Rare Disease institutes, grant programs and the passage of orphan drug legislation by various countries. At the same time, it must be pointed out that the 300 orphan drugs and devices approved in the last 25 years are a drop in the bucket compared with the many thousands of orphan diseases that manifested in that same period.

The assignment of orphan status to a disease is vital in terms of the potential for a treatment to be developed. This assignment is a matter of public policy in many countries. This has happily resulted in medical breakthroughs that may not have otherwise been achieved given the economics of drug research and development for private pharmaceutical companies. In the case of orphan drugs, it seems clear that the sales of such a product would never exceed the investment in the development of the product. The market is by definition too small to pay its way. This is a significant challenge to not only improving but also even maintaining our response to orphan diseases.

At the same time, there is also room for optimism, as there is potential for growth opportunities in the global orphan drugs market. The global market is expected to grow at a compound annual growth rate (CAGR) of nearly 6%. The U.S. market is expected to grow at 8%. Biological drugs account for a major share (64.3%) of the orphan drug market. The size of the biological orphan drug market is projected to grow at a 6.9%. Orphan drugs for the cancer sector generate the largest amount of revenue.


Let us hope that both private pharmaceutical companies and public actors continue to act independently, and, ideally, together, to develop orphan drugs for those people who suffer from orphan diseases now, and in the future. 

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