Getting to Know “Orphan Drugs”

If someone were to ask you what the most common drugs are in the country, what would you say? Chances are, this survey would produce a narrow range of results – drugs for cholesterol, erectile dysfunction, birth control, high blood pressure, and maybe a few other common and low-cost prescriptions. And while it's wonderful that we have treatments for even our most trivial needs, there's still a huge, mostly unmet need within the pharmaceutical market.

Rare conditions receive very little attention from BigPharma, mostly because the ailments are too rare and too costly to make a drug profitable to produce. And while it sounds abhorrent to rank diseases based on profitability, pharmaceutical companies are a business just like every other industry. If they can't scrounge up enough money to support the R&D for these often poorly understood afflictions, it doesn't make sense to bother trying – especially if the number of patients who could benefit from the undoubtedly expensive medicine wouldn't pay anywhere close to what the business would need to recoup its investment.

Luckily, governments can – and do – play an important role in these matters. In the United States, the 1983 Orphan Drug Act was enacted to stimulate funding for rare diseases and encourage research in areas that would otherwise be a money pit. It also created the FDA Office of Orphan Products Development, which evaluates potential new medications and determines their efficacy in the treatment of low-incidence conditions.

To clarify, a "rare disease" is one that affects fewer than 200,000 people in the United States, or one that affects more than 200,000 people but is so expensive to treat that without external funding, pharmaceutical companies would stay far away. In the last 30 years, the act has enabled medical researchers to develop more than 400 unique drugs, although most of the nearly 7,000 conditions covered under the legislation are still lacking effective treatment options.

Although many people have never heard of the term "orphan drug," its importance for the healthcare industry is huge. Many of these rare conditions – including Creutzfeldt-Jakob Disease, Duchenne Muscular Dystrophy, Narcolepsy, and Trigeminal Neuralgia – are far more serious than an ailment like high blood pressure, in that they are either fatal or lead to substantially compromised functioning in the afflicted.

Unfortunately, many healthcare providers are unaware of both the conditions and their potential treatments. It can take many years and a highly qualified specialist to recognize a rare condition, and even then, practitioners need to know how to get their hands on these valuable orphan drugs. In some cases, it is not as simple as calling a prescription into CVS or Rite Aid.

Healthcare administrators can do their part by organizing educational seminars on rare conditions, sending doctors and nurses to conferences within their specialty, and educating themselves on the financial costs of treating the unlucky patients. Administrators who work in research should see orphan drugs as an opportunity – tax incentives, marketing rights, clinical research subsidies, and other forms of government assistance are all available to companies that are willing to partake. Lastly, healthcare administrators who work in the insurance industry arguably need to stay informed more than anyone; without knowledge of who needs these expensive medications and just how badly they need them, many insurance agents are quick to deny coverage or push for less expensive (and less effective) alternatives.

For more information, see an overview of the global report for 2015 here.

Orphan Drug Act
FDA Office of Orphan Products Development
Global Genes: Rare Diseases List
Orphan Drugs Market

About the Author:
Iris Stone is a freelance writer, editor, and business owner who has written on a range of topics. She has experience covering content on medicine, healthcare, and career training, as well as education. Iris is also interested in science and mathematics and is currently studying to be a physicist. Check out her Google+ Profile.

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