In the United States, rare diseases are those which occur in 200,000 or less people. Some rare diseases may be able to be prevented or cured by vaccination. However, these special vaccines, called orphan vaccines, are expensive to research and develop, and since they have limited use, pharmaceutical (drug) companies don't make money from them. Drug companies are therefore reluctant to engage in orphan vaccine research.

To encourage companies to take on the humanitarian work of developing vaccines, the Orphan Drug Act of 1983 was passed. Under the Act's provisions, drug companies receive a 50% deduction tax credit for clinical trial expenses of orphan drug and vaccine testing, assistance in speeding up the FDA approval process, and exclusive marketing rights for an approved drug for 7 years.

Few vaccines developed
As of 2000, only 8 orphan vaccines were registered with the FDA, seven of which are for treating or curing (such as for cancer or sickle cell anemia); one vaccine is for preventing an infectious disease called Japanese encephalitis.

What you can do
So, what does all this mean for you, a person living with, or at risk for, a rare disease, or a concerned caregiver, friend, or relative? It means that for now, vaccines to prevent rare diseases are a long way off, but there are ways you can help with the process.

One problem drug researchers have with evaluating orphan vaccines is finding enough people who have the rare disease they're studying. You can join (or start) a network of people with the disease you're concerned about (see below for helpful links). You can ask your doctor about clinical trials you could take part in, and check out online listings of clinical trials for current information.

Another way you can help is by spreading the work about orphan drugs and vaccines that are on the market, so that the drugs can get to the people who would benefit from them. The National Organization for Rare Disorders Orphan Drug Database has information about orphan drugs for you and your doctor, and information you can share with people in your contact network.

Finally, advocacy for more orphan drug and vaccine development is needed. It is well known that patient groups' efforts are responsible for the increasing number of orphan drugs authorized for testing. In 1997, 152 orphan drugs were authorized--almost five times the number (34) authorized in the previous 14 years. If you want to get involved in a campaign, check out your national disease association or network. As Margaret Mead said, "Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it's the only thing that ever has."

Information for this article was taken from:
"Development of Orphan Vaccines: An Industry Perspective," by Jean Lang and Susan C. Wood, in Emerging Infectious Diseases, vol. 5, no. 6, 1999.

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