RARE Toolkits

Below is a list of all of our current RARE Toolkits.

Differences in Drug Development


Rare disease patients and advocates need safe and effective new treatments and drugs fast! There are 7,000 rare diseases and yet there are only around 400 drugs approved by the FDA to treat them.  To add to the urgency, the National Institutes of Health estimates that 50 percent of people affected by rare diseases are children, making rare diseases one of the most deadly and debilitating for children worldwide.

For many years, rare diseases were not attractive to biotech and pharmaceutical companies as the diseases are extremely difficult to treat and the numbers of patients affected is extremely small. Rare disease does not fit the blockbuster drug development model.

The Orphan Drug Act of 1983

Recognizing the scarcity of medicines to treat rare diseases and the unique challenges with developing drugs and therapies for very small patient populations, the Orphan Drug Act of 1983 provided incentives to companies developing rare disease treatments. Since that time, about 400 medicines representing more than 447 separate indications have been approved to treat rare diseases, compared to fewer than 10 in the 1970’s.


Incentives provided by the government to rare disease drug companies today include:

•Seven years of exclusive marketing, which means that no other drug company can receive approval of the same drug for the same disease, unless the new drug can demonstrate greater safety, greater efficacy, or a major contribution to patient care;

•A 50 percent tax credit for qualifying research expenses;

•Grants supporting the development of any drug used to treat a rare disease;

•Companies developing new drugs for rare diseases do not have to pay the same fees as companies developing drugs for common diseases; and

•Special assistance from the FDA regarding studies to support a rare disease. The FDA provides extensive and timely guidance to drug companies about the data that are needed for approval of a drug to treat a rare disease.

FDA has also put in place special processes that can speed up the approval of treatments for rare diseases: