FDA Fights Rare Diseases: New Help for Patients Without Treatments
Years ago, people diagnosed with a rare disease might have found themselves
with little hope for a medical treatment. This changed in 1983 when Congress
passed the Orphan Drug Act, a law that offered drug companies special
incentives to develop products for treating diseases with fewer than 200,000
patients a year. The inducements include seven-year marketing exclusivity, tax
credit for the product-associated clinical research, research design assistance
by the FDA, and grants of up to $300,000 per year.
Thanks to the drug sponsors' gratifying response to this program, which can
be applied to up to 6,000 rare diseases, the FDA has so far approved more than
200 so-called "orphan" drugs.
Here are some of the rare diseases that now can be treated with "orphan"
medications the FDA approved in recent years:
- Sickle cell anemia, an inherited blood disorder that causes chronic
anemia and pain.
- Cystic fibrosis, an inherited disorder affecting children and young
- Pneumocystis carinii pneumonia, an infection that
strikes high-risk, HIV-infected patients.
- Hansen's disease (leprosy), a disease that attacks the skin and
- T-cell lymphoma, a type of blood cancer.
Other rare diseases that now have an orphan treatment include ovarian cancer,
Lou Gehrig's disease, neonatal respiratory distress, hemophilia, organ transplant
rejection, Huntington's disease, and juvenile rheumatoid arthritis.
For more information, please contact the FDA's Office of Orphan Product
Development at 301-827-3666 or 1-800-300-7469.
Humanitarian Use Devices
The success of the orphan drug program encouraged congressional authorization
in 1997 for the FDA's humanitarian use device program, which offers incentives
for the development of medical devices for patient populations too small to
justify full-scale product evaluation. Devices approved under this mandate require
only evidence that their probable health benefit is greater than the risk of
use, a standard which is less costly to achieve than the level of safety and
effectiveness required for regular devices. The FDA approvals under this program
include a stent to treat urinary tract obstruction in unborn babies and a cardiac
patch for repair of holes in the heart-products that would not be available
without this program.