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
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 adults.
- Pneumocystis carinii pneumonia,
an infection that strikes high-risk, HIV-infected patients.
- Hansen's disease (leprosy), a disease that
attacks the skin and nervous system.
- 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
For more information, please contact
the FDA's Office of Orphan Product Development at 301-827-3666
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.