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 American 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
    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 arthritis.


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