For years, patients suffering from orphan diseases such as Gaucher's disease, rare cancers, hemophilia, multiple sclerosis, and Parkinson's disease simply were out of luck. Without financial incentives, pharmaceutical companies said they could not risk the time and money to develop orphan products. Other possible drug developers, such as universities or research hospitals, lacked the capital or business acumen to develop treatments for small patient groups.
Congress passed the Orphan Drug Act in 1983. This law gives tax breaks, special monopoly protections, and other incentives to companies who support research and sponsor New Drug Applications for orphan drugs, i.e., existing chemicals that are at least believed to be effective for orphan diseases, but lack a sponsor.
By the year 2000, the FDA had approved 182 orphan products — including drugs and biologicals. Sponsors had submitted 1252 applications for orphan designation, of which the FDA had granted designation to 917. Examples include:
• Crohn's disease — Remicade (infliximab), approved in August 1998, was the first approved specific (i.e., more than symptomatic) treatment for this chronic, incurable inflammatory bowel disease.
• Hansen's disease (leprosy) — The FDA cleared thalidomide to treat a serious inflammatory symptom seen in Hansen's patients. Because of its well-known potential for causing birth defects, the drug was approved with tight restrictions on its use. Thalidomide also has received orphan designation, though not approval yet, for treating primary brain tumors and Karposi's sarcoma, an AIDS-related cancer.
• Sickle cell anemia — Under the orphan program, a decades-old cancer drug, hydroxyurea (Droxia), was approved to treat adults who suffer from this inherited blood disorder that causes chronic anemia and periodic episodes of pain.
• Cutaneous T-cell lymphoma — Ontak (denileukin diftitox) treats this slow-growing form of non-Hodgkin's lymphoma when other therapies have not worked.
• Pneumocystis carinii pneumonia (PCP) — Mepron (atovaquone) treats this infection that strikes high-risk, HIV-infected patients.
"How encouraging it is that a medical tragedy [thalidomide birth defects in the 1960s] led to a medical breakthrough that will likely help people with many diseases. Nobody would have done research on this aspect of thalidomide without the Orphan Drug Act."*
* Henkel, J., Orphan Drug Law Matures into Medical Mainstay, FDA Consumer Magazine, May-June 1999.
However, there is controversy about the incentives provided by the Orphan Drug Act. According to James Love, "What is needed are more targeted incentives to conduct essential medical research, with greater public accountability."* In his view, the Orphan Drug Act has been written to qualify a very wide range of drugs as orphans, including, for example, all AIDS medicines in the United States, plus drugs for countless other severe illnesses. He feels that the Orphan Drug Act is a very blunt instrument that is often wasteful, costly to consumers and taxpayers, and sometimes counterproductive (by discouraging investments by rivals once markets become legally exclusive).
* Love, J., Brief note on the abuse of Orphan Drug programs in creating monopolies, http:// www.cptech.org/ip/health/orphan/.
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