Scientific Publications by FDA Staff

Drug Info J 1994 Apr;28(2):489-94

Unique Aspects of Orphan Drug Safety

Haffner ME

In December 1992, the Orphan Drug Regulations: Final Rule were published. These regulations describe the role orphan products play in developing safer drugs, and discovering safer uses for products in special populations. In many instances, the Orphan Drug Act is responsible for the discovery of better ways of producing already developed products for safe treatment of special patient populations. 1. In general, orphan drugs are as safe as nonorphan drugs. No changes in safety parameters are considered based on the orphan status of a product. 2. Many orphan drugs are used in serious and/or life-threatening situations where, because of the seriousness of the disease or the lack of any other therapy, toxicity testing may be waived for a period of time. 3. Drugs previously approved are being studied to determine their safe use for orphan indications specific to women, pregnant women, and children. 4. Orphan drug studies are aiding in the development of recombinant forms of biologics for treating rare disorders. For example, Recombinant Factors VIII and IX, which greatly diminish the risk of hepatitis from this biologic product, are used to treat patients with hemophilia. 5. Orphan drug grant clinical studies are being conducted using potentially harmful drugs (such as known teratogens) to treat illnesses in small populations of patients that are no longer fertile. 6. Pediatric studies are taking place using products for which no data exists for treatment of diseases in children, for example, using digitalis in pediatric patient populations