There is no limit to the number of disincentives and obstacles that could be described for developing orphan drugs. Many have already been mentioned. Selected commonly encountered examples are described below.
1. The tax credit offered in the USA for developing orphan drugs is not much more than the tax credit for research and development of any new pharmaceutical.
2. Resources of the company could be applied to developing more profitable drugs (opportunity costs).
3. Orphan drug development may not be required if the drug is already marketed for a more common use. This implies off-label orphan use.
4. Because the safety and quality standards of manufacturing are the same, creation of a special formulation for orphan use may create too many technical problems and costs.
5. The medical need for the drug may not be great and/or the clinical effectiveness of the drug may not be strong.
6. The regulatory authority may require more data than the company thinks is warranted.
7. The liability risks may be unacceptably high. A drug that causes a serious adverse effect used to treat patients with a rare disease could increase the exposure of the company to a major court suit in return for minimal revenues.
8. There are difficulties in finding a small number of patients widely dispersed through the USA (or other countries) for conducting clinical trials or for marketing products.
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