Benefits of Orphan Drugs from a Development Perspective

The most obvious benefit for a company is that the number of clinical trials and the quantity of clinical data required for marketing approval will usually be less for an orphan drug, primarily based on the limited number of patients available for clinical trials. However, even though the numbers are fewer, the data must be convincing and the standards of trial design are often unchanged. Such standards may be modified for extremely rare diseases, where a company may be limited to obtaining a number of individual case studies. A further possible benefit in some drug development programs is that less toxicology data may be required later on, when there is already substantial human experience with the drug.

Standards of manufacturing and quality control for orphan drugs are generally identical to those of non-orphan drugs. Sometimes, fewer validation batches may be required, and stability tests may occasionally be allowed to continue while the drug is being evaluated by the regulatory authority or, in exceptional cases, even after the product is on the market. Thus, the time to develop the chemistry and technical package of data for the regulatory submission may or may not differ from that needed if the drug was for a common disease.

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