Development of therapies for rare diseases is a priority


The development of new drugs for orphan diseases may not be commercially as appealing as for common diseases, such as breast cancer. Therefore, governmental agencies and institutions provide several incentives for orphan drugs to facilitate their development. For example, Australia provides 5 years, FDA 7 years, and the EMA and Japan 10 years of market exclusivity from the date of approval for a new orphan drug product. Japan, the FDA, and the EMA (varies by country) provide a fee waiver for companies plus taxation incentives. In addition, the agencies provide protocol assistance, and may provide a fast track status for approval. Orphan drugs may be approved based on one pivotal phase 3 trial by the FDA and the EMA, whereas for other drugs at least two phase 3 trials may be needed for approval. A smaller sample size is often accepted in clinical trials.