What is an orphan drug?

let us start our discussion with the word “orphan “, it means a child without any parents or support, like that for some diseases Also there will be no support for research because its prevalence is much less due to which companies won’t come forward to make drugs that cure these rare diseases, the main reason is the cost associated with research is very high and nobody comes forward to sponsor the research, and one of the primary reason is rare diseases affect a tiny number of population.

Why companies won’t make drugs for rare diseases

1. These affect a tiny group of population
2. less market
3. companies cannot recover the cost associated with research
4. fewer sponsors to support the research

To solve the above issue, the FDA amended incentives established in the ” Orphan Drug Act ” to make companies step forward to make promising drugs for rare diseases.

These incentives include

1. Regulatory assistance from the FDA.

2. Tax credit which covers 50% of clinical drug testing expenses.

3. Exemption from user fees.

4. Potential seven years of market exclusivity after approval.

Then how a drug is considered an orphan – To get orphan drug designation sponsor should submit a request to the FDA. and the request can be made in one of the three ways listed here

1. Through the CDER NEXTGEN PORTAL
2. By emailing the required information to orphan@fda.hhs.gov
3. By mailing the required information to:
   Office of Orphan Products Development

To grant an orphan drug designation request the disease or condition in question must have occurred or is present in not more than 20,000 persons in the United States. also, the sponsors should show information regarding the lack of profitability for making the drug.