What is required for orphan drug designation?
Criteria for Orphan Drug Designation Adequate documentation or prevalence data must demonstrate that the intended condition is rare. There must be a scientific rationale establishing a medically plausible basis for the use of the product for the rare condition.
What does FDA orphan drug designation mean?
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.
What is orphan drug exclusivity?
Orphan drug exclusivity (ODE; 21 CFR 316.31) is used as an incentive to promote the development of products intended to diagnose or treat rare diseases or conditions. As defined by the Orphan Drug Act, rare diseases are those that affect fewer than 200,000 individuals in the US.
What is orphan exclusivity?
• The exclusivity granted to orphan drugs provides seven years without generic competition for the approved orphan designation but does not prevent generic competition for other approved uses of the medicine. • Orphan exclusivity continues longer than patent protection in only 60 of the 503 orphan-designated medicines.
When do you apply for orphan drug designation?
In fact, the orphan application can be filed anytime in the drug development process before NDA/BLA submission, even prior to IND filing. Not only can there be multiple orphan designations per indication; more than one sponsor can receive an orphan designation for the same drug/indication.
How do I apply for orphan drug designation?
Requests for an orphan drug designation can be submitted through the FDA Form 4035. The FDA will complete a review of the orphan drug designation request within 90 days of its receipt. The components required for submission of Form 4035 include: Information about the sponsor and drug product.
What are some examples of orphan drugs?
Rosuvastatin (brand name Crestor) is an example of a drug that received Orphan Drug funding but was later marketed to a large consumer base.
What are the benefits of orphan drug designation?
Companies that receive orphan drug designation are entitled to several advantages, including the possibility of free of charge advice from the FDA and certain financial benefits, such as R&D tax credits (which is not part of the company’s current plans) and exemptions or reductions in regulatory submission fees.
What is an orphan drug application?
The definition of orphan product was extended to products other than drugs and in particular : biologics, medical devices and medical foods, mainly parenteral nutrition and nutraceuticals. The product must be part of the basis of a Marketing Authorisation submitted before application for orphan drugs status.
What are orphan drugs?
Orphan drug. An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance.