Skip to main content

Orphan Drug Designation

BayBiotech.NET

Orphan Drug is a special status designated to a drug or biologics to treat a rare disease or condition. In order to obtain this designation, drug or biologics must meet certain criteria.

Orphan designation qualifies the sponsor for the tax credit and marketing incentives as per Orphan Drug Act. In addition, orphan drug is not subject to a prescription drug user fee unless used for other indications.

For application purposes, sponsor shall submit two copies of a completed, dated, and signed request for designation that contains the following:

(1) A statement that the sponsor requests orphan drug designation for a rare disease or condition.

(2) The name and address of the sponsor; the name of the sponsor's primary contact person.

(3) A description of the rare disease or condition for which the drug will be investigated.

(4) A scientific rationale for the use of the drug for the rare disease or condition.

(5) Where the sponsor of an already approved orphan drug seeks orphan drug designation for the subsequent drug for the same rare disease or condition, an explanation of why the proposed variation may be clinically superior to the first drug.

(6) If a drug is under development for only a subset of persons with a particular disease or condition, a justification that the subset is medically plausible.

(7) A summary of the regulatory status and marketing history of the drug in the United States and in foreign countries.

Application must have supportive documents to demonstrate that the disease or condition for which the drug is intended affects fewer than 200,000 people.
More information about Orphan Drug Status and tips for application can be found at:
Designating an Orphan Product: Drugs and Biologics, http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm

Comments

Post a Comment

Popular posts from this blog

Group C (Treatment IND) Drugs

BayBiotech.NET Since 1976, National Cancer Institute (NCI) in agreement with FDA has established the Group C classification system to allow access to certain drugs for the cancer patients specifically falling under a category that adequate alternative therapy or if the available alternative therapy has significant toxic effects. Each Group C drug protocol specifies patient eligibility and drug use information. Group C drugs are provided only to properly trained physicians who have registered themselves with NCI using a special form to assure that their patient qualifies under guidelines - or protocols - for the drug. Physicians using drugs under Group C have no reporting requirements to the NCI other than the obligation to report adverse drug reactions. Group C drugs are provided free of charge, and the Centers for Medicare and Medicaid Services provides coverage for care associated with Group C therapy. Making Group C drugs available to the critically ill patients not only provi...
Post a Comment
Read more

Good Machine Learning Practices

BayBiotech.NET A joint effort by FDA,  United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada have developed guiding principles to help promote utilization of medical devices that are safe and effective and utilize artiificial intelligence and machine learning. To find out more details check out the link here!
Post a Comment
Read more

Harmonization by Doing (HBD): Japan & U.S. Collaboration

BayBiotech.NET HBD is an international cooperative effort by Japan and US for regulatory convergence for Medical Devices. The efforts are focused on to develop global clinical trials and address regulatory barriers for timely device approvals. To address the needs for additional evaluation, the HBD initiative is a pilot project launched jointly by FDA and MHLW-PMDA for the premarket review of device cardiovascular technology. Instead of taking a theoretical approach to harmonization, HBD is focused on Proof of concept by utilizing parallel development, application submissions and review of actual medical device projects. HBD Study intends to collect and analyze regulatory submission data from multiple applications in the U.S. and Japan. The purpose of the study is to further understand differences that may exist with format and content, to define best practices and to improve globally harmonized processes. To read more about the HBD program, follow the link: http://www.fda.gov/M...
Post a Comment
Read more