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The Food and Drug Administration's Orphan Drug Program


Designating an Orphan Product: Drugs and Biological Products | FDA

The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition.

The Food and Drug Administration's Orphan Drug Program - NCBI

New business models for the development of products to treat rare and neglected diseases have developed within a legal and regulatory framework that has ...

Medical products for rare diseases and conditions - FDA

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and ...

Orphan Drug Approval Laws - StatPearls - NCBI Bookshelf

In 1983, the United States Congress passed the Orphan Drug Law Act (ODA), which incentivized pharmaceutical companies to develop drugs aimed specifically at ...

The Orphan Drug Act: Legal Overview and Policy Considerations

drug would be for the treatment of that disease or condition. ... FDA's administration of the Orphan Drug Designation. Program. These ...

Orphan Drugs - HRSA

Covered entities and their partners may need to analyze drugs on the OOPD list to decide which orphan drugs to exclude from the 340B Program and ...

What is an orphan drug?

An orphan drug is a drug intended for use in a rare disease. The World Health Organization defines a rare disease—sometimes referred to as an ...

Orphan Drug Status: What it Means, How it Works, Example

Orphan drug status (designated by the FDA) gives a company exclusive marketing rights for a seven-year period, along with other benefits to recoup the costs of ...

Unlocking the Benefits of FDA's Orphan Drug Designation

What Is the FDA ODD? ODD provides incentives such as tax credits, marketing exclusivity, fee waivers, and the opportunity to apply for grants to ...

US FDA Orphan Drug Designation

Orphan drug designations are typically given early on in development and orphan drugs must still follow the proper clinical development and regulatory process.

21 CFR Part 316 -- Orphan Drugs - eCFR

(10) Orphan drug means a drug intended for use in a rare disease or condition as defined in section 526 of the act. (11) Orphan-drug designation means FDA's act ...

Using four decades of FDA orphan drug designations to describe ...

The orphan drug designation is administered by the Office of Orphan Products Development (OOPD) within the FDA. Drug developers must request and ...

Orphan Drugs: FDA Could Improve Designation Review Consistency

Drugs granted this designation treat rare diseases and may receive various incentives under the Orphan Drug Act (ODA). As the number of orphan ...

Orphan drug - Wikipedia

Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and ...

Knowledge on rare diseases and orphan drugs - Orphanet

Orphan drugs are used in diseases or circumstances which occur so infrequently in the USA, that there is no reasonable expectation that the cost ...

Orphan Drug Regulations - Federal Register

The Food and Drug Administration (FDA) is issuing final regulations amending the 1992 Orphan Drug Regulations issued to implement the Orphan ...

FDA Faulted For Lapses In Orphan Drug Program - NPR

The FDA's rare-disease program began after Congress overwhelmingly passed the 1983 Orphan Drug Act to motivate pharmaceutical companies to ...

Orphan Drug Label Expansions: Analysis Of Subsequent Rare And ...

As of July 2018, the FDA no longer grants orphan designation for rare pediatric subsets of common diseases, meaning that going forward, these ...

Orphan Drugs: Understanding the FDA Approval Process

Sponsors of an orphan drug can make use of expedited Food and Drug Administration (FDA) programs such as the Fast Track, Breakthrough ...

GAO-19-83, ORPHAN DRUGS: FDA Should Ensure Designation ...

5 However, the growth in orphan designations and marketing approvals has coincided with questions about FDA's orphan drug program, including ...