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Orphan Drug Status


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.

Definition of orphan drug designation - NCI Dictionary of Cancer ...

NCI's Dictionary of Cancer Terms provides easy-to-understand definitions for words and phrases related to cancer and medicine.

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

Orphan drug status offers companies researching rare diseases an incentive to pursue a cure via tax incentives and marketing exclusivity.

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 - 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 ...

Search for an orphan drug - Orphanet

Orphan designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, ...

Orphan Drugs - HRSA

Section 340B(e) of the Public Health Service Act (PHSA) states the term "covered outpatient drugs" doesn't include a drug selected by the ...

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 ...

Orphan designation | European Medicines Agency (EMA)

A status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine.

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 ...

Knowledge on rare diseases and orphan drugs - Orphanet

Orphan drugs are covered by a specific individual legal system endowing them with a special status. Granting orphan drug status is based upon an ...

Benefits of FDA Orphan Drug Designation: What You Need to Know

Under the Orphan Drug Act, drug companies can apply for Orphan Drug Designation (ODD), and if granted, the drug will have a status which ...

What Is the Orphan Drug Status, and When Is It Designated? - GoodRx

Medications are expensive to make. Unfortunately, there's not much financial incentive for manufacturers to make medications for rare ...

21 CFR Part 316 -- Orphan Drugs - eCFR

(11) Orphan-drug designation means FDA's act of granting a request for designation under section 526 of the act. (12) Orphan-drug exclusive approval or ...

US FDA Orphan Drug Designation

The SAC hopes that drug companies take advantage of incentives and expedited pathways set up by regulatory agencies such as the orphan drug designation to ...

Orphan designation: Overview | European Medicines Agency (EMA)

Designated orphan medicines are eligible for conditional marketing authorisation. In some cases, designated orphan medicines may be allowed to be administered ...

Orphan Drug Status and Indication - Curonz Limited

The clinical phases are condensed with Phase I & Phase II combined into a Phase I/II trial and parts of Phase II and Phase III are combined into a Phase II/III ...

What Is "Orphan Drug Status?" - Buffalo, NY

“orphan status” to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders.

Precision Medicines' Impact on Orphan Drug Designation - PMC

This analysis suggests that the increasing number of orphan drug designations over recent years is not driven by precision medicines nor subsetting for ...

Overview of Orphan Drug/Medical Device Designation System

Applications for orphan drug/medical device designation can be submitted at any time. After the receipt of application, the designation will be determined based ...


Orphan drug

An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions.