Definition: Orphan drugs is drugs used to treat rare diseases; not normally produced because potential sales are small.

What Are Orphan Drugs?

An orphan drug is a pharmaceutical agent developed to treat, prevent, or diagnose a rare medical condition, often referred to as an orphan disease. Due to the small market potential, these conditions, affecting a relatively low number of patients, are typically unattractive for the pharmaceutical industry under normal market conditions.

Importance

The term "orphan drug" stems from the fact that these drugs are "orphaned" from the drug development pipeline, receiving less attention from pharmaceutical companies. Governments and international organizations have developed legal frameworks to incentivize the development of orphan drugs. See Orphan Drug Act of 1983 and the European Medicines Agency's policy on orphan drugs for detailed legislative information.

Criteria for Orphan Drug Status

A drug is designated as an orphan drug when it is intended for the treatment of a condition that affects fewer than 200,000 individuals in the United States, or a condition that affects more than 200,000 persons but for which the cost of developing and making available in the United States is not expected to be recovered from sales. Other countries have similar but varying criteria, which can be found under Rare Disease Act and Drug Development Regulations.

Incentives for Development

To stimulate the development of orphan drugs, governments offer a range of incentives to pharmaceutical companies. These may include tax credits, research grants, reduced fees for regulatory processes, and market exclusivity once the drug is developed. Refer to Pharmaceutical Incentives for a comprehensive list of incentives available in different regions.

Challenges in Orphan Drug Development

Despite incentives, orphan drug development faces significant challenges, including small patient populations for clinical trials, limited understanding of the pathophysiology of rare diseases, and complex manufacturing processes. Detailed challenges are discussed in the article Clinical Trials for Rare Diseases.

Examples of Orphan Drugs

Some notable orphan drugs that have made a substantial impact include:

Imatinib (Gleevec) – used for treating chronic myeloid leukemia (CML), a rare form of cancer.
Eculizumab (Soliris) – for the treatment of paroxysmal nocturnal hemoglobinuria, a rare blood disease.
Xyrem and. Xywav - for treatment of narcolepsy and idiopathic hypersomnia

For a more comprehensive list, see List of Orphan Drugs below.

Ethical Considerations

The high cost of orphan drugs poses ethical concerns regarding accessibility and affordability. The article Ethics in Pharmaceutical Pricing examines these concerns in detail.

Orphan Drug Act and Amendments

The Orphan Drug Act of 1983 in the United States was a pivotal moment in orphan drug development, providing the incentives necessary to encourage pharmaceutical companies to invest in these drugs. Subsequent amendments and international legislation have continued to evolve, which are discussed in Legislation on Orphan Drugs.

Future Directions

The future of orphan drug research is promising due to advances in genetic research and biotechnology. For more on this topic, review Biotechnology in Orphan Drug Research and Genomics and Drug Development.

Partial list of US FDA designated orphan drugs

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List of orphan drugs