Orphan drug

An Orphan Drug refers to a pharmaceutical agent developed specifically to treat, diagnose, or prevent diseases that are so rare they are often overlooked by the mainstream pharmaceutical industry. The United States Food and Drug Administration (FDA) grants Orphan Drug designation to encourage development of treatments for diseases affecting fewer than 200,000 individuals in the country.

History

The concept of orphan drugs arose in the 1980s, with the United States Congress passing the Orphan Drug Act (ODA) in 1983. This legislation was created to address the need for drugs for rare diseases, which were not being adequately developed by the pharmaceutical industry due to the limited potential for profitability.

Orphan Drug Designation

The FDA's Orphan Drug Designation program provides various incentives for drug developers, including tax credits for clinical trial costs, assistance in the drug development process, and exclusive marketing rights upon approval.

Global Impact

Following the success of the ODA in the United States, similar regulatory frameworks have been established worldwide, including in Europe under the European Medicines Agency (EMA) and in Japan through the Ministry of Health, Labour, and Welfare.

Challenges and Controversies

While the Orphan Drug Act has significantly boosted the development of treatments for rare diseases, it has also faced criticism. Concerns include high prices of orphan drugs and companies exploiting the system for financial gain.

Orphan disease

An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States.

Importance

Orphan diseases are often serious or life threatening.

US law

In 1983, the U.S. government passed a law, called the Orphan Drug Act, to give drug companies certain financial benefits for developing orphan drugs that are safe and effective.

Glossary

• Orphan disease - A disease that affects a small percentage of the population, often lacking sufficient drug development under normal market conditions due to a small potential market.
• Food and Drug Administration (FDA) - The federal agency responsible for protecting public health by ensuring the safety, efficacy, and security of drugs, biological products, and medical devices in the United States.
• European Medicines Agency (EMA) - An agency of the European Union (EU) responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU.
• Clinical trials - Research studies performed on human volunteers designed to answer specific health questions, including testing the efficacy and safety of new treatments.
• Market exclusivity - A period during which the FDA promises not to approve generic versions of an orphan drug, typically lasting 7 years in the United States, to encourage development.
• Rare disease - A condition affecting a small portion of the population, defined in the U.S. as affecting fewer than 200,000 people nationwide.
• Tax credits - Part of the incentives provided under the Orphan Drug Act, allowing companies to reduce their tax liability as a reward for conducting qualified clinical trials.
• Regulatory framework - The system of regulations and guidelines that govern the development, approval, and marketing of drugs and therapies.
• Pharmaceutical industry - The sector of the health care industry that deals with the development, production, and marketing of medications.
• Drug development - The process of bringing a new pharmaceutical drug to the market once a lead compound has been identified through the process of drug discovery.

Also see

• List of orphan drugs

Resources

Frequently asked questions

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Orphan drug Resources
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