The term "orphan drug" refers to a medication developed to treat a rare disease that affects fewer than 200,000 patients in the US. The word "orphan" in this context comes from the fact that these diseases are often neglected by drug companies due to their small patient populations. The spelling of "orphan" is pronounced ˈɔːfən in IPA phonetic transcription, with the stressed syllable being the first one. The word "drug" is pronounced drʌɡ, with the stressed syllable being the second one. Together, they form the compound word "orphan drug."
An orphan drug refers to a medication or therapeutic product developed specifically for the treatment of rare diseases or medical conditions, which affect a relatively small number of people in a given population. These conditions are commonly referred to as "orphan diseases" or "rare diseases." The term "orphan" signifies that the drug is intended for a small target population that otherwise would not be profitable enough for pharmaceutical companies to develop drugs for.
To be classified as an orphan drug, a medication or therapy must meet specific criteria set by regulatory authorities, such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). These criteria typically include a disease prevalence threshold (e.g., affecting fewer than a certain number of individuals in a specific population) and the absence of existing adequate treatment options.
The development and approval process for orphan drugs may differ from standard drug development procedures. Governments often provide incentives to encourage pharmaceutical companies to invest in orphan drug research and development, such as tax credits, grants, extended exclusivity periods, and expedited regulatory review. This framework encourages the advancement of innovative therapies and ensures patients with rare diseases have access to potentially life-saving treatments. Orphan drugs have significantly improved the outlook for individuals affected by rare diseases, addressing unmet medical needs and providing hope for those who previously had limited or no treatment options.
The word "Orphan Drug" originated from the term "orphan diseases" or "rare diseases", which are medical conditions that affect only a small number of people.
The term was first coined by Dr. Harry Meyer, a former chief of the Endocrine Section at the National Institutes of Health (NIH) in the United States. In the late 1970s, Dr. Meyer realized that pharmaceutical companies were unlikely to invest in developing treatments for rare diseases due to the limited market potential. As a result, patients suffering from these neglected diseases had no access to life-saving or life-altering drugs.
In response to this issue, Dr. Meyer proposed a solution during a speech at a conference in 1983, where he referred to these conditions as "orphan diseases". He likened the patients with rare diseases to orphans, as they were often neglected and lacked necessary medical attention.
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