ORPHAN DISEASE Meaning and
Definition
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An orphan disease, also known as a rare disease, refers to a condition or disorder that affects a relatively small number of individuals within a population. The term "orphan" is used to describe these diseases because they are often neglected or overlooked by the medical research community, pharmaceutical companies, and healthcare systems due to their rarity and the limited potential for financial gain.
In order to be classified as an orphan disease, a condition typically affects fewer than 200,000 people in the United States, or less than 1 in 2,000 individuals in the European Union. However, there are also many diseases that are even rarer, affecting only a handful or even a single individual worldwide.
Orphan diseases can encompass a wide range of medical conditions, including genetic disorders, autoimmune diseases, infectious diseases, and certain types of cancers. Due to their rarity, diagnosis and treatment of orphan diseases can be extremely challenging. In many cases, there is limited scientific knowledge about the disease, and patients may face delays or misdiagnosis before accurate treatment can be provided.
Recognizing the unique challenges faced by individuals with orphan diseases, many countries have implemented special regulatory measures to encourage research and development of treatments or therapies. These measures often include financial incentives or grants for pharmaceutical companies and researchers, as well as expedited approval processes for drug or therapy development. The aim of these efforts is to provide hope and support for individuals affected by orphan diseases, who often face limited treatment options and significant medical and socioeconomic burdens.
Etymology of ORPHAN DISEASE
The word "orphan disease" was derived from the term "orphan drug". The term "orphan drug" was coined in the United States in 1983 when the Orphan Drug Act was passed. An orphan drug refers to a pharmaceutical product developed to treat a rare medical condition or disease, also known as an orphan disease.
The term "orphan" in this context implies that these diseases were neglected or "orphaned" by the pharmaceutical industry due to their rarity and the lack of financial incentives for developing treatments. Additionally, the word "orphan" refers to the fact that these conditions often lack a large patient population or support compared to more common diseases.
Over time, the concept of orphan drugs expanded to include the broader term "orphan disease" or "rare disease" to refer to the medical conditions themselves.
