Orphan drugs, often referred to as the pharmaceutical underdogs, are medications developed to treat rare diseases that affect a small number of individuals. These conditions, termed orphan diseases, typically lack attention and funding due to their limited prevalence, making them commercially unattractive for large pharmaceutical companies.
The term "orphan" in this context doesn't denote the drugs' efficacy but rather the economic challenge of developing treatments for small patient populations. To incentivize research and development in this crucial area, governments often provide incentives such as extended market exclusivity and tax credits. The significance of orphan drugs lies in their ability to bring hope to individuals facing conditions that might otherwise lack effective treatments. By addressing rare diseases, these drugs contribute to a more inclusive and compassionate healthcare landscape, emphasizing the importance of providing solutions for all, regardless of the rarity of their medical conditions.
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