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An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive.〔〔 The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.〔 According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investing in orphan drug Research and Development partly due to the U. S. Orphan Drug Act of 1983 (ODA) and similar Acts in other regions of the world and also driven by "high-profile philanthropic funding." The period between 2001 to 2011 was the "most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals."〔 For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an "impressive 25.8 percent, compared to only 20.1 percent for a matched control group of non-orphan drugs."〔 By 2012 the market for orphan drugs was worth USD$637 million compared to the USD$638 million matched control group of non-orphan drugs, Thomson Reuters.〔 By 2012, ==Orphan drug legislation== Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable for pharmaceutical companies, government intervention is often required to motivate a manufacturer to address the need for an orphan drug.〔 In 2015 Gibson et al published the results of their qualitative study of "34 key Canadian stakeholders which including drug regulators, funders, scientists, policy experts, pharmaceutical industry representatives, and patient advocates in which they investigated contributing factors to the growing interest of the pharmaceutical industry in "niche markets" such as orphan drugs. The intervention by government on behalf of orphan drug development can take a variety of forms: * Tax incentives. * Enhanced patent protection and marketing rights. * Clinical research financial subsidization. * Creating a government-run enterprise to engage in research and development (see Crown corporation). Currently there are more than 400 orphan designated drugs in clinical trial process. Majority of these drugs are being developed in the US followed by Europe. The US dominate the development of orphan drugs with more than 300 orphan designated drugs being under clinical trial process.〔 * Orphan Designated Drugs in Clinical Phase: 600〔 * Marketed Orphan Designated Drugs: 281〔 * Highest Number of Drugs in Phase-2 Trial: 231〔 * US Dominates Clinical Trial Process: 350 in Pipeline (Research till Registration)〔 * Indication for Clinical Trials for Orphan Drug: More than 30% for Cancer Treatment * Share of Biological in Orphan Drug: More than 60%〔 * Key Market: US (Sales > US$ 40 billion) 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「orphan drug」の詳細全文を読む スポンサード リンク
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