罕見(jiàn)病用藥指用于治療、診斷和預(yù)防罕見(jiàn)病或罕見(jiàn)癥狀的藥物。近年來(lái)罕見(jiàn)病用藥的研發(fā)逐漸成為一個(gè)可獲利的研發(fā)策略，受到高度關(guān)注和重視。從目前國(guó)外罕見(jiàn)病藥品市場(chǎng)情況、參與罕見(jiàn)病藥物研發(fā)的公司和重點(diǎn)品種、美國(guó)和歐盟等發(fā)達(dá)國(guó)家和地區(qū)罕見(jiàn)病藥物的指定和批準(zhǔn)情況等方面對(duì)罕見(jiàn)病用藥的現(xiàn)狀及發(fā)展趨勢(shì)進(jìn)行簡(jiǎn)述。通過(guò)借鑒這些國(guó)家和地區(qū)罕見(jiàn)病藥物發(fā)展的成功經(jīng)驗(yàn)，為制定中國(guó)罕見(jiàn)病藥物開發(fā)的刺激措施提出建議，為相關(guān)研發(fā)人員提供參考。

The orphan drug is a pharmaceutical product aimed at curing, diagnosing, and preventing rare diseases or disorders. The research and development of orphan drugs has been a profitable development strategy gradually, and has drawn people's special attentions in recent years. This article highlights the trends in prescription sales for orphan drugs, orphan products and company performance, and the designation and approval analysis in US and Europe. The successful implementation experiences of developed countries in this field have provided suggestions for the establishment of incentive measures for facilitating orphan drugs in China.