日本における新薬の臨床開発と承認審査の実績 2000～2010年承認品目
福島 達也（医薬産業政策研究所 主任研究員）
小野 俊介（東京大学大学院薬学系研究科 医薬品評価科学講座 准教授）
（No.51：2011年11月発行）
2010年は承認品目が100品目を超え、例年以上に多くの新医薬品が承認されたが、臨床開発期間の中央値は35.9ヵ月（3.0年）、審査期間の中央値は14.8ヵ月（1.2年）であり、2009年に比べ、それぞれ12.3ヵ月、4.3ヵ月の短縮がみられるとともに、2000～2010年の間で最も短い期間であった。
近年、治験の国際化とともに、国際共同治験のデータを評価資料として提出した品目が増加しつつあり、臨床開発の一層の効率化が期待される。また、2005年以降、審査期間は短縮傾向にあり、公知申請品目の増加等、未承認薬・適応外薬検討会議のスキームも相まって、引き続き審査期間の短縮が予想される。しかし、今後、通常の審査プロセスを踏んだ申請品目のみであっても、継続的に審査期間の目標値を達成できるよう、審査体制や審査プロセスの更なる整備が必要であり、アンケート調査の回答企業からも改善に向けた多くの意見、要望が寄せられた。
加えて、製薬企業とともに早期に新医薬品の安全対策を講ずべく、審査部門と安全性部門の連携、今後、重要性が増すと考えられるコンパニオン診断薬を伴う新医薬品審査における医療機器審査部門との連携、国際共同治験や海外との同時申請も増加する中、FDAやEMAを中心とした国際連携、情報共有の強化等に関する意見、要望も多くみられ、今後、これらを踏まえた対応を検討していく必要があると考えられる。
Over one hundred of new drugs were approved in 2010, which was more than usual. The median clinical development time and review time in 2010 were 35.9 months (3.0 years) and 14.8 months (1.2 years) respectively which were the shortest between 2000 and 2010, and the former was shortened by 12.3 months and the latter by 4.3 months, compared with 2009.
In recent years, the number of NDAs utilizing data of multinational clinical trials is increasing according to globalization of clinical trials, and it would be expected to further streamline process for clinical development. Since 2005, review times have been becoming shorter, and would shorten continuously along with scheme of "Non-Approved and Off-Label Drugs Review Meeting", because of increasing the number of NDAs based on common knowledge and facts on off-label use or much use experiences abroad in some diseases without conducting clinical trials overall or partially in Japan, that is "Kouchi Application". But further improving the review processes and systems should be considered to meet annual target of review time continuously in the future even without "Kouchi Application", and there are many comments and requests for the improvement from respondents of the survey, as described in the tenth chapter.
In addition, there are opinions on review system from the respondents such as further cooperation between the Office of New Drugs/Biologics and the Office of Safety to tighten safety measures for new drugs earlier with applicants, as well as between the Office of New Drugs/Biologics and the Office of Medical Devices when new drugs accompanied with companion diagnostics, which would become more important, are reviewed, international cooperation with other regulatory agencies such as the FDA and the EMA as the numbers of multinational clinical trials and simultaneous NDA submissions have been increasing, and so on. In the future, discussions on a regulatory approval system considering the quality of the regulatory review based on these comments and requests are also essential.