CDISCまでの長い道のり [2]

カテゴリー: CDISCラボ


CDER Small Business Chroniclesというサイトがあります。この10月付けのリリースに、PDUFA Vの概要がとてもよくまとめられています。CDISCに関係しそうな点を列挙しましょう。まずは最も基本的な概念から。

 ・PDUFA Vにより、FDAは申請料を徴収できるようになった
 ・その結果、Timely Mannerで申請処理ができる

これは各所で繰り返し述べられているJustification(正当な理由の主張)です。FDAへお金を払うことできちんとしたメリットがあること、お金を徴収するFDAに相応の義務や目標があることが分かります。PDUFA Vはお金に絡んだ決め事です。そこに正当な理由があるのは必須です。

 Priority Review:90%の申請を6ヶ月以内に処理


ここまではPDUFA Vの概要です。上記の目標を達成するには、それなりの実現方法が必要です。その一つがElectronic Submission。そして、その時に一定のフォーマットに従ったデータ提出が必要になります。ここでCDISCのような標準が力を発揮するコトになります。

このElectronic Submissionについて詳細を眺めてみましょう。まず、FDASIAという法律の解説書 A Reference Guide to the Food and Drug Administration Safety and Innovation Actで1136節の説明を見ると、次のようなことが分かります。

FDASIA prohibits FDA from requiring only electronic drug and biologic submissions for at least 2 years after the issuance of a final guidance, except for expanded access drugs and biologics, under this section.

 ・FDAはFinal Guidanceを作成する
 ・Final Guidance完成後、2年間は電子申請を強制できない

Final Guidanceって何?という疑問が出ます。この疑問を解決するのがPDUFA V Performance Goals Letterの12章です。


A. To enhance the quality and efficiency of FDA’s review of NDAs, BLAs, and INDs, FDA shall consult with stakeholders, including pharmaceutical manufacturers and other research sponsors, to issue draft guidance on the standards and format of electronicsubmission of applications by December 31, 2012.

B. FDA will issue final guidance no later than 12 months from the close of the public comment period on the draft guidance. Such final guidance and any subsequent revisions to the final guidance shall be binding on sponsors, applicants, and manufacturers no earlier than twenty-four months after issuance of the final guidance.

C. Requirements for electronic submission shall be phased in according to the followingschedule:

1. Twenty-four (24) months after publication of the final guidance: All new original NDA and BLA submissions, all new NDA and BLA efficacy supplements and amendments, all new NDA and BLA labeling supplements and amendments, all new manufacturing supplements and amendments, and all other new NDA submissions.

2. Thirty-six (36) months after publication of the final guidance: All original commercial INDs and amendments, except for submissions described in section 561 of the Federal Food, Drug, and Cosmetic Act.

D. Because of the significant investments required to change regulatory submission and review software, initial FDA guidance shall specify the format of electronic submission of applications using eCTD version 3.2.2 unless, after notice and an opportunity for stakeholder comment, FDA determines that another version will provide for more efficient and effective applicant submission or FDA review. In general, when FDA revises final guidance requiring submission using a new version of electronic standards or formats, FDA shall also accept submissions using the previous version for no less than twenty-four (24) months.

E. Clinical Terminology Standards: Using a public process that allows for stakeholder input, FDA shall develop standardized clinical data terminology through open standards development organizations (i.e., the Clinical Data Interchange Standards Consortium (CDISC)) with the goal of completing clinical data terminology and detailed implementation guides by FY 2017.

1. FDA shall develop a project plan for distinct therapeutic indications, prioritizing clinical terminology standards development within and across review divisions. FDA shall publish a proposed project plan for stakeholder review and comment by June 30, 2013. FDA shall update and publish its project plan annually.

F. Development of terminology standards for data other than clinical data: To address FDA-identified nonclinical data standards needs, FDA will request public input on the use of relevant already-existing data standards and the involvement of existing standards development organizations to develop new standards or refine existing standards. FDA will obtain this input via publication of a Federal Register notice that specifies a 60-day comment period.

G. FDA shall periodically publish final guidance specifying the completed data standards, formats, and terminologies that sponsors must use to submit data in applications. In the case of standards for study data, new data standards and terminology shall be applicable prospectively and only required for studies that begin 12 months after issuance of FDA's final guidance on the applicable data standards and terminology.

重要と思われる部分を太字にしてあります。まず、Final Guidanceの作成の前にDraft Guidanceが作成されることが分かります(2012年12月31日までに)。その後、Public Commentを受け付けてから1年以内にガイダンスが完成します。ガイダンスが完成してから24ヶ月後から、NDA・BLAへの適用が始まります。36ヵ月後にはINDへの適用が開始されます。また、Guidanceの内容は段階的に改定されることが分かります。

用語集作成、Theraputic Areaごとの標準作成も見え隠れしています。これは既に始まっている領域別CDISC標準の策定を指していると理解して問題ないと考えられます。


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