[Federal Register: December 30, 2004 (Volume 69, Number 250)]
[Page 78468-78469]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]



Food and Drug Administration

[Docket No. 2004N-0545]

Nonclinical and Clinical Datasets; Notice of Pilot Project

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.


SUMMARY: The Food and Drug Administration (FDA), Center for Biologics 
Evaluation and Research (CBER), is seeking volunteers to participate in 
a pilot project involving the evaluation of various analysis tools to 
facilitate the use of electronic datasets for analysis of animal and 
human data submitted to FDA by applicants of biologics license 
applications (BLAs). These analysis tools will allow a reviewer to more 
efficiently capture and evaluate nonclinical and clinical datasets 
submitted in electronic format.

DATES: Submit written requests to participate in the pilot project by 
February 28, 2005. Comments on this pilot project may be submitted at 
any time.

ADDRESSES: Submit written requests to participate and comments 
regarding the pilot project to the Division of Dockets Management (HFA-
305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, 
Rockville, MD 20852. Submit electronic comments to http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments

FOR FURTHER INFORMATION CONTACT: Richard Diamond, Center for Biologics 
Evaluation and Research (HFM-6), Food and Drug Administration, 1401 
Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-0372.


I. Background

    Under current FDA regulations (21 CFR 601.2), applicants must 
provide nonclinical and clinical data in BLAs. CBER provided 
recommendations for the electronic submission of BLAs, as well as new 
drug applications (NDAs), in the ``Guidance for Industry: Providing 
Regulatory Submissions to the Center for Biologics Evaluation and 
Research (CBER) in Electronic Format--Biologics Marketing 
Applications'' dated November 1999. A joint CBER and Center for Drug 
Evaluation and Research (CDER) document entitled ``Guidance for 
Industry: Providing Regulatory Submissions in Electronic Format--
General Considerations'' dated January 1999 provided recommendations 
for the file formats for nonclinical and clinical datasets.
    FDA announced on July 21, 2004, a standard format, called the Study 
Data Tabulation Model (SDTM) developed by the Clinical Data Interchange 
Standards Consortium (CDISC), that sponsors of human drug clinical 
trials can use to submit data to the agency. It is expected that this 
step will lead to greater efficiencies in clinical research and FDA 
reviews of NDAs and BLAs. CDISC is an open, multidisciplinary, 
nonprofit organization including members from pharmaceutical companies, 
biotechnology companies, contract research organizations, and software 
vendors. CDISC committed to the development of industry standards to 
support the electronic acquisition, exchange, submission and archiving 
of clinical trials data and metadata for medical and biopharmaceutical 
product development. CDISC is currently facilitating the work on 
similar standards for nonclinical and clinical datasets. Where 
possible, the standards developed for clinical datasets and metadata 
should be used in the development of standardized

[[Page 78469]]

presentations of the datasets for routine toxicology studies (e.g., 
chronic toxicology and carcinogenicity studies). Version 3.1 (V3.1) of 
the CDISC Submission Data Domain Standards was officially released on 
June 25, 2004. These standards and the accompanying Implementation 
Guide can be viewed on the CDISC Web site at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.cdisc.org.\1\


    \1\FDA has verified the Web site address, but is not responsible 
for subsequent changes to the Web site after this document publishes 
in the Federal Register.

    FDA has performed some initial pilot testing of nonclinical and 
clinical data applicable to drugs. The purpose of this pilot project is 
to evaluate the Version 3.1 of the CDISC SDTM and the Implementation 
Guide to determine applicability to clinical and nonclinical data 
required for submission of CBER regulated BLAs, to help in the 
refinement of analysis tools designed to facilitate the review and 
evaluation of electronic nonclinical and clinical datasets, and to 
obtain feedback from reviewers and pharmaceutical companies on the 
creation and use of standardized nonclinical and clinical data and 
metadata in a format that is applicable to BLA submissions.

II. Pilot Project Description

    This pilot project is part of an effort to improve the process for 
submitting nonclinical and clinical data. Eventually, FDA expects to 
recommend detailed data standards for the submission of nonclinical and 
clinical data. Participants in this pilot project will have the 
opportunity not only to assist the agency in testing the use of various 
analysis tools and standardized nonclinical and clinical data and 
metadata, but would also be able to familiarize themselves with the 
process at an early stage of development. Only a few participants are 
needed for this pilot.

A. Initial Approach

    Because a limited group of voluntary participants are needed, the 
agency will use its discretion in choosing volunteers, based on their 
having previously submitted nonclinical and clinical datasets to FDA 
and having demonstrated familiarity with our recommendations for 
creating nonclinical and clinical datasets as presented in the 
``Guidance for Industry: Providing Regulatory Submissions to the Center 
for Biologics Evaluation and Research (CBER) in Electronic Format--
Biologics Marketing Applications.'' During the pilot project, specific 
technical instructions for providing the nonclinical and clinical data 
for testing will be made available to pilot participants. Participants 
in the pilot project will be asked to provide nonclinical and clinical 
datasets as described in the technical instructions and to provide 
technical feedback.

B. Scope

    Existing requirements for the submission of CBER nonclinical and 
clinical data will not be waived, suspended, or modified for purposes 
of this pilot project. The pilot project will test the preparation and 
use of the submitted nonclinical and clinical electronic datasets.

C. How to Participate

    Written requests to volunteer should be submitted to the Division 
of Dockets Management (see ADDRESSES). Requests are to be identified 
with the docket number found in brackets in the heading of this 

III. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic comments regarding this pilot 
project. Submit a single copy of electronic comments or two paper 
copies of any mailed comments, except that individuals may submit one 
paper copy. Comments are to be identified with the docket number found 
in brackets in the heading of this document. We will consider all 
received comments in making a determination on electronic filing and 
when drafting a guidance document for submitting CBER nonclinical and 
clinical study data as electronic datasets. Received comments may be 
seen in the Division of Dockets Management between 9 a.m. and 4 p.m., 
Monday through Friday.

    Dated: December 16, 2004.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-28579 Filed 12-29-04; 8:45 am]