[Federal Register: December 10, 2001 (Volume 66, Number 237)]
[Page 63712-63713]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]



Food and Drug Administration

[Docket No. 01N-0496 ]

Patient Profile Viewer; Notice of Pilot Project

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.


SUMMARY: The Food and Drug Administration (FDA), Center for Drug 
Evaluation and Research (CDER), is seeking volunteers to participate in 
a pilot project involving the testing of the Patient Profile Viewer 
(PPV). The PPV is computer software that allows a reviewer to display 
data collected from case report tabulations (CRTs) submitted in 
electronic format. We are working with PPD Informatics to develop the 
PPV under a Cooperative Research and Development Agreement (CRADA) in 
an effort to improve review efficiency, develop standards for 
submission of data, and eliminate the need for the submission of 
patient profiles by applicants of new drug applications (NDAs). To help 
in this development, we are seeking volunteers to provide CRT datasets 
from clinical studies to test the PPV. Data supplied during the pilot 
project will not replace any regulatory requirements for submitting 

DATES: Submit written or electronic requests to participate in the 
pilot project by January 9, 2002. Comments on the pilot project may be 
submitted at any time.

ADDRESSES: Submit written requests to participate and comments 
regarding this pilot project to the Dockets Management Branch (HFA-
305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, 
Rockville, MD 20852. Submit written comments on the pilot project to 
the Dockets Management Branch (address above). 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.

Evaluation and Research (HFD-001), Food and Drug Administration, 5600 
Fishers Lane, Rockville, MD 20857, 301-594-5411, e-mail: 


I. Background

    Under current FDA regulations (21 CFR 314.50), applicants must 
provide CRTs with NDAs. Since November 1997, under 21 CFR part 11, we 
have accepted CRTs in electronic format instead of paper.
    We have published several guidance documents that provide 
recommendations concerning electronic submissions. In the Federal 
Register of January 28, 1999 (64 FR 4432), CDER published the guidance 
entitled ``Providing Regulatory Submissions in Electronic Format--
NDAs.'' This guidance describes how applicants can provide CRTs as 
electronic datasets. This guidance also offers recommendations on how 
to organize the datasets and how to provide descriptive information on 
the datasets and the data variables (metadata). In the Federal Register 
of November 12, 1999 (64 FR 61647), the Center for Biologics Evaluation 
and Research (CBER) provides similar recommendations for biologic 
license applications (BLAs) in their guidance entitled ``Providing 
Regulatory Submissions in Electronic Format--BLAs.'' A joint CBER and 
CDER guidance for industry entitled ``Providing Regulatory Submissions 
in Electronic Format--General Considerations,'' provides 
recommendations for the file formats for clinical datasets (64 FR 4433, 
January 28, 1999).
    The datasets described in these guidance documents are organized by 
domain (e.g., labs, adverse events). For NDAs, however, we also 
recommend the submission of CRTs organized by individual patients--a 
format we call patient profiles. Patient profiles are provided in 
portable document format (PDF) and not as electronic datasets. Patient 
profiles are not recommended for submissions to CBER. CDER is working 
with CBER to update the guidance documents with more detailed standards 
for the submission of CRT datasets and metadata.
    Recently, we have received recommendations for a standard 
presentation of the most common CRT datasets and metadata from the 
Clinical Data Interchange Standards Consortium, Inc. (CDISC). CDISC is 
a nonprofit organization and its members are from pharmaceutical 
companies, biotechnology companies, contract research organizations, 
and software vendors.
    CDER has also entered into a CRADA with PPD Informatics (PPD) to 
develop a module for PPD's commercially available CrossGraphs software 
that will generate patient profiles directly from CRT datasets provided 
with NDA submissions. The use of standardized datasets and metadata 
reduces the amount of preparation required by the reviewer to generate 
patient profiles and would eliminate the need for applicants to provide 
patient profiles in PDF. The purpose of the pilot project is to test 
the PPV module with standardized datasets and metadata and to obtain 
feedback from reviewers and pharmaceutical

[[Page 63713]]

companies on the creation and use of standardized clinical data and 

II. Pilot Project Description

    The pilot project is part of an effort to improve the standards for 
submission of clinical data. Eventually, we expect to recommend 
detailed clinical data and metadata standards for the submission of 
CRTs. Participants in this PPV pilot project will not only assist us in 
testing the use of the PPV and standard clinical data and metadata but 
will also 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 number of voluntary participants are needed, the 
agency will use its discretion in choosing volunteers, based on their 
experience with providing CRTs and their familiarity with the standards 
recommended by CDISC. During the pilot project, the agency will make 
available to the public specific technical instructions for providing 
the clinical data and metadata for testing. See the Electronic Access 
section for instructions. Participants in the pilot project will be 
asked to provide clinical trial datasets and metadata as described in 
the technical instructions and to provide technical feedback.

B. Scope

    The pilot project will test the PPV module and the preparation and 
use of the submitted data and metadata. Existing requirements for the 
submission of CRT datasets will not be waived, suspended, or modified 
for purposes of this pilot project.

III. Pilot Project Participation

    Written requests to volunteer for the pilot project should be 
submitted to the Dockets Management Branch (address above). Requests 
are to be identified with the docket number found in brackets in the 
heading of this document.

IV. Comments

    Interested persons may submit to the Dockets Management Branch 
(mail and electronic addresses above) written comments regarding this 
pilot project. Two copies of any comments are to be submitted, except 
that individuals may submit one copy. Comments are to be identified 
with the docket number found in brackets in the heading of this 
document. FDA will consider comments in making a determination on 
electronic filing and in drafting a guidance document for submitting 
clinical trial data and metadata electronically. Received comments may 
be seen in the Dockets Management Branch between 9 a.m. and 4 p.m., 
Monday through Friday.

V. Electronic Access

    These instructions will be available on the Internet at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cder/regulatory/ersr/default.htm.

    Dated: December 3, 2001.
Margaret M. Dotzel,
Associate Commissioner for Policy.
[FR Doc. 01-30430 Filed 12-5-01; 11:21 am]