[Federal Register Volume 73, Number 79 (Wednesday, April 23, 2008)]
[Notices]
[Pages 21963-21964]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E8-8772]



[[Page 21963]]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2008-N-0234]


Developing Guidance on Conducting Scientifically Sound 
Pharmacoepidemiologic Safety Studies Using Large Electronic Healthcare 
Data Sets; Public Workshop; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop; request for comments.

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SUMMARY: The Center for Drug Evaluation and Research (CDER) and the 
Center for Biologics Evaluation and Research (CBER) at the Food and 
Drug Administration (FDA) are announcing a public workshop entitled 
``Developing Guidance on Conducting Scientifically Sound 
Pharmacoepidemiologic Safety Studies Using Large Electronic Healthcare 
Data Sets.'' The purpose of the public workshop is to solicit 
information and views from interested persons on best practices and 
principles for the design and evaluation of pharmacoepidemiologic 
safety studies using large electronic healthcare data sets. The input 
from this workshop will be used to develop a draft Guidance to 
Industry, and to provide consistent review criteria for FDA to use in 
evaluating protocols and study reports submitted to the agency.

DATES: The public workshop will be held on Wednesday, May 7, 2008, from 
8:30 a.m. to 5 p.m. See section III of this document for information on 
the deadline and on how to attend or present at the meeting. Written or 
electronic comments must be submitted to the docket by June 7, 2008.

ADDRESSES: The public workshop will be held in the Ballroom at the 
Crowne Plaza Hotel Washington DC-Silver Spring, MD at 8777 Georgia 
Ave., Silver Spring, MD 20910.
    Regardless of attendance at the public workshop, interested persons 
may submit written electronic comments 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://www.regulations.gov. Comments are to be identified with the docket 
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Lana Pauls, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave. 
Bldg. 51, rm. 6196, Silver Spring, MD 20903, 301-796-0518, FAX: 301-
847-8753, e-mail: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    As part of the reauthorization of the Prescription Drug User Fee 
Act (PDUFA IV), FDA committed to certain performance goals (see http://www.fda.gov/oc/pdufa4/pdufa4goals.html). In one of these goals, FDA 
agreed to identify, with input from academia, industry, and others from 
the general public, epidemiology best practices and to develop 
guidance(s) describing these practices. In addition, in the Food and 
Drug Administration Amendments Act of 2007 (FDAAA, Public Law 110-85, 
121 Stat. 823 et seq.), Congress directed FDA to develop and implement 
a postmarket risk identification and analysis system that would 
include, among other things, advanced analysis of drug safety data 
(FDAAA, section 905, 121 Stat. 944). This workshop represents the first 
step in meeting the PDUFA goal and will provide valuable information as 
we build our active postmarket surveillance system.
    New technologies and the ability to assemble large data sets for 
use in epidemiologic research of drug safety issues have precipitated a 
great deal of discussion over the appropriate use of these data in 
conducting pharmacoepidemiologic studies. FDA is committed to 
developing guidance to identify and encourage the use of best practices 
in the conduct of epidemiologic studies of drug safety issues by 
industry, FDA, and academic researchers. Experts from industry, 
academia, and the general public are invited to contribute ideas and 
concepts for consideration.
    The workshop objectives are as follows: (1) Initiate constructive 
dialogue and information-sharing among regulators, researchers, the 
pharmaceutical industry, health organizations, and individuals about 
the design, conduct and interpretation of pharmacoepidemiologic safety 
studies using electronic healthcare data sets; (2) share current FDA 
experiences regarding the evaluation of protocols and study reports 
submitted to the agency; and (3) obtain input on developing consistent 
review criteria for FDA to use in evaluating protocols and study 
reports submitted to the agency.
    Two panel discussions will focus on areas in which the agency 
requests input.
    Panel 1 will focus on characteristics of electronic data used to 
conduct pharmacoepidemiologic studies for use in regulatory assessment 
of product safety. Topics include: differences in health care coverage, 
determinants of enrollment, country or region of data collection, 
characteristics of various healthcare systems and how these might 
impact on the interpretation and the generalizability of the results to 
the U.S. patient population. Specific questions include:
    1. What information and what level of detail are needed for FDA to 
ensure the appropriateness of the data source to address the product 
safety questions being asked? How does this differ by type of data 
source (electronic medical records (EMR) vs. claims)?
    2. What are the challenges of using enrollment data for defining 
study populations in claims databases? Describe effective strategies 
for addressing the absence of formal enrollment data in some EMR 
systems.
    3. Under what circumstances should FDA consider studies using non-
U.S. electronic data sources in its assessment of product safety 
questions?
    Panel 2 will focus on characteristics related to study design, 
conduct and interpretation of pharmacoepidemiologic safety studies, 
specifically those using electronic healthcare data sources. Topics 
include issues pertinent to definition of exposure, ascertainment of 
outcome, analysis of data, and interpretation of study findings and 
will address the following questions:
    1. How can FDA assure that the study design accurately captures the 
clinical events, exposures of interest, and confounding factors needed 
to answer the product safety question under investigation?
    2. What are effective strategies to address confounding by 
indication and the effect of measured and unmeasured confounders?
    3. What are other challenges to internal and external validity in 
studies using EMR and claims databases? What are the best practices for 
addressing them?
    FDA is working to refine the workshop agenda and to invite panel 
members. We are seeking broad participation by safety researchers, 
health system officials, the pharmaceutical industry, and others. We 
anticipate issuing a summary of the workshop, including a discussion of 
implications and next steps for further development.

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II. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic comments regarding this document. 
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. Received comments may be seen in the 
Division of Dockets Management between 9 a.m. and 4 p.m., Monday 
through Friday.
    Please note that on January 15, 2008, the FDA Division of Dockets 
Management Web site transitioned to the Federal Dockets Management 
System (FDMS). FDMS is a Government-wide, electronic docket management 
system. Electronic comments or submissions will be accepted by FDA only 
through FDMS at http://www.regulations.gov.

III. Attendance and Registration

    The Workshop facility, the Ballroom in the Crowne Plaza Hotel at 
8777 Georgia Ave. in Silver Spring, MD is not a secure facility. 
Seating will be made available on a first-come basis. Individual 
interested in attending the workshop need not register.
    Individuals who wish to speak during the public workshop must 
register on or before April 7, 2008. You should identify the subject 
matter you wish to address during the public workshop. Please specify 
Panel 1, or Panel 2 (see I. Background). To register to speak, contact 
[email protected] or call 301-796-0518.
    Ample time will be allowed during the scheduled agenda for 
attendees to ask questions of panelists. In addition, we strongly 
encourage written comments to the docket.
    If you need special accommodations because of disability, please 
contact Lana Pauls (see FOR FURTHER INFORMATION CONTACT) at least 7 
days before the workshop.

IV. Workshop Transcripts

    Please be advised that as soon as a transcript is available, it 
will be accessible at http://www.fda.gov/ohrms/dockets/ac/acmenu.htm. 
It may be viewed at the Division of Dockets Management (see ADDRESSES). 
A transcript will also be available in either hardcopy or on CD-ROM, 
after submission of a Freedom of Information request. Written requests 
are to be sent to Division of Freedom of Information (HFI-35), Office 
of Management Programs, Food and Drug Administration, 5600 Fishers 
Lane, rm. 6-30, Rockville, MD 20857.

    Dated: April 17, 2008.
Jeffrey Shuren,
Associate Commissioner for Policy and Planning.
[FR Doc. E8-8772 Filed 4-22-08; 8:45 am]
BILLING CODE 4160-01-S