[Federal Register Volume 77, Number 86 (Thursday, May 3, 2012)]
[Notices]
[Pages 26292-26294]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-10646]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2012-N-0408]
Risk Evaluation and Mitigation Strategy Assessments: Social
Science Methodologies to Assess Goals Related to Knowledge; Public
Workshop; Issue Paper
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
workshop entitled ``Risk Evaluation and Mitigation Strategy
Assessments: Social Science Methodologies to Assess Goals Related to
Knowledge.'' The purpose of the public workshop is to initiate
constructive dialogue and information-sharing among regulators,
researchers, the pharmaceutical industry, health care organizations,
health care providers, and others from the general public about survey
methodologies and instruments that can be used to evaluate patients'
and health care providers' knowledge about the risks of drugs marketed
with an approved Risk Evaluation and Mitigation Strategy (REMS). The
input from this workshop will be used to develop guidance for industry
describing the best practices for conducting an assessment of a REMS
goal regarding patient and/or health care provider knowledge about a
drug's risk(s). To assist in the workshop discussion and the ultimate
development of the guidance, FDA is making available an issue paper
that discusses our experience with knowledge assessments for REMS and
contains specific questions we hope to receive input on. FDA is also
opening a public docket to receive written comments.
Date and Time: The public workshop will be held on June 7, 2012,
from 8 a.m. to 5 p.m.
Location: The public workshop will be held at FDA's White Oak
Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the
Great Room (rm. 1503), Silver Spring, MD 20993-0002. Entrance for the
public workshop participants (non-FDA employees) is through Building 1,
where routine security check procedures will be performed. For parking
and security information, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm. Participants are encouraged to arrive early to ensure
time for parking and security screening before the workshop.
Contact Person: Colleen O'Malley, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
22, Rm. 4305, Silver Spring, MD 20993-0002, 301-796-1786, FAX: 301-796-
9832, email: [email protected].
Registration and Requests for Oral Presentations: There is no fee
to attend the workshop, and attendees who do not wish to make a formal
presentation do not need to register. Seating will be on a first-come,
first-served basis. Individuals who wish to make a presentation at the
public workshop must register and provide an abstract of your
presentation by 5 p.m. on May 21, 2012.
Submit electronic registration requests to make a presentation to
[email protected]. Submit written registration
requests to make a presentation to Colleen O'Malley (see Contact
Person). Please provide your name, title, business affiliation (if
applicable), address, telephone, FAX number, and email address.
Identify the Panel number(s) for the question(s) you will discuss in
your presentation (see section IV of this document).
FDA will do its best to accommodate requests to speak. Individuals
and organizations with common interests are urged to consolidate or
coordinate their presentations and request time for a joint
presentation. FDA will determine the amount of time allotted to each
presenter and the approximate time that each oral presentation is
scheduled to begin. Persons registered to make a formal presentation
should check in before the workshop. Time will be allowed during the
scheduled agenda for attendees to ask questions of the panelists. In
addition, we strongly
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encourage electronic or written comments to the docket.
FDA has developed an issue paper entitled ``Risk Evaluation and
Mitigation Strategy Assessments: Social Science Methodologies to Assess
Goals Related to Knowledge'' that discusses our experience with
knowledge assessments for REMS. The issue paper also contains a number
of specific questions that we hope to receive input on. The issue paper
can be found on the Internet at http://www.fda.gov/Drugs/NewsEvents/ucm292337.htm.
Background information on the public workshop, registration
information, the agenda, and other relevant information will be posted
on the Internet at http://www.fda.gov/Drugs/NewsEvents/ucm132703.htm as
it becomes available.
If you need special accommodations due to a disability, please
contact Colleen O'Malley (see Contact Person) at least 7 days before
the workshop.
Comments: FDA is opening a docket to allow for public comments to
be submitted to the Agency on the issues and questions presented in the
issue paper or at the workshop. Regardless of attendance at the public
workshop, interested persons may submit to the Division of Dockets
Management either electronic or written comments by July 7, 2012, to
receive consideration. Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
rm. 1061, Rockville, MD 20852. It is only necessary to send one set of
comments. Identify comments 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.
SUPPLEMENTARY INFORMATION:
I. Background
Title IX, Subtitle A, section 901 of the Food and Drug
Administration Amendments Act (FDAAA) (Pub. L. 110-85) \1\ created new
section 505-1 of the Federal Food, Drug, and Cosmetic Act (FD&C Act)
(21 U.S.C. 355-1), which authorizes FDA to require persons submitting
new drug applications (NDAs) or abbreviated new drug applications
(ANDAs) for prescription products, or biologics license applications
(BLAs), to submit and implement a REMS if FDA determines that a REMS is
necessary to ensure the benefits of a drug outweigh the risks of the
drug. To require a REMS for an already approved drug, FDA must have
become aware of new safety information as defined in the statute.
Elements for REMS approved for NDAs and BLAs may include a Medication
Guide, a communication plan, and/or elements to assure safe use
(ETASU), and an implementation system, if specific statutory criteria
are met. All approved REMS for products approved under an NDA or BLA
must include a timetable for submission of assessments of the REMS.
FDAAA contains provisions that are specifically directed to REMS for
ANDAs, and these REMS may include only a Medication Guide and/or ETASU
and an implementation system.
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\1\ See http://www.gpo.gov/fdsys/pkg/PLAW-110publ85/pdf/PLAW-110publ85.pdf.
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Because most REMS include a goal related to knowledge, such as to
inform or educate patients and/or health care providers about the
serious risks associated with and safe use of a drug, assessments for a
drug subject to a REMS frequently include assessments of patients' and
providers' knowledge. To conduct this assessment, most applicants have
undertaken cross-sectional surveys of patients who have taken the drug
and health care providers who have prescribed or dispensed the drug.
As a result of FDA's review of the surveys that are included as
components of a REMS assessment, the Agency has identified certain
challenges to conducting these types of studies. FDA has specific
questions about the methodology for obtaining survey data and
presenting the results, including about appropriate sample size;
methods to ensure representativeness; how to determine endpoints;
questionnaire design and analyses; and presentation of survey results.
To date, FDA has worked with individual applicants to attempt to
introduce and sustain some measure of consistency in methods and
expectations. Although absolute uniformity is not possible, the Agency
seeks to solicit information and feedback about valid survey methods
that can improve the quality and consistency of REMS assessment
surveys. In addition, FDA seeks feedback on whether methodologies other
than surveys could be used to obtain this information. Finally, FDA
seeks to solicit information about using surveys other than knowledge
assessment surveys as a tool to assess whether the elements of a REMS
are meeting its goals including: (1) Changes in behavior for both
patients and prescribers such as whether a drug is used to a large
degree in patients at higher risk of an adverse reaction; (2) burden on
the health care system, which could include the time required to
accomplish REMS-related activities; and (3) adverse effects on patient
access to the drug, such as substantial delays between the time of
presentation of a prescription and the time of drug dispensing or
prescribers choosing not to prescribe the drug anymore.
II. Why are we holding this public workshop?
FDA is soliciting information and feedback to optimize the
assessment of REMS goals related to knowledge. Because we have received
only surveys that assess knowledge, the workshop will invest
considerable time in identifying best methodological practices for
conducting REMS assessment surveys. However, FDA is also encouraging
discussion of alternatives to surveys, given the issues we have
observed, as discussed in the issue paper. Feedback received in the
docket and resulting from this workshop will assist the Agency in
developing guidance for industry.
The workshop objectives are as follows: (1) Initiate constructive
dialogue and information-sharing about survey methodologies and
instruments used to evaluate patients' and healthcare providers'
knowledge about drugs' risks; (2) share current FDA experience
regarding social science assessments of surveys as a component of REMS
Assessment Plans; (3) obtain information that will be used to develop
standardized survey methodologies for evaluating patient and health
care provider knowledge under a REMS; (4) discuss alternative
methodologies to surveys to assess knowledge; and (5) discuss the use
of surveys as a tool to assess patient and prescriber behavior changes,
burden on the health care system, and patient access to the drug under
a REMS.
III. Who is the target audience and who should attend this public
workshop?
Although the workshop is open to all interested parties, the target
audience includes social science professionals; statisticians;
regulators; researchers; and representatives from academia, the
pharmaceutical industry, and the scientific community who may be
interested in improving the quality and consistency of methodology for
evaluating REMS goals related to knowledge.
IV. What are the topics we intend to discuss at the public workshop?
The workshop will include panel discussions and individual and/or
joint presentations. The key issues to be addressed are: (1) How should
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assessments of knowledge be structured to achieve valid, reliable, and
informative results; (2) how can surveys be used to assess changes in
patient and prescriber behavior, burden to the health care system, and
patient access to the drug; and (3) what are appropriate alternatives
to surveys to assess educational components of REMS? Two panel
discussions will focus on areas in which the Agency requests specific
input.
Panel 1 will focus on using surveys to assess knowledge.
Topics will include, but are not limited to, recruiting a
representative sample, sample size, question design, process, and
endpoints.
Panel 2 will focus on alternatives to surveys and the use
of surveys to assess patient and prescriber behavior changes, burden on
the health care system, and patient access to the drug. Topics will
include, but are not limited to, recruiting a representative sample,
question design, interpretation of results, and specific pros and cons
of the alternatives.
V. Transcripts
Please be advised that as soon as a transcript of the workshop is
available, it will be accessible at http://www.regulations.gov. It may
be viewed at the Division of Dockets Management (see Comments). 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 the Division of Freedom of Information (ELEM-1029),
Food and Drug Administration, 12420 Parklawn Dr., Element Bldg.,
Rockville, MD 20857.
Dated: April 27, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-10646 Filed 5-2-12; 8:45 am]
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