[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]
BILLING CODE 4160-01-P