Risk Evaluation and Mitigation Strategy Assessments: Social Science Methodologies to Assess Goals Related to Knowledge; Public Workshop; Issue Paper, 26292-26294 [2012-10646]
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26292
Federal Register / Vol. 77, No. 86 / Thursday, May 3, 2012 / Notices
FDA has determined that the
applicable regulatory review period for
LASTACAFT is 2,189 days. Of this time,
1,886 days occurred during the testing
phase of the regulatory review period,
while 303 days occurred during the
approval phase. These periods of time
were derived from the following dates:
1. The date an exemption under
section 505(i) of the Federal Food, Drug,
and Cosmetic Act (the FD&C Act) (21
U.S.C. 355(i)) became effective: August
1, 2004. The applicant claims July 31,
2004, as the date the investigational new
drug application (IND) became effective.
However, FDA records indicate that the
IND effective date was August 1, 2004,
which was 30 days after FDA receipt of
the IND.
2. The date the application was
initially submitted with respect to the
human drug product under section
505(b) of the FD&C Act: September 29,
2009. The applicant claims September
28, 2009, as the date the new drug
application (NDA) for LASTACAFT
(NDA 22–134) was initially submitted.
However, FDA records indicate that
NDA 22–134 was submitted on
September 29, 2009.
3. The date the application was
approved: July 28, 2010. FDA has
verified the applicant’s claim that NDA
22–134 was approved on July 28, 2010.
This determination of the regulatory
review period establishes the maximum
potential length of a patent extension.
However, the U.S. Patent and
Trademark Office applies several
statutory limitations in its calculations
of the actual period for patent extension.
In its application for patent extension,
this applicant seeks 1,246 days of patent
term extension.
Anyone with knowledge that any of
the dates as published are incorrect may
submit to the Division of Dockets
Management (see ADDRESSES) either
electronic or written comments and ask
for a redetermination by July 2, 2012.
Furthermore, any interested person may
petition FDA for a determination
regarding whether the applicant for
extension acted with due diligence
during the regulatory review period by
October 30, 2012. To meet its burden,
the petition must contain sufficient facts
to merit an FDA investigation. (See H.
Rept. 857, part 1, 98th Cong., 2d sess.,
pp. 41–42, 1984.) Petitions should be in
the format specified in 21 CFR 10.30.
Interested persons may submit to the
Division of Dockets Management (see
ADDRESSES) electronic or written
comments and written petitions. It is
only necessary to send one set of
comments. However, if you submit a
written petition, you must submit three
copies of the petition. Identify
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Jkt 226001
comments with the docket number
found in brackets in the heading of this
document.
Comments and petitions that have not
been made publicly available on
https://www.regulations.gov may be
viewed in the Division of Dockets
Management between 9 a.m. and 4 p.m.,
Monday through Friday.
Dated: April 16, 2012.
Jane A. Axelrad,
Associate Director for Policy, Center for Drug
Evaluation and Research.
[FR Doc. 2012–10694 Filed 5–2–12; 8:45 am]
BILLING CODE 4160–01–P
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.
Notice of public workshop;
request for comments.
ACTION:
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 informationsharing 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.
SUMMARY:
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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 https://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: colleen.omalley@
fda.hhs.gov.
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
KnowledgeAssessmentWorkshop@fda.
hhs.gov. 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
E:\FR\FM\03MYN1.SGM
03MYN1
Federal Register / Vol. 77, No. 86 / Thursday, May 3, 2012 / Notices
wreier-aviles on DSK7SPTVN1PROD with NOTICES
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
https://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
https://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 https://
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
1 See https://www.gpo.gov/fdsys/pkg/PLAW110publ85/pdf/PLAW-110publ85.pdf.
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Jkt 226001
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.
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 crosssectional 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
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26293
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
E:\FR\FM\03MYN1.SGM
03MYN1
26294
Federal Register / Vol. 77, No. 86 / Thursday, May 3, 2012 / Notices
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 https://
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Government-Owned Inventions;
Availability for Licensing
National Institutes of Health,
Public Health Service, HHS.
ACTION: Notice.
wreier-aviles on DSK7SPTVN1PROD with NOTICES
AGENCY:
The inventions listed below
are owned by an agency of the U.S.
Government and are available for
licensing in the U.S. in accordance with
35 U.S.C. 207 to achieve expeditious
commercialization of results of
SUMMARY:
VerDate Mar<15>2010
15:32 May 02, 2012
Jkt 226001
federally-funded research and
development. Foreign patent
applications are filed on selected
inventions to extend market coverage
for companies and may also be available
for licensing.
FOR FURTHER INFORMATION: Licensing
information and copies of the U.S.
patent applications listed below may be
obtained by writing to the indicated
licensing contact at the Office of
Technology Transfer, National Institutes
of Health, 6011 Executive Boulevard,
Suite 325, Rockville, Maryland 20852–
3804; telephone: 301–496–7057; fax:
301–402–0220. A signed Confidential
Disclosure Agreement will be required
to receive copies of the patent
applications.
Mouse Monoclonal Antibody Targeting
Human NOX1, a Target for Cancer and
Inflammation
Description of Technology: Available
for licensing is a mouse monoclonal
antibodies targeting human
nicotinamide adenine dinucleotide
phosphate-oxidase (NAPH) oxidase 1
(NOX1) enzyme. NOX mediates the
homeostasis of reactive oxygen species,
which play a critical regulatory role in
cancer cell signal transduction and
tumor cell differentiation. NOX1generated hydrogen peroxide can trigger
an ‘‘angiogenic switch’’ that includes
the induction of angiogenic factors that
promote tumor cell vascularization.
Additionally, NOX1 may play a role in
inflammation.
Investigators at the National Cancer
Institute found NOX1 is significantly
expressed more in colon and gastric
cancers compared with adjacent normal
bowel and gastric mucosa respectively.
To the best of NIH’s knowledge, this is
the only monoclonal antibody that can
be used to detect human NOX1. This
antibody detects endogenous levels of
the NOX1 protein and could potentially
be used in biochemical laboratory
studies as well as diagnostic tests that
involve the functional significance of
NOX1 in human physiology and
pathophysiology, particularly its role in
cancer and inflammation.
Potential Commercial Applications:
• Research tool to study cancer and
inflammation
• Method to diagnose colon and
gastric cancer
• Treatment for cancer and
inflammation
Competitive Advantages: To the best
of NIH’s knowledge, this is the only
available monoclonal antibody to detect
human NOX1.
Development Stage:
• Early-stage
• In vitro data available
PO 00000
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Inventors: James Doroshaw,
Krishnendu Roy, Guojian Jiang, Jiamo
Lu, and Smitha Antony (all of NCI).
Intellectual Property: HHS Reference
No. E–097–2012/0—Research Tool.
Patent protection is not being pursued
for this technology.
Licensing Contact: Sabarni K.
Chatterjee, Ph.D.; 301–435–5587;
chatterjeesa@mail.nih.gov.
A Non-Invasive Post-Treatment
Strategy for Stroke by Intranasal
Delivery of Cocaine- and
Amphetamine-Regulated Transcript
(CART)
Description of Technology: Cocaine
and amphetamine-regulated transcript
(CART) is a neuropeptide known to
protect against ischemic brain injury
when administered before the onset of
stroke in mice, both in vivo and in vitro.
Utilizing a classic stroke model in
rodents, middle cerebral artery
occlusion (MCAo), inventors at NIDA
discovered a novel post-stroke
therapeutic approach involving the
intranasal administration of CART. This
new non-invasive treatment strategy for
stroke patients is effective when
initiated three days after stroke,
providing a longer treatment window.
Nasal delivery of CART improved
behavioral recovery and reduced
neurological scores in stroke animals.
CART, given after stroke, modifies
endogenous neural repair in stroke brain
by facilitating neuroprogenitor cell
proliferation and migration, enhancing
reinnervation, and improving the
functional recovery.
Potential Commercial Applications:
Method of treating stroke
Competitive Advantages:
• New treatment strategy for stroke
patients
• Non-invasive (nasal spray)
• Longer treatment window (3 days
post-stroke)
• Current strategies aim to protect
lesion site from damage, whereas this
method helps brain repair
Development Stage:
• Early-stage
• Pre-clinical
• In vitro data available
• In vivo data available (animal)
Inventors: Yun Wang, Hui Shen,
Seong Jin Yu, Yihong Yang (all of
NIDA).
Publications: Manuscript in
preparation.
Intellectual Property: HHS Reference
No. E–058–2012/0—U.S. Provisional
Application No. 61/592,761 filed 31 Jan
2012.
Licensing Contact: Betty B. Tong,
Ph.D.; 301–594–6565;
tongb@mail.nih.gov.
E:\FR\FM\03MYN1.SGM
03MYN1
]]>Agencies
[Federal Register Volume 77, Number 86 (Thursday, May 3, 2012)]
[Notices]
[Pages 26292-26294]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-10646]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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 https://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: colleen.omalley@fda.hhs.gov.
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
KnowledgeAssessmentWorkshop@fda.hhs.gov. 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
[[Page 26293]]
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 https://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 https://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 https://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.
---------------------------------------------------------------------------
\1\ See https://www.gpo.gov/fdsys/pkg/PLAW-110publ85/pdf/PLAW-110publ85.pdf.
---------------------------------------------------------------------------
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 https://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
