[Federal Register Volume 77, Number 76 (Thursday, April 19, 2012)]
[Notices]
[Pages 23485-23486]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-9418]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA 2012-N-0001]


Food and Drug Administration Patient Network Annual Meeting; 
Input Into Food and Drug Administration Benefit-Risk Decisionmaking: 
Opportunities and Challenges; Hosted by the Food and Drug 
Administration Office of Special Health Issues; Notice of Meeting

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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    The Food and Drug Administration (FDA) is announcing a meeting for 
patients, caregivers, independent patient advocates and patient 
advocate groups, and health professional groups to explore ways to more 
effectively include patient input in regulatory decisionmaking on drug, 
device, and biological products. The meeting will serve as a forum for 
FDA's patient stakeholders and the general public, including health 
professionals, academia, and industry to learn about the regulatory 
process related to the medical product life cycle, analyze where in the 
process patient input may be most practical and most valuable, and 
explore practicable approaches to collecting and incorporating 
meaningful input that well represents broad patient perspectives into 
regulatory decisions.

DATES: Date and Time: The meeting will be held on May 18, 2012, from 9 
a.m. to 4:30 p.m. Register at http://fda.contractmeetings.com/home on 
or before May 4, 2012. Please include the name and title of the person 
attending, the name of the organization, the role within the 
organization, email address, and telephone number. There is no 
registration fee for this conference. Early registration is suggested 
because space is limited. We request that organizations limit the 
number of representatives to two. For further registration information 
or problems with the Web site, call Cindy de Sales, 1-240-316-3200, 
ext. 207.
    If you need special accommodations due to a disability, please 
contact Steve Morin at least 7 days in advance.
    Location: The meeting will be held at the FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room 
(Rm. 1503) Silver Spring, MD 20993.
    Contact Person: Steve Morin, Office of Special Health Issues, 10903 
New Hampshire Ave., Silver Spring, MD 20993, 301-796-0161, FAX: 301-
847-8623, [email protected].

SUPPLEMENTARY INFORMATION:

I. FDA Patient Network

    This is the inaugural FDA Patient Network Annual Meeting hosted by 
the FDA Office of Special Health Issues, the Agency's liaison to the 
patient and health professional communities. This annual meeting is 
being hosted in conjunction with the launch of the overarching FDA 
Patient Network program. The FDA Patient Network is a new resource for 
patients, caregivers, independent patient advocates, and patient 
advocate groups that seek to:
     Educate and inform patient stakeholders about FDA, its 
regulatory

[[Page 23486]]

authorities and processes, its initiatives and programs, etc.; and,
     Provide a venue for advocacy for patient stakeholders 
within FDA and be transparent to patients about Agency actions.
    In addition to an annual meeting, the FDA Patient Network consists 
of other activities, including the:
     FDA Patient Network Web site--A new, patient-centered Web 
site that contains educational modules, centralized Agency information, 
and multidirectional communication tools;
     Biweekly FDA Patient Network News email newsletter; and 
hosting of periodic meetings, briefings, and listening session between 
patient advocates and FDA staff.

II. Patient Perspectives in Regulatory Decisionmaking

    Establishing a means for obtaining input from patients and patient 
advocate groups will allow FDA to further enhance its benefit-risk 
assessment in regulatory decisionmaking. Patients who live with a 
disease have a direct stake in the outcomes of the review process and 
are in a unique position to contribute to the weighing of benefit-risk 
considerations that can occur throughout the medical product 
development process. Though several programs exist that facilitate 
patient representation on Advisory Committees or participation in 
selected review meetings, there are currently few venues in which the 
patient perspective is discussed outside of a specific product's 
marketing application review. The medical product review process could 
benefit from a more scientific, systematic, and expansive approach to 
obtaining input from patients who are experiencing a particular disease 
condition.
    As part of the proposed agreements for Prescription Drug User Fee 
Act (PDUFA) V, FDA plans to conduct meetings with patients and patient 
advocacy groups to gather broader patient input. This meeting kicks off 
these efforts and provides an opportunity to gain feedback on how FDA 
can best structure these upcoming meetings.
    FDA seeks public discussion based on the following questions. These 
questions are intended to frame patient input at the May 18, 2012, 
meeting and there will be time at the meeting to discuss the following 
issues.
    (1) How can FDA ensure gathering a broad range of representative 
patient input that is relevant to a specific disease area during its 
meetings with patients? For example, who should serve as 
representatives of patients?
    (2) What methodological and practical issues should FDA consider as 
it develops its strategy for eliciting the patient perspective? For 
instance, FDA is interested in addressing topics including, but not 
limited to, the following:
    (a) Are there particular advantages or disadvantages to utilizing 
face-to-face meetings versus web-based or other methods in obtaining 
the patient perspective on a particular disease condition and its 
treatment?
    (b) How can FDA ensure that certain subpopulations, such as 
patients with the most severe form of the disease, are represented?

III. Center for Drug Evaluation and Research and Center for Biologics 
Evaluation and Research Efforts

    Center for Drug Evaluation and Research and Center for Biologics 
Evaluation and Research plan to conduct a series of patient-focused 
drug development meetings to gather patient input on the clinical 
context of a disease and its impact on a patient's daily life. These 
considerations, which would include an analysis of the severity of the 
disease condition and the current state of the available treatment 
options, can be critical in regulatory decisionmaking. FDA is 
interested in obtaining patient input on the context of specific 
disease areas through the patient-focused drug development meetings. 
The following questions are examples of topics for which FDA believes 
the patient perspective could add valuable insight. They are presented 
in this document for general discussion at the Patient Network 
Conference.

A. Understanding the Disease Condition

    (1) What are the clinical manifestations of the disease that have 
the greatest impact on patients?
    (2) Are there other aspects of the disease that have a significant 
impact on a patient's daily life? (e.g., impaired mobility, sleep 
problems, etc.)
    (3) How do the clinical manifestations change with disease 
progression?
    (4) How do the other aspects of the disease change with disease 
progression?

B. Assessment of Treatment Options

    (1) How effective are approved therapies at treating the clinical 
manifestations of the disease?
    (2) How well do approved therapies mitigate the other aspects of 
the disease?
    (3) How does the effectiveness of approved therapies change with 
progression of the disease?
    (4) Does therapy effectiveness vary by patient subpopulation?
    FDA is continuing to make plans for its efforts and will be able to 
provide more detail on the patient-focused drug development meetings at 
the Patient Network Conference.
IV. Center for Devices and Radiologic Health Efforts
    Center for Devices and Radiologic Health is interested in a public 
discussion on issues related to risk associated with medical products, 
and on avenues for patients to provide input into regulatory 
decisionmaking related to the amount of risk patients may be willing to 
accept in exchange for a potential treatment benefit. The following 
questions are presented in this document for general discussion at the 
Patient Network Conference.
    (1) How do patients perceive and weigh risks associated with 
medical treatment in light of the risk associated with the underlying 
condition being treated and the potential benefit from the treatment?
    (2) Under what circumstances and in which populations would various 
levels of risk be appropriate/acceptable?
    (3) How can medical device companies, government, academia, 
community physicians and patients collaborate to account for the level 
of risk acceptable to patients affected by serious or life threatening 
illnesses?
    (4) What mechanisms would be appropriate for patients to provide 
input into regulatory decisionmaking for new therapeutic and diagnostic 
products--e.g., web-based survey instruments? Patient representation at 
advisory committee meetings? Patient input to medical device companies 
during clinical trial design? Who (FDA, patient advocate groups, 
medical device companies, etc.) could sponsor such surveys?
    (5) Are patients willing to accept responsibility for the level of 
risk to which they may be exposed if patient input increases risk 
tolerance?

    Dated: April 13, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-9418 Filed 4-18-12; 8:45 am]
BILLING CODE 4160-01-P