[Federal Register Volume 77, Number 185 (Monday, September 24, 2012)]
[Notices]
[Pages 58849-58851]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-23454]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0967]


Prescription Drug User Fee Act Patient-Focused Drug Development; 
Public Meeting and Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

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SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
meeting and an opportunity for public comment related to FDA's patient-
focused drug development initiative. This initiative is being conducted 
to fulfill FDA performance commitments made as part of the fifth 
authorization of the Prescription Drug User Fee Act (PDUFA V). This 
effort provides for a more systematic approach under PDUFA V for 
obtaining patient perspective on the disease severity and the currently 
available treatments for a set of disease areas. FDA is publishing a 
preliminary list of nominated disease areas for the patient-focused 
drug development initiative and the criteria used for nomination. The 
public is invited to comment on this preliminary list through a public 
docket and at a public meeting where FDA will provide an overview of 
the patient-focused drug development initiative with discussion of the 
nominated disease areas.

DATES: Submit either electronic or written comments by November 1, 
2012. The public meeting will be held on October 25, 2012, from 9 a.m. 
to 12:30 p.m. Registration to attend the meeting must be received by 
October 18, 2012. See the SUPPLEMENTARY INFORMATION

[[Page 58850]]

section for information on how to register for the meeting.

ADDRESSES: 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 20993-0002. Entrance for the public meeting 
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.
    Submit electronic comments to 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. All 
comments should be identified with the docket number found in brackets 
in the heading of this document.

FOR FURTHER INFORMATION CONTACT:
Andrea Tan, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1168, Silver 
Spring, MD 20993, 301-796-7641, FAX: 301-847-8443, 
[email protected],
or

Robert Yetter, Center for Biologics Evaluation and Research (HFM-25), 
1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448, 301-827-
0373.

SUPPLEMENTARY INFORMATION: 

I. Background

    On July 9, 2012, the President signed into law the Food and Drug 
Administration Safety and Innovation Act of 2012 (FDASIA). Title I of 
FDASIA reauthorizes the Prescription Drug User Fee Act (PDUFA) that 
provides FDA with the necessary user fee resources to maintain an 
efficient review process for human drug and biologic products. The 
reauthorization of PDUFA includes performance goals and procedures that 
represent FDA's commitments during fiscal years (FY) 2013-2017. These 
commitments are referred to in section 101 of FDASIA and are available 
on the FDA Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.
    Section X of these commitments relates to enhancing benefit-risk 
assessment in regulatory decisionmaking. A key part of regulatory 
decisionmaking is establishing the context in which the particular 
decision is made. In drug regulation, this context includes a thorough 
understanding of the severity of the treated condition and the adequacy 
of the existing treatment options. Patients who live with a disease 
have a direct stake in the outcome of the review process and are in a 
unique position to contribute to weighing benefit-risk considerations 
that can occur throughout the medical product development process. 
Though several programs exist to facilitate patient representation, 
there are currently few venues in which the patient perspective is 
discussed outside of a specific product's marketing application review. 
The human drug and biologic review process could benefit from a more 
systematic and expansive approach to obtaining input from patients who 
experience a particular disease or condition.
    FDA is committed to obtaining input from patients and, as set out 
in the commitment letter, will conduct public meetings to consider 20 
different disease areas over the 5-year authorization of the program. 
For each disease area, FDA will conduct a public meeting to discuss 
such topics as the impact of the disease on patients, the spectrum of 
severity for those who have the disease, the measures of benefit that 
matter most to patients, and the adequacy of the existing treatment 
options for patients. These meetings will include participation of FDA 
review divisions, the relevant patient advocacy community, and other 
interested stakeholders. FDA seeks public comment on the set of disease 
areas that will be discussed at these meetings throughout PDUFA V. A 
preliminary list of possible disease areas and the criteria used to 
identify these disease areas are published in this notice for public 
comment.
    FDA recognizes that there is significant interest in patient-
focused drug development within the patient community. To ensure that 
patient stakeholders have an opportunity to contribute as this 
initiative moves forward in PDUFA V, FDA also is convening an 
additional series of patient consultation meetings with patient 
stakeholders to discuss key process questions for patient-focused drug 
development. These consultation meetings will be separate from the 
disease-specific meetings that are part of FDA's commitments in PDUFA 
V. FDA has published a separate notice elsewhere in this issue of the 
Federal Register requesting that patient stakeholders notify FDA if 
they intend to participate in the patient consultation meetings.

II. Disease Area Nomination

    FDA is nominating the following disease areas as potential 
candidates for the focus of one of the 20 future public meetings and 
invites public comment on this preliminary list. In your comments, 
please identify the disease areas that you consider to be of greatest 
priority and explain the rationale for your recommendation.
     Pulmonary arterial hypertension.
     Heart failure.
     Primary glomerular diseases.
     Narcolepsy.
     Huntington's Disease.
     Depression.
     Autism.
     Peripheral neuropathy.
     Fibromyalgia.
     Obesity.
     Nocturia.
     Chronic fatigue syndrome.
     Irritable bowel syndrome.
     Inflammatory bowel disease.
     Alopecia areata.
     Diabetic ulcers.
     Female sexual dysfunction.
     Interstitial cystitis/painful bladder syndrome.
     Fracture healing.
     Diabetic foot infections.
     Hepatitis C.
     HIV.
     Patients who have experienced an organ transplant.
     Sickle cell disease.
     Chronic graft versus host disease.
     Amyloidosis.
     Aplastic anemia.
     Melanoma.
     Lung cancer.
     Cancer and young patients.
     Cancer treatment in pregnancy.
     Cancer and sexual dysfunction.
     Cancer and depression.
     Clotting disorders (e.g., hemophilia A (factor VIII 
deficiency) and von Willebrand disease).
     Thrombotic disorders (e.g., antithrombin deficiency and 
protein C deficiency).
     Primary humoral immune deficiencies (e.g., common variable 
immune deficiency).
     Neurologic disorders treated with immune globulins (e.g., 
chronic inflammatory demyelinating polyneuropathy).
     Hereditary angioedema.
     Alpha-1 antitrypsin deficiency.
    FDA is also interested in public comment on disease areas that are 
not represented on this preliminary list. The Agency used several 
criteria to develop the preliminary list of potential disease areas. 
FDA requests that when proposing additional disease areas for 
consideration, please describe how you

[[Page 58851]]

applied the identified criteria in making recommendations for 
additional disease areas to consider.
    FDA also welcomes public comment on the criteria for disease area 
selection. These criteria include the following:
     Disease areas that are chronic, symptomatic, or affect 
functioning and activities of daily living;
     Disease areas that reflect a range of severity;
     Disease areas for which aspects of the disease are not 
formally captured in clinical trials;
     Disease areas that have a severe impact on identifiable 
subpopulations (such as children or the elderly);
     Disease areas that represent a broad range in terms of 
size of the affected population; or
     Disease areas for which there are currently no therapies 
or very few therapies, or the available therapies do not directly 
affect how a patient feels, functions, or survives.
    FDA will consider the public comments received at the public 
meeting and through the docket and post the set of disease areas for FY 
2013-2015 on the FDA Web site. By the end of FY 2015, FDA will initiate 
a public process for determining the list of disease areas for FY 2016-
2017.

III. Public Meeting

    FDA is holding a public meeting that will begin FDA's patient-
focused drug development initiative in PDUFA V. The purpose of this 
meeting will be to obtain public comment on the preliminary list of 
potential disease areas and the criteria used to develop the list. In 
addition, recognizing that there are many more disease areas than can 
be addressed in the 20 planned FDA meetings for PDUFA V, FDA will also 
discuss strategies that have already been pursued by patient and other 
public stakeholder collaborations outside of FDA to address the types 
of questions being considered under the PDUFA patient-focused drug 
development effort, to review lessons learned and identify a roadmap 
that could be used by patient-focused private collaborations going 
forward.
    If you wish to attend this meeting, please register by email to 
[email protected] by October 18, 2012. Your email should 
contain complete contact information, including name, title, 
affiliation, address, email address, and phone number. Seating will be 
limited, so early registration is recommended. Registration is free and 
will be on a first-come, first-served basis. However, FDA may limit the 
number of participants from each organization based on space 
limitations. Registrants will receive confirmation once they have been 
accepted. Onsite registration on the day of the meeting will be based 
on space availability. If you need special accommodations because of 
disability, please contact Andrea Tan (see FOR FURTHER INFORMATION 
CONTACT) at least 7 days before the meeting.

    Dated: September 14, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-23454 Filed 9-21-12; 8:45 am]
BILLING CODE 4160-01-P