[Federal Register Volume 79, Number 195 (Wednesday, October 8, 2014)]
[Notices]
[Pages 60857-60859]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-23965]


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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; 
Request for Comments

AGENCY: Food and Drug Administration, HHS.

[[Page 60858]]


ACTION: Request for comments.

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SUMMARY: The Food and Drug Administration (FDA) is announcing 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 the patient 
perspective on disease severity and currently available treatments for 
a set of disease areas. FDA is publishing a preliminary list of 
nominated disease areas for consideration in patient-focused drug 
development meetings during fiscal years (FYs) 2016-2017. The public is 
invited to comment on this preliminary list through a public docket.

DATES: Submit either electronic or written comments by December 5, 
2014.

ADDRESSES: 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. All comments should be identified with the docket 
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Pujita Vaidya, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1144, Silver Spring, MD 20993, 301-796-
7641, FAX: 301-796-0684, [email protected]; or Christopher 
Joneckis, Center for Biologics Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7316, Silver 
Spring, MD 20993-0002, 240-402-8083, [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    On July 9, 2012, the President signed into law the Food and Drug 
Administration Safety and Innovation Act (FDASIA) of 2012. 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 FYs 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 the patient perspective on 20 
different disease areas during the course of PDUFA V (FY 2013-2017). 
For each disease area, the Agency is conducting a public meeting to 
discuss the disease, its impact on patients' daily lives, the types of 
treatment benefit that matter most to patients, and patients' 
perspectives on the adequacy of available therapies. These meetings 
include participation of FDA review divisions, the relevant patient 
community, and other interested stakeholders.
    On April 11, 2013, FDA published a Federal Register notice (78 FR 
21613) announcing the disease areas for meetings in FYs 2013-2015, the 
first 3 years of PDUFA V. In selecting the set of disease areas, FDA 
carefully considered the public comments received and the perspectives 
of review divisions at FDA. FDA is initiating a second public process 
for determining the disease areas for FYs 2016-2017. A preliminary list 
of possible disease areas and the criteria used to identify these 
disease areas are published in this document for public comment. FDA 
will consider the public comments received through the public docket 
and publish the set of disease areas for FYs 2016-2017 in a Federal 
Register notice.

II. Disease Area Nomination

    FDA is nominating the following disease areas as potential 
candidates for the focus of the remaining public meetings in FYs 2016-
2017 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.

 Achondroplasia
 Alopecia areata
 Autism
 Autoimmune disorders treated with immune globulins
 Depression
 Diabetic foot infection
 Hereditary angiodema
 Melanoma, specifically unresectable loco-regional disease
 Neurologic disorders treated with immune globulins
 Nontuberculous mycobacterial infections
 Ovarian cancer
 Patients who have received an organ transplant
 Primary humoral immune deficiencies
 Pruritis
 Sarcopenia
 Thrombotic disorders

    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. In 
the series of disease area meetings, the final disease set should 
reflect a range of diseases with respect to disease severity (less 
severe to more severe) and represent a broad range in terms of the size 
of the affected population (e.g., including more prevalent diseases as 
well as rare diseases). FDA requests that when proposing additional 
disease areas for consideration, please describe how you applied the 
identified criteria in making recommendations for additional disease 
areas to consider. The criteria include the following:
     Disease areas that are chronic, symptomatic, or affect 
functioning and activities of daily living;
     disease areas for which aspects of the disease are not 
formally captured in clinical trials;
     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; and
     disease areas that have a severe impact on identifiable 
subpopulations (such as children or the elderly).

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III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). 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, and will be posted to the docket at http://www.regulations.gov.

    Dated: October 2, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-23965 Filed 10-7-14; 8:45 am]
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