[Federal Register Volume 80, Number 127 (Thursday, July 2, 2015)]
[Notices]
[Pages 38216-38217]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-16359]


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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; 
Announcement of Disease Areas for Meetings Conducted in Fiscal Years 
2016-2017

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the selection of disease areas to be addressed during fiscal years 
(FYs) 2016-2017of its Patient-Focused Drug Development Initiative. This 
initiative is being conducted to fulfill FDA's performance commitments 
under the fifth authorization of the Prescription Drug User Fee Act 
(PDUFA V). This effort provides a more systematic approach under PDUFA 
V for obtaining the patients' perspective on disease severity and 
currently available treatments for a set of disease areas. FDA selected 
these disease areas based on a careful consideration of the public 
comments received after publication of a preliminary list of disease 
areas in the Federal Register on October 8, 2014.

ADDRESSES: The general schedule of FYs 2016-2017 Patient-Focused Drug 
Development meetings, along with materials from past meetings (such as 
transcripts and webcast recordings) from past meetings, can be found at 
the Web site for Patient-Focused Drug Development, http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. Individual 
comments may be viewed at http://www.regulations.gov/#!documentDetail;D=FDA-2012-N-0967-0595 or by visiting the Division of 
Dockets Management (HFA-305), Food and Drug Administration, 5630 
Fishers Lane, Rm. 1061, Rockville, MD 20852, between 9 a.m. and 4 p.m., 
Monday through Friday.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993, 301-796-
5003, FAX: 301-847-8443, email: [email protected], or Stephen 
Ripley, Center for Biologics Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver 
Spring, MD 20993, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    On July 9, 2012, the President signed into law the Food and Drug 
Administration Safety and Innovation Act (FDASIA) (Pub. L. 112-144). 
Title I of FDASIA reauthorizes the Prescription Drug User Fee Act 
(PDUFA), which 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 
assessments in regulatory decision making. A key part of regulatory 
decision making is establishing the context in which the particular 
decision is made. For purposes of drug marketing approval, this 
includes an understanding of the severity of the treated condition and 
the adequacy of the available therapies. Patients who live with a 
disease have a direct stake in the outcome of FDA's decisions and are 
in a unique position to contribute to the Agency's understanding of 
their disease.
    FDA has committed to obtaining the patient perspective on at least 
20 disease areas during the course of PDUFA V. For each disease area, 
the Agency will conduct a public meeting to

[[Page 38217]]

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.

II. Disease Area Selection

    On October 8, 2014, FDA published a Federal Register notice (79 FR 
60857) that requested public comment on potential disease areas to be 
addressed in FYs 2016-2017. In that notice, based on several criteria 
listed, FDA identified 16 disease areas as potential candidates for 
remaining public meetings and invited public comment on the preliminary 
list and on disease areas that were not listed.
    Following publication of the notice, almost 2,700 comments 
addressing over 50 disease areas were submitted by patients, patient 
advocates and advocacy groups, caregivers, healthcare providers, 
professional societies, scientific and academic experts, pharmaceutical 
companies, and others. The majority of comments received were submitted 
by individual patients. The comments focused generally on nominating 
individual disease areas or groups of disease areas to be addressed and 
on providing general suggestions for the Patient-Focused Drug 
Development Initiative. The comments received also discussed the impact 
of these nominated diseases on the patients' daily lives, the symptoms 
that were most concerning to patients, and the nature of (or lack of) 
specific treatments for these diseases. The majority of comments 
received concerned lewy body dementia, frontotemporal lobar 
degeneration, and neuropathies. Other disease areas, such as hereditary 
angioedema, dystonia, temporomandibular disorders, lupus, alopecia 
areata, chronic lymphocytic leukemia, trigeminal neuralgia, and 
arachnoiditis, also received a significant number of comments.
    In selecting the disease areas of focus for the Patient-Focused 
Drug Development Initiative of FYs 2016-2017, FDA carefully considered 
the valuable public comments received, the perspectives of reviewing 
divisions at FDA, and the following selection criteria, which were 
published in the October 8, 2014, Federal Register notice:
     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).
    FDA's selection also reflects disease areas from FDA review 
divisions that were not covered by the meetings held during FYs 2013-
15. For its FYs 2016-2017 list of disease areas, FDA has added a broad 
range of diseases based upon disease severity (less severe to more 
severe) and upon the size of the affected population (rare diseases to 
more prevalent diseases). FDA has identified the following diseases to 
be the focus of meetings scheduled in FYs 2016-2017:

 Alopecia areata
 Autism
 Hereditary angioedema
 Non-tuberculous mycobacterial infections
 Patients who have received an organ transplant
 Psoriasis
 Neuropathic pain associated with peripheral neuropathy
 Sarcopenia

    A schedule of the meetings planned can be found at the FDA Patient-
Focused Drug Development Web site, which is described in section III of 
this notice. The Agency recognizes that there are many more disease 
areas than can be addressed in the planned FDA meetings under the 
formal PDUFA V commitment, and FDA will seek other opportunities to 
gather public input on disease areas not addressed through this 
Patient-Focused Drug Development Initiative. FDA encourages 
stakeholders to identify and organize patient-focused collaborations to 
generate public input on other disease areas using the process 
established through this Patient-Focused Drug Development Initiative as 
a model. Information on additional opportunities for gathering patient 
input can be found on the Patient-Focused Drug Development Web site.

III. Patient-Focused Drug Development Web Site

    FDA's Web site on Patient-Focused Drug Development is available 
online at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. This Web site contains the 
general schedule of upcoming meetings for FYs 2016-2017, information on 
how stakeholders can prepare for these upcoming meetings, and 
information on how stakeholders may leverage the Patient-Focused Drug 
Development Initiative to generate input on disease areas not addressed 
through the Patient-Focused Drug Development PDUFA V commitment. The 
Web site will be updated as new information becomes available.

    Dated: June 26, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-16359 Filed 7-1-15; 8:45 am]
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