80 FR 38216 - Prescription Drug User Fee Act Patient-Focused Drug Development; Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2016-2017

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 127 (July 2, 2015)

Page Range		38216-38217
FR Document		2015-16359

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.

Federal Register, Volume 80 Issue 127 (Thursday, July 2, 2015)

[Federal Register Volume 80, Number 127 (Thursday, July 2, 2015)]
[Notices]
[Pages 38216-38217]
From the Federal Register Online  [www.thefederalregister.org]
[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]
BILLING CODE 4164-01-P

38216 Federal Register / Vol. 80, No. 127 / Thursday, July 2, 2015 / Notices

manufacturing or distributing listed 2. Cortazzo, J. A. and A. D. Lichtman, ForIndustry/UserFees/
products in response to this notice. All ‘‘Methemoglobinemia: A Review and PrescriptionDrugUserFee/
firms are required to electronically Recommendations for Management.’’ ucm326192.htm. Individual comments
update the listing of their products Journal of Cardiothoracic and Vascular may be viewed at http://
Anesthesia, vol. 28(4), pp. 1055–1059.
under section 510(j) of the FD&C Act to 2014.
www.regulations.gov/
reflect discontinuation of unapproved 3. Lieberthal, A. S., A. E. Carroll, T. #!documentDetail;D=FDA-2012-N-0967-
products covered by this notice (21 CFR Chonmaitree, et al., ‘‘The Diagnosis and 0595 or by visiting the Division of
207.21(b)). Questions on electronic drug Management of Acute Otitis Media,’’ Dockets Management (HFA–305), Food
listing updates should be sent to Pediatrics, vol. 131, pp. e964–e999, and Drug Administration, 5630 Fishers
eDRLS@fda.hhs.gov. In addition to the 2013. Lane, Rm. 1061, Rockville, MD 20852,
required update, firms can also notify 4. Wood, D. N., N. Naas, and C. W. Gregory, between 9 a.m. and 4 p.m., Monday
the Agency of product discontinuation ‘‘Clinical Trials Assessing Ototopical through Friday.
by sending a letter, signed by the firm’s Agents in the Treatment of Pain
Associated with Acute Otitis Media in FOR FURTHER INFORMATION CONTACT:
chief executive officer and fully Graham Thompson, Center for Drug
Children,’’ International Journal of
identifying the discontinued product(s), Otorhinolaryngology, vol. 76, pp. 1229– Evaluation and Research, Food and
including the product NDC number(s), 1335, 2012. Drug Administration, 10903 New
and stating that the manufacturing and/ Hampshire Ave., Bldg. 51, Rm. 1146,
or distribution of the product(s) have Dated: June 26, 2015.
Leslie Kux,
Silver Spring, MD 20993, 301–796–
been discontinued. The letter should be 5003, FAX: 301–847–8443, email:
sent electronically to Kathleen Joyce Associate Commissioner for Policy.
PatientFocused@fda.hhs.gov, or Stephen
(see ADDRESSES). FDA plans to rely on [FR Doc. 2015–16360 Filed 7–1–15; 8:45 am]
Ripley, Center for Biologics Evaluation
its existing records, including its drug BILLING CODE 4164–01–P and Research, Food and Drug
listing records, the results of any Administration, 10903 New Hampshire
subsequent inspections, or other Ave., Bldg. 71, Rm. 7301, Silver Spring,
available information when considering DEPARTMENT OF HEALTH AND MD 20993, 240–402–7911.
enforcement action. HUMAN SERVICES
SUPPLEMENTARY INFORMATION:
VI. Reformulated Products Food and Drug Administration I. Background
FDA cautions firms against [Docket No. FDA–2012–N–0967]
reformulating their products into On July 9, 2012, the President signed
unapproved new drugs without into law the Food and Drug
Prescription Drug User Fee Act
benzocaine; benzocaine and antipyrine; Administration Safety and Innovation
Patient-Focused Drug Development;
benzocaine, antipyrine, and zinc Act (FDASIA) (Pub. L. 112–144). Title I
Announcement of Disease Areas for
acetate; benzocaine, chloroxylenol, and of FDASIA reauthorizes the Prescription
Meetings Conducted in Fiscal Years
hydrocortisone; chloroxylenol and Drug User Fee Act (PDUFA), which
2016–2017
pramoxine; or chloroxylenol, provides FDA with the necessary user
pramoxine, and hydrocortisone and AGENCY: Food and Drug Administration, fee resources to maintain an efficient
marketing them under the same name or HHS. review process for human drug and
substantially the same name (including ACTION: Notice of availability. biologic products. The reauthorization
a new name that contains the old name) of PDUFA includes performance goals
in anticipation of an enforcement action SUMMARY: The Food and Drug and procedures that represent FDA’s
based on this notice. As stated in the Administration (FDA or Agency) is commitments during FYs 2013–2017.
Marketed Unapproved Drugs CPG, FDA announcing the selection of disease These commitments are referred to in
intends to give higher priority to areas to be addressed during fiscal years section 101 of FDASIA and are available
enforcement actions involving (FYs) 2016–2017of its Patient-Focused on the FDA Web site at http://
unapproved drugs that are reformulated Drug Development Initiative. This www.fda.gov/downloads/ForIndustry/
to evade an anticipated FDA initiative is being conducted to fulfill UserFees/PrescriptionDrugUserFee/
enforcement action but have not been FDA’s performance commitments under UCM270412.pdf.
brought into compliance with the law. the fifth authorization of the Section X of these commitments
In addition, reformulated products Prescription Drug User Fee Act (PDUFA relates to enhancing benefit-risk
marketed under a name previously V). This effort provides a more assessments in regulatory decision
identified with a different active systematic approach under PDUFA V making. A key part of regulatory
ingredient have the potential to confuse for obtaining the patients’ perspective decision making is establishing the
healthcare practitioners and harm on disease severity and currently context in which the particular decision
patients. available treatments for a set of disease is made. For purposes of drug marketing
areas. FDA selected these disease areas approval, this includes an
VIII. References based on a careful consideration of the understanding of the severity of the
The following references have been public comments received after treated condition and the adequacy of
placed on display in the Division of publication of a preliminary list of the available therapies. Patients who
Dockets Management (see ADDRESSES) disease areas in the Federal Register on live with a disease have a direct stake
and may be seen by interested persons October 8, 2014. in the outcome of FDA’s decisions and
asabaliauskas on DSK5VPTVN1PROD with NOTICES

between 9 a.m. and 4 p.m., Monday ADDRESSES: The general schedule of FYs are in a unique position to contribute to
through Friday, and are available 2016–2017 Patient-Focused Drug the Agency’s understanding of their
electronically at http:// Development meetings, along with disease.
www.regulations.gov. materials from past meetings (such as FDA has committed to obtaining the
1. Logan, B. K. and A.M. Gordon, ‘‘Case transcripts and webcast recordings) patient perspective on at least 20
Report: Death of an Infant Involving from past meetings, can be found at the disease areas during the course of
Benzocaine,’’ Journal of Forensic Web site for Patient-Focused Drug PDUFA V. For each disease area, the
Sciences, vol. 50, pp. 1486–1488, 2005. Development, http://www.fda.gov/ Agency will conduct a public meeting to

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Federal Register / Vol. 80, No. 127 / Thursday, July 2, 2015 / Notices 38217

discuss the disease, its impact on • Disease areas for which aspects of prepare for these upcoming meetings,
patients’ daily lives, the types of the disease are not formally captured in and information on how stakeholders
treatment benefit that matter most to clinical trials; may leverage the Patient-Focused Drug
patients, and patients’ perspectives on • Disease areas for which there are Development Initiative to generate input
the adequacy of available therapies. currently no therapies or very few on disease areas not addressed through
These meetings include participation of therapies, or the available therapies do the Patient-Focused Drug Development
FDA review divisions, the relevant not directly affect how a patient feels, PDUFA V commitment. The Web site
patient community, and other interested functions, or survives; and will be updated as new information
stakeholders. • Disease areas that have a severe becomes available.
impact on identifiable subpopulations Dated: June 26, 2015.
II. Disease Area Selection
(such as children or the elderly). Leslie Kux,
On October 8, 2014, FDA published a FDA’s selection also reflects disease
Associate Commissioner for Policy.
Federal Register notice (79 FR 60857) areas from FDA review divisions that
[FR Doc. 2015–16359 Filed 7–1–15; 8:45 am]
that requested public comment on were not covered by the meetings held
during FYs 2013–15. For its FYs 2016– BILLING CODE 4164–01–P
potential disease areas to be addressed
in FYs 2016–2017. In that notice, based 2017 list of disease areas, FDA has
on several criteria listed, FDA identified added a broad range of diseases based
upon disease severity (less severe to DEPARTMENT OF HEALTH AND
16 disease areas as potential candidates HUMAN SERVICES
for remaining public meetings and more severe) and upon the size of the
invited public comment on the affected population (rare diseases to [Document Identifier: HHS–OS–0937–0166–
preliminary list and on disease areas more prevalent diseases). FDA has 60D]
that were not listed. identified the following diseases to be
the focus of meetings scheduled in FYs Agency Information Collection
Following publication of the notice, 2016–2017: Activities; Proposed Collection; Public
almost 2,700 comments addressing over Comment Request
50 disease areas were submitted by • Alopecia areata
patients, patient advocates and • Autism AGENCY: Office of Population Affairs,
advocacy groups, caregivers, healthcare • Hereditary angioedema Office of the Assistant Secretary for
providers, professional societies, • Non-tuberculous mycobacterial Health, HHS.
scientific and academic experts, infections
ACTION: Notice.
pharmaceutical companies, and others. • Patients who have received an organ
The majority of comments received transplant SUMMARY: In compliance with section
• Psoriasis 3506(c)(2)(A) of the Paperwork
were submitted by individual patients.
• Neuropathic pain associated with Reduction Act of 1995, the Office of the
The comments focused generally on
peripheral neuropathy Secretary (OS), Department of Health
nominating individual disease areas or
• Sarcopenia and Human Services, announces plans
groups of disease areas to be addressed
and on providing general suggestions for A schedule of the meetings planned to submit an Information Collection
the Patient-Focused Drug Development can be found at the FDA Patient- Request (ICR), described below, to the
Initiative. The comments received also Focused Drug Development Web site, Office of Management and Budget
discussed the impact of these which is described in section III of this (OMB). The ICR is for extending the use
nominated diseases on the patients’ notice. The Agency recognizes that there of the approved information collection
daily lives, the symptoms that were are many more disease areas than can be assigned OMB control number 0937–
most concerning to patients, and the addressed in the planned FDA meetings 0166, which expires on October 31,
nature of (or lack of) specific treatments under the formal PDUFA V 2015. Prior to submitting the ICR to
for these diseases. The majority of commitment, and FDA will seek other OMB, OS seeks comments from the
comments received concerned lewy opportunities to gather public input on public regarding the burden estimate,
body dementia, frontotemporal lobar disease areas not addressed through this below, or any other aspect of the ICR.
degeneration, and neuropathies. Other Patient-Focused Drug Development DATES: Comments on the ICR must be
disease areas, such as hereditary Initiative. FDA encourages stakeholders received on or before August 31, 2015.
angioedema, dystonia, to identify and organize patient-focused
ADDRESSES: Submit your comments to
temporomandibular disorders, lupus, collaborations to generate public input
Information.CollectionClearance@
alopecia areata, chronic lymphocytic on other disease areas using the process
hhs.gov or by calling (202) 690–6162.
leukemia, trigeminal neuralgia, and established through this Patient-
Focused Drug Development Initiative as FOR FURTHER INFORMATION CONTACT:
arachnoiditis, also received a significant Information Collection Clearance staff,
number of comments. a model. Information on additional
opportunities for gathering patient input Information.CollectionClearance@
In selecting the disease areas of focus can be found on the Patient-Focused hhs.gov or (202) 690–6162.
for the Patient-Focused Drug Drug Development Web site. SUPPLEMENTARY INFORMATION: When
Development Initiative of FYs 2016– submitting comments or requesting
2017, FDA carefully considered the III. Patient-Focused Drug Development information, please include the
valuable public comments received, the Web Site document identifier HHS–OS–0937–
asabaliauskas on DSK5VPTVN1PROD with NOTICES

perspectives of reviewing divisions at FDA’s Web site on Patient-Focused 0166–60D for reference.
FDA, and the following selection Drug Development is available online at Information Collection Request Title:
criteria, which were published in the http://www.fda.gov/ForIndustry/ HHS 42 CFR part 50, subpart B;
October 8, 2014, Federal Register UserFees/PrescriptionDrugUserFee/ Sterilization of Persons in Federally
notice: ucm326192.htm. This Web site contains Assisted Family Planning Projects—
• Disease areas that are chronic, the general schedule of upcoming OMB No. 0937–0166–Extension—
symptomatic, or affect functioning and meetings for FYs 2016–2017, OASH, Office of Population Affairs—
activities of daily living; information on how stakeholders can Office of Family Planning

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Document Created: 2015-12-15 13:15:13
Document Modified: 2015-12-15 13:15:13

Category		Regulatory Information
Collection		Federal Register
sudoc Class		AE 2.7: GS 4.107: AE 2.106:
Publisher		Office of the Federal Register, National Archives and Records Administration
Section		Notices
Action		Notice of availability.
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.
FR Citation		80 FR 38216

80 FR 38216 - Prescription Drug User Fee Act Patient-Focused Drug Development; Announcement of Disease Areas for Meetings Conducted in Fiscal Years 2016-2017

DEPARTMENT OF HEALTH AND HUMAN SERVICESFood and Drug Administration

Federal Register Volume 80, Issue 127 (July 2, 2015)

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration