80 FR 31048 - Patient-Focused Drug Development for Alpha-1 Antitrypsin Deficiency; Public Meeting
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 104 (June 1, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 104 (June 1, 2015)
| Page Range | 31048-31050 | |
| FR Document | 2015-13063 |
[Federal Register Volume 80, Number 104 (Monday, June 1, 2015)] [Notices] [Pages 31048-31050] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2015-13063] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2015-N-1798] Patient-Focused Drug Development for Alpha-1 Antitrypsin Deficiency; Public Meeting AGENCY: Food and Drug Administration, HHS. ACTION: Notice of public meeting; request for comments. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing a public meeting and an opportunity for public comment on Patient- Focused Drug Development for Alpha-1 Antitrypsin Deficiency (AATD). Patient-Focused Drug Development is an FDA performance commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to provide FDA with patients' perspectives on the impact on daily life of AATD. FDA also is seeking patients' perspectives on the available therapies for this disorder. DATES: The public meeting will be held on September 29, 2015, from 9 a.m. to 3:30 p.m. Registration to attend the meeting must be received by September 15, 2015. Registration from those individuals interested in presenting comments as part of the panel discussions should be received by July 31, 2015. See the SUPPLEMENTARY INFORMATION section for instructions on how to register for the meeting. Submit either electronic or written comments by November 30, 2015. ADDRESSES: The public meeting will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the Great Room (rm. 1503), Silver Spring, MD 20993. Entrance for public meeting participants (non-FDA employees) is through Building 1, where routine security checks 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 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: Barbara Kass, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, rm. 1125, Silver Spring, MD 20993, 240-402- 6887, FAX: 301-595-1243, email: PatientFocused_CBER@fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background on Patient-Focused Drug Development FDA has selected AATD as the focus of a public meeting under the Patient-Focused Drug Development initiative. This initiative involves obtaining a better understanding of patients' perspectives on the challenges posed by AATD and the impact of current therapies for this condition. The Patient-Focused Drug Development initiative is being conducted to fulfill FDA performance commitments that are part of the PDUFA reauthorization under Title I of the Food and Drug Administration Safety and Innovation Act (Pub. L. 112-144). The full set of performance commitments is available on the FDA Web site at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf. FDA has committed to obtaining the patient perspective on 20 disease areas during the course of PDUFA V. For each disease area, the Agency will conduct a public meeting to discuss the disease and its impact on patients' daily lives, the types of treatment benefits that matter most to patients, and patients' perspectives on the adequacy of the available therapies. These meetings will include participation of FDA review divisions, the relevant patient communities, and other interested stakeholders. On April 11, 2013, FDA published a notice in the Federal Register (78 FR 21613) that announced the disease areas for meetings in fiscal years (FY) 2013-2015, the first 3 years of the 5-year PDUFA V timeframe. The Agency used several criteria outlined in the April 11, 2013, notice to develop the list of disease areas. FDA obtained public comment on the Agency's proposed criteria and potential disease areas through a public docket and a public meeting that was convened on October 25, 2012. In selecting the set of disease areas, FDA carefully considered the public comments received and the perspectives of review divisions at FDA. FDA has initiated a second public process for determining the disease areas for meetings in FY 2016-2017 and published a notice in the Federal Register on October 8, 2014 (79 FR 60857). More information, including the list of disease areas and a general schedule of meetings, is posted on FDA's Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. II. Purpose and Scope of the Meeting The purpose of this Patient-Focused Drug Development meeting is to obtain input on the symptoms and other impacts that matter most to patients with AATD. FDA also intends to seek patients' perspectives on current approaches to treating this disorder. FDA expects that this information will come directly from patients, caregivers, and patient advocates. Individuals with AATD have low serum levels of Alpha-1-Antitrypsin (AAT, also known as Alpha-1 proteinase inhibitor (A1-PI)) and increased risks of developing a form of chronic obstructive lung disease called emphysema and, less frequently, liver disease. Some AATD patients with emphysema have symptoms of asthma. There are different genetic forms of the disease, but even among people with the same genetic form and similar levels of AAT in their blood, there is tremendous diversity in [[Page 31049]] clinical severity. A substantial percentage of individuals with severe AATD never develop symptomatic lung disease during their lifetimes. Others may develop the first signs and symptoms of lung disease between the ages of approximately 25 and 50 years, or older. Affected individuals often develop emphysema, which is a lung disease caused by damage to the small air sacs in the lung. Progression of emphysema in AATD may lead to respiratory failure, a need for lung transplantation, and eventually death. The only specific medication approved for raising the blood levels of AAT in severe AATD patients with emphysema is weekly intravenous treatment with A1-PI (Human) purified from human blood plasma. Severe AATD patients may also develop liver disease as infants, during childhood, or as adults. There is wide variation in the severity of liver disease among affected patients. Currently, no specific therapy for AATD-related liver disease is available other than liver transplantation, so the focus in these patients is on the prevention and management of other types of liver damage. The questions that will be asked of patients and patient caregivers at the meeting are provided in this document. The meeting will be divided into two main topics: (1) The effects of Alpha-1 Antitrypsin Deficiency that matter most to you, and (2) perspectives on current approaches to treatment. For each topic, a brief patient panel discussion will begin the dialogue. This will be followed by a facilitated discussion inviting comments from other patient and patient caregiver participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through electronic or written comments, which can be submitted to the Division of Dockets Management (see ADDRESSES). For context, please indicate if you are commenting as a patient with AATD or on behalf of a child or loved one. Topic 1: The effects of Alpha-1 Antitrypsin Deficiency that matter most to you.Of all of the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life? (Examples may include: (a) For lung disease: shortness of breath during specific activities or at rest, chronic cough, wheezing, weight loss, exacerbations of particular symptoms; (b) For liver disease: abdominal pain, loss of appetite, height and weight concerns.) Are there specific activities that are important to you, but that you cannot do at all, or as well as you would like, because of your condition? Please describe, using specific examples. (Examples may include: Participating in physical activities, attending work/school and family/social activities.) How have your condition and its symptoms changed over time? What worries you most about your condition? Topic 2: Perspectives on current approaches to treatment. What are you currently doing to treat your condition or its symptoms? (Examples may include: (a) For lung disease: Inhaled bronchodilators, inhaled corticosteroids, intravenous augmentation therapy with A1-PI (Human) on a regular or intermittent basis; (b) For liver disease: Ursodiol.) [cir] How well do these treatments work for you? [cir] What are the most significant disadvantages or complications of your current treatments, and how do they affect your daily life? [cir] How has your treatment changed over time and why? [cir] What aspects of your condition are not improved by your current treatment regimen? [cir] What treatment has had the most positive impact on your life? If you could create your ideal treatment, what would it do for you (i.e., what specific things would you look for in an ideal treatment)? If you had the opportunity to consider participating in a clinical trial studying experimental treatments, what things would you consider when deciding whether or not to participate? III. Attendance and Registration If you wish to attend this meeting, visit https://www.eventbrite.com/e/public-meeting-on-patient-focused-drug-development-for-alpha-1-antitrypsin-deficiency-tickets-15617092143. If you do not have access to the Internet, you may mail or fax your registration information (including name, title, affiliation, address, email address, telephone, and fax numbers) to Barbara Kass (see FOR FURTHER INFORMATION CONTACT) by September 15, 2015. There is no registration fee for the public meeting. Early registration is recommended because seating is limited. FDA may limit the number of participants from each organization based on space limitations. Registrants will receive confirmation once they have been accepted. Registration on the day of the public meeting will be provided on a space available basis beginning at 8 a.m. Those who are unable to attend the meeting in person can register to view a live Web cast of the meeting. You will be asked to indicate in your registration if you plan to attend in person or via the Web cast. If you need special accommodations because of disability, please contact Barbara Kass at least 7 days in advance. FDA will post the agenda approximately 5 days before the meeting at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/MeetingsMeetingsConferences/ucm435242.htm. IV. Comments Patients and patient caregivers who are interested in presenting comments as part of the panel discussions should register by July 31, 2015. You will be asked to indicate in your registration which topic(s) you wish to address and to send a brief summary of responses to the topic questions to PatientFocused_CBER@fda.hhs.gov by July 31, 2015. Panelists will be notified of their selection soon after August 28, 2015. FDA will try to accommodate all patients and patient caregivers who wish to speak, either through the panel discussion or audience participation; however, the duration of comments may be limited by time constraints. Regardless of attendance at the public meeting, interested persons may submit either electronic or written responses to any or all of the questions pertaining to topics 1 and 2 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. To ensure consideration, submit comments by November 30, 2015. 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. V. Transcripts Please be advised that as soon as a transcript is available, it will be accessible at http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/ucm435242.htm and at http://www.regulations.gov. It may also be viewed at the Division of Dockets Management (see ADDRESSES). A transcript will also be available in either hardcopy or on CD-ROM, after submission of a Freedom of Information request. Written requests are to be sent [[Page 31050]] to the Division of Freedom of Information (ELEM-1029), Food and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857. Dated: May 26, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015-13063 Filed 5-29-15; 8:45 am] BILLING CODE 4164-01-P
![31048 Federal Register / Vol. 80, No. 104 / Monday, June 1, 2015 / Notices
TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN—Continued
Number of Average
Number of Total annual
FDA center/21 CFR section/FDA form responses per burden per Total
respondents responses
respondent response
All Centers Form FDA 3500B ..................................... 13,750 1 13,750 0.46 (30 minutes) ... 6,325
Total ...................................................................... ........................ ........................ ........................ ................................ 909,395
Dated: May 27, 2015. Building 1, where routine security include participation of FDA review
Leslie Kux, checks will be performed. For parking divisions, the relevant patient
Associate Commissioner for Policy. and security information, please refer to communities, and other interested
[FR Doc. 2015–13102 Filed 5–29–15; 8:45 am] http://www.fda.gov/AboutFDA/ stakeholders.
BILLING CODE 4164–01–P
WorkingatFDA/BuildingsandFacilities/ On April 11, 2013, FDA published a
WhiteOakCampusInformation/ notice in the Federal Register (78 FR
ucm241740.htm. 21613) that announced the disease areas
DEPARTMENT OF HEALTH AND Submit electronic comments to for meetings in fiscal years (FY) 2013–
HUMAN SERVICES http://www.regulations.gov. Submit 2015, the first 3 years of the 5-year
written comments to the Division of PDUFA V timeframe. The Agency used
Food and Drug Administration Dockets Management (HFA–305), Food several criteria outlined in the April 11,
and Drug Administration, 5630 Fishers 2013, notice to develop the list of
[Docket No. FDA–2015–N–1798]
Lane, rm. 1061, Rockville MD 20852. disease areas. FDA obtained public
Patient-Focused Drug Development for All comments should be identified with comment on the Agency’s proposed
Alpha-1 Antitrypsin Deficiency; Public the docket number found in brackets in criteria and potential disease areas
Meeting the heading of this document. through a public docket and a public
FOR FURTHER INFORMATION CONTACT: meeting that was convened on October
AGENCY: Food and Drug Administration, Barbara Kass, Center for Biologics 25, 2012. In selecting the set of disease
HHS. Evaluation and Research, Food and areas, FDA carefully considered the
ACTION: Notice of public meeting; Drug Administration, 10903 New public comments received and the
request for comments. Hampshire Ave., Bldg. 71, rm. 1125, perspectives of review divisions at FDA.
Silver Spring, MD 20993, 240–402– FDA has initiated a second public
SUMMARY: The Food and Drug process for determining the disease
6887, FAX: 301–595–1243, email:
Administration (FDA or Agency) is areas for meetings in FY 2016–2017 and
PatientFocused_CBER@fda.hhs.gov.
announcing a public meeting and an published a notice in the Federal
SUPPLEMENTARY INFORMATION:
opportunity for public comment on Register on October 8, 2014 (79 FR
Patient-Focused Drug Development for I. Background on Patient-Focused Drug 60857). More information, including the
Alpha-1 Antitrypsin Deficiency (AATD). Development list of disease areas and a general
Patient-Focused Drug Development is FDA has selected AATD as the focus schedule of meetings, is posted on
an FDA performance commitment under of a public meeting under the Patient- FDA’s Web site at http://www.fda.gov/
the fifth authorization of the Focused Drug Development initiative. ForIndustry/UserFees/
Prescription Drug User Fee Act (PDUFA This initiative involves obtaining a PrescriptionDrugUserFee/
V). The public meeting is intended to better understanding of patients’ ucm326192.htm.
provide FDA with patients’ perspectives perspectives on the challenges posed by
on the impact on daily life of AATD. AATD and the impact of current II. Purpose and Scope of the Meeting
FDA also is seeking patients’ therapies for this condition. The Patient- The purpose of this Patient-Focused
perspectives on the available therapies Focused Drug Development initiative is Drug Development meeting is to obtain
for this disorder. being conducted to fulfill FDA input on the symptoms and other
DATES: The public meeting will be held performance commitments that are part impacts that matter most to patients
on September 29, 2015, from 9 a.m. to of the PDUFA reauthorization under with AATD. FDA also intends to seek
3:30 p.m. Registration to attend the Title I of the Food and Drug patients’ perspectives on current
meeting must be received by September Administration Safety and Innovation approaches to treating this disorder.
15, 2015. Registration from those Act (Pub. L. 112–144). The full set of FDA expects that this information will
individuals interested in presenting performance commitments is available come directly from patients, caregivers,
comments as part of the panel on the FDA Web site at http:// and patient advocates.
discussions should be received by July www.fda.gov/downloads/forindustry/ Individuals with AATD have low
31, 2015. See the SUPPLEMENTARY userfees/prescriptiondruguserfee/ serum levels of Alpha-1-Antitrypsin
INFORMATION section for instructions on ucm270412.pdf. (AAT, also known as Alpha-1 proteinase
how to register for the meeting. Submit FDA has committed to obtaining the inhibitor (A1–PI)) and increased risks of
either electronic or written comments patient perspective on 20 disease areas developing a form of chronic obstructive
by November 30, 2015. during the course of PDUFA V. For each lung disease called emphysema and,
Lhorne on DSK2VPTVN1PROD with NOTICES
ADDRESSES: The public meeting will be disease area, the Agency will conduct a less frequently, liver disease. Some
held at the FDA White Oak Campus, public meeting to discuss the disease AATD patients with emphysema have
10903 New Hampshire Ave., Building and its impact on patients’ daily lives, symptoms of asthma. There are different
31 Conference Center, the Great Room the types of treatment benefits that genetic forms of the disease, but even
(rm. 1503), Silver Spring, MD 20993. matter most to patients, and patients’ among people with the same genetic
Entrance for public meeting participants perspectives on the adequacy of the form and similar levels of AAT in their
(non-FDA employees) is through available therapies. These meetings will blood, there is tremendous diversity in
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![31050 Federal Register / Vol. 80, No. 104 / Monday, June 1, 2015 / Notices
to the Division of Freedom of the Internet at http://www.reginfo.gov/ Development, Center for Biologics
Information (ELEM–1029), Food and public/do/PRAMain. Evaluation and Research (CBER), Food
Drug Administration, 12420 Parklawn Dated: May 21, 2015. and Drug Administration, 10903 New
Dr., Element Bldg., Rockville, MD Leslie Kux,
Hampshire Ave., Bldg. 71, Rm. 3128,
20857. Silver Spring, MD 20993–0002. Send
Associate Commissioner for Policy.
Dated: May 26, 2015. one self-addressed adhesive label to
[FR Doc. 2015–13064 Filed 5–29–15; 8:45 am]
Leslie Kux, assist the office in processing your
BILLING CODE 4164–01–P requests. The draft guidance may also be
Associate Commissioner for Policy.
obtained by mail by calling CBER at 1–
[FR Doc. 2015–13063 Filed 5–29–15; 8:45 am]
800–835–4709 or 240–402–7800. See
BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND
the SUPPLEMENTARY INFORMATION section
HUMAN SERVICES
for electronic access to the draft
Food and Drug Administration guidance document.
DEPARTMENT OF HEALTH AND
Submit electronic comments on the
HUMAN SERVICES [Docket No. FDA–2015–D–1659] draft guidance to http://
Food and Drug Administration www.regulations.gov. Submit written
Established Conditions: Reportable comments to the Division of Dockets
[Docket No. FDA–2005–N–0161] Chemistry, Manufacturing, and Management (HFA–305), Food and Drug
Controls Changes for Approved Drug Administration, 5630 Fishers Lane, Rm.
Agency Information Collection and Biologic Products; Draft Guidance 1061, Rockville, MD 20852.
Activities; Announcement of Office of for Industry; Availability
Management and Budget Approval; FOR FURTHER INFORMATION CONTACT:
Current Good Manufacturing Practices AGENCY: Food and Drug Administration, Ashley Boam, Center for Drug
for Blood and Related Regulations for HHS. Evaluation and Research, Food and
and Blood Components; and ACTION: Notice. Drug Administration, 10903 New
Requirements for Donor Testing, Hampshire Ave., Silver Spring, MD
SUMMARY: The Food and Drug 20993, 301–796–2400; or Stephen
Donor Notification, and ‘‘Lookback’’ Administration (FDA) is announcing the Ripley, Center for Biologics Evaluation
AGENCY: Food and Drug Administration, availability of a draft guidance for and Research, Food and Drug
HHS. industry entitled ‘‘Established Administration, 10903 New Hampshire
ACTION: Notice. Conditions: Reportable CMC Changes Ave., Bldg. 71, Rm. 7301, Silver Spring,
for Approved Drug and Biologic MD 20993, 240–402–7911.
SUMMARY: The Food and Drug Products.’’ The purpose of this guidance SUPPLEMENTARY INFORMATION:
Administration (FDA) is announcing is to provide applicants of new drug
that a collection of information entitled, applications, abbreviated new drug I. Background
‘‘Current Good Manufacturing Practices applications, and biologic license FDA is announcing the availability of
for Blood and Related Regulations for applications with FDA’s current a draft guidance for industry entitled
and Blood Components; and thinking on established conditions (i.e., ‘‘Established Conditions: Reportable
Requirements for Donor Testing, Donor the chemistry, manufacturing, and CMC Changes for Approved Drug and
Notification, and ‘Lookback’ ’’ has been controls (CMC) information in a Biologic Products.’’ The current
approved by the Office of Management submission that would require reporting regulations for drugs and biologics
and Budget (OMB) under the Paperwork to FDA if changed for approved drug require applicants with approved drug
Reduction Act of 1995. and biologic products, per the current or biologic products to notify FDA about
FOR FURTHER INFORMATION CONTACT: FDA regulations). This guidance also each change in each condition
PRA Staff, Office of Operations, Food describes those sections of a common established in the approved application
and Drug Administration, 8455 technical document formatted beyond the variations already provided
Colesville Rd., COLE–14526, Silver application that typically contain for in the application (see 21 CFR
Spring, MD 20993–0002, PRAStaff@ information that meets the definition of 314.70) or each change in the product,
fda.hhs.gov. established conditions, and provides production process, quality controls,
SUPPLEMENTARY INFORMATION: On March considerations for managing changes to equipment, facilities, responsible
11, 2015, the Agency submitted a established conditions over the life personnel, or labeling established in the
proposed collection of information cycle of an approved product. approved license application (see 21
entitled, ‘‘Current Good Manufacturing DATES: Although you can comment on CFR 601.12). FDA guidance documents
Practices for Blood and Related any guidance at any time (see 21 CFR clarify the recommended reporting
Regulations for and Blood Components; 10.115(g)(5)), to ensure that the Agency mechanism (i.e., supplement, annual
and Requirements for Donor Testing, considers your comment on this draft report) for postapproval CMC changes.
Donor Notification, and ‘Lookback’ ’’ to guidance before it begins work on the This draft guidance has been developed
OMB for review and clearance under 44 final version of the guidance, submit to address the lack of clarity with
U.S.C. 3507. An Agency may not either electronic or written comments respect to what CMC information in an
conduct or sponsor, and a person is not on the draft guidance by July 31, 2015. application constitutes an established
required to respond to, a collection of ADDRESSES: Submit written requests for condition.
information unless it displays a single copies of the draft guidance to the A better understanding of which
Lhorne on DSK2VPTVN1PROD with NOTICES
currently valid OMB control number. Division of Drug Information, Center for elements of the CMC information
OMB has now approved the information Drug Evaluation and Research, Food constitute established conditions to
collection and has assigned OMB and Drug Administration, 10001 New FDA and where in an application these
control number 0910–0116. The Hampshire Ave., Hillandale Building, are generally expected to be described
approval expires on May 31, 2018. A 4th Floor, Silver Spring, MD 20993– will allow for a more effective
copy of the supporting statement for this 0002; or to the Office of postapproval submission strategy (e.g.,
information collection is available on Communication, Outreach and effective use of risk management
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Document Created: 2015-12-15 15:18:36
Document Modified: 2015-12-15 15:18:36
| 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 public meeting; request for comments. | |
| Dates | The public meeting will be held on September 29, 2015, from 9 a.m. to 3:30 p.m. Registration to attend the meeting must be received by September 15, 2015. Registration from those individuals interested in presenting comments as part of the panel discussions should be received by July 31, 2015. See the SUPPLEMENTARY INFORMATION section for instructions on how to register for the meeting. Submit either electronic or written comments by November 30, 2015. | |
| Contact | Barbara Kass, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, rm. 1125, Silver Spring, MD 20993, 240-402- 6887, FAX: 301-595-1243, email: [email protected] | |
| FR Citation | 80 FR 31048 |
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