81 FR 24818 - Public Meeting on Patient-Focused Drug Development for Patients Who Have Received an Organ Transplant
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 81, Issue 81 (April 27, 2016)
Food and Drug Administration
Federal Register Volume 81, Issue 81 (April 27, 2016)
| Page Range | 24818-24820 | |
| FR Document | 2016-09785 |
[Federal Register Volume 81, Number 81 (Wednesday, April 27, 2016)] [Notices] [Pages 24818-24820] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2016-09785] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-N-2016-1134] Public Meeting on Patient-Focused Drug Development for Patients Who Have Received an Organ Transplant 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 patients who have received an organ transplant. Patient-Focused Drug Development is part of FDA's performance commitments made as part of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patient perspectives on the impact of receiving an organ transplant on daily life and patient views on treatment approaches; the input from this public meeting will help in developing topics for further discussion. FDA is also interested in discussing issues related to scientific challenges in developing drugs to manage organ transplantation. In the afternoon, FDA will hold a workshop and provide information for and gain perspective from patients and patient advocacy organizations, health care providers, academic experts, and industry on various aspects of clinical development of drug products intended to manage organ transplantation. DATES: The public meeting will be held on September 27, 2016, from 9 a.m. to 5 p.m. Please register here for the meeting by September 20, 2016: http://organtransplantpfdd.eventbrite.com. Submit electronic or written comments to the public docket by November 27, 2016. ADDRESSES: The meeting and workshop 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. Participants must enter through Building 1 and undergo security screening. For more information on parking and security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm. You may submit comments as follows: Electronic Submissions Submit electronic comments in the following way:Federal eRulemaking Portal: http://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to http://www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or [[Page 24819]] anyone else's Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on http://www.regulations.gov. If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see ``Written/Paper Submissions'' and ``Instructions''). Written/Paper Submissions Submit written/paper submissions as follows: Mail/Hand delivery/Courier (for written/paper submissions):Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ``Instructions.'' Instructions: All submissions received must include the Docket No. FDA-2016-N-1134 for ``Public Meeting on Patient-Focused Drug Development for Patients Who Have Received an Organ Transplant.'' Received comments will be placed in the docket and, except for those submitted as ``Confidential Submissions,'' publicly viewable at http://www.regulations.gov or at the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday. Confidential Submissions--To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION''. The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on http://www.regulations.gov. Submit both copies to the Division of Dockets Management. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as ``confidential.'' Any information marked as ``confidential'' will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA's posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: http://www.fda.gov/regulatoryinformation/dockets/default.htm. Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to http://www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ``Search'' box and follow the prompts and/or go to the Division of Dockets Management, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. FDA will post the agenda approximately 5 days before the meeting at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm495933.htm. 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 protected]. SUPPLEMENTARY INFORMATION: I. Background on Patient-Focused Drug Development FDA has selected patients who have received an organ transplant as the focus of a public meeting under Patient-Focused Drug Development, an initiative that involves obtaining a better understanding of patient perspectives on the severity of a disease and the available therapies for these conditions. Patient-Focused Drug Development is being conducted to fulfill FDA performance commitments that are part of the reauthorization of the PDUFA under Title I of the Food and Drug Safety and Innovation Act (Pub. L. 112-144). The full set of performance commitments is available at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf. FDA committed to obtain 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 benefit 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 July 2, 2015, FDA published a notice (80 FR 32816) in the Federal Register announcing the disease areas for meetings in fiscal years 2016-2017, final 2 years of PDUFA V time frame. The Agency used several criteria outlined in that 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. In selecting the set of disease areas, FDA carefully considered the public comments received and the perspectives of review divisions at FDA. More information, including the list of disease areas and a general schedule of meetings, is posted at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. II. Public Meeting and Workshop Information A. Purpose and Scope of the Meeting The purpose of this Patient-Focused Drug Development meeting is to obtain input on organ transplantation and current approaches to management of organ transplantation. In 2015, over 25,000 people in the United States received an organ transplant. Organ transplantation requires pharmacologic and non-pharmacologic management before and after receipt. There are FDA-approved therapies used to assist the immune system in responding properly to the transplanted organ. Treatment requires a combination of drugs given for the lifetime of a transplanted organ. FDA is committed to working with all stakeholders to develop safe and effective therapies for affected individuals. The questions that will be asked of patients and patient stakeholders at the meeting are listed in this section, organized by topic. For each topic, a brief initial patient panel discussion will begin the dialogue. This will be followed by a facilitated discussion inviting comments from other patient and patient stakeholder participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through written comments, which can be submitted to the public docket (see ADDRESSES). When submitting comments, if you are commenting on behalf of a child, please indicate that you are doing so and [[Page 24820]] answer the following questions as much as possible from the patient's perspective. Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients 1. What have been the most significant changes in your overall health since you received your transplanted organ? (a) How long has it been since you received your transplant? 2. Focusing on symptoms related to your organ transplant and post- transplant effects, which 1-3 symptoms have the most significant impact on your life? (Examples may include pain, infection, anxiety, etc.) 3. Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your transplant? (Examples of activities may include sleeping through the night, driving, walking/running, exercising, etc.) (a) How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days? (Examples may include limitations on the ability to undertake physically strenuous activities, restrictions on the ability to travel, lack of appetite, fatigue, etc.) 4. How has your experience with your transplanted organ changed over time? Do particular symptoms come and go as your duration of time with a transplanted organ has increased? If so, do you know of anything that makes your symptoms better? Worse? 5. What worries you most about your health post-transplant? Topic 2: Patients' Perspectives on Transplant and Treatment Impacts 1. What are you currently doing to maintain your transplanted organ or treat related health concerns following transplantation? (Examples may include immunosuppressants, antibiotics, antivirals, over-the- counter products, and other therapies including non-drug therapies) (a) How has your post-transplant treatment regimen changed over time, and why? 2. How well does your current treatment regimen manage the most significant symptoms you experience post-transplantation? (a) How well do these treatments improve your ability to do specific activities that are important to you in your daily life? (b) How well have these treatments worked for you as your experiences post-transplant have changed over time? 3. What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples of downsides may include bothersome side effects, need for multiple medications, risk of infection, need for hospitalization, etc.) (a) What are the biggest challenges you face in maintaining your post-transplant treatment regimen? (Examples of challenges may be bothersome side effects, need for multiple medications, etc.) 4. What specific things would you look for in an ideal treatment for managing your transplanted organ? In the afternoon, discussion will be related to scientific topics, with the goal of understanding issues that may affect the development of drugs for the treatment of organ transplantation and identifying topics for future discussion. Discussion topics for the afternoon will include the following: Current treatment considerations, adherence, clinical trial designs, and clinical trial endpoints. B. Meeting Attendance and Participation If you wish to attend this meeting, visit http://organtransplantpfdd.eventbrite.com. Please register by September 20, 2016. If you are unable to attend the meeting in person, you can register to view a live Webcast of the meeting. You will be asked to indicate in your registration if you plan to attend in person or via the Webcast. 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 a disability, please contact Graham Thompson (see FOR FURTHER INFORMATION CONTACT) at least 7 days before the meeting. Patients who are interested in presenting comments as part of the initial panel discussions will be asked to indicate in their registration which topic(s) they wish to address. These patients also must send to [email protected] a brief summary of responses to the topic questions by September 12, 2016. Panelists will be notified of their selection approximately 7 days before the public meeting. We will try to accommodate all patients and patient stakeholders who wish to speak, either through the panel discussion or audience participation; however, the duration of comments may be limited by time constraints. FDA will hold an open public comment period to give the public an opportunity to comment. Registration for open public comment will occur at the registration desk on the day of the meeting and workshop on a first-come, first-served basis. Docket Comments: Regardless of if you attend the public meeting, you can submit electronic or written responses to the questions pertaining to Topics 1 and 2 to the public docket (see ADDRESSES) by November 27, 2016. 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. Transcripts: As soon as a transcript is available, FDA will post it at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm495933.htm. Dated: April 21, 2016. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2016-09785 Filed 4-26-16; 8:45 am] BILLING CODE 4164-01-P
![24818 Federal Register / Vol. 81, No. 81 / Wednesday, April 27, 2016 / Notices
estimates for the implementation and requested extension period is estimated Thus, burden hours for all components
cost study and the impact study to be two years and three months, from are annualized over two years and three
components of the current request. The July 1, 2016 to September 30, 2018. months.
IMPLEMENTATION AND COST STUDY
Total Number of Average Total Total annual
Instrument number of responses per burden hours burden hours burden hours a
respondents respondent per response
Staff interview topic guide .................................................... 120 1 1 120 53
Study MIS to track program participation ............................ 200 468.75 0.0333 3,125 1,390
Impact Study
Introductory script:
Grantee staff ................................................................. 120 9 0.1667 180 80
Program applicants b ..................................................... 1,050 1 0.1667 175 78
Baseline survey .................................................................... 1,000 1 0.5833 583 259
Study MIS to conduct random assignment ......................... 120 9 0.1667 180 80
Protocol for collecting administrative records ...................... 32 1 8 256 114
12 month follow-up survey ................................................... 1,476 1 0.75 1,107 492
a All
burden estimates are annualized over 2.25 years.
b Fivepercent of program applicants are not expected to agree to participate in the study; thus there are 5% more program applicants than
study participants.
Estimated Total Annual Burden DEPARTMENT OF HEALTH AND development of drug products intended
Hours: 2,546. HUMAN SERVICES to manage organ transplantation.
In compliance with the requirements DATES: The public meeting will be held
of Section 506(c)(2)(A) of the Paperwork Food and Drug Administration on September 27, 2016, from 9 a.m. to
Reduction Act of 1995, the 5 p.m. Please register here for the
Administration for Children and [Docket No. FDA–N–2016–1134] meeting by September 20, 2016: http://
Families is soliciting public comment organtransplantpfdd.eventbrite.com.
on the specific aspects of the Public Meeting on Patient-Focused Submit electronic or written comments
information collection described above. Drug Development for Patients Who to the public docket by November 27,
Copies of the proposed collection of Have Received an Organ Transplant 2016.
information can be obtained and
comments may be forwarded by writing AGENCY: Food and Drug Administration, ADDRESSES: The meeting and workshop
to the Administration for Children and HHS. will be held at the FDA White Oak
Families, Office of Planning, Research ACTION: Notice of public meeting; Campus, 10903 New Hampshire Ave.,
and Evaluation, 330 C Street SW., request for comments. Bldg. 31 Conference Center, the Great
Washington, DC 20201. Attn: ACF Room (Rm.1503), Silver Spring, MD
Reports Clearance Officer. Email SUMMARY: The Food and Drug 20993–0002. Participants must enter
address: infocollection@acf.hhs.gov. All Administration (FDA or Agency) is through Building 1 and undergo
requests should be identified by the title announcing a public meeting and an security screening. For more
of the information collection. opportunity for public comment on information on parking and security
The Department specifically requests Patient-Focused Drug Development for procedures, please refer to http://
comments on: (a) Whether the proposed patients who have received an organ www.fda.gov/AboutFDA/
collection of information is necessary transplant. Patient-Focused Drug WorkingatFDA/BuildingsandFacilities/
for the proper performance of the Development is part of FDA’s WhiteOakCampusInformation/
functions of the agency, including performance commitments made as part ucm241740.htm.
whether the information shall have of the fifth authorization of the You may submit comments as
practical utility; (b) the accuracy of the Prescription Drug User Fee Act (PDUFA follows:
agency’s estimate of the burden of the V). The public meeting is intended to
allow FDA to obtain patient Electronic Submissions
proposed collection of information; (c)
the quality, utility, and clarity of the perspectives on the impact of receiving Submit electronic comments in the
information to be collected; and (d) an organ transplant on daily life and following way:
ways to minimize the burden of the patient views on treatment approaches; • Federal eRulemaking Portal: http://
collection of information on the input from this public meeting will www.regulations.gov. Follow the
respondents, including through the use help in developing topics for further instructions for submitting comments.
of automated collection techniques or discussion. FDA is also interested in Comments submitted electronically,
discussing issues related to scientific including attachments, to http://
asabaliauskas on DSK3SPTVN1PROD with NOTICES
other forms of information technology.
Consideration will be given to challenges in developing drugs to www.regulations.gov will be posted to
comments and suggestions submitted manage organ transplantation. In the the docket unchanged. Because your
within 60 days of this publication. afternoon, FDA will hold a workshop comment will be made public, you are
and provide information for and gain solely responsible for ensuring that your
Robert Sargis, perspective from patients and patient comment does not include any
Reports Clearance Officer. advocacy organizations, health care confidential information that you or a
[FR Doc. 2016–09803 Filed 4–26–16; 8:45 am] providers, academic experts, and third party may not wish to be posted,
BILLING CODE 4184–01–P industry on various aspects of clinical such as medical information, your or
VerDate Sep<11>2014 17:29 Apr 26, 2016 Jkt 238001 PO 00000 Frm 00036 Fmt 4703 Sfmt 4703 E:\FR\FM\27APN1.SGM 27APN1](https://thefederalregister.org/doc/81_FR_24899/page/1.svg)
![24820 Federal Register / Vol. 81, No. 81 / Wednesday, April 27, 2016 / Notices
answer the following questions as much and how do they affect your daily life? FDA will hold an open public
as possible from the patient’s (Examples of downsides may include comment period to give the public an
perspective. bothersome side effects, need for opportunity to comment. Registration
multiple medications, risk of infection, for open public comment will occur at
Topic 1: Disease Symptoms and Daily
need for hospitalization, etc.) the registration desk on the day of the
Impacts That Matter Most to Patients (a) What are the biggest challenges meeting and workshop on a first-come,
1. What have been the most you face in maintaining your post- first-served basis.
significant changes in your overall transplant treatment regimen? Docket Comments: Regardless of if
health since you received your (Examples of challenges may be you attend the public meeting, you can
transplanted organ? bothersome side effects, need for submit electronic or written responses
(a) How long has it been since you multiple medications, etc.) to the questions pertaining to Topics 1
received your transplant? 4. What specific things would you and 2 to the public docket (see
2. Focusing on symptoms related to look for in an ideal treatment for ADDRESSES) by November 27, 2016.
your organ transplant and post- managing your transplanted organ? Received comments may be seen in the
transplant effects, which 1–3 symptoms In the afternoon, discussion will be Division of Dockets Management
have the most significant impact on related to scientific topics, with the goal between 9 a.m. and 4 p.m., Monday
your life? (Examples may include pain, of understanding issues that may affect through Friday, and will be posted to
infection, anxiety, etc.) the development of drugs for the the docket at http://
3. Are there specific activities that are treatment of organ transplantation and www.regulations.gov.
important to you but that you cannot do identifying topics for future discussion. Transcripts: As soon as a transcript is
at all or as fully as you would like Discussion topics for the afternoon will available, FDA will post it at http://
because of your transplant? (Examples include the following: Current treatment www.fda.gov/ForIndustry/UserFees/
of activities may include sleeping considerations, adherence, clinical trial PrescriptionDrugUserFee/
through the night, driving, walking/ designs, and clinical trial endpoints. ucm495933.htm.
running, exercising, etc.) B. Meeting Attendance and Dated: April 21, 2016.
(a) How do your symptoms and their Participation Leslie Kux,
negative impacts affect your daily life Associate Commissioner for Policy.
on the best days? On the worst days? If you wish to attend this meeting,
visit http:// [FR Doc. 2016–09785 Filed 4–26–16; 8:45 am]
(Examples may include limitations on
the ability to undertake physically organtransplantpfdd.eventbrite.com. BILLING CODE 4164–01–P
strenuous activities, restrictions on the Please register by September 20, 2016. If
ability to travel, lack of appetite, fatigue, you are unable to attend the meeting in
person, you can register to view a live DEPARTMENT OF HEALTH AND
etc.) HUMAN SERVICES
4. How has your experience with your Webcast of the meeting. You will be
transplanted organ changed over time? asked to indicate in your registration if
you plan to attend in person or via the Food and Drug Administration
Do particular symptoms come and go as
your duration of time with a Webcast. Seating will be limited, so [Docket No. FDA–2013–N–0514]
transplanted organ has increased? If so, early registration is recommended.
Registration is free and will be on a first- Agency Information Collection
do you know of anything that makes Activities; Proposed Collection;
your symptoms better? Worse? come, first-served basis. However, FDA
may limit the number of participants Comment Request; Requests for
5. What worries you most about your
from each organization based on space Clinical Laboratory Improvement
health post-transplant?
limitations. Registrants will receive Amendments Categorization
Topic 2: Patients’ Perspectives on confirmation once they have been AGENCY: Food and Drug Administration,
Transplant and Treatment Impacts accepted. Onsite registration on the day HHS.
1. What are you currently doing to of the meeting will be based on space ACTION: Notice.
maintain your transplanted organ or availability. If you need special
treat related health concerns following accommodations because of a disability, SUMMARY: The Food and Drug
transplantation? (Examples may include please contact Graham Thompson (see Administration (FDA) is announcing an
immunosuppressants, antibiotics, FOR FURTHER INFORMATION CONTACT) at opportunity for public comment on the
antivirals, over-the-counter products, least 7 days before the meeting. proposed collection of certain
and other therapies including non-drug Patients who are interested in information by the Agency. Under the
therapies) presenting comments as part of the Paperwork Reduction Act of 1995 (the
(a) How has your post-transplant initial panel discussions will be asked PRA), Federal Agencies are required to
treatment regimen changed over time, to indicate in their registration which publish notice in the Federal Register
and why? topic(s) they wish to address. These concerning each proposed collection of
2. How well does your current patients also must send to information, including each proposed
treatment regimen manage the most PatientFocused@fda.hhs.gov a brief extension of an existing collection of
significant symptoms you experience summary of responses to the topic information, and to allow 60 days for
post-transplantation? questions by September 12, 2016. public comment in response to the
(a) How well do these treatments Panelists will be notified of their notice. This notice solicits comments on
asabaliauskas on DSK3SPTVN1PROD with NOTICES
improve your ability to do specific selection approximately 7 days before requests for Clinical Laboratory
activities that are important to you in the public meeting. We will try to Improvement Amendments of 1998
your daily life? accommodate all patients and patient (CLIA) categorization of in vitro
(b) How well have these treatments stakeholders who wish to speak, either diagnostic tests when a premarket
worked for you as your experiences through the panel discussion or review is not needed.
post-transplant have changed over time? audience participation; however, the DATES: Submit either electronic or
3. What are the most significant duration of comments may be limited by written comments on the collection of
downsides to your current treatments, time constraints. information by June 27, 2016.
VerDate Sep<11>2014 17:29 Apr 26, 2016 Jkt 238001 PO 00000 Frm 00038 Fmt 4703 Sfmt 4703 E:\FR\FM\27APN1.SGM 27APN1](https://thefederalregister.org/doc/81_FR_24899/page/3.svg)
Document Created: 2018-02-07 13:55:31
Document Modified: 2018-02-07 13:55:31
| 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 27, 2016, from 9 a.m. to 5 p.m. Please register here for the meeting by September 20, 2016: http://organtransplantpfdd.eventbrite.com. Submit electronic or written comments to the public docket by November 27, 2016. | |
| 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 protected] | |
| FR Citation | 81 FR 24818 |
2025 Federal Register | Disclaimer | Privacy Policy
USC | CFR | eCFR