Sickle Cell Disease Public Meeting on Patient-Focused Drug Development, 66747-66748 [2013-26548]

Download as PDF Federal Register / Vol. 78, No. 215 / Wednesday, November 6, 2013 / Notices Dated: October 30, 2013. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2013–26547 Filed 11–5–13; 8:45 am] BILLING CODE 4160–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–N–2013–1328] Sickle Cell Disease Public Meeting on Patient-Focused Drug Development AGENCY: Food and Drug Administration, HHS. Notice of public meeting; request for comments. ACTION: The Food and Drug Administration (FDA) is announcing a public meeting and an opportunity for public comment on Patient-Focused Drug Development for sickle cell disease. Patient-Focused Drug Development is part of FDA’s performance commitments in the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The public meeting is intended to allow FDA to obtain patients’ perspectives on the impact of sickle cell disease on daily life and on available therapies for sickle cell disease. DATES: The public meeting will be held on February 7, 2014; from 10 a.m. to 4 p.m. Registration to attend the meeting must be received by January 27, 2014. See the SUPPLEMENTARY INFORMATION section for information on how to register for the meeting. Submit electronic or written comments by April 8, 2014. ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, in Sections B and C of the Great Room (Rm. 1503), Silver Spring, MD 20993. Entrance for the public meeting participants is through Building 1, where routine security check procedures will be performed. For more information on parking and security procedures, please refer to http://www.fda.gov/AboutFDA/ WorkingatFDA/BuildingsandFacilities/ WhiteOakCampusInformation/ ucm241740.htm. Submit electronic comments to 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. mstockstill on DSK4VPTVN1PROD with NOTICES SUMMARY: VerDate Mar<15>2010 17:25 Nov 05, 2013 Jkt 232001 FDA will post the agenda approximately 5 days before the meeting at: http://www.fda.gov/ForIndustry/ UserFees/PrescriptionDrugUserFee/ ucm370867.htm. FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1199, Silver Spring, MD 20993, 301–796– 5003, Fax: 301–847–8443, email: Graham.Thompson@fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background on Patient-Focused Drug Development FDA has selected sickle cell disease to be the focus of a meeting under PatientFocused Drug Development, an initiative that involves obtaining a better understanding of patients’ perspectives on the severity of the disease and the available therapies for the condition. Patient-Focused Drug Development is being conducted to fulfill FDA’s performance commitments made as part of the authorization of PDUFA V under Title I of the Food and Drug 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 obtain the patient perspective in 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 available therapies for sickle cell disease. These meetings will include participation of FDA review divisions, the relevant patient community, and other interested stakeholders. On April 11, 2013, FDA published a notice (78 FR 21613) in the Federal Register announcing the disease areas for meetings in fiscal years (FYs) 2013 through 2015, the first 3 years of the 5year PDUFA V timeframe. To develop the list of disease areas, the Agency used several criteria that were outlined in the April 2013 notice. The Agency obtained public comment on these criteria and potential disease areas through a notice for public comment published in the Federal Register on September 24, 2012 (77 FR 58849), and through a public meeting held on October 25, 2012. In selecting the disease areas, FDA carefully considered the public comments received and the PO 00000 Frm 00067 Fmt 4703 Sfmt 4703 66747 perspectives of its review divisions. By the end of FY 2015, FDA will initiate another public process for determining the disease areas for FYs 2016 and 2017. 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. Public Meeting Information A. Purpose and Scope of the Meeting As part of Patient-Focused Drug Development, FDA will obtain patient and patient stakeholder input on sickle cell disease and on current approaches to treatment. Approximately 100,000 people in the United States, and millions of people worldwide, have sickle cell disease. Sickle cell disease is an inherited red blood cell disorder resulting from a mutation in the beta globin gene. Red blood cells are more prone to an abnormal shape and rigidity, causing multi-organ damage over time. Some of the effects of sickle cell disease are painful crises, increased risk of infections, stroke, pulmonary hypertension, acute chest syndrome, recurrent priapism, gallstones, and kidney dysfunction. Therapies to prevent the complications of sickle cell disease are limited and can include prescription medications and blood transfusions. Bone marrow transplantation is an option for some patients. Other therapies, such as pain medications, antibiotics, supplemental oxygen, and vitamin supplements, are used to manage specific health effects of the disease. New approaches to treating sickle cell disease or preventing its complications are being explored, including new medications, advances in transplantation, and gene therapies. FDA is interested in obtaining a better understanding of patients’ perspectives on sickle cell disease, including the symptoms that matter most to patients, limitations to current treatment approaches, opportunities for new treatment approaches, and specific considerations regarding sickle cell disease in pediatric patients. 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 patient panel discussion will begin the dialogue, 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 E:\FR\FM\06NON1.SGM 06NON1 66748 Federal Register / Vol. 78, No. 215 / Wednesday, November 6, 2013 / Notices through written comments that can be submitted to the public docket (see ADDRESSES). When submitting comments to the docket, please provide some context to your comment by indicating whether you are an adolescent or young adult, or older adult. If you are commenting on behalf of a child or other loved one who has sickle cell disease, please indicate that and answer the following questions as much as possible from the patient’s perspective. mstockstill on DSK4VPTVN1PROD with NOTICES Topic 1: The Effects of Sickle Cell Disease That Matter Most to You 1. Of all of the ways that sickle cell disease affects your health, which one to three effects have the greatest impact on your life? (Examples may include pain crises, breathing problems, difficulty concentrating, tiredness, infections, and others.) 2. How does sickle cell disease affect your life on an ‘‘average’’ day? a. Are there activities that you cannot do at all or as well as you would like on these ‘‘average’’ days? Please describe, using specific examples. (Examples may include sleeping through the night, concentrating at work or at school, participating in physical activities, and others.) 3. How does sickle cell disease affect your life on the ‘‘worst’’ days, such as days when you have a pain crisis or have to be hospitalized for some reason? a. Are there activities that you cannot do at all or as well as you would like on these ‘‘worst’’ days? Please describe, using specific examples. 4. What worries you most about how sickle cell disease could affect your health in the future? 5. What specific concerns do you have about sickle cell disease: a. In infants and young children? b. In adolescents and young adults? c. In older adults? Topic 2: Perspectives on Treatments for Sickle Cell Disease 1. Are you currently using any prescription medicines or medical treatments to prevent or treat any negative effects of your sickle cell disease? Please describe these treatments, which may include blood transfusions, supplemental oxygen and prescription medications such as hydroxyurea, antibiotics, pain medications, and others. a. How well do these treatments work for you? For example, how well do they reduce your number of pain crises, hospitalizations, or strokes? How well do they help you manage your pain, breathing difficulties, or other health effects? VerDate Mar<15>2010 17:25 Nov 05, 2013 Jkt 232001 b. What are the biggest problems with these treatments? (Examples may include side effects of medicine, going to the hospital for treatment, frequent blood tests, etc.) How do these problems affect your daily life? 2. Besides prescription medications, what else do you do to prevent or treat any negative effects of your sickle cell disease? Please describe any medications purchased at a store without a prescription, home remedies, diet changes, massages, or other therapies. a. What specific parts of your sickle cell disease do these treatments address? b. How well do these treatments work for you? c. What are the biggest problems with these treatments? 3. What parts of your sickle cell disease do your current treatments not treat at all or not as well as you would like? 4. Assuming that there is no cure for sickle cell disease, what specific things would you look for in an ideal treatment? 5. If you had the opportunity to consider participating in a clinical trial studying experimental treatments for sickle cell disease, what things would you consider when deciding whether or not to participate? Examples may include how severe your sickle cell disease is, how well current treatments are working for you, your concern about serious risks, and other things. 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. They will also be asked to send a brief summary of responses to the topic questions to PatientFocused@fda.hhs.gov. Panelists will be notified of their selection soon after the close of registration on January 27, 2014. FDA 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. Interested members of the public, including those who attend the meeting in person or through the Webcast, are invited to provide electronic or written responses to the questions pertaining to Topics 1 and 2 to the public docket (see ADDRESSES). Comments may be submitted until April 8, 2014. B. Meeting Attendance and/or Participation If you wish to attend this meeting, visit https://patientfocusedsickle cell.eventbrite.com. Please register by January 27, 2014. Those who are unable to attend the meeting in person can register to participate in a live Webcast of the meeting. You will be asked to indicate in your registration whether you plan to attend in person or via the Webcast. Your registration should also contain your complete contact information, including name, title, affiliation, address, email address, and phone number. Seating will be limited, so early registration is recommended. Registration is free and will be on a firstcome, 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 disability, please contact Graham Thompson (see [Docket No. FDA–2013–N–1285] PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 FOR FURTHER INFORMATION CONTACT) Dated: October 31, 2013. Leslie Kux, Assistant Commissioner for Policy. [FR Doc. 2013–26548 Filed 11–5–13; 8:45 am] BILLING CODE 4160–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration Smith Miller and Patch Inc. et al.; Proposal to Withdraw Approval of 14 New Drug Applications; Opportunity for a Hearing AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA) is announcing an opportunity to request a hearing on the Agency’s proposal to withdraw approval of 14 new drug applications (NDAs) from multiple sponsors. The basis for the proposal is that the sponsors have repeatedly failed to file required annual reports for these applications. DATES: Submit written requests for a hearing by December 6, 2013; submit data and information in support of the hearing request by January 6, 2014. ADDRESSES: Identify your requests for a hearing, supporting data, and other comments with Docket No. FDA–2013– N–1285, and submit this information to the Division of Dockets Management (HFA–305), Food and Drug SUMMARY: E:\FR\FM\06NON1.SGM 06NON1

Agencies

[Federal Register Volume 78, Number 215 (Wednesday, November 6, 2013)]
[Notices]
[Pages 66747-66748]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-26548]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-N-2013-1328]


Sickle Cell Disease Public Meeting on Patient-Focused Drug 
Development

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
meeting and an opportunity for public comment on Patient-Focused Drug 
Development for sickle cell disease. Patient-Focused Drug Development 
is part of FDA's performance commitments in the fifth authorization of 
the Prescription Drug User Fee Act (PDUFA V). The public meeting is 
intended to allow FDA to obtain patients' perspectives on the impact of 
sickle cell disease on daily life and on available therapies for sickle 
cell disease.

DATES: The public meeting will be held on February 7, 2014; from 10 
a.m. to 4 p.m. Registration to attend the meeting must be received by 
January 27, 2014. See the SUPPLEMENTARY INFORMATION section for 
information on how to register for the meeting. Submit electronic or 
written comments by April 8, 2014.

ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903 
New Hampshire Ave., Bldg. 31 Conference Center, in Sections B and C of 
the Great Room (Rm. 1503), Silver Spring, MD 20993. Entrance for the 
public meeting participants is through Building 1, where routine 
security check procedures will be performed. For more information on 
parking and security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Submit electronic comments to 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.
    FDA will post the agenda approximately 5 days before the meeting 
at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm370867.htm.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1199, Silver Spring, MD 20993, 301-796-
5003, Fax: 301-847-8443, email: Graham.Thompson@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: 

I. Background on Patient-Focused Drug Development

    FDA has selected sickle cell disease to be the focus of a meeting 
under Patient-Focused Drug Development, an initiative that involves 
obtaining a better understanding of patients' perspectives on the 
severity of the disease and the available therapies for the condition. 
Patient-Focused Drug Development is being conducted to fulfill FDA's 
performance commitments made as part of the authorization of PDUFA V 
under Title I of the Food and Drug 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 obtain the patient perspective in 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 available therapies for 
sickle cell disease. These meetings will include participation of FDA 
review divisions, the relevant patient community, and other interested 
stakeholders.
    On April 11, 2013, FDA published a notice (78 FR 21613) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years (FYs) 2013 through 2015, the first 3 years of the 5-year PDUFA V 
timeframe. To develop the list of disease areas, the Agency used 
several criteria that were outlined in the April 2013 notice. The 
Agency obtained public comment on these criteria and potential disease 
areas through a notice for public comment published in the Federal 
Register on September 24, 2012 (77 FR 58849), and through a public 
meeting held on October 25, 2012. In selecting the disease areas, FDA 
carefully considered the public comments received and the perspectives 
of its review divisions. By the end of FY 2015, FDA will initiate 
another public process for determining the disease areas for FYs 2016 
and 2017. 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. Public Meeting Information

A. Purpose and Scope of the Meeting

    As part of Patient-Focused Drug Development, FDA will obtain 
patient and patient stakeholder input on sickle cell disease and on 
current approaches to treatment. Approximately 100,000 people in the 
United States, and millions of people worldwide, have sickle cell 
disease. Sickle cell disease is an inherited red blood cell disorder 
resulting from a mutation in the beta globin gene. Red blood cells are 
more prone to an abnormal shape and rigidity, causing multi-organ 
damage over time. Some of the effects of sickle cell disease are 
painful crises, increased risk of infections, stroke, pulmonary 
hypertension, acute chest syndrome, recurrent priapism, gallstones, and 
kidney dysfunction.
    Therapies to prevent the complications of sickle cell disease are 
limited and can include prescription medications and blood 
transfusions. Bone marrow transplantation is an option for some 
patients. Other therapies, such as pain medications, antibiotics, 
supplemental oxygen, and vitamin supplements, are used to manage 
specific health effects of the disease. New approaches to treating 
sickle cell disease or preventing its complications are being explored, 
including new medications, advances in transplantation, and gene 
therapies. FDA is interested in obtaining a better understanding of 
patients' perspectives on sickle cell disease, including the symptoms 
that matter most to patients, limitations to current treatment 
approaches, opportunities for new treatment approaches, and specific 
considerations regarding sickle cell disease in pediatric patients.
    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 patient panel discussion will begin the 
dialogue, 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

[[Page 66748]]

through written comments that can be submitted to the public docket 
(see ADDRESSES). When submitting comments to the docket, please provide 
some context to your comment by indicating whether you are an 
adolescent or young adult, or older adult. If you are commenting on 
behalf of a child or other loved one who has sickle cell disease, 
please indicate that and answer the following questions as much as 
possible from the patient's perspective.
Topic 1: The Effects of Sickle Cell Disease That Matter Most to You
    1. Of all of the ways that sickle cell disease affects your health, 
which one to three effects have the greatest impact on your life? 
(Examples may include pain crises, breathing problems, difficulty 
concentrating, tiredness, infections, and others.)
    2. How does sickle cell disease affect your life on an ``average'' 
day?
    a. Are there activities that you cannot do at all or as well as you 
would like on these ``average'' days? Please describe, using specific 
examples. (Examples may include sleeping through the night, 
concentrating at work or at school, participating in physical 
activities, and others.)
    3. How does sickle cell disease affect your life on the ``worst'' 
days, such as days when you have a pain crisis or have to be 
hospitalized for some reason?
    a. Are there activities that you cannot do at all or as well as you 
would like on these ``worst'' days? Please describe, using specific 
examples.
    4. What worries you most about how sickle cell disease could affect 
your health in the future?
    5. What specific concerns do you have about sickle cell disease:
    a. In infants and young children?
    b. In adolescents and young adults?
    c. In older adults?
Topic 2: Perspectives on Treatments for Sickle Cell Disease
    1. Are you currently using any prescription medicines or medical 
treatments to prevent or treat any negative effects of your sickle cell 
disease? Please describe these treatments, which may include blood 
transfusions, supplemental oxygen and prescription medications such as 
hydroxyurea, antibiotics, pain medications, and others.
    a. How well do these treatments work for you? For example, how well 
do they reduce your number of pain crises, hospitalizations, or 
strokes? How well do they help you manage your pain, breathing 
difficulties, or other health effects?
    b. What are the biggest problems with these treatments? (Examples 
may include side effects of medicine, going to the hospital for 
treatment, frequent blood tests, etc.) How do these problems affect 
your daily life?
    2. Besides prescription medications, what else do you do to prevent 
or treat any negative effects of your sickle cell disease? Please 
describe any medications purchased at a store without a prescription, 
home remedies, diet changes, massages, or other therapies.
    a. What specific parts of your sickle cell disease do these 
treatments address?
    b. How well do these treatments work for you?
    c. What are the biggest problems with these treatments?
    3. What parts of your sickle cell disease do your current 
treatments not treat at all or not as well as you would like?
    4. Assuming that there is no cure for sickle cell disease, what 
specific things would you look for in an ideal treatment?
    5. If you had the opportunity to consider participating in a 
clinical trial studying experimental treatments for sickle cell 
disease, what things would you consider when deciding whether or not to 
participate? Examples may include how severe your sickle cell disease 
is, how well current treatments are working for you, your concern about 
serious risks, and other things.

B. Meeting Attendance and/or Participation

    If you wish to attend this meeting, visit https://patientfocusedsicklecell.eventbrite.com. Please register by January 27, 
2014. Those who are unable to attend the meeting in person can register 
to participate in a live Webcast of the meeting. You will be asked to 
indicate in your registration whether you plan to attend in person or 
via the Webcast. Your registration should also contain your complete 
contact information, including name, title, affiliation, address, email 
address, and phone number.
    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 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. They will also be 
asked to send a brief summary of responses to the topic questions to 
PatientFocused@fda.hhs.gov. Panelists will be notified of their 
selection soon after the close of registration on January 27, 2014. FDA 
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.
    Interested members of the public, including those who attend the 
meeting in person or through the Webcast, are invited to provide 
electronic or written responses to the questions pertaining to Topics 1 
and 2 to the public docket (see ADDRESSES). Comments may be submitted 
until April 8, 2014.

    Dated: October 31, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-26548 Filed 11-5-13; 8:45 am]
BILLING CODE 4160-01-P