Patient-Focused Drug Development for Nontuberculous Mycobacterial Lung Infections; Public Meeting, 46026-46027 [2015-18919]

Download as PDF 46026 Federal Register / Vol. 80, No. 148 / Monday, August 3, 2015 / Notices DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2012–N–0967] Patient-Focused Drug Development for Nontuberculous Mycobacterial Lung Infections; Public Meeting AGENCY: Food and Drug Administration, HHS. Notice of public meeting; request for comments. ACTION: 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 nontuberculous mycobacterial (NTM) lung infections. 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 NTM lung infections on daily life and patient views on treatment approaches. FDA is also interested in discussing issues related to scientific challenges in developing drugs to treat NTM lung infections. 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 treat NTM lung infections. The input from this public meeting will help in developing topics for further discussion. DATES: The public meeting will be held on October 15, 2015, from 9 a.m. to 5 p.m. Please register for the meeting by October 7, 2015. Registration from those individuals interested in presenting comments as part of the panel discussions should be received by September 28, 2015. See the SUPPLEMENTARY INFORMATION section for instructions on how to register for the meeting. Submit electronic or written comments to the public docket by December 15, 2015. 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:// mstockstill on DSK4VPTVN1PROD with NOTICES SUMMARY: VerDate Sep<11>2014 18:35 Jul 31, 2015 Jkt 235001 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/Drugs/ NewsEvents/ucm453877.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, graham.thompson@fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background on Patient-Focused Drug Development FDA has selected NTM lung infections 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 PDUFA under Title I of the Food and Drug Administration Safety and Innovation Act (FDASIA) (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 38216) in the Federal Register announcing the disease areas for meetings in fiscal years 2016–2017, the final 2 years of the PDUFA V time frame. The Agency used several criteria PO 00000 Frm 00095 Fmt 4703 Sfmt 4703 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. 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 of NTM lung infections that matter most to patients, as well as perspectives on current approaches to treating this condition. NTM infections can affect all organs in the body; however, NTM infections primarily affect the lungs, especially in patients with underlying lung disease. Common causes of NTM lung infections include Mycobacterium avium-intracellulare and Mycobacterium abscessus. Symptoms of NTM lung infections include chronic cough, shortness of breath, blood in sputum, fever, fatigue, loss of appetite, night sweats, and weight loss. There are no FDA-approved therapies for NTM lung infections. Treatment requires a combination of drugs given for prolonged duration. The antibacterial drugs used can cause severe side effects that make treatment of this condition difficult. 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 answer the following questions as much as possible from the patient’s perspective. E:\FR\FM\03AUN1.SGM 03AUN1 Federal Register / Vol. 80, No. 148 / Monday, August 3, 2015 / Notices Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients 1. Of all the symptoms that you experience because of your condition, which 1–3 symptoms have the most significant impact on your life? (Examples may include cough, increased sputum production, shortness of breath, difficulty breathing, chest pain) 2. 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 condition? (Examples of activities may include sleeping through the night, daily hygiene, driving, walking/running, exercising, etc.) • 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, inability to sleep, lack of appetite, fatigue, etc.) 3. How has your condition and its symptoms changed over time? • Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse? 4. What worries you most about your condition? mstockstill on DSK4VPTVN1PROD with NOTICES Topic 2: Patients’ Perspectives on Current Approaches To Treating NTM Lung Infections 1. What are you currently doing to help treat your condition or its symptoms? (Examples may include prescription medicines, over-thecounter products, nebulizers, and other therapies including non-drug therapies) • What specific symptoms do your treatments address? • How has your treatment regimen changed over time, and why? 2. How well does your current treatment regimen treat the most significant symptoms of your disease? • How well do these treatments stop or slow the progression of your disease? • How well do these therapies improve your ability to do specific activities that are important to you in your daily life? • How well have these treatments worked for you as your condition has 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, need for injections, going to the hospital for treatment, etc.) 4. Assuming there is no complete cure for your condition, what specific things VerDate Sep<11>2014 18:35 Jul 31, 2015 Jkt 235001 would you look for in an ideal treatment for your condition? 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 NTM lung infections and identifying topics for future discussion. Discussion topics for the afternoon will include the following: Epidemiology and natural history of NTM lung infections, current treatment considerations, clinical trial designs, and clinical trial endpoints. III. Attendance and Registration If you wish to attend this meeting, visit http://ntmpfdd.eventbrite.com. Please register by October 7, 2015. 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 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 a disability, please contact Graham Thompson at least 7 days before the meeting. IV. Comments 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 PatientFocused@fda.hhs.gov a brief summary of responses to the topic questions by September 28, 2015. 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. Regardless of attendance at the public meeting, you can submit electronic or written responses to the questions PO 00000 Frm 00096 Fmt 4703 Sfmt 4703 46027 pertaining to topics 1 and 2 to the Division of Dockets Management (see ADDRESSES) by December 15, 2015. 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. 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 As soon as a transcript is available, FDA will post it at http://www.fda.gov/ Drugs/NewsEvents/ucm453877.htm. Dated: July 28, 2015. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2015–18919 Filed 7–31–15; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2012–N–1182] Joint Food and Drug Administration/ Health Canada Quantitative Assessment of the Risk of Listeriosis From Soft-Ripened Cheese Consumption in the United States and Canada AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA or we) is announcing the availability of the ‘‘Joint Food and Drug Administration/Health ´ Canada—Sante Canada Quantitative Assessment of the Risk of Listeriosis From Soft-Ripened Cheese Consumption in the United States and Canada.’’ We are making available an interpretative summary, a technical Quantitative Risk Assessment (QRA) report with appendices, a riskassessment model, and a document responding to public comments that we received regarding the 2013 ‘‘Draft Joint Food and Drug Administration/Health ´ Canada—Sante Canada Quantitative Assessment of the Risk of Listeriosis From Soft-Ripened Cheese Consumption in the United States and Canada.’’ The purpose of the QRA is to evaluate the effect of factors such as the microbiological status of milk, cheesemanufacturing steps, and conditions during distribution and storage on the overall risk of invasive listeriosis to the consumer of soft-ripened cheese in the United States or Canada. The QRA SUMMARY: E:\FR\FM\03AUN1.SGM 03AUN1

Agencies

[Federal Register Volume 80, Number 148 (Monday, August 3, 2015)]
[Notices]
[Pages 46026-46027]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-18919]



[[Page 46026]]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0967]


Patient-Focused Drug Development for Nontuberculous Mycobacterial 
Lung Infections; 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 nontuberculous mycobacterial (NTM) lung 
infections. 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 
NTM lung infections on daily life and patient views on treatment 
approaches. FDA is also interested in discussing issues related to 
scientific challenges in developing drugs to treat NTM lung infections. 
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 treat NTM 
lung infections. The input from this public meeting will help in 
developing topics for further discussion.

DATES: The public meeting will be held on October 15, 2015, from 9 a.m. 
to 5 p.m. Please register for the meeting by October 7, 2015. 
Registration from those individuals interested in presenting comments 
as part of the panel discussions should be received by September 28, 
2015. See the SUPPLEMENTARY INFORMATION section for instructions on how 
to register for the meeting. Submit electronic or written comments to 
the public docket by December 15, 2015.

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.
    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/Drugs/NewsEvents/ucm453877.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, graham.thompson@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

    FDA has selected NTM lung infections 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 PDUFA under Title I of the Food and Drug 
Administration Safety and Innovation Act (FDASIA) (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 38216) in the 
Federal Register announcing the disease areas for meetings in fiscal 
years 2016-2017, the final 2 years of the 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. 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 of NTM lung infections 
that matter most to patients, as well as perspectives on current 
approaches to treating this condition. NTM infections can affect all 
organs in the body; however, NTM infections primarily affect the lungs, 
especially in patients with underlying lung disease. Common causes of 
NTM lung infections include Mycobacterium avium-intracellulare and 
Mycobacterium abscessus. Symptoms of NTM lung infections include 
chronic cough, shortness of breath, blood in sputum, fever, fatigue, 
loss of appetite, night sweats, and weight loss. There are no FDA-
approved therapies for NTM lung infections. Treatment requires a 
combination of drugs given for prolonged duration. The antibacterial 
drugs used can cause severe side effects that make treatment of this 
condition difficult. 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 answer the following questions as much as possible from the 
patient's perspective.

[[Page 46027]]

Topic 1: Disease Symptoms and Daily Impacts That Matter Most to 
Patients

    1. Of all the symptoms that you experience because of your 
condition, which 1-3 symptoms have the most significant impact on your 
life? (Examples may include cough, increased sputum production, 
shortness of breath, difficulty breathing, chest pain)
    2. 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 
condition? (Examples of activities may include sleeping through the 
night, daily hygiene, driving, walking/running, exercising, etc.)
     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, inability to sleep, 
lack of appetite, fatigue, etc.)
    3. How has your condition and its symptoms changed over time?
     Do your symptoms come and go? If so, do you know of 
anything that makes your symptoms better? Worse?
    4. What worries you most about your condition?

Topic 2: Patients' Perspectives on Current Approaches To Treating NTM 
Lung Infections

    1. What are you currently doing to help treat your condition or its 
symptoms? (Examples may include prescription medicines, over-the-
counter products, nebulizers, and other therapies including non-drug 
therapies)
     What specific symptoms do your treatments address?
     How has your treatment regimen changed over time, and why?
    2. How well does your current treatment regimen treat the most 
significant symptoms of your disease?
     How well do these treatments stop or slow the progression 
of your disease?
     How well do these therapies improve your ability to do 
specific activities that are important to you in your daily life?
     How well have these treatments worked for you as your 
condition has 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, need for injections, going to the hospital for treatment, 
etc.)
    4. Assuming there is no complete cure for your condition, what 
specific things would you look for in an ideal treatment for your 
condition?
    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 NTM lung infections and identifying 
topics for future discussion. Discussion topics for the afternoon will 
include the following: Epidemiology and natural history of NTM lung 
infections, current treatment considerations, clinical trial designs, 
and clinical trial endpoints.

III. Attendance and Registration

    If you wish to attend this meeting, visit http://ntmpfdd.eventbrite.com. Please register by October 7, 2015. 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 at least 7 days before the meeting.

IV. Comments

    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 PatientFocused@fda.hhs.gov a brief summary of responses to 
the topic questions by September 28, 2015. 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.
    Regardless of attendance at the public meeting, you can submit 
electronic or written responses to the questions pertaining to topics 1 
and 2 to the Division of Dockets Management (see ADDRESSES) by December 
15, 2015. 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. 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

    As soon as a transcript is available, FDA will post it at http://www.fda.gov/Drugs/NewsEvents/ucm453877.htm.

    Dated: July 28, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-18919 Filed 7-31-15; 8:45 am]
BILLING CODE 4164-01-P