80 FR 46026 - Patient-Focused Drug Development for Nontuberculous Mycobacterial Lung Infections; Public Meeting
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 80, Issue 148 (August 3, 2015)
Food and Drug Administration
Federal Register Volume 80, Issue 148 (August 3, 2015)
| Page Range | 46026-46027 | |
| FR Document | 2015-18919 |
[Federal Register Volume 80, Number 148 (Monday, August 3, 2015)] [Notices] [Pages 46026-46027] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2015-18919] [[Page 46026]] ----------------------------------------------------------------------- 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
![46026 Federal Register / Vol. 80, No. 148 / Monday, August 3, 2015 / Notices
DEPARTMENT OF HEALTH AND www.fda.gov/AboutFDA/ outlined in that notice to develop the
HUMAN SERVICES WorkingatFDA/BuildingsandFacilities/ list of disease areas. FDA obtained
WhiteOakCampusInformation/ public comment on the Agency’s
Food and Drug Administration ucm241740.htm. proposed criteria and potential disease
Submit electronic comments to areas through a public docket. In
[Docket No. FDA–2012–N–0967]
www.regulations.gov. Submit written selecting the set of disease areas, FDA
Patient-Focused Drug Development for comments to the Division of Dockets carefully considered the public
Nontuberculous Mycobacterial Lung Management (HFA–305), Food and Drug comments received and the perspectives
Infections; Public Meeting Administration, 5630 Fishers Lane, Rm. of review divisions at FDA. More
1061, Rockville, MD 20852. All information, including the list of disease
AGENCY: Food and Drug Administration, comments should be identified with the areas and a general schedule of
HHS. docket number found in brackets in the meetings, is posted at http://
ACTION: Notice of public meeting; heading of this document. www.fda.gov/ForIndustry/UserFees/
request for comments. FDA will post the agenda PrescriptionDrugUserFee/
approximately 5 days before the meeting ucm326192.htm.
SUMMARY: The Food and Drug at http://www.fda.gov/Drugs/
Administration (FDA or Agency) is NewsEvents/ucm453877.htm. II. Purpose and Scope of the Meeting
announcing a public meeting and an FOR FURTHER INFORMATION CONTACT:
opportunity for public comment on The purpose of this Patient-Focused
Graham Thompson, Center for Drug Drug Development meeting is to obtain
Patient-Focused Drug Development for Evaluation and Research, Food and
nontuberculous mycobacterial (NTM) input on the symptoms and other
Drug Administration, 10903 New impacts of NTM lung infections that
lung infections. Patient-Focused Drug Hampshire Ave., Bldg. 51, Rm. 1146,
Development is part of FDA’s matter most to patients, as well as
Silver Spring, MD 20993, 301–796– perspectives on current approaches to
performance commitments made as part 5003, FAX: 301–847–8443,
of the fifth authorization of the treating this condition. NTM infections
graham.thompson@fda.hhs.gov. can affect all organs in the body;
Prescription Drug User Fee Act (PDUFA
SUPPLEMENTARY INFORMATION: however, NTM infections primarily
V). The public meeting is intended to
allow FDA to obtain patient I. Background on Patient-Focused Drug affect the lungs, especially in patients
perspectives on the impact of NTM lung Development with underlying lung disease. Common
infections on daily life and patient causes of NTM lung infections include
FDA has selected NTM lung Mycobacterium avium-intracellulare
views on treatment approaches. FDA is infections as the focus of a public
also interested in discussing issues and Mycobacterium abscessus.
meeting under Patient-Focused Drug Symptoms of NTM lung infections
related to scientific challenges in Development, an initiative that involves
developing drugs to treat NTM lung include chronic cough, shortness of
obtaining a better understanding of breath, blood in sputum, fever, fatigue,
infections. In the afternoon, FDA will patient perspectives on the severity of a
hold a workshop and provide loss of appetite, night sweats, and
disease and the available therapies for weight loss. There are no FDA-approved
information for and gain perspective these conditions. Patient-Focused Drug
from patients and patient advocacy therapies for NTM lung infections.
Development is being conducted to Treatment requires a combination of
organizations, health care providers, fulfill FDA performance commitments
academic experts, and industry on drugs given for prolonged duration. The
that are part of the reauthorization of antibacterial drugs used can cause
various aspects of clinical development PDUFA under Title I of the Food and
of drug products intended to treat NTM severe side effects that make treatment
Drug Administration Safety and of this condition difficult. FDA is
lung infections. The input from this Innovation Act (FDASIA) (Pub. L. 112–
public meeting will help in developing committed to working with all
144). The full set of performance
topics for further discussion. stakeholders to develop safe and
commitments is available at http://
DATES: The public meeting will be held effective therapies for affected
www.fda.gov/downloads/forindustry/
on October 15, 2015, from 9 a.m. to 5 individuals.
userfees/prescriptiondruguserfee/
p.m. Please register for the meeting by ucm270412.pdf. The questions that will be asked of
October 7, 2015. Registration from those FDA committed to obtain the patient patients and patient stakeholders at the
individuals interested in presenting perspective on 20 disease areas during meeting are listed in this section,
comments as part of the panel the course of PDUFA V. For each organized by topic. For each topic, a
discussions should be received by disease area, the Agency will conduct a brief initial patient panel discussion
September 28, 2015. See the public meeting to discuss the disease will begin the dialogue. This will be
SUPPLEMENTARY INFORMATION section for and its impact on patients’ daily lives, followed by a facilitated discussion
instructions on how to register for the the types of treatment benefit that inviting comments from other patient
meeting. Submit electronic or written matter most to patients, and patients’ and patient stakeholder participants. In
comments to the public docket by perspectives on the adequacy of the addition to input generated through this
December 15, 2015. available therapies. These meetings will public meeting, FDA is interested in
ADDRESSES: The meeting and workshop include participation of FDA review receiving patient input addressing these
will be held at the FDA White Oak divisions, the relevant patient questions through written comments,
mstockstill on DSK4VPTVN1PROD with NOTICES
Campus, 10903 New Hampshire Ave., communities, and other interested which can be submitted to the public
Bldg. 31 Conference Center, the Great stakeholders. docket (see ADDRESSES). When
Room (Rm. 1503), Silver Spring, MD On July 2, 2015, FDA published a submitting comments, if you are
20993–0002. Participants must enter notice (80 FR 38216) in the Federal commenting on behalf of a child please
through Building 1 and undergo Register announcing the disease areas indicate that you are doing so and
security screening. For more for meetings in fiscal years 2016–2017, answer the following questions as much
information on parking and security the final 2 years of the PDUFA V time as possible from the patient’s
procedures, please refer to http:// frame. The Agency used several criteria perspective.
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![Federal Register / Vol. 80, No. 148 / Monday, August 3, 2015 / Notices 46027
Topic 1: Disease Symptoms and Daily would you look for in an ideal treatment pertaining to topics 1 and 2 to the
Impacts That Matter Most to Patients for your condition? Division of Dockets Management (see
In the afternoon, discussion will be ADDRESSES) by December 15, 2015. It is
1. Of all the symptoms that you
related to scientific topics, with the goal only necessary to send one set of
experience because of your condition,
of understanding issues that may affect comments. Identify comments with the
which 1–3 symptoms have the most
the development of drugs for the docket number found in brackets in the
significant impact on your life?
treatment of NTM lung infections and heading of this document. Received
(Examples may include cough,
identifying topics for future discussion. comments may be seen in the Division
increased sputum production, shortness
Discussion topics for the afternoon will of Dockets Management between 9 a.m.
of breath, difficulty breathing, chest include the following: Epidemiology and 4 p.m., Monday through Friday, and
pain) and natural history of NTM lung will be posted to the docket at http://
2. Are there specific activities that are
infections, current treatment www.regulations.gov.
important to you but that you cannot do
considerations, clinical trial designs,
at all or as fully as you would like V. Transcripts
and clinical trial endpoints.
because of your condition? (Examples of As soon as a transcript is available,
activities may include sleeping through III. Attendance and Registration FDA will post it at http://www.fda.gov/
the night, daily hygiene, driving, If you wish to attend this meeting, Drugs/NewsEvents/ucm453877.htm.
walking/running, exercising, etc.) visit http://ntmpfdd.eventbrite.com. Dated: July 28, 2015.
• How do your symptoms and their Please register by October 7, 2015. If you
negative impacts affect your daily life Leslie Kux,
are unable to attend the meeting in
on the best days? On the worst days? Associate Commissioner for Policy.
person, you can register to view a live
(Examples may include limitations on [FR Doc. 2015–18919 Filed 7–31–15; 8:45 am]
Webcast of the meeting. You will be
the ability to undertake physically asked to indicate in your registration if BILLING CODE 4164–01–P
strenuous activities, restrictions on the you plan to attend in person or via the
ability to travel, inability to sleep, lack Webcast. Seating will be limited, so
of appetite, fatigue, etc.) DEPARTMENT OF HEALTH AND
early registration is recommended.
3. How has your condition and its HUMAN SERVICES
Registration is free and will be on a first-
symptoms changed over time? come, first-served basis. However, FDA Food and Drug Administration
• Do your symptoms come and go? If may limit the number of participants
so, do you know of anything that makes from each organization based on space [Docket No. FDA–2012–N–1182]
your symptoms better? Worse? limitations. Registrants will receive
4. What worries you most about your Joint Food and Drug Administration/
confirmation once they have been Health Canada Quantitative
condition? accepted. Onsite registration on the day Assessment of the Risk of Listeriosis
Topic 2: Patients’ Perspectives on of the meeting will be based on space From Soft-Ripened Cheese
Current Approaches To Treating NTM availability. Consumption in the United States and
Lung Infections If you need special accommodations
Canada
because of a disability, please contact
1. What are you currently doing to Graham Thompson at least 7 days before AGENCY: Food and Drug Administration,
help treat your condition or its the meeting. HHS.
symptoms? (Examples may include ACTION: Notice.
prescription medicines, over-the- IV. Comments
counter products, nebulizers, and other Patients who are interested in SUMMARY: The Food and Drug
therapies including non-drug therapies) presenting comments as part of the Administration (FDA or we) is
• What specific symptoms do your initial panel discussions will be asked announcing the availability of the ‘‘Joint
treatments address? to indicate in their registration which Food and Drug Administration/Health
• How has your treatment regimen topic(s) they wish to address. These Canada—Santé Canada Quantitative
changed over time, and why? patients also must send to Assessment of the Risk of Listeriosis
2. How well does your current PatientFocused@fda.hhs.gov a brief From Soft-Ripened Cheese
treatment regimen treat the most summary of responses to the topic Consumption in the United States and
significant symptoms of your disease? questions by September 28, 2015. Canada.’’ We are making available an
• How well do these treatments stop Panelists will be notified of their interpretative summary, a technical
or slow the progression of your disease? selection approximately 7 days before Quantitative Risk Assessment (QRA)
• How well do these therapies the public meeting. We will try to report with appendices, a risk-
improve your ability to do specific accommodate all patients and patient assessment model, and a document
activities that are important to you in stakeholders who wish to speak, either responding to public comments that we
your daily life? through the panel discussion or received regarding the 2013 ‘‘Draft Joint
• How well have these treatments audience participation; however, the Food and Drug Administration/Health
worked for you as your condition has duration of comments may be limited by Canada—Santé Canada Quantitative
changed over time? time constraints. Assessment of the Risk of Listeriosis
3. What are the most significant FDA will hold an open public From Soft-Ripened Cheese
downsides to your current treatments, comment period to give the public an Consumption in the United States and
mstockstill on DSK4VPTVN1PROD with NOTICES
and how do they affect your daily life? opportunity to comment. Registration Canada.’’ The purpose of the QRA is to
(Examples of downsides may include for open public comment will occur at evaluate the effect of factors such as the
bothersome side effects, need for the registration desk on the day of the microbiological status of milk, cheese-
multiple medications, need for meeting and workshop on a first-come, manufacturing steps, and conditions
injections, going to the hospital for first-served basis. during distribution and storage on the
treatment, etc.) Regardless of attendance at the public overall risk of invasive listeriosis to the
4. Assuming there is no complete cure meeting, you can submit electronic or consumer of soft-ripened cheese in the
for your condition, what specific things written responses to the questions United States or Canada. The QRA
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Document Created: 2018-02-23 10:51:55
Document Modified: 2018-02-23 10:51:55
| 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 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. | |
| 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 | 80 FR 46026 |
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