Public Meeting on Patient-Focused Drug Development for Sarcopenia; Request for Comments

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 Sarcopenia.” 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 sarcopenia on daily life as well as patient views on treatment approaches for sarcopenia.

DATES:

The public meeting will be held on April 6, 2017, from 1 p.m. to 5 p.m. Registration to attend the meeting must be received by March 27, 2017 (see SUPPLEMENTARY INFORMATION for instructions). Public comments will be accepted through June 6, 2017. See the ADDRESSES section for information about submitting comments to the public docket.

ADDRESSES:

The public meeting 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. Entrance for the public meeting participants (non-FDA employees) 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.

You may submit comments as follows:

Electronic Submissions

Submit electronic comments in the following way:

• Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://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 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 https://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-4198 for “Public Meeting on Patient-Focused Drug Development for Sarcopenia; Request for Comments.” Received comments will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://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 https://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 https://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/ucm527587.htm.

FOR FURTHER INFORMATION CONTACT:

Meghana Chalasani, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1146, Silver Spring, MD 20993-0002, 240-402-6525, FAX: 301-847-8443, Meghana.Chalasani@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background on Patient-Focused Drug Development

FDA has selected sarcopenia 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 that condition. 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 Administration 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 at least 20 disease areas during the course of PDUFA V. For each disease area, the Agency is conducting 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 April 11, 2013, FDA published a notice in the Federal Register (78 FR 21613) announcing the disease areas for meetings in fiscal years (FYs) 2013-2015, the first 3 years of the 5-year 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 and a public meeting that was convened on October 25, 2012. In selecting the set of disease areas, FDA carefully considered the public comments received and the perspectives of review divisions at FDA. FDA initiated a second public process for determining the disease areas for FY 2016-2017, and published a notice in the Federal Register on July 2, 2015 (80 FR 38216), announcing the selection of eight disease areas. 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 Information

As part of Patient-Focused Drug Development, FDA will obtain patient and patient stakeholder input on the symptoms of sarcopenia that matter most to patients and on current approaches to treating sarcopenia. Sarcopenia is a condition characterized by loss of muscle mass and loss of muscle function or strength that occurs with age. While there is currently no cure, treatments for sarcopenia are primarily non-drug therapies including exercise and nutrition. FDA is interested in the perspectives of patients with sarcopenia on (1) symptoms and the daily impacts of their condition, (2) current approaches to treatment, and (3) decision factors taken into account when selecting a treatment.

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).

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 one to three symptoms have the most significant impact on your life? (Examples may include difficulty walking, feeling unsteady and falling frequently, having a decreased level of activity, etc.)

(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 participation in social activities, household chores, daily hygiene, etc.)

(3) How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?

(4) How have your condition and its symptoms changed over time?

(a) Would you define your condition today as being well managed?

(5) What worries you most about your condition?

Topic 2: Patients' Perspectives on Current Approaches to Treatment

(1) What are you currently doing to help treat your condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification.)

(a) What specific symptoms do your treatments address?

(b) How has your treatment regimen changed over time, and why?

(2) How well does your current treatment regimen control your condition?

(a) How well do your treatments address specific activities that are important to you in your daily life?

(b) 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 going to the hospital or clinic for treatment, time devoted to treatment, etc.)

(4) What specific things would you look for in an ideal treatment for your condition?

(a) What would you consider to be a meaningful improvement (for example, symptom improvements or functional improvements) in your condition that a treatment could provide?

III. Meeting Attendance and Participation

If you wish to attend this meeting, visit https://sarcopeniapfdd.eventbrite.com. Please register by March 27, 2017. 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 Meghana Chalasani (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 PatientFocused@fda.hhs.gov a brief summary of responses to the topic questions by March 20, 2017. 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.

Transcripts: Please be advised that as soon as a transcript of the public meeting is available, it will be accessible at https://www.regulations.gov. It may be viewed at the Division of Dockets Management (see ADDRESSES). A link to the transcript will also be available on the Internet at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm527587.htm.