2019 Public Meeting on Center for Drug Evaluation and Research Standard Core Sets: Clinical Outcome Assessments and Endpoints Grant Program; Public Meeting; Request for Comments

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public meeting; request for comments.

SUMMARY:

The Food and Drug Administration (FDA, the Agency, or we) is announcing the following public meeting entitled “2019 Public Meeting on CDER Standard Core Sets: Clinical Outcome Assessments and Endpoints Grant Program.” The purpose of the public meeting is to help ensure that as standard core sets of clinical outcome assessments (COAs) are developed as part of the FDA pilot grant program, the identified concepts, COAs, and endpoints reflect what is most important to patients and relevant to regulatory and potentially other stakeholder decision making. To facilitate this, stakeholders including patients, care partners, FDA reviewers, drug developers, other government and academic researchers, health care providers, health technology assessors and health payers are encouraged to attend the meeting.

DATES:

The public meeting will be held on December 5, 2019, from 8:30 a.m. to 12 p.m. Submit either electronic or written comments on this public meeting by January 6, 2020. See the SUPPLEMENTARY INFORMATION section for registration date and information.

ADDRESSES:

The public meeting will be held at FDA's White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD 20993. Entrance for the public meeting participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

You may submit comments as follows. Please note that late, untimely filed comments will not be considered. Electronic comments must be submitted on or before January 6, 2020. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of January 6, 2020. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are postmarked or the delivery service acceptance receipt is on or before that date.

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): Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
• For written/paper comments submitted to the Dockets Management Staff, 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-2019-N-5157 for “2019 Public Meeting on CDER Standard Core Sets: Clinical Outcome Assessments and Endpoints Grant Program.” Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff 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 Dockets Management Staff. 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: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.

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 Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT:

Meena Savani, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6306, Silver Spring, MD 20993-0002, 240-402-1348, CDER_StandardCoreCOAs@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

As part of our Patient Focused Drug Development efforts, FDA developed a pilot grant program to support the development of publicly available standard core set(s) of COAs and their related endpoints for specific disease indications. On September 11, 2019, the FDA made three awards under this grant program in the areas of: (1) Migraine, (2) acute pain in infants and young children, and (3) physical function across a range of chronic conditions.

The purpose of this public meeting is to ensure that, as these standard core sets of clinical outcome assessments are developed, the identified concepts, COAs, and endpoints reflect what is most important and relevant to patients and support regulatory and potentially other stakeholder decision making.

COAs are often endpoints in clinical trials used to support drug approval and labeling claims or other communications regarding clinical benefit. Clinical benefit is defined as a positive clinically meaningful effect of an intervention on how an individual feels, functions, or survives. FDA uses COAs primarily to determine whether a drug has been shown to provide clinical benefit to patients. Severity of side effects or treatment burden can also be measured by COAs.

A standard core set of COAs can include different types of COAs such as patient-reported outcome (PRO), clinician-reported outcome (ClinRO), observer-reported outcome (ObsRO), and performance outcome (PerfO) instruments and their related endpoints. These sets should assess a minimum list of impacts that matter most to patients, are likely to demonstrate change (including differences in trial arms related to disease burden, treatment burden, and if applicable, physical function), and should be assessed during a clinical trial. A standard core set might be relevant across several disease populations or subgroups or be focused on attributes of a specific disease.

II. Topics for Discussion at the Public Meeting

This meeting will provide an opportunity for grantees funded as part of the FDA Standard Core COAs and Endpoints Pilot Grant Program to share their development plans for the standard core COA sets and to receive feedback from stakeholders. FDA will provide an introduction and discuss plans for the pilot grant program including future public meetings.

III. Participating in the Public Meeting

Registration: Persons interested in attending this public meeting in person or via webcast must register online at https://standard_core_coa_grant_program.eventbrite.com by December 2, 2019, at 11:59 p.m. Eastern Time. Registration is free and based on space availability, with priority given to early registrants. Early registration for in person attendance is recommended because seating is limited; therefore, FDA may limit the number of participants from each organization. Registrants will receive confirmation when they have been accepted. If time and space permit, onsite registration on the day of the public meeting/public workshop will be provided beginning at 8 a.m.

If you need special accommodations due to a disability, please contact Meena Savani no later than November 29, 2019.

Streaming Webcast of the Public Meeting: This public meeting will also be webcast. Webcast information will be provided upon completion of registration.

If you have never attended a Connect Pro event before, test your connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Connect Pro program, visit https://www.adobe.com/go/connectpro_overview. FDA has verified the website addresses in this document, as of the date this document publishes in the Federal Register, but websites are subject to change over time.

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 Dockets Management Staff (see ADDRESSES). A link to the transcript will also be available on the internet at https://www.fda.gov/drugs/news-events-human-drugs/public-meeting-cder-standard-core-sets-clinical-outcome-assessments-and-endpoints-grant-program.