2018-17272. Patient-Focused Drug Development Guidance: Methods To Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments; Public Workshop; Request for Comments

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public workshop; request for comments.

SUMMARY:

The Food and Drug Administration (FDA) is announcing a 2-day public workshop to convene a discussion on methodological approaches that may be used to identify what is most important to patients and caregivers with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient's disease, and best practices for selecting, developing, or modifying fit-for-purpose clinical outcome assessments (COAs) to measure the patient experience in clinical trials. This workshop will inform development of patient-focused drug development guidance as required by the 21st Century Cures Act (Cures Act) and as part of commitments made by FDA under the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI). FDA will publish discussion documents approximately 1 month before the workshop date. FDA is interested in seeking information and comments on the approaches proposed in the discussion documents. FDA is also interested in input on examples, which could be illustrated in the draft guidance, where the approaches proposed in the discussion document have been successfully applied.

DATES:

The public workshop will be held on October 15 and 16, 2018, from 9 a.m. to 5 p.m. Submit either electronic or written comments on this public workshop by December 14, 2018. See the SUPPLEMENTARY INFORMATION section for registration date and information.

ADDRESSES:

The public workshop 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 workshop 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. Workshop updates, agenda, and discussion documents will be made available at: https://www.fda.gov/​Drugs/​NewsEvents/​ucm607276.htm prior to the workshop.

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 December 14, 2018. The https://www.regulations.gov electronic filing system will accept comments until midnight Eastern Time at the end of December 14, 2018. 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-2018-N-2455 for “Patient-Focused Drug Development Guidance: Methods to Identify What is Important to Patients and Select, Develop or Modify Fit-for-Purpose Clinical Outcome Assessments; Public Workshop; Request for Comments.” 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:

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: [phone], [e-mail].

SUPPLEMENTARY INFORMATION:

I. Background

This public workshop is intended to support FDA implementation of requirements for guidance development under section 3002 of the Cures Act (Pub. L. 114-255) and to meet a performance goal included in the sixth authorization of PDUFA VI. Section 3002 of Title III Subtitle A of the Cures Act directs FDA to develop patient-focused drug development guidance to address a number of areas including methodological approaches that may be used to develop and identify what is most important to patients with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient's disease; and approaches to identifying and developing methods to measure impacts to patients that will help facilitate collection of patient experience data in clinical trials.

In addition, FDA committed to meet certain performance goals under PDUFA VI. This reauthorization, part of the FDA Reauthorization Act of 2017 signed by the President on August 18, 2017, includes a number of performance goals and procedures that are documented in the PDUFA VI Commitment Letter, which is available at https://www.fda.gov/​downloads/​ForIndustry/​UserFees/​PrescriptionDrugUserFee/​UCM511438.pdf. These goal commitments were developed in consultation with patient and consumer advocates, healthcare professionals, and other public stakeholders, as part of negotiations with regulated industry. Section J.1 of the commitment letter, “Enhancing the Incorporation of the Patient's Voice in Drug Development and Decision-Making,” (https://www.fda.gov/​downloads/​ForIndustry/​UserFees/​PrescriptionDrugUserFee/​UCM511438.pdf) outlines work, including the development of a series of guidance documents and associated public workshops to facilitate the advancement and use of systematic approaches to collect and utilize robust and meaningful patient and caregiver input that can more consistently inform drug development, and, as appropriate, regulatory decision making.

Prior to the issuance of each guidance, as part of the development, FDA will conduct a public workshop to gather input from the wider community of patients, patient advocates, academic researchers, expert practitioners, drug developers, and other stakeholders.

II. Purpose and Scope of Meeting

FDA is announcing a 2-day public workshop to convene a discussion on: (1) Methodological approaches that may be used to develop and identify what is most important to patients and caregivers with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient's disease and (2) best practices for selecting, developing, or modifying fit-for-purpose COAs to measure the patient experience in clinical trials. The purpose of this public workshop is to obtain feedback from stakeholders on: (1) Methods to identify what is important to patients; (2) best practices for eliciting information on which aspects of disease symptoms, signs, impacts, and other issues are important and meaningful to patients; (3) measuring symptoms, signs, impacts and other issues of a disease or condition in a meaningful way; and (4) selecting, developing, or modifying fit-for-purpose COAs to measure the patient experience in clinical trials. FDA is seeking information and comments from a broad range of stakeholders, including patients, patient advocates, academic and medical researchers, expert practitioners, drug developers, and other interested persons. FDA will publish discussion documents outlining the topic areas that will be addressed in the draft guidances. These documents will be published approximately 1 month before the workshop date on the website at: https://www.fda.gov/​Drugs/​NewsEvents/​ucm607276.htm. FDA is interested in seeking information and comments on the approaches and considerations proposed in the discussion documents. FDA is also interested in seeking information and comments on additional examples, which could be included in guidance, where the approaches proposed in the discussion document have been successfully applied. After this public workshop, FDA will take into consideration the stakeholder input from the workshop and the public docket and publish draft guidance(s).

III. Participating in the Public Workshop

Registration: Interested parties are encouraged to register early. To register electronically, please visit: https://pfddmethods.eventbrite.com. Registration for in-person attendance will close on October 10, 2018. Registration for the webcast will remain open until the day of the meeting. Persons without access to the internet can call 240-402-6525 to register. 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.

Request for Oral Presentations: There will be time allotted during the workshop for open public comment. Sign-up for this session will be on a first-come, first-served basis on the day of the workshop. Individuals and organizations with common interests are urged to consolidate or coordinate, and request time for a joint presentation. No commercial or promotional material will be permitted to be presented or distributed at the public workshop.

Transcripts: As soon as a transcript is available, FDA will post it at https://www.fda.gov/​Drugs/​NewsEvents/​ucm607276.htm.

Dated: August 8, 2018.

Leslie Kux,

Associate Commissioner for Policy.