2020-20436. Eosinophilic Esophagitis: Developing Drugs for Treatment; Guidance for Industry; Availability

AGENCY:

Food and Drug Administration, Health and Human Services (HHS).

ACTION:

Notice of availability.

SUMMARY:

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Eosinophilic Esophagitis: Developing Drugs for Treatment.” This guidance is intended to describe the Agency's current thinking and recommended approach for the development of drugs and therapeutic biologics for the treatment of patients with eosinophilic esophagitis. This guidance finalizes the draft guidance of the same name issued February 6, 2019.

DATES:

The announcement of the guidance is published in the Federal Register on September 16, 2020.

ADDRESSES:

You may submit either electronic or written comments on Agency guidances at any time 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): 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-D-0177 for “Eosinophilic Esophagitis: Developing Drugs for Treatment.” 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 Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.

• 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.govinfo.gov/​content/​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, 240-402-7500.

You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)).

Submit written requests for single copies of this guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 20993-0002. Send one self-addressed adhesive label to assist that office in processing your requests. See the SUPPLEMENTARY INFORMATION section for electronic access to the guidance document.

FOR FURTHER INFORMATION CONTACT:

Erica Lyons, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 5373, Silver Spring, MD 20993-0002, 301-796-8023.

SUPPLEMENTARY INFORMATION:

I. Background

FDA is announcing the availability of a final guidance for industry entitled “Eosinophilic Esophagitis: Developing Drugs for Treatment.” This guidance provides FDA's current recommendations regarding clinical trials for drugs and therapeutic biologics for the treatment of eosinophilic esophagitis, including attributes of patients for enrollment, trial designs, efficacy considerations, safety assessments, and pediatric considerations.

This guidance finalizes the draft guidance of the same name issued February 6, 2019 (84 FR 2237). Changes to the guidance took into consideration public comments received. Major changes included:

• Removal of the recommendation for the proton pump inhibitor trial before patient enrollment;
• removal of the recommendation for exclusion of patients with significant strictures;
• addition of a clarification that FDA does not recommend a randomized withdrawal design for trials of drugs with the potential to induce immunogenicity;
• addition of a recommendation to report eosinophil density per square millimeter (mm²) as well as per high-power field;
• creation of a statistical section with recommendations on estimands; and
• addition of a clarification on the recommendation for the number of adolescent patients to be included in adult trials.

This guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The guidance represents the current thinking of FDA on “Eosinophilic Esophagitis: Developing Drugs for Treatment.” It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

This final guidance contains no collection of information. Therefore, clearance by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not required.

However, this final guidance refers to previously approved collections of information found in FDA regulations. These collections of information are subject to review by OMB under the PRA. The collections of information in 21 CFR part 312 (investigational new drug applications) have been approved under OMB control number 0910-0014. The collections of information in part 314 (new drug applications) have been approved under OMB control number 0910-0001. The collections of information in part 601 (biologics license applications) have been approved under OMB control number 0910-0338. The collections of information in parts 50 and 56 (protection of human subjects and institutional review boards) have been approved under OMB control number 0910-0130. The collections of information in the guidance for industry entitled “Expedited Programs for Serious Conditions—Drugs and Biologics” (available at https://www.fda.gov/​media/​86377/​download) have been approved under OMB control number 0910-0765.

III. Electronic Access

Persons with access to the internet may obtain the guidance at either https://www.fda.gov/​drugs/​guidance-compliance-regulatory-information/​guidances-drugs or https://www.regulations.gov.

Dated: September 11, 2020.

Lowell J. Schiller,

Principal Associate Commissioner for Policy.