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Food and Drug Administration, HHS.
Notice of availability.
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.” The purpose of this draft guidance is to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-investigational new drug application (pre-IND) meetings. Drug development for rare diseases has many challenges related to the nature of these diseases. This draft guidance is intended to advance and facilitate the development of drugs and biological products for the treatment of rare diseases.
Submit either electronic or written comments on the draft guidance by December 17, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
You may submit comments on any guidance at any time as follows:
Submit electronic comments in the following way:
Submit written/paper submissions as follows:
Instructions: All submissions received must include the Docket No. FDA-2018-D-3268 for “Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings; Draft Guidance for Industry.” 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.
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.
You may submit comments on any guidance at any time (see 21 CFR 10.115(g)(5)).
Submit written requests for single copies of the draft guidance to the Division of Drug Information, Center for Drug Evaluation and Research, Food and Drug Administration, 10001 New Hampshire Ave., Hillandale Building, 4th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, or Office of Communication, Outreach, and Development, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, 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 draft guidance document.
Lucas Kempf, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6460, Silver Spring, MD 20993-0002, 301-796-1140; or Stephen Ripley, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.
FDA is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.” This guidance is intended to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-IND meetings through a discussion of selected issues commonly encountered in the early phases of rare disease drug development. Although these issues are encountered in other drug development programs, the issues are frequently more difficult to address in the context of a rare disease than in the context of a common disease, of which there is often greater and more widespread medical experience. A rare disease is defined in section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(2)) as a disease or condition that affects fewer than 200,000 people in the United States or affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug. Most rare diseases affect far fewer than 200,000 people.
Most rare disorders are serious conditions with no approved treatments, and rare disease patients often have considerable unmet medical needs. Collectively, rare diseases are highly diverse. FDA is committed to helping sponsors of drugs for rare diseases have successful pre-IND meetings that address the particular challenges posed by each drug.
This guidance addresses the following important topics related to pre-IND meetings:
Early consideration of these issues allows sponsors to efficiently and adequately plan for a productive pre-IND meeting with FDA.
This draft guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on “Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.” 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. This guidance is not subject to Executive Order 12866.
This guidance refers to previously approved collections of information that are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The collections of information in 21 CFR parts 312 and 314 have been approved under OMB control numbers 0910-0014 and 0910-0001, respectively. The collection of information resulting from the draft guidance for industry “Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products” (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm590547.pdf) has been approved under OMB control number 0910-0429. The collection of information resulting from the guidance for industry “Expedited Programs for Serious Conditions—Drugs and Biologics” (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm358301.pdf) has been approved under OMB control number 0910-0765.
Persons with access to the internet may obtain the draft guidance at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or https://www.regulations.gov.
Dated: October 10, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-22435 Filed 10-15-18; 8:45 am]
BILLING CODE 4164-01-P
6 comments have been received at Regulations.gov.
Agencies review all submissions and may choose to redact, or withhold, certain submissions (or portions thereof). Submitted comments may not be available to be read until the agency has approved them.