1156 Federal Register / Vol. 84, No. 22 / Friday, February 1, 2019 / Notices

DEPARTMENT OF HEALTH AND confidential information that you or a available, you can provide this HUMAN SERVICES third party may not wish to be posted, information on the cover sheet and not such as medical information, your or in the body of your comments and you Food and Drug Administration anyone else’s Social Security number, or must identify this information as [Docket No. FDA–2015–D–2818] confidential business information, such ‘‘confidential.’’ Any information marked as a manufacturing process. Please note as ‘‘confidential’’ will not be disclosed Rare Diseases: Common Issues in that if you include your name, contact except in accordance with 21 CFR 10.20 Drug Development; Draft Guidance for information, or other information that and other applicable disclosure law. For Industry; Availability identifies you in the body of your more information about FDA’s posting comments, that information will be of comments to public dockets, see 80 AGENCY: Food and Drug Administration, posted on https://www.regulations.gov. FR 56469, September 18, 2015, or access HHS. • If you want to submit a comment the information at: https://www.gpo.gov/ ACTION: Notice of availability. with confidential information that you fdsys/pkg/FR-2015-09-18/pdf/2015- do not wish to be made available to the SUMMARY: The Food and Drug 23389.pdf. public, submit the comment as a Administration (FDA or Agency) is Docket: For access to the docket to written/paper submission and in the announcing the availability of a draft read background documents or the manner detailed (see ‘‘Written/Paper guidance for industry entitled ‘‘Rare electronic and written/paper comments Submissions’’ and ‘‘Instructions’’). Diseases: Common Issues in Drug received, go to https:// Development.’’ This draft guidance Written/Paper Submissions www.regulations.gov and insert the docket number, found in brackets in the assists sponsors of drug and biological Submit written/paper submissions as products intended to treat or prevent heading of this document, into the follows: ‘‘Search’’ box and follow the prompts rare diseases in conducting more • Mail/Hand Delivery/Courier (for and/or go to the Dockets Management efficient and successful development written/paper submissions): Dockets Staff, 5630 Fishers Lane, Rm. 1061, programs through discussions of Management Staff (HFA–305), Food and Rockville, MD 20852. selected issues commonly encountered Drug Administration, 5630 Fishers You may submit comments on any in rare disease drug development. This Lane, Rm. 1061, Rockville, MD 20852. draft guidance addresses the following • For written/paper comments guidance at any time (see 21 CFR important aspects of drug development: submitted to the Dockets Management 10.115(g)(5)). Adequate description and Staff, FDA will post your comment, as Submit written requests for single understanding of the disease’s natural well as any attachments, except for copies of the draft guidance to the history, adequate understanding of the information submitted, marked and Division of Drug Information, Center for pathophysiology of the disease and the identified, as confidential, if submitted Drug Evaluation and Research, Food drug’s proposed , as detailed in ‘‘Instructions.’’ and Drug Administration, 10001 New nonclinical pharmacotoxicology Instructions: All submissions received Hampshire Ave., Hillandale Building, considerations to support the proposed must include the Docket No. FDA– 4th Floor, Silver Spring, MD 20993– clinical investigation or investigations, 2015–D–2818 for ‘‘Rare Diseases: 0002, or Office of Communication, reliable endpoints and outcome Common Issues in Drug Development; Outreach, and Development, Center for assessment, standard of evidence to Draft Guidance for Industry; Biologics Evaluation and Research, establish safety and effectiveness, drug Availability.’’ Received comments will Food and Drug Administration, 10903 manufacturing considerations during be placed in the docket and, except for New Hampshire Ave., Bldg. 71, Rm. drug development, participation of those submitted as ‘‘Confidential 3128, Silver Spring, MD 20993–0002. patients, caretakers, and advocates in Submissions,’’ publicly viewable at Send one self-addressed adhesive label development programs, and interactions https://www.regulations.gov or at the to assist that office in processing your with the Agency. This guidance revises Dockets Management Staff between 9 requests. See the SUPPLEMENTARY and replaces the draft guidance of the a.m. and 4 p.m., Monday through INFORMATION section for electronic same name issued on August 17, 2015. Friday. access to the draft guidance document. DATES: Submit either electronic or • Confidential Submissions—To FOR FURTHER INFORMATION CONTACT: written comments on the draft guidance submit a comment with confidential Lucas Kempf, Center for Drug by April 2, 2019 to ensure that the information that you do not wish to be Evaluation and Research, Food and Agency considers your comment on this made publicly available, submit your Drug Administration, 10903 New draft guidance before it begins work on comments only as a written/paper Hampshire Ave., Bldg. 22, Rm. 6460, the final version of the guidance. submission. You should submit two Silver Spring, MD 20993–0002, 301– ADDRESSES: You may submit comments copies total. One copy will include the 796–1140, or Stephen Ripley, Center for on any guidance at any time as follows: information you claim to be confidential Biologics Evaluation and Research, with a heading or cover note that states Food and Drug Administration, 10903 Electronic Submissions ‘‘THIS DOCUMENT CONTAINS New Hampshire Ave., Bldg. 71, Rm. Submit electronic comments in the CONFIDENTIAL INFORMATION.’’ The 7301, Silver Spring, MD 20993–0002. following way: Agency will review this copy, including SUPPLEMENTARY INFORMATION: • Federal eRulemaking Portal: the claimed confidential information, in https://www.regulations.gov. Follow the its consideration of comments. The I. Background instructions for submitting comments. second copy, which will have the FDA is announcing the availability of Comments submitted electronically, claimed confidential information a draft guidance for industry entitled including attachments, to https:// redacted/blacked out, will be available ‘‘Rare Diseases: Common Issues in Drug www.regulations.gov will be posted to for public viewing and posted on Development.’’ This draft guidance the docket unchanged. Because your https://www.regulations.gov. Submit assists sponsors of drug and biological comment will be made public, you are both copies to the Dockets Management products intended to treat or prevent solely responsible for ensuring that your Staff. If you do not wish your name and rare diseases in conducting more comment does not include any contact information to be made publicly efficient and successful development

VerDate Sep<11>2014 21:23 Jan 31, 2019 Jkt 247001 PO 00000 Frm 00110 Fmt 4703 Sfmt 4703 E:\FR\FM\01FEN1.SGM 01FEN1 Federal Register / Vol. 84, No. 22 / Friday, February 1, 2019 / Notices 1157

programs through a discussion of information found in FDA regulations. have been approved under OMB control selected issues commonly encountered These collections of information are number 0910–0167. in rare disease drug development. This subject to review by the Office of The collections of information under draft guidance addresses the following Management and Budget (OMB) under § 314.80 for submitting postmarketing important aspects of drug development: the Paperwork Reduction Act of 1995 reporting of adverse drug experiences • Adequate description and (44 U.S.C. 3501–3520). have been approved under OMB control understanding of the disease’s natural The following collections of number 0910–0230. history information in the draft guidance have The collections of information under • Adequate understanding of the been approved under OMB control §§ 312.47 and 312.82 for requesting pathophysiology of the disease and number 0910–0001: meetings with FDA about drug the drug’s proposed mechanism of • Submitting under 21 CFR development programs have been action 314.50(c)(1)(iv) and (d)(3) approved under OMB control number • Nonclinical pharmacotoxicology (§ 314.50(c)(1)(iv) and (d)(3)) a summary 0910–0429. considerations to support the of the nonclinical and The following collections of proposed clinical investigation or section and the human information have been approved under investigations and bioavailability OMB control number 0910–0765: (1) • Reliable endpoints and outcome section of new drug application (NDAs); Requests under 21 CFR part 314, assessment • Submitting under § 314.50(d)(1)(i) subpart H to grant accelerated approval • Standard of evidence to establish chemistry, manufacturing, and controls for INDs to treat rare diseases that are safety and effectiveness serious or life threatening and (2) as a • information, including the drug Drug manufacturing considerations substance, for the content and format of basis for accelerated approval requests, during drug development a NDA for rare diseases; and submissions of evidence to support that This guidance revises and replaces the • Submitting under § 314.50(d)(5) and an endpoint reasonably likely to predict draft guidance for industry of the same (d)(5)(iv) clinical data of a drug, clinical benefit. name issued on August 17, 2015 (80 FR including a description of any other III. Electronic Access 49246). This revision includes the data information relevant to an following: Persons with access to the internet evaluation of the safety and may obtain the guidance at https:// • Updates to the natural history section effectiveness of a drug. www.fda.gov/Drugs/ • Inclusion of issues for evaluation of The following collections of biomarkers for consideration as GuidanceCompliance information in the draft guidance have RegulatoryInformation/Guidances/ surrogate endpoints been approved under OMB control • Description of nonclinical flexibility default.htm, https://www.fda.gov/ • number 0910–0014: Biologics;BloodVaccines/ Additional information on historical • Submitting under 21 CFR (external) controls and early GuidanceCompliance 312.23(a)(6)(i) (§ 312.23(a)(6)(i)) a RegulatoryInformation/Guidances/ randomization protocol for the duration of a trial and • Addition of safety section default.htm, or https:// the criteria to enter a trial and under • Retitled Chemistry, Manufacturing, www.regulations.gov. § 312.23(a)(6)(i), (a)(6)(iii)(d) and (g) a and Controls section to description of an estimate of patients Dated: January 16, 2019. Pharmaceutical Quality that will be involved in a trial, Leslie Kux, Considerations Associate Commissioner for Policy. • Additional information on changes to including a description of the safety drug substance or manufacturing exclusions and a description of clinical [FR Doc. 2019–00677 Filed 1–31–19; 8:45 am] process with clarification on areas of procedures, laboratory, or other BILLING CODE 4164–01–P methods; flexibility • • Addition of a considerations section Submitting under § 312.23(a)(3)(i) a DEPARTMENT OF HEALTH AND addressing several topics including brief introductory statement and general HUMAN SERVICES participation of patients, caretakers, investigational plan, including the route and advocates; consideration of of administration of a drug; • Food and Drug Administration pediatric issues; and interactions with Submitting under § 312.23(a)(7) and FDA (a)(7)(iv)(a) chemistry, manufacturing, [Docket No. FDA–2018–D–1387] This draft guidance is being issued and controls information for the content Safety and Performance Based consistent with FDA’s good guidance and format of an investigational new Pathway; Guidance for Industry and practices regulation (21 CFR 10.115). drug application (IND) and the safety Food and Drug Administration Staff; The draft guidance, when finalized, will and effectiveness of such information; Availability represent the current thinking of FDA • Submitting under § 312.23(a)(8) and on ‘‘Rare Diseases: Common Issues in (a)(8)(i) pharmacology, toxicology, and AGENCY: Food and Drug Administration, Drug Development.’’ It does not drug disposition information for rare HHS. diseases; establish any rights for any person and ACTION: Notice of availability. is not binding on FDA or the public. • Submitting under 312.23(a)(10)(iii) You can use an alternative approach if plans for assessing pediatric safety and SUMMARY: The Food and Drug it satisfies the requirements of the effectiveness; Administration (FDA or Agency) is applicable statutes and regulations. This • Submitting under § 312.32(c)(1) IND announcing the availability of a final guidance is not subject to Executive safety reports; guidance entitled ‘‘Safety and Order 12866. • Submissions under §§ 312.305(b) Performance Based Pathway,’’ which and 312.310(b) for expanded access uses was previously issued in draft version II. The Paperwork Reduction Act of and treatment of an individual patient. entitled ‘‘Expansion of the Abbreviated 1995 The collections of information in 21 510(k) Program: Demonstrating This draft guidance refers to CFR part 316 for submitting the content Substantial Equivalence Through previously approved collections of and format of NDAs for orphan drugs Performance Criteria.’’ This final

VerDate Sep<11>2014 21:23 Jan 31, 2019 Jkt 247001 PO 00000 Frm 00111 Fmt 4703 Sfmt 4703 E:\FR\FM\01FEN1.SGM 01FEN1