[Federal Register Volume 61, Number 16 (Wednesday, January 24, 1996)]
[Notices]
[Pages 1939-1940]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 96-943]



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DEPARTMENT OF HEALTH AND HUMAN SERVICES
[Docket No. 95D-0386]


Guidance for Industry; Content and Format of Investigational New 
Drug Applications (IND's) for Phase 1 Studies of Drugs, Including Well-
Characterized, Therapeutic, Biotechnology-Derived Products; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a guidance entitled ``Guidance for Industry; Content 
and Format of Investigational New Drug Applications (IND's) for Phase 1 
Studies of Drugs, Including Well-Characterized, Therapeutic, 
Biotechnology-derived Products.'' The guidance clarifies data 
requirement issues related to the initial entry of an unapproved drug 
into human studies in the United States. The guidance is intended to 
expedite the entry of new drugs into clinical studies by eliminating 
ambiguities in IND requirements and by decreasing inconsistencies in 
IND reviews.

DATES: Written comments on the guidance may be submitted at any time.

ADDRESSES: Submit written requests for single copies of the guidance 
entitled ``Guidance for Industry; Content and Format of Investigational 
New Drug Applications (IND's) for Phase 1 Studies of Drugs, Including 
Well-Characterized, Therapeutic, Biotechnology-derived Products'' to 
the Consumer Affairs Branch (formerly the CDER Executive Secretariat 
Staff), Center for Drug Evaluation and Research (HFD-210), Food and 
Drug Administration, 7500 Standish Pl., Rockville, MD 20855, or the 
Congressional and Consumer Affairs Branch, Center for Biologics 
Evaluation and Research (HFM-12), Food and Drug Administration, 1401 
Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-594-1800 or 
800-835-4709. Send two self-addressed adhesive labels to assist the 
offices in processing your requests. A copy of the guidance document is 
also available from CDER's FAX On Demand. To obtain a copy from FAX On 
Demand, call 1-800-342-2722 or locally 301-827-0577. An electronic 
version of the guidance document is also available via Internet. 
Requesting persons should connect to the CDER file transfer protocol 
(FTP) server (CDVS2.CDER.FDA.GOV) using the FTP protocol. The guidance 
is available in WordPerfect versions 5.2 and 6.0. Submit written 
comments on the guidance to the Dockets Management Branch (HFA-305), 
Food and Drug Administration, 12420 Parklawn Dr., rm. 1-23, Rockville, 
MD 20857. Requests and comments should be identified with the docket 
number found in brackets in the heading of this document. A copy of the 
guidance and received comments are available for public examination in 
the Dockets Management Branch between 9 a.m. and 4 p.m., Monday through 
Friday.

FOR FURTHER INFORMATION CONTACT: Murray M. Lumpkin, Center for Drug 
Evaluation and Research (HFD-2), Food and Drug Administration, 1451 
Rockville Pike, Rockville, MD 20852, 301-594-6740, or Rebecca Devine, 
Center for Biologics Evaluation and Research (HFM-10), Food and Drug 
Administration, 1401 Rockville Pike, Rockville, MD 20852, 301-827-0373.
SUPPLEMENTARY INFORMATION: FDA is announcing the availability of a 
guidance entitled ``Guidance for Industry; Content and Format of 
Investigational New Drug Applications (IND's) for Phase 1 Studies of 
Drugs, Including Well-Characterized, Therapeutic, Biotechnology-derived 
Products.'' Any use in humans in the United States of a drug product 
not 

[[Page 1940]]
previously authorized for marketing in the United States first requires 
the submission of an IND to FDA. FDA regulations in Secs. 312.22 and 
312.23 (21 CFR 312.22 and 312.23) contain the general principles 
underlying the IND submission and the general requirements for an IND's 
content and format. This guidance clarifies these requirements related 
to the initial entry of an unapproved drug, including well-
characterized, therapeutic, biotechnology-derived products. -
    Because of the manufacturing and toxicologic differences between 
well-characterized, therapeutic, biotechnology-derived products and 
other biologic products, the guidance only applies to drugs that are 
not also biologics and to well-characterized, therapeutic, 
biotechnology-derived biologic products. For products not covered by 
the guidance the center responsible for the product should be contacted 
for guidance.
    The requirements in Secs. 312.22 and 312.23 permit a great deal of 
flexibility in the amount and depth of data to be submitted in an IND, 
depending in large part on the phase of the investigation and the 
specific human testing proposed. In some cases, the extent of that 
flexibility and the limited data needed has not been appreciated. FDA 
believes that clarification of these requirements will decrease the 
submission of unnecessary data and help expedite the entry of new drugs 
into clinical testing by increasing transparency and reducing ambiguity 
and inconsistencies. These clarifications will reduce the amount of 
information ordinarily submitted in an IND, yet continue to provide the 
agency with the data it needs to assess the safety of the proposed 
Phase 1 study.
    The most significant clarifications contained in the guidance are 
FDA's willingness to accept an integrated summary report of toxicology 
findings as initial support for human studies based upon unaudited, 
draft, toxicological reports of completed animal studies, as well as 
specific manufacturing data that FDA will accept as appropriate for a 
Phase 1 study. This guidance applies equally to both commercial and 
individual investigator sponsors of IND's.
    As part of the President's Reinventing Government Initiative, FDA 
has been reviewing its regulatory processes to determine which 
requirements could be reduced or eliminated without lowering health and 
safety standards. These clarifications of the IND requirements have 
been identified during this review and should significantly reduce the 
burden on industry regarding data submitted in Phase 1 IND's without 
sacrificing the quality of FDA's review of the IND.
    In addition to this guidance, FDA is preparing an advance notice of 
proposed rulemaking (ANPR) that will describe proposed revisions to the 
IND regulations that FDA is contemplating to facilitate further the 
entry of drugs into clinical studies so that safe and effective drugs 
can be made available in the United States more quickly. The ANPR is 
expected to be published in the first quarter of 1996 and will address 
the possibility of: (1) A specific single dose IND with limited data 
requirements and (2) reducing or eliminating the IND submission 
requirements for individual investigators who would like to use 
products already in Phase 2 of commercial development.
     Although this guidance does not create or confer any rights for or 
on any person and does not operate to bind FDA or the industry, it does 
represent the agency's current thinking on data requirement issues 
related to the initial entry of an unapproved drug into human studies 
in the United States.
    Although the guidance is being implemented immediately because it 
merely clarifies existing regulations and is expected to reduce the 
data submission burden on the industry, FDA is soliciting comments on 
the guidance that will be taken into account in making further 
revisions or clarifications to the IND process. FDA is particularly 
interested in comments on how the guidance could be extended to cover 
biological products other than well-characterized, therapeutic, 
biotechnology-derived products or whether a separate guidance should be 
developed for those products.
    Interested persons may, at any time, submit to the Dockets 
Management Branch (address above) written comments on the guidance. Two 
copies of any comments are to be submitted, except that individuals may 
submit one copy. Comments are to be identified with the docket number 
found in brackets in the heading of this document. The guidance and 
received comments may be seen in the office above between 9 a.m. and 4 
p.m., Monday through Friday. -

    Dated: January 8, 1996.
William B. Schultz,
Deputy Commissioner for Policy.
[FR Doc. 96-943 Filed 1-23-96; 8:45 am]
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