75566 Federal Register / Vol. 79, No. 243 / Thursday, December 18, 2014 / Notices 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2014–D–0313] 

Agency Information Collection 

Activities; Submission for Office of 

Management and Budget Review; 

Comment Request; Draft Guidance for 

Industry, Researchers, Patient Groups, 

and Food and Drug Administration 

Staff on Meetings With the Office of 

Orphan Products Development 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice. 

SUMMARY: The Food and Drug 

Administration (FDA) is announcing 

that a proposed collection of 

information has been submitted to the 

Office of Management and Budget 

(OMB) for review and clearance under 

the Paperwork Reduction Act of 1995. 

DATES: Fax written comments on the 

collection of information by January 20, 

2015. 

ADDRESSES: To ensure that comments on 

the information collection are received, 

OMB recommends that written 

comments be faxed to the Office of 

Information and Regulatory Affairs, 

OMB, Attn: FDA Desk Officer, FAX: 

202–395–7285, or emailed to 

oira_submission@omb.eop.gov. All 

comments should be identified with the 

OMB control number 0910–NEW and 

title, ‘‘Draft Guidance for Industry, 

Researchers, Patient Groups, and Food 

and Drug Administration Staff on 

Meetings With the Office of Orphan 

Products Development.’’ Also include 

the FDA docket number found in 

brackets in the heading of this 

document. 

FOR FURTHER INFORMATION CONTACT: FDA 

PRA Staff, Office of Operations, Food 

and Drug Administration, 8455 

Colesville Rd., COLE–14526, Silver 

Spring, MD 20993–0002, 

PRAStaff@fda.hhs.gov. 

SUPPLEMENTARY INFORMATION: In 

compliance with 44 U.S.C. 3507, FDA 

has submitted the following proposed 

collection of information to OMB for 

review and clearance. Draft Guidance 

for Industry, Researchers, Patient 

Groups, and Food and Drug 

Administration Staff on Meetings With 

the Office of Orphan Products 

Development—(OMB Control Number 

0910–NEW). 

FDA is issuing a draft guidance on the 

procedures for requesting meetings with 

the Office of Orphan Products 

Development (OOPD) on issues related 

to orphan drug designation requests, 

Humanitarian Use Device (HUD) 

designation requests, rare pediatric 

disease designation requests, funding 

opportunities through the Orphan 

Products Grants Program and the 

Pediatric Device Consortia Grants 

Program, and orphan product patientrelated topics of concern. The draft 

guidance describes procedures for 

requesting, scheduling, conducting, and 

documenting such meetings. 

The draft guidance describes three 

collections of information: (1) The 

submission of a meeting request (for 

informal and formal meetings), (2) the 

submission of a meeting package (for 

formal meetings), and (3) the 

submission of draft meeting minutes (for 

formal and certain informal meetings). 

These collections of information will be 

used by the Agency to schedule and 

prepare for meetings on the issues 

described previously in this document 

and will provide for more productive 

meetings with stakeholders. This draft 

guidance refers to previously approved 

collections of information found in FDA 

regulations. Agency regulations at part 

316 (21 CFR part 316) describe 

information that should be submitted in 

support of an orphan drug designation 

request. The information collection 

provisions of part 316 have been 

approved under OMB control number 

0910–0167. Agency regulations at 

§ 814.102 (21 CFR 814.102) describe 

information that should be submitted in 

support of a HUD designation request. 

The information collection provisions 

of § 814.102 have been approved under 

OMB control number 0910–0332. 

A. Request for a Meeting 

Under the draft guidance, a 

stakeholder interested in meeting with 

OOPD should submit a meeting request: 

• For specific designation requests or 

grant applications, by emailing the 

identified point of contact for the 

designation request or grant application 

with the subject heading ‘‘Meeting 

Request’’; or 

• For other issues, by emailing the 

general OOPD inbox at 

orphan@fda.hhs.gov with the subject 

heading ‘‘Meeting Request’’ or by 

emailing the point of contact for each 

OOPD Program Area listed in the 

‘‘Contact FDA’’ section of the OOPD’s 

Web site (http://www.fda.gov/orphan), 

again with the subject heading ‘‘Meeting 

Request.’’ In the draft guidance, FDA 

recommends that the meeting request, at 

a minimum, include (1) a brief 

statement of the meeting purpose, (2) 

whether the stakeholder prefers an 

informal or formal meeting, (3) 

suggested dates and times for the 

meeting, (4) preferred format of the 

meeting, and (5) the email address(es) to 

which OOPD should send a response to 

the meeting request (if different from the 

email address from which the request 

was sent) and telephone number for the 

primary contact for the stakeholder. 

Before scheduling a meeting, OOPD may 

ask the stakeholder for more 

information about the proposed meeting 

to help determine whether an informal 

or formal meeting is most appropriate 

and who from OOPD should attend. For 

informal meetings, the information in 

the meeting request may suffice, 

although OOPD may ask for 

supplemental information via email or 

telephone. 

B. Meeting Package 

If a formal meeting is scheduled, FDA 

recommends that stakeholders submit a 

meeting package to OOPD at least 2 

weeks before the meeting. Stakeholders 

are encouraged to submit the package 

electronically by email to the OOPD 

program contact who scheduled the 

meeting. In the draft guidance, FDA 

recommends that the meeting package 

contain the following information: (1) 

The date, time, and subject of the 

meeting; (2) an explanation of the 

meeting purposes; (3) basic information 

about the product to be discussed (e.g., 

product name or identifier, designation 

or application number (if applicable), 

proposed rare disease or condition, brief 

background about the product); (4) 

proposed meeting agenda; (5) any data, 

information, or presentation materials to 

support the discussion (if needed); and 

(6) a list of all individuals, with their 

titles and affiliations, who are expected 

to participate in the meeting on behalf 

of the stakeholder. 

C. Draft Meeting Minutes 

Under the draft guidance, a 

stakeholder should prepare a draft of 

summary meeting minutes for all formal 

meetings and certain informal meetings. 

These draft minutes should be sent to 

the OOPD program contact by email 

with the subject heading ‘‘Draft Meeting 

Minutes.’’ The draft minutes should 

summarize the meeting discussion 

points, agreements, disagreements, and 

action items. OOPD will review and 

provide any revisions to the draft 

meeting minutes via email, and the 

stakeholder will then either accept the 

version as final and notify OOPD to that 

effect or will follow-up with questions 

and/or further revisions. 

Description of Respondents: 

Individuals from industry, researchers, 

patient groups, and other stakeholders 

who seek a meeting with OOPD 

regarding orphan drug designation 

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Federal Register / Vol. 79, No. 243 / Thursday, December 18, 2014 / Notices 75567 

requests, HUD designation requests, rare 

pediatric disease designation requests, 

funding opportunities through the 

Orphan Products Grants Program and 

the Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. 

Burden estimate: Table 1 of this 

document provides an estimate of the 

annual reporting burden for the 

preparation and submission of meeting 

requests, meeting packages, and meeting 

minutes under the guidance. 

Request for a meeting: Based upon 

information collected from OOPD 

program areas, approximately 2,120 

informal and 46 formal meetings were 

requested with OOPD in fiscal year (FY) 

2013 regarding orphan drug designation 

requests, HUD designation requests, rare 

pediatric disease designation requests, 

funding opportunities through the 

Orphan Products Grants Program and 

the Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. FDA anticipates that the 

number of meeting requests and 

stakeholders will remain the same or 

will only slightly increase, and therefore 

estimates the total number of meeting 

requests will be 2,166 annually (2120 

informal and 46 formal meetings). The 

hours per response, which is the 

estimated number of hours that a 

stakeholder would spend preparing the 

information to be submitted with a 

meeting request in accordance with the 

draft guidance, is estimated to be 

approximately 3 hours for informal 

meetings and approximately 10 hours 

for formal meetings. Based on FDA’s 

experience, the Agency expects that it 

will take stakeholders this amount of 

time to gather and copy brief statements 

about the product and a description of 

the purpose and details of the meeting. 

Therefore, the Agency estimates that 

stakeholders will spend 6,820 hours per 

year (6,360 hours for informal meetings 

and 460 hours for formal meetings) 

preparing meeting requests to OOPD 

regarding orphan drug designation 

requests, HUD designation requests, rare 

pediatric disease designation requests, 

funding opportunities through the 

Orphan Products Grants Program and 

the Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. 

Meeting package: Based upon 

information collected from OOPD 

program areas, OOPD held 

approximately 46 formal meetings in FY 

2013 regarding orphan drug designation 

requests, HUD designation requests, rare 

pediatric disease designation requests, 

funding opportunities through the 

Orphan Products Grants Program and 

the Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. FDA anticipates that the 

number of formal meetings, and 

therefore meeting packages, may 

increase only slightly as a result of this 

guidance; thus, the Agency estimates 

that the total responses will be 46 

annually. As stated previously, it is 

current practice for stakeholders to 

submit meeting packages to the Agency 

in advance of any such formal meeting. 

The hours per response, which is the 

estimated number of hours that a 

stakeholder would spend preparing the 

meeting package in accordance with this 

draft guidance, is estimated to be 

approximately 18 hours. Based on 

FDA’s experience, the Agency expects it 

will take stakeholders this amount of 

time to gather and copy brief statements 

about the product, a description of 

details for the anticipated meeting, and 

data and information that generally 

would already have been compiled for 

submission to the Agency. Therefore, 

the Agency estimates that stakeholders 

will spend 828 hours per year 

submitting meeting packages to the 

Agency prior to a formal meeting 

regarding orphan drug designation 

requests, HUD designation requests, rare 

pediatric disease designation requests, 

funding opportunities through the 

Orphan Products Grants Program and 

the Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. 

Draft meeting minutes: Based upon 

information collected from OOPD 

program areas, OOPD received 

approximately 46 draft meeting minutes 

for formal meetings and 21 draft 

meeting minutes for informal meetings 

in FY 2013 regarding orphan drug 

designation requests, HUD designation 

requests, rare pediatric disease 

designation requests, funding 

opportunities through the Orphan 

Products Grants Program and the 

Pediatric Device Consortia Grants 

Program, and orphan product patientrelated issues. FDA anticipates that the 

number of stakeholders submitting draft 

meeting minutes may remain the same 

or increase only slightly; thus, the 

Agency estimates that the total number 

of respondents will be 67 annually. As 

stated previously, it is current practice 

for stakeholders to submit draft meeting 

minutes to the Agency after all formal 

meetings and certain informal meetings. 

The hours per response, which is the 

estimated number of hours that a 

stakeholder would spend preparing 

draft meeting minutes in accordance 

with this draft guidance, is estimated to 

be approximately 8 hours. Based on 

FDA’s experience, the Agency expects it 

will take stakeholders this amount of 

time to summarize the meeting 

discussion points, agreements, 

disagreements, and action items. 

Therefore, the Agency estimates that 

stakeholders will spend 536 hours per 

year submitting draft meeting minutes 

to the Agency documenting the meeting 

outcomes, agreements, disagreements, 

and action items as follow-up to all 

formal and certain informal meetings. 

In the Federal Register of April 9, 

2014 (79 FR 19623), FDA published a 

60-day notice requesting public 

comment on the proposed collection of 

information. No comments were 

received. 

FDA estimates the burden of this 

collection of information as follows: 

TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN

Activity Number of 

respondents 

Number of 

responses per 

respondent 

Total annual 

responses 

Average 

burden per 

response 

Total hours 

Meeting Requests (informal) ................................................ 2120 1 2120 3 6360 

Meeting Requests (formal) .................................................. 46 1 46 10 460 

Meeting Packages ............................................................... 46 1 46 18 828 

Meeting Minutes ................................................................... 67 1 67 8 536 

Total .............................................................................. ........................ ........................ ........................ ........................ 8,184 

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75568 Federal Register / Vol. 79, No. 243 / Thursday, December 18, 2014 / Notices 

1Available at http://www.fda.gov/forindustry/

datastandards/studydatastandards/default.htm. 

2For purposes of this guidance, the study start 

date for clinical studies is the earliest date of 

informed consent among any subject that enrolled 

in the study. For example, see Study Start Date (also 

known as the study initiation date) in the SDTM 

Trial Summary Domain (TSPARMCD = SSTDTC). 

For nonclinical studies, the study start date is the 

date on which the study protocol or plan is 

Dated: December 11, 2014. 

Leslie Kux, 

Associate Commissioner for Policy. 

[FR Doc. 2014–29612 Filed 12–17–14; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2012–D–0097] 

Providing Regulatory Submissions in 

Electronic Format—Standardized 

Study Data; Guidance for Industry; 

Availability 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice. 

SUMMARY: The Food and Drug 

Administration (FDA) is announcing the 

availability of a guidance for industry 

entitled ‘‘Providing Regulatory 

Submissions in Electronic Format— 

Standardized Study Data.’’ The 

guidance announced in this document 

is being issued in accordance with the 

Food and Drug Administration Safety 

and Innovation Act (FDASIA), which 

amended the Federal Food, Drug, and 

Cosmetic Act (the FD&C Act) to require 

that certain submissions under the 

FD&C Act and Public Health Service Act 

(PHS Act) be submitted in electronic 

format, beginning no earlier than 24 

months after issuance of final guidance 

on that topic. The guidance describes 

how FDA plans to implement the 

requirements for the electronic 

submission of standardized study data 

contained in certain submissions under 

new drug applications (NDAs), 

abbreviated new drug applications 

(ANDAs), biologics license applications 

(BLAs), and investigational new drug 

applications (INDs). This finalizes the 

revised draft guidance that was issued 

on February 6, 2014. 

DATES: Submit either electronic or 

written comments on Agency guidances 

at any time. 

ADDRESSES: Submit written requests for 

single copies of the documents 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; or 

the Office of Communication, Outreach 

and Development, Center for Biologics 

Evaluation and Research (CBER), 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 documents. 

Submit electronic comments on the 

guidance to http://www.regulations.gov. 

Submit written comments to the 

Division of Dockets Management (HFA– 

305), Food and Drug Administration, 

5630 Fishers Lane, Rm. 1061, Rockville, 

MD 20852. 

FOR FURTHER INFORMATION CONTACT: Ron 

Fitzmartin, Center for Drug Evaluation 

and Research, Food and Drug 

Administration, 10903 New Hampshire 

Ave., Bldg. 51, Rm. 1192, Silver Spring, 

MD 20993–0002, ronald.fitzmartin@

fda.hhs.gov; or Stephen Ripley, Center 

for Biologics Evaluation and Research, 

Food and Drug Administration, Bldg. 

71, rm. 7301, Silver Spring, MD 20993, 

stephen.ripley@fda.hhs.gov. 

SUPPLEMENTARY INFORMATION: 

I. Background 

FDASIA (Pub. L. 112–144), signed by 

the President on July 9, 2012, amended 

the FD&C Act to add section 745A, 

entitled ‘‘Electronic Format for 

Submissions.’’ Section 745A(a)(1) of the 

FD&C Act requires that submissions 

under section 505(b), (i), or (j) of the 

FD&C Act (21 U.S.C. 355(b), (i), or (j)) 

and submissions under sections 351(a) 

or (k) of the PHS Act (42 U.S. C. 262(a) 

or (k)) be submitted to FDA in electronic 

format no earlier than 24 months after 

FDA issues final guidance on that topic. 

In accordance with section 745A(a)(1) 

of the FD&C Act, FDA is issuing this 

guidance, announcing its determination 

that the study data contained in the 

submission types identified in this 

guidance must be submitted 

electronically (except for submissions 

that are exempted), in a format that FDA 

can process, review, and archive. 

Currently, the Agency can process, 

review, and archive electronic 

submissions of study data that use the 

standards, formats, and terminologies 

specified in the Data Standards Catalog 1

posted to the FDA’s Study Data 

Standards Resources Web page. 

In the Federal Register of February 6, 

2014 (79 FR 7201), FDA announced the 

availability of the revised draft guidance 

entitled ‘‘Providing Regulatory 

Submissions in Electronic Format— 

Standardized Study Data.’’ The 

comment period on the revised draft 

guidance ended on May 6, 2014. We 

reviewed all comments received on the 

draft guidance and revised several 

sections of the guidance. The updates 

include: 

Section II.A: (1) Clarified which INDs 

and BLAs are addressed in this 

guidance. Specifically, a footnote was 

added to clarify the meaning of 

‘‘certain’’ in the context of BLAs and 

INDs and states that the guidance is not 

applicable to INDs for devices that are 

regulated by CBER as biological 

products under section 351 of the PHS 

Act and to INDs that are 

noncommercial. Further, the guidance is 

not applicable to those devices that are 

regulated by CBER as biological 

products under section 351 of the PHS 

Act. Examples are provided in this 

regard. (2) Clarified that both clinical 

and nonclinical study data are within 

the scope of the guidance. 

Section II.C: (1) Clarified that the 

Agency may refuse to file an NDA or 

BLA or refuse to receive an ANDA 

containing study data that are not in 

conformance with the required 

standards. (2) Clarified that both the 

Clinical Data Interchange Standards 

Consortium (CDISC) Study Data 

Tabulation Model (SDTM) and Standard 

Exchange for Nonclinical Data (SEND) 

are examples of study data standards for 

tabulations data. (3) Clarified that some 

controlled terminologies are extensible 

and permit additions to existing code 

lists. It is the expectation that sponsors 

or applicants will use the controlled 

terminologies maintained by external 

organizations as the standard. 

Section II.D: Clarified the waiver 

process. 

Section II.E: (1) Clarified that FDA 

recognizes that version updates to 

standards may be released in the 

interval between the start of a study and 

the submission of study data to the 

Agency and the Data Standards Catalog 

may list more than one version of a 

supported standard. (2) Specified the 

definition of study start date for both 

clinical and nonclinical studies. (3) 

Revised terminology to more clearly 

state when a particular requirement 

becomes required. 

This guidance implements the 

electronic submission requirements of 

section 745A(a) of the FD&C Act by 

specifying the format for electronic 

submission of study data contained in 

NDA, ANDA, BLA, and IND 

submissions. With the publication of 

this Federal Register notice of 

availability, all studies with a start 

date 2 24 months or later after the 

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