Document ID: FDA-2014-N-0386-0005
Agency: fda
Document Type: Notice
Title: Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Orphan Drugs
Posted Date: 2020-12-23T05:00Z

[Federal Register Volume 85, Number 247 (Wednesday, December 23, 2020)]
[Notices]
[Pages 83971-83972]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-28349]

-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0386]

Agency Information Collection Activities; Submission for Office 
of Management and Budget Review; Comment Request; Orphan Drugs

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA, Agency, or we) 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: Submit written comments (including recommendations) on the 
collection of information by January 22, 2021.

ADDRESSES: To ensure that comments on the information collection are 
received, OMB recommends that written comments be submitted to https://www.reginfo.gov/public/do/PRAMain. Find this particular information 
collection by selecting ``Currently under Review--Open for Public 
Comments'' or by using the search function. The OMB control number for 
this information collection is 0910-0167. Also include the FDA docket 
number found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: JonnaLynn Capezzuto, Office of 
Operations, Food and Drug Administration, Three White Flint North, 10A-
12M, 11601 Landsdown St., North Bethesda, MD 20852, 301-796-3794, 
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.

Orphan Drugs

OMB Control Number 0910-0167--Revision

    This information collection supports FDA regulations implementing 
sections 525, 526, 527, and 528 of the Federal Food, Drug and Cosmetic 
Act (FD&C Act) (21 U.S.C. 360aa, 360bb, 360cc, and 360dd), as well as 
related guidance. Sections 525, 526, 527, and 528 of the FD&C Act 
pertain to the development of drugs for rare diseases or conditions, 
including biological products and antibiotics, otherwise known or 
referred to as ``Orphan Drugs.'' Specifically, section 525 of the FD&C 
Act requires written recommendations on studies required for approval 
of a marketing application for a drug for a rare disease or condition. 
The information collection in 21 CFR 316.10, 316.12, and 316.14 is 
approved under OMB control numbers 0910-0001 and 0910-0014. Section 526 
of the FD&C Act provides for designation of drugs as orphan drugs when 
certain conditions are met, section 527 provides conditions under which 
a sponsor of an approved orphan drug enjoys exclusive FDA marketing 
approval for that drug for the orphan indication for a period of 7 
years, and, finally, section 528 is intended to encourage sponsors to 
make investigational orphan drugs available for treatment of persons in 
need on an open protocol basis before the drug has been approved for 
general marketing. Open protocols may permit patients who are not part 
of the formal clinical investigation to obtain treatment where adequate 
supplies exist and no alternative effective therapy is available.
    We have issued regulations in part 316 (21 CFR part 316) to 
implement the Orphan Drug provisions of the FD&C Act and to set forth 
procedures and requirements related to requesting recommendations for 
investigations of drugs for rare diseases or conditions, requesting 
designation of a drug for a rare disease or condition, or requesting 
exclusive approval for a drug for a rare disease or condition. To 
assist respondents and to be consistent with Sec.  316.50, our Office 
of Orphan Products Development (OOPD) maintains and makes publicly 
available guidance documents that apply to the Orphan Drug provisions 
of the FD&C Act and regulations in part 316. The list is maintained on 
the internet and guidance documents are issued in accordance with our 
Good Guidance Practice regulations in 21 CFR 10.115, which provide for 
public comment at any time.
    Accordingly, we are revising the information collection to include 
Agency guidance. The document entitled ``Meetings with the Office of 
Orphan Products Development: Guidance for Industry, Researchers, 
Patient Groups, and Food and Drug Administration Staff'' provides 
recommendations to industry, researchers, patient groups, and other 
stakeholders interested in requesting a meeting, including a 
teleconference, with OOPD on issues related to orphan drug designation 
requests, humanitarian use device 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 patient-related topics of concern. It is 
also intended to assist OOPD staff in addressing such meeting requests. 
This guidance describes procedures for requesting, preparing, 
scheduling, conducting, and documenting such meetings and discusses 
background information we recommend be included in such requests. 
Information collection attendant to recommendations in the guidance are 
currently approved under OMB control number 0910-0787; however, for 
efficiency of Agency operations, we are consolidating it into this 
related information collection. The guidance is available at https://www.fda.gov/media/92815/download.
    The FDA Orphan Drug Designation Request Form (Form FDA 4035) is 
intended to benefit sponsors who desire to seek orphan designation of 
drugs intended for rare diseases or conditions from only FDA. The form 
is a simplified

[[Page 83972]]

method for sponsors to provide only the information required by Sec.  
316.20 for FDA to make a decision.
    During this public health emergency associated with the COVID-19 
pandemic, the OOPD is providing sponsors with increased flexibility for 
submission of orphan drug designation requests and related submissions 
(amendments, annual reports, etc.). During this public health 
emergency, orphan drug designation, humanitarian use device 
designation, and rare pediatric disease designation requests and 
submissions may be submitted electronically by email to the OOPD. When 
transmitting information to the Orphan Drug Designation Program via 
email, please utilize the mailbox orphan@fda.hhs.gov. We recommend 
using the automated read receipt feature to avoid having to call to 
verify receipt of the email. We also strongly encourage sponsors and 
others who plan to email information to FDA that is considered to be 
private, sensitive, proprietary, or commercial confidential to send it 
from an FDA-secured email address, which is provided by FDA, so the 
transmission is encrypted. The OOPD will assume that the addresses of 
emails received or email addresses provided as a point of contact are 
FDA secure when responding to those email addresses.
    In the Federal Register of October 2, 2020 (85 FR 62306), 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 1
----------------------------------------------------------------------------------------------------------------
                                                     Number of                        Average
    21 CFR section; activity         Number of     responses per   Total annual     burden per      Total hours
                                    respondents     respondent       responses       response
----------------------------------------------------------------------------------------------------------------
Content and format of a request              534            1.25             668             135          90,180
 for designation; request for
 verification of status;
 amendment to designation.......
Sec.  Sec.   316.20, 316.21,                 534            1.25             668              32          21,376
 316.26 (Form FDA 4035).........
Sec.   316.22; Notifications of              132               1             132               2             264
 changes in agents..............
Sec.   316.24(a); Deficiency                  20               1              20               2              40
 letters and granting orphan-
 drug designation...............
Sec.   316.27; Submissions to                104               1             104               5             520
 change ownership of orphan-drug
 designation....................
Sec.   316.30; Annual reports...             744               1             744               3           2,232
Sec.   316.36; Assurance of the                1               3               3              15              45
 availability of sufficient
 quantities of the orphan drug;
 holder's consent for the
 approval of other marketing
 applications for the same drug.
Guidance Recommendations:                  2,508               1           2,508           3.595           9,016
 Meeting requests to OOPD and
 related submission packages....
                                 -------------------------------------------------------------------------------
    Total.......................  ..............  ..............  ..............  ..............         123,673
----------------------------------------------------------------------------------------------------------------
\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

    Based on our evaluation, we have adjusted the currently approved 
burden estimate we attribute to information collection activities 
associated with our Orphan Drug program to reflect an increase in 
submissions. This notice corrects the mathematical error published in 
the 60-day notice, which indicated that the total burden was 123,623.

    Dated: December 17, 2020.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2020-28349 Filed 12-22-20; 8:45 am]
BILLING CODE 4164-01-P