[Federal Register Volume 85, Number 105 (Monday, June 1, 2020)]
[Notices]
[Pages 33163-33165]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-11655]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-N-0837]


Rare Disease Clinical Trial Networks; Request for Information and 
Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; request for information and comments.

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[[Page 33164]]

SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is 
announcing the establishment of a docket to obtain information and 
comments from patients, patient advocates, the scientific community, 
health professionals, other regulatory and health authorities in the 
global community, regulated industry, and the general public regarding 
practical steps and successful approaches to establish a rare disease 
clinical trials network.

DATES: Submit written or electronic comments and information on the 
notice by July 31, 2020.

ADDRESSES: You may submit comments as follows. Please note that late, 
untimely filed comments will not be considered. Electronic comments 
must be submitted on or before July 31, 2020. The https://www.regulations.gov electronic filing system will accept comments until 
11:59 p.m. Eastern Time at the end of July 31, 2020. Comments received 
by mail/hand delivery/courier (for written/paper submissions) will be 
considered timely if they are postmarked or the delivery service 
acceptance receipt is on or before that date.

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand Delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2019-N-5464 for ``Rare Disease Clinical Trial Networks; Request for 
Information and Comments.'' Received comments, those filed in a timely 
manner (see ADDRESSES), 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.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. If you do not wish 
your name and contact information to be made publicly available, you 
can provide this information on the cover sheet and not in the body of 
your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    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 240-402-7500.

FOR FURTHER INFORMATION CONTACT: Meghana Chalasani, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 6304, Silver Spring, MD 20993-0002, 240-
402-6525, meghana.chalasani@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: 

I. Background

    Over the past decade, progress has been made in planning and 
conducting clinical trials for rare disease drug development. In 2018, 
for the first time ever, a majority of new molecular entities approved 
by the FDA were orphan drugs to treat rare diseases. However, of the 
approximately 7,000 known rare diseases, less than 10 percent have an 
FDA-approved treatment available. Rare disease drug development 
continues to be challenged by the small numbers of patients and limited 
understanding of the variability and progression of each disease.
    To support innovation and quality in the drug development pipeline 
for rare diseases, FDA has proposed establishment of a ``Rare Disease 
Cures Accelerator.'' The Rare Disease Cures Accelerator would provide a 
more centralized infrastructure and common platform(s) and approaches 
to support: (1) Rare disease characterization, (2) development of 
standard core sets of clinical outcome assessments and endpoints 
relevant to rare conditions, and (3) support conduct of clinical trials 
in rare disease populations. Following FDA CDER receipt of $10 million 
in FY 2019 Congressional appropriations for investment and innovation 
for rare diseases, FDA launched a set of efforts to begin building 
capabilities for the first two of these three components. To learn 
more, please visit FDA's Rare Disease Cures Accelerator Homepage 
[https://www.fda.gov/drugs/regulatory-science-research-and-education/rare-disease-cures-accelerator].
    With this request for information and comments, FDA is interested 
in understanding what work is currently being done and what work needs 
to be done to address the third component of its Rare Disease Cures 
Accelerator--improving the design, conduct, and completion of rare 
disease clinical trials. FDA is particularly interested in learning 
practical steps and successful approaches related to startup, 
implementation, and sustainment of clinical trials networks for rare 
diseases, including specific considerations for establishing such 
networks for a range

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of rare diseases. Because of the small size of rare disease populations 
and global occurrence of rare conditions, it is considered that the 
networks needed to support rare disease drug development would also 
have global reach and operations.

II. Requested Information and Comments

    FDA requests input on practical steps and successful approaches to 
startup, implement, and sustain global clinical trials networks, 
including specific considerations for establishing such networks for a 
range of rare diseases. Questions that could be addressed include, but 
are not limited to, those listed below. It is not necessary to answer 
all the questions below.
    1. What should be the immediate (<3 years) and long-term objectives 
of a global clinical trials network?
    2. How could a global clinical trials network for rare disease be 
organizationally structured (e.g., what mix of scientific and clinical 
disciplines are engaged to staff it; what process or guidance is 
followed for study protocol design; what standard procedures are 
employed for conduct of trials, and related protection of study 
participants and study data, etc.)? For example:
     Are there experiences that can be shared regarding 
networks integrating a disease-specific development center with a 
disease-agnostic operations center?
     Are there experiences that can be shared regarding 
networks focused on a broad group of rare diseases and collaboration 
with regional or disease-specific networks?
    3. What kind of investigator experience is needed to start up and 
expand to implement a global clinical trial network (e.g., experience 
with clinical trial research administration, clinical trial operations, 
working with pharmaceutical companies in the design, conduct and 
management of clinical trials)?
    4. What are successful models of governance for global clinical 
trial networks (e.g., role, responsibilities, and composition of 
various governing bodies)?
    5. What are potential opportunities to leverage and/or complement 
other existing networks (e.g., Institute for Advanced Clinical Trials 
for Children Network, Duke Clinical Research Institute Pediatric Trial 
Network, National Institutes of Health (NIH) Rare Diseases Clinical 
Research Network, NIH Experimental Therapeutics Clinical Trials 
Network, European Network of Paediatric Research at the European 
Medicines Agency)?
    6. What infrastructure is required to startup, implement, and 
sustain a global clinical trials network (e.g., required 
administrative, financial and physical resources, centralized 
functions, data coordination and network operations, global 
interoperability)?
    7. What level of funding would be needed to establish a network, 
potentially expand a network, and sustain the network over the long 
term (e.g., at least 5 years and longer)? A a range of estimates (e.g., 
startup costs, annual operating costs) and associated assumptions would 
be helpful.
    8. What are the key milestones and associated timelines for 
starting up and expanding to implement a global clinical trials 
network?
    9. What are potential challenges or barriers to starting up, 
implementing, and sustaining a global rare disease clinical trials 
network?

    Dated: May 26, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-11655 Filed 5-29-20; 8:45 am]
 BILLING CODE 4164-01-P


