Rare Disease Clinical Trial Networks; Request for Information and Comments, 33163-33165 [2020-11655]
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Federal Register / Vol. 85, No. 105 / Monday, June 1, 2020 / Notices
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-201509-18/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.
You may submit comments on any
guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single
copies of the guidance to the Policy and
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Veterinary Medicine, Food and Drug
Administration, 7519 Standish Pl.,
Rockville, MD 20855. Send one selfaddressed adhesive label to assist that
office in processing your requests. See
the SUPPLEMENTARY INFORMATION section
for electronic access to the guidance
document.
For
questions regarding this document,
contact Tom Modric, Center for
Veterinary Medicine (HFV–216), Food
and Drug Administration, 7519 Standish
Place, Rockville, Rockville, MD 20855,
240–402–5853, tomislav.modric@
fda.hhs.gov or AskCVM@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
jbell on DSKJLSW7X2PROD with NOTICES
FOR FURTHER INFORMATION CONTACT:
I. Background
In the Federal Register of March 12,
2018 (83 FR 10732), FDA published the
notice of availability for a draft guidance
entitled ‘‘Proprietary Names for New
Animal Drugs,’’ giving interested
persons until May 11, 2018, to comment
on the draft guidance. FDA received
comments on the draft guidance, and
those comments were considered as the
guidance was finalized. Changes made
include revisions to the definitions. In
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addition, editorial changes were made
to improve clarity. The guidance
announced in this notice finalizes the
draft guidance dated March 2018.
II. Significance of Guidance
This level 1 guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
The guidance represents the current
thinking of FDA on proprietary names
for new animal drugs. It does not
establish any rights for any person and
is not binding on FDA or the public.
You can use an alternative approach if
it satisfies the requirements of the
applicable statutes and regulations.
III. Paperwork Reduction Act of 1995
FDA concludes that there are no
collections of information under the
Paperwork Reduction Act of 1995. This
guidance refers to previously approved
collections of information found in FDA
regulations. These collections of
information are subject to review by the
Office of Management and Budget
(OMB) under the Paperwork Reduction
Act of 1995 (44 U.S.C. 3501–3521). The
collections of information in 21 CFR
part 514 have been approved under
OMB control numbers 0910–0032 and
0910–0699; the collections of
information in 21 CFR part 511 have
been approved under OMB control
number 0910–0117.
IV. Electronic Access
Persons with access to the internet
may obtain the guidance at either
https://www.fda.gov/AnimalVeterinary/
GuidanceComplianceEnforcement/
GuidanceforIndustry/default.htm or
https://www.regulations.gov.
Dated: May 26, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–11679 Filed 5–29–20; 8:45 am]
BILLING CODE 4164–01–P
33163
The Federal Food, Drug, and Cosmetic
Act (FD&C Act), as amended by the
Food and Drug Administration Safety
and Innovation Act (FDASIA),
authorizes FDA to award priority review
vouchers to sponsors of approved rare
pediatric disease product applications
that meet certain criteria. FDA is
required to publish notice of the
issuance of vouchers as well as the
approval of products redeeming a
voucher. FDA has determined that
NURTEC ODT (rimegepant), approved
February 27, 2020, meets the
redemption criteria.
FOR FURTHER INFORMATION CONTACT:
Althea Cuff, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–4061, Fax: 301–796–9858,
email: althea.cuff@fda.hhs.gov.
SUPPLEMENTARY INFORMATION: Under
section 529 of the FD&C Act (21 U.S.C.
360ff), which was added by FDASIA,
FDA will report the issuance of rare
pediatric disease priority review
vouchers and the approval of products
for which a voucher was redeemed.
FDA has determined that NURTEC ODT
(rimegepant), approved February 27,
2020, meets the redemption criteria.
For further information about the Rare
Pediatric Disease Priority Review
Voucher Program and for a link to the
full text of section 529 of the FD&C Act,
go to https://www.fda.gov/ForIndustry/
DevelopingProductsforRare
DiseasesConditions/RarePediatric
DiseasePriorityVoucherProgram/
default.htm. For further information
about NURTEC ODT (rimegepant),
approved February 27, 2020, go to the
‘‘Drugs@FDA’’ website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
Dated: May 26, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–11681 Filed 5–29–20; 8:45 am]
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–N–1262]
Notice of Approval of Product Under
Voucher: Rare Pediatric Disease
Priority Review Voucher
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
The Food and Drug
Administration (FDA) is announcing the
issuance of approval of a product
redeeming a priority review voucher.
SUMMARY:
PO 00000
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BILLING CODE 4164–01–P
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.
Notice; request for information
and comments.
ACTION:
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33164
Federal Register / Vol. 85, No. 105 / Monday, June 1, 2020 / Notices
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.
SUMMARY:
jbell on DSKJLSW7X2PROD with NOTICES
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
VerDate Sep<11>2014
19:40 May 29, 2020
Jkt 250001
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-201509-18/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
PO 00000
Frm 00086
Fmt 4703
Sfmt 4703
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/regulatoryscience-research-and-education/raredisease-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
E:\FR\FM\01JNN1.SGM
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Federal Register / Vol. 85, No. 105 / Monday, June 1, 2020 / Notices
jbell on DSKJLSW7X2PROD with NOTICES
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 diseasespecific 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)?
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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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2020–N–1069]
Approved Drug Products With
Therapeutic Equivalence Evaluations
(the ‘‘Orange Book’’); Establishment of
a Public Docket; Request for
Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice; establishment of a
public docket; request for comments.
ACTION:
The Food and Drug
Administration (FDA or Agency) is
announcing the establishment of a
public docket to solicit comments on
FDA’s publication entitled ‘‘Approved
Drug Products With Therapeutic
Equivalence Evaluations’’ (commonly
known as the ‘‘Orange Book’’). The
Orange Book identifies drug products
approved by FDA under the Federal
Food, Drug, and Cosmetic Act (FD&C
Act) and includes related information.
As part of FDA’s Drug Competition
Action Plan and our continued effort to
improve transparency and provide
useful information to regulated industry
and the public, we are seeking
comments on how stakeholders and the
public use the Orange Book and
whether it can be improved.
DATES: Submit either electronic or
written comments by August 31, 2020.
SUMMARY:
PO 00000
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ADDRESSES:
33165
You may submit comments
as follows:
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–
2020–N–1069 for ‘‘Approved Drug
Products With Therapeutic Equivalence
Evaluations (the ’Orange Book’);
Establishment of a Public Docket;
Request for 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
E:\FR\FM\01JNN1.SGM
01JNN1
]]>Agencies
[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]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
[[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, [email protected].
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
[[Page 33165]]
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
