Critical Path Innovation Meetings

Administrative guidance, not enacted legislation. This captured publisher edition preserves the listed document and attachments; later developments are not independently consolidated.

Critical Path
Innovation Meetings
Guidance for Industry
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
April 2015
Procedural

Critical Path
Innovation Meetings
Guidance for Industry
Additional copies are available from:
Office of Communications, Division of Drug Information
Center for Drug Evaluation and Research
Food and Drug Administration
10001 New Hampshire Ave., Hillandale Bldg., 4th Floor
Silver Spring, MD 20993-0002
Tel: 855-543-3784 or 301-796-3400; Fax: 301-431-6353; Email: druginfo@fda.hhs.gov
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
April 2015
Procedural

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TABLE OF CONTENTS
I. 	INTRODUCTION............................................................................................................. 1
II. 	BACKGROUND ............................................................................................................... 2
III. 	SCOPE, CONTENT, AND OUTCOMES ...................................................................... 2
A. 	Scope ............................................................................................................................................... 2
B. 	Potential Topics for a CPIM ......................................................................................................... 2
C. 	Outcomes ........................................................................................................................................ 3
IV. 	PROCEDURES ................................................................................................................. 4
A. 	General Considerations ................................................................................................................. 4
B. 	CPIM Requests .............................................................................................................................. 4
C. 	CPIM Preparation Packages ........................................................................................................ 4
D. 	Postmeeting Summaries ................................................................................................................ 5

Contains Nonbinding Recommendations
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Critical Path Innovation Meetings
Guidance for Industry1
This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on
this topic. It does not create 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. To
discuss an alternative approach, contact the FDA staff responsible for this guidance as listed on the title
page.
I. 	INTRODUCTION
This guidance describes the purpose, scope, documentation, and administrative procedures for a
Critical Path Innovation Meeting (CPIM), including how to request such a meeting. The CPIM is a
means by which the Center for Drug Evaluation and Research (CDER or we) and investigators from
industry, academia, patient advocacy groups, and government can communicate to improve
efficiency and success in drug development. The goals of the CPIM are to discuss a methodology or
technology proposed by the meeting requester and for CDER to provide general advice on how this
methodology or technology might enhance drug development. CDER will identify some of the larger
gaps in existing knowledge that requesters might consider addressing in the course of their work. The
discussions and background information submitted through the CPIM are nonbinding on both FDA
and CPIM requesters.
This guidance provides some examples of topics appropriate for a CPIM. It also describes the
information that should be provided to CDER in preparation for a meeting and potential outcomes
from the CPIM.
In general, FDA’s guidance documents do not establish legally enforceable responsibilities.
Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only
as recommendations, unless specific regulatory or statutory requirements are cited. The use of
the word should in Agency guidances means that something is suggested or recommended, but
not required.
1 This guidance has been prepared by the Office of Translational Sciences and the Office of New Drugs in the
Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration.

Contains Nonbinding Recommendations
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II. 	BACKGROUND
In 2004, FDA published the report Innovation or Stagnation: Challenge and Opportunity on the
Critical Path to New Medical Products, which called attention to a “slowdown . . . in innovative
medical therapies reaching patients.” 2 The report identified several areas of product
development in need of improvement, including “technical methods such as animal or computer-
based predictive models, biomarkers for safety and effectiveness, and new clinical evaluation
techniques,” and cited a need “to create better tools for developing medical technologies [and] a
knowledge base built not just on ideas from biomedical research, but on reliable insights into the
pathway to patients.” Through the Critical Path Initiative (CPI), FDA works to foster innovation
through collaborations among government, industry, academia, patient advocacy groups, and
other external stakeholders. CDER has created programs such as the Voluntary eXploratory
Data Submission program (VXDS)3 and Drug Development Tools (DDT) qualification
programs4,5 for biomarkers, clinical outcome assessments, and animal models under the Animal
Rule.6 FDA continues to work to identify opportunities to advance drug development efforts.7
III. 	SCOPE, CONTENT, AND OUTCOMES
A. 	Scope
The CPIM is broad in scope. It is a general discussion of challenges in drug development and
innovative strategies to address them. Appropriate FDA experts from CDER offices and other centers
will participate as resources and time permit.
B. 	Potential Topics for a CPIM
Potential topics for a CPIM include, but are not limited to, the following:
2 Available at www.fda.gov/ScienceResearch/SpecialTopics/CriticalPathInitiative/CriticalPathOpportunitiesReports
under Challenges and Opportunities Report – March 2004.
3 See the guidance for industry Pharmacogenomics Data Submissions. The guidances referenced in this document
are available on the FDA Drugs guidance Web page at
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm. We update
guidances periodically. To make sure you have the most recent version of a guidance, check the FDA Drugs
guidance Web page.
4 See the Drug Development Tools (DDT) Qualification Programs Web page,
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/.
5 See the guidance for industry and FDA staff Qualification Process for Drug Development Tools.
6 See the draft guidance for industry Product Development Under the Animal Rule. When final, this guidance will
represent the FDA’s current thinking on this topic.
7 Identifying CDER’s Science and Research Needs Report (2011), available at
http://www.fda.gov/Drugs/ScienceResearch/ucm264327.htm.

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• 	Biomarkers in the early phase of development and not yet ready for the Biomarker
Qualification Program (BQP): The CPIM can be a venue for a discussion of the
potential of proposed biomarkers. The discussion can help requesters understand some of
the more important questions FDA may have related to proposed biomarkers and prepare
prospective submitters for the BQP.
• 	Clinical outcome assessments in the early phase of development and not yet ready
for the Clinical Outcome Assessment Qualification Program: Clinical outcome
assessments (COAs) include patient-reported outcomes, clinician-reported outcomes,
observer-reported outcomes, and performance outcomes. The CPIM can be a venue for a
discussion of the potential approaches to developing COAs to provide evidence of
treatment benefit to support marketing approval and labeling claims. The discussion can
help requesters understand the needs and goals for COA qualification and answer
questions they may have related to the development or selection of COAs in preparation
for the qualification process.
• 	Natural history study designs and implementation: The CPIM can assist in the design
of natural history studies to increase the potential for the data generated by these studies
to help in the design of interventional clinical trials and drug development programs.
• 	Emerging technologies (other than manufacturing technology) or new uses of
existing technologies: The CPIM may help developers understand the strengths and
weaknesses of these technologies in relation to the various potential uses at different
stages of drug development.
• 	Innovative conceptual approaches to clinical trial design and analysis: The CPIM can
be a forum for the discussion of conceptual and general regulatory issues concerning
various design and analytical approaches to clinical trials.
FDA will not give regulatory advice on specific product development programs at a CPIM.
Meetings relating to a specific drug development program should be requested through the
appropriate review division in accordance with the FDA guidance for industry Formal Meetings
Between the FDA and Sponsors or Applicants. The CPIM is not intended to replace meetings
that should be held through formal DDT qualification programs (see Background). Requesters
interested in discussions related to the DDT qualification programs should contact the relevant
DDT qualification program directly. CPIMs are not intended to discuss therapeutic product-
specific data or result in binding agreements.
C. 	Outcomes
Through the CPIM, CDER intends to provide our perspective on the potential for use of
proposed new tools and methods in drug development. Based on CDER experience, we may
advise requesters of issues to consider in pursuing their work, propose joint efforts through
existing consortia, or discuss the potential to form new consortia. The CPIM may also lead to
recommendations for public workshops or other avenues for engaging with the wider scientific
community. CDER expects that the CPIM will also provide FDA with exposure to methods and
techniques that may have value in drug development.

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The CPIM is not intended to be a venue for ongoing, recurrent discussions with FDA. However,
we will consider requests for a subsequent meeting on a given topic on a case-by-case basis.
CDER will post the topics discussed on a quarterly basis at
http://www.fda.gov/drugs/developmentapprovalprocess/druginnovation/ucm395888.htm
IV. 	PROCEDURES
A. 	General Considerations
The CPIM is administered by the Office of Translational Sciences (OTS), which is the point of
contact for all communications.
CDER will discuss with the requester, as needed, whether the issues raised in the CPIM request
would be better addressed through other venues (see Scope). A request for a CPIM should not
be submitted to a particular regulatory application (e.g., investigational new drug application
(IND), new drug application (NDA), biologics license application (BLA)).
Requesters should clearly identify confidential or other proprietary information.
B. 	CPIM Requests
The CPIM request should be submitted electronically. Information about how to submit the
request electronically is available at:
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm395888.htm .
The request should include the following information:
• 	Name of requester
• 	Date of request
• 	Description of organization
• 	A document, no more than 5-6 pages in length, containing the background and purpose of
the meeting, steps taken in advancing the project, and specific questions for FDA (if
needed)
• 	Desired outcome of the meeting
Requests should provide enough information appropriate for a discussion of conceptual drug
development issues and should not be focused on a particular regulatory submission.
CDER plans to respond to the requester within 14 days of receipt of the meeting request and
discuss with the requester the appropriateness of the CPIM.
C. 	CPIM Preparation Packages
If a request for a CPIM is granted, the requester should submit a final preparation package no
later than 2 weeks before the meeting date.

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The CPIM preparation package should be submitted to the CDER Document Room and include
the following elements:
• 	Objective of the meeting
• 	Proposed agenda
• 	Presentation slides, if any
• 	Proposed attendees and respective affiliations
Send the CPIM package to the following address:
Food and Drug Administration
10903 New Hampshire Avenue
WO Building 21-4547
Silver Spring, MD 20993-0002
D. 	Postmeeting Summaries
We will send a meeting summary to the requester within 60 days of the meeting.