Posted: 02 Apr 2015 08:26 PM PDT
By James E. Valentine*, Josephine M. Torrente & Frank J. Sasinowski –
On March 31, 2015, FDA’s Center for Drug Evaluation and Research (“CDER” or the “Center”) finalized its Guidance for Industry: Critical Path Innovation Meetings, establishing an enduring function of the Center to facilitate Critical Path Innovation Meetings (“CPIMs”). The CPIM program is a tool for external stakeholders, including industry, academia, patient advocacy groups, or the government, to request a meeting to discuss a drug development methodology or technology with CDER outside of the context of review for any particular product or application.
CPIMs are a venue to seek “[FDA’s] perspective on the potential use of proposed new tools and methods in drug development” and “advise requesters of issues to consider in pursuing their work . . . .” FDA Guidance for Industry: Critical Path Innovation Meetings, at page 3 (Apr. 2015). CPIMs also provide an opportunity to discuss with CDER follow-up activities, such as initiatives with consortia and/or wider engagement of the scientific community (e.g., public workshops). The scope of the CPIM program is broad, and can concentrate on addressing any number of challenges in drug development and strategies.
Meeting Topics
While the program’s focus may change over time, CDER provided a preliminary list of topics:
1. Biomarkers in the early phase of development.
CPIMs can serve as a venue for discussing the potential of proposed biomarkers, as well as the type of questions FDA may have related to proposed biomarkers. This type of meeting may be of particular interest to prospective submitters to the Biomarker Qualification Program.
2. Clinical outcome assessments (“COAs”) in the early phase of development.
CPIMs can also provide a forum for discussing potential approaches to developing COAs, such as patient-reported outcomes, that can support marketing approval and labeling claims. This type of meeting could answer questions related to the development or selection of COAs in preparation for the qualification process.
3. Natural history study designs and implementation.
Here, CPIMs could assist those interested in the design of natural history studies in ways that maximize the potential to generate data, which will help in the design of interventional clinical trials and drug development programs. Dr. Janet Woodcock, the director of CDER, has recently been advocating for greater adoption of patient registries, particularly by patient groups, to systematically collect natural history information to aid drug developers and FDA in determining if a drug has an effect. One such use of natural history information is to leverage it as a historical control in a single-arm clinical trial.
4. Emerging technologies or new uses of existing technologies.
These types of meetings could help drug developers understand the strengths and weaknesses of technologies in relation to their various potential uses at different stages of drug development. For example, on March 20, 2015, the National Institutes of Health and FDA hosted a public scientific workshop to discuss dystrophin protein quantification methodologies for use in Duchenne Muscular Dystorphy (“DMD”) therapy developemnt. Under the CPIM program, stakeholders in any disease area may be able to initiate discussions with CDER that could ultimately result in similar public workshops.
5. Innovative conceptual approaches to clinical trial design and analysis.
Such CPIMs could provide a venue to discuss conceptual and general regulatory issues concerning various design and analytical approaches to clinical trials. In particular, CPIMs may be a valuable forum for discussing, for instance, novel adaptive or enrichment study designs for classes of therapeutic targets.
Requesting a Meeting
To request a CPIM, one must first develop a proposal containing background material, the purpose of the meeting, steps that have already been taken to advance the project, any specific questions for FDA, and the desired outcome of the meeting. Requests can be submitted electronically here. The program is administered by the Office of Translational Sciences, which will respond to requests within 14 days.
If a meeting request is granted, the requester will also be responsible for developing a final preparation package to submit to FDA at least two weeks before the meeting date. CDER commits to sending a meeting summary to the requester within 60 days of the meeting.
*Admitted only in Maryland. Work supervised by the Firm while D.C. application is pending.
On March 31, 2015, FDA’s Center for Drug Evaluation and Research (“CDER” or the “Center”) finalized its Guidance for Industry: Critical Path Innovation Meetings, establishing an enduring function of the Center to facilitate Critical Path Innovation Meetings (“CPIMs”). The CPIM program is a tool for external stakeholders, including industry, academia, patient advocacy groups, or the government, to request a meeting to discuss a drug development methodology or technology with CDER outside of the context of review for any particular product or application.
CPIMs are a venue to seek “[FDA’s] perspective on the potential use of proposed new tools and methods in drug development” and “advise requesters of issues to consider in pursuing their work . . . .” FDA Guidance for Industry: Critical Path Innovation Meetings, at page 3 (Apr. 2015). CPIMs also provide an opportunity to discuss with CDER follow-up activities, such as initiatives with consortia and/or wider engagement of the scientific community (e.g., public workshops). The scope of the CPIM program is broad, and can concentrate on addressing any number of challenges in drug development and strategies.
Meeting Topics
While the program’s focus may change over time, CDER provided a preliminary list of topics:
1. Biomarkers in the early phase of development.
CPIMs can serve as a venue for discussing the potential of proposed biomarkers, as well as the type of questions FDA may have related to proposed biomarkers. This type of meeting may be of particular interest to prospective submitters to the Biomarker Qualification Program.
2. Clinical outcome assessments (“COAs”) in the early phase of development.
CPIMs can also provide a forum for discussing potential approaches to developing COAs, such as patient-reported outcomes, that can support marketing approval and labeling claims. This type of meeting could answer questions related to the development or selection of COAs in preparation for the qualification process.
3. Natural history study designs and implementation.
Here, CPIMs could assist those interested in the design of natural history studies in ways that maximize the potential to generate data, which will help in the design of interventional clinical trials and drug development programs. Dr. Janet Woodcock, the director of CDER, has recently been advocating for greater adoption of patient registries, particularly by patient groups, to systematically collect natural history information to aid drug developers and FDA in determining if a drug has an effect. One such use of natural history information is to leverage it as a historical control in a single-arm clinical trial.
4. Emerging technologies or new uses of existing technologies.
These types of meetings could help drug developers understand the strengths and weaknesses of technologies in relation to their various potential uses at different stages of drug development. For example, on March 20, 2015, the National Institutes of Health and FDA hosted a public scientific workshop to discuss dystrophin protein quantification methodologies for use in Duchenne Muscular Dystorphy (“DMD”) therapy developemnt. Under the CPIM program, stakeholders in any disease area may be able to initiate discussions with CDER that could ultimately result in similar public workshops.
5. Innovative conceptual approaches to clinical trial design and analysis.
Such CPIMs could provide a venue to discuss conceptual and general regulatory issues concerning various design and analytical approaches to clinical trials. In particular, CPIMs may be a valuable forum for discussing, for instance, novel adaptive or enrichment study designs for classes of therapeutic targets.
Requesting a Meeting
To request a CPIM, one must first develop a proposal containing background material, the purpose of the meeting, steps that have already been taken to advance the project, any specific questions for FDA, and the desired outcome of the meeting. Requests can be submitted electronically here. The program is administered by the Office of Translational Sciences, which will respond to requests within 14 days.
If a meeting request is granted, the requester will also be responsible for developing a final preparation package to submit to FDA at least two weeks before the meeting date. CDER commits to sending a meeting summary to the requester within 60 days of the meeting.
*Admitted only in Maryland. Work supervised by the Firm while D.C. application is pending.
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