Workshop - What to Expect Workshop - What to Expect
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Workshop – What to Expect Workshop – What to Expect
Workshop - What to Expect • This session is being recorded • On demand resources at SHIP-MD.org • In case of outage, we will email you with instructions • Audio for pre-recorded video broadcasts is only available through computer audio, not phone audio • Please share our event on social media • Recommend to not use VPN • Please direct questions using the Q&A panel and direct to “All Panelists”
Hospitals
System of Hospitals for
Innovation In Pediatrics –
Medical Devices (SHIP-MD)
The National Innovation Ecosystem For Pediatric
3
Medical Device Development
Presenters
Kolaleh Eskandanian, PhD, MBA, PMP Jonathan Davis, MD
Vice President and Chief Innovation Officer Vice-Chair of Pediatrics
Children’s National Hospital Chief of Newborn Medicine
Floating Hospital for Children at Tufts Medical Center
As Vice President and Chief Innovation Officer, Dr. Eskandanian leads the strategic
and operation management of novel product development at Children’s National, with
Dr. Jonathan Davis is Vice-Chair of Pediatrics and Chief of Newborn
a focus on translating pediatric innovation to patients and families. She is the
Medicine at the Floating Hospital for Children at Tufts Medical Center and
Managing Director of Innovation Ventures, the commercialization arm of Children’s
Professor of Pediatrics at Tufts University School of Medicine. His research
National, the Executive Director of the Sheikh Zayed Institute for Pediatric Surgical
has focused on neonatal drug development for the prevention and
Innovation, and Principal Investigator of the National Capital Consortium for Pediatric
treatment of a variety of neonatal conditions. He has authored over 200
Device Innovation, an FDA-funded initiative. Most recently, Dr. Eskandanian played
manuscripts and book chapters and received numerous grant awards from
critical leadership role in the development of the Children’s National Research and
the NIH, the FDA, the March of Dimes, and many others. He is currently
Innovation Campus, housing Children’s National Research Institute as well as JLABS
funded by NIH to develop better outcome measures for clinical trials and
@ Washington, DC and Virginia Tech as the initial anchors of the campus. Dr.
new and existing therapeutics to improve outcomes in children.
Eskandanian works with a large network of small and large businesses, nonprofits,
and government agencies to maximize the commercial potential of pediatric novel
Dr. Davis is Chair of the Neonatal Advisory Committee in the Office of the
products. Since early 2014, over 160 pediatric medical device companies have been
Commissioner at FDA, the Director of the International Neonatal
funded and/or received technical expertise through initiatives led by Dr. Eskandanian.
Consortium (FDA, EMA), and a member of Pediatric Policy Council
Her own innovations are in the market space for decades, used by millions. Dr.
representing the American Pediatric Society. These positions permit him to
Eskandanian is the lead inventor of the first-ever adverse event reporting system,
work closely with governments, academic leaders, industry and families
initially deployed in a research academic environment. She has had leadership roles
worldwide to promote the development of important therapeutics for infants
in the development of the first web-based consumer trouble-entry management
and children.
system for a Fortune 100 company in the telecommunications sector. In her role in
academia, she has been instrumental in securing over $100 million in peer-reviewed
government funding.
2Workstream Participants
Key Contributors: C-Path Leads:
• Kolaleh Eskandanian (co-lead) • Kanwaljit Singh
• Jonathan Davis (co-lead) • Laura Butte
• Peter Armstrong • Sarah Spieth
• Matthew Cooper • Karen Stamm
• Chester Koh SHIP-MD Executive Committee:
• Susan Rahman • Vasum Peiris
• Michael Adeyanju • Tara Federici
Panel Discussion Participants: • Geoffrey Rosenthal
• Jonathan Davis • Jonathan Davis
• Kolaleh Eskandanian • Joseph Scheeren
• Sue Rahman *SHIP-MD Coordinating Committee
• Peter Armstrong
Members
3SHIP-MD
The National Innovation Ecosystem for Pediatric Medical Devices
To improve public health for children, SHIP-MD is aimed
at transforming the pediatric medical device ecosystem by de-
risking and accelerating developmental processes to stimulate
investment and innovation in pediatric devices.
4Overview – Phases of SHIP-MD
Phase I – Pre-Consortium/Conceptual Phase – Current Phase
• A team of volunteers, divided into five unique but related workstreams, has worked to develop
and articulate the anticipated value proposition of the SHIP-MD framework.
• During this workshop the framework and value proposition will be presented and deliberated
• Feedback from workshop will factor into a go/no-go decision regarding Phase II
Phase II – Consortium Phase
• A Strategic Plan that incorporates short, medium, and long-term goals will need to be
implemented to understand and undertake the action steps required to implement the
framework and launch SHIP-MD.
Phase III – Implement the SHIP-MD framework, stand-alone SHIP-MD
entity
5Executive Summary
In selecting hospitals, consider:
Regional, patient, and disease-related diversity
Infrastructure to monitor clinical studies including:
o Local IRB/use of a central IRB
o COI Committee
o Clinical Trial Management System (CTMS)
Infrastructure to conduct clinical studies:
o Space (e.g. Clinical Research Center [CRC] for Hub-and-Spoke
inpatient/outpatient studies)
Model
o Clinical Research Coordinators, database
management personnel
o Biostatistics and study design support
Evidence generation via EMR/EHR, data registries
Experience with clinical trial performance metrics
Administrative infrastructure to manage contracts
6Device Sponsor Priorities
Ability to enroll eligible subjects in a timely manner
Meeting timelines outlined in the agreement and the
protocol
Ability to provide high quality data (verifiable per source
documentation) including timely follow-up
Investigators vested in the study and available for
meetings, consultations, discussions Hub-and-Spoke
Investigators with significant interest in the product being Model
tested (sufficient equipoise that a product is needed)
Sufficient site staff available for support who are actively
engaged in the process
Open to collaboration/brainstorming with sponsor
regarding obstacles in enrollment, follow-up, protocol
adherence, etc.
7• Confirmation of unmet pediatric device needs/Identification of candidate pediatric devices
Reimbursement models/Financing models
• IP provisions
• Central IRB
SHIP Coordinating • PR and communications
Center • Monitoring the progress of the Hub/Spoke entities
• Collection of metrics/report to Congress
• Template for legal agreements (e.g., IIA, CTA, MTA, etc.)
• Established clinical trials infrastructure (IRB, COI,
Hub(s) CTMS, payments)
• Existing clinical research unit (space, study design,
Shall ensure regional pool of coordinators)
and patient population Hospital 1 Hospital… Hospital n • Ability for evidence generation via EMR-
EHR/Registries
inclusion
• Institutional commitment from C-suite to cost-share
• Ability to manage sub-awardees (the spokes)
Spoke(s) Hospital/ Hospital/ Hospital/ Hospital/
Practice 1 Practice 2 Practice 3 Practice 4
• Demonstrated experience in conducting device trials
Shall ensure regional • Human subject protection program (IRB, ethics board)
and patient population • Access to subspecialty patient populations
inclusion Hospital/ Hospital/ Hospital/ Hospital/
Practice 4 Practice 5 Practice.. Practice n
8Questions
• What are the barriers to conducting device trials?
Group by hospital, industry/device sponsor, regulators, patient
advocacy group perspectives, adequate resources
• What are the top criteria hospitals must satisfy to participate in
the SHIP-MD ecosystem?
• Will a Hub-and-Spoke Model be a good fit?
9Challenges of Pediatric Device Trials
• In contrast with drug trials, device trials are poorly understood in
most academic sites. Adding to this complexity is the nature of
the pediatric population that requires additional considerations,
such as:
Relatively small number of children affected
Broad range of device sizes often required
Accommodation for growth
Off-label use of adult devices
10Pediatric Considerations | Age Group in Device
Studies
The Federal Food, Drug, and Cosmetic Act (FD&C Act) defines
pediatric patients as persons aged 21 or younger at the time of their
diagnosis or treatment. Pediatric subpopulations are further
categorized as follows:
Neonates - from birth through the first 28 days of life
Infants - 29 days to less than 2 years
Children - 2 years to less than 12 years
Adolescents - aged 12 through 21 (up to but not including the
22nd birthday)
11Drugs 21 CFR 312 vs. Devices 21 CFR 812 |
Phases of Clinical Trials
Drugs Devices
Phase I Pilot
Aimed at safety and tolerance Smaller population with disease or condition
Healthy volunteers (20-100 subjects) (10-30 subjects)
Determine dosing and major adverse effects Determine preliminary safety and
performance information
Phase II
Aimed at safety and effectiveness Pivotal
Small population with disease or condition (50-200 Larger population with disease or condition
subjects) (150-300 subjects)
Confirm dosing and major adverse effects Determine effectiveness and adverse effects
Phase III Post Approval
Aimed at safety and effectiveness Collect long-term data and adverse effects
Large population with disease or condition (100s to
1000s of subjects)
Determine drug-drug interactions and minor adverse
effect
Phase IV
Post approval study
Collect long-term data and adverse effects 12Drugs 21 CFR 312 vs. Devices 21 CFR 812 |
Trials Comparison
• Safety and efficacy • Device testing is stratified
conducted in Phase I based on the class of
through Phase III clinical device and level of risk.
trials approach. • Minimal risk devices do not
• Drug studies always require clinical trials
require clinical trials • Devices have no Phase I
conducted in phases. equivalent to drugs
• Training focuses on • Hands-on device training in
protocol requirements, addition to protocol
mechanism of action of training
Devices
drug, side effects • Devices are expensive to
Drugs
• Investigational drugs are produce, so charges apply
provided free (mostly)
Safeguard research participants while bringing
safe and effective products to market
13Pediatric Considerations | Special Protections
When a proposed research study involves children and is supported or
conducted by HHS, the research institution's Institutional Review Board
(IRB) must take into consideration the special regulatory
requirements (45 CFR 46) that provide additional protection for the
children who would be involved in the research. If the proposed
research involves FDA-regulated products, then FDA's parallel
regulations (21 CFR 50) apply.
Source: Office for Human Research Protections
14Pediatric Considerations | Ethical Factors
• Guardianship
• Racial, ethnic, socioeconomic diversity
• Researcher competencies
• Barriers to recruitment
• Role of compensation
• Commercial sponsorship
15Conclusions/Recommendations
• Create a safe environment for medical device development for children
Ensure the hospitals have the breadth and depth of infrastructure, experience,
and support
C-suite support and commitment
• Create a dynamic, adaptive, and sustainable national evidence generation
infrastructure for pediatric device development
Streamlined clinical trials
Potential for enterprise-wide EHR/RWE/data management systems
• SHIP-MD as the coordinating unit and ”go-to” for participating hospitals
Economies of scale reduced cost of conducting studies and reduced timelines
o Cost-sharing will allow small companies to have the advantage of BIG
MedTech
Single signature contracting for entire network within a defined time period
16Thank you!
Next, our panelists will join us in answering your questions
17Workshop - What to Expect • This session is being recorded • On demand resources at SHIP-MD.org • In case of outage, we will email you with instructions • Audio for pre-recorded video broadcasts is only available through computer audio, not phone audio • Please share our event on social media • Recommend to not use VPN • Please direct questions using the Q&A panel and direct to “All Panelists”
Panelists
• Jonathan Davis – Vice Chair of Pediatrics, Floating Hospital
for Children at Tufts Medical Center
• Kolaleh Eskandanian – Vice President and Chief Innovation
Officer Children’s National Hospital
• Sue Rahman – Director of Health Care Innovation,
Children’s Mercy Kansas City
• Peter Armstrong – Chief Medical Officer, OrthoPediatrics
18Thank you! Coming up next: Workshop – What to Expect
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