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RAPID
National Evaluation System for
health Technology (NEST)
William Maisel, MD, MPHDeputy Director for Science and Chief Scientist
Center for Devices and Radiological HealthU.S. Food and Drug Administration
www.fda.gov
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Patients are at the Heart of
What We Do
CDRH Vision: Patients in the U.S. have access to high-quality, safe, and effective medical devices of public health importance first in the world
William H. Maisel, MD, MPH – RAPID 2017
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Novel Device Approvals
2428
5561
49
67
8091
0
20
40
60
80
100
2009 2010 2011 2012 2013 2014 2015 2016
Number of Novel Devices
* Novel devices include original PMAs, panel track supplement PMAs, and de novos
>3-fold Increase in # of Novel Device Approvals
Calendar Year
William H. Maisel, MD, MPH – RAPID 2017
We face a critical public health
challenge
The U.S. regulatory standard for market approval protects patients by setting a high public health bar but imposes costs that make the U.S. marketplace less attractive for innovators thereby delaying patient access to important technologies
The solution is to reduce the time
and cost of the total product life
cycle…
device development, assessment, review, manufacturing, monitoring, and reimbursement – without compromising the reasonable assurance of safety and effectiveness standard
Vision
“Patients in the U.S.
have access to high-
quality, safe and
effective medical
devices of public health
importance first in the
world.”
William H. Maisel, MD, MPH – RAPID 2017
Learning Medical Device Ecosystem
Postmarket
Surveillance
TIME TO MARKET
Premarket
Review
Pre
marke
t D
ec
isio
n
INFORMATION FLOW
Total Product Life Cycle (TPLC) Framework
5William H. Maisel, MD, MPH – RAPID 2017
Learning Medical Device Ecosystem
Postmarket
Surveillance
TIME TO MARKET
Expedited
Access
Pathway
Premarket
Review
Pre
marke
t D
ec
isio
nBenefit -Risk
INFORMATION FLOW
Total Product Life Cycle (TPLC) Framework
6William H. Maisel, MD, MPH – RAPID 2017
Learning Medical Device Ecosystem
National Evaluation
System (NEST)
Real-World
Evidence
TIME TO MARKET
Expedited
Access
Pathway
Premarket
Review
Pre
marke
t D
ec
isio
n
Benefit -Risk
INFORMATION FLOW
“Safety Net”
Total Product Life Cycle (TPLC) Framework
7William H. Maisel, MD, MPH – RAPID 2017
Learning Medical Device Ecosystem
Postmarket
Surveillance
National Evaluation
System (NEST)
Real-World
Evidence
Expedited
Access
Pathway
Premarket
Review
Benefit -Risk “Safety Net”
EVOLUTION OF BENEFIT–RISK EVIDENCE
Total Product Life Cycle (TPLC) Framework
INTERNATIONAL HARMONIZATION
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Patient Access
NEST
Clinical
Research
Incorporated
Into Routine
Clinical
Practice
William H. Maisel, MD, MPH – RAPID 2017
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Foundational Work
William H. Maisel, MD, MPH – RAPID 2017
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Use of Real-World Data
Adapted from Galson S and Simon G. Available at: https://nam.edu/wp-content/uploads/2016/10/Real-World-Evidence-to-Guide-the-Approval-and-Use-of-New-Treatments.pdf
William H. Maisel, MD, MPH – RAPID 2017
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Potential Benefits of Better
Leveraging Real World Evidence
More Efficient and Timely Data Collection
Reduce Cost of Evidence Generation
Bring Devices to Patients More Quickly
Better Reflection of Real World Performance
Reduce Other Regulatory Burdens
Meet Other Stakeholder (Patient, Clinician, Payer, etc.) Needs
William H. Maisel, MD, MPH – RAPID 2017
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Evolving Use of Registries
Postmarket Surveillance
Leverage Infrastructure for Premarket
Studies
“Real World” Use to
Support Expanded Indications
Link to Other Databases -
EHR, Claims, etc.
Useful to Multiple
Stakeholders
William H. Maisel, MD, MPH – RAPID 2017
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The Value Proposition for NEST
Patients/Clinicians
• More timely access to safer, more effective devices
• Better information about the use of a given device in practice
• Improved quality• Reliable assurances of
safety• Possibly, reduced
reporting requirements
• Access to high-quality evidence on device performance in clinical practice
PayersHospitals,
Health Systems
Medical Device
Industry
• High-quality evidence at lower cost, in less time, to support premarket approval/clearance, payer coverage
• Meet or reduce the need for postmarket study and adverse event reporting requirements
• Potential for premarket-postmarket shift owing to strong assurances that postmarket RWE would be generated
• May obviate the need for FDA premarket review of some device modifications because more timely and informative routine data collection
William H. Maisel, MD, MPH – RAPID 2017
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Transcatheter Heart Valves
Use of Registry Data for Post Market
Surveillance and Label Expansion
Intermediate Risk
High Risk
Inoperable
William H. Maisel, MD, MPH – RAPID 2017
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Registry Assessment of Peripheral
Interventional Devices
Infrainguinal Arterial Occlusive Disease
Treatment Heterogeneity
Patient Conditions
Disease Severity
Disease Location
Specialty, Training,
Experience
Treatment Options
Device Heterogeneity
Angioplasty Balloons
Stents
Atherect. Devices
Total Occlusion Crossing Devices
Other Devices Used for PAD
William H. Maisel, MD, MPH – RAPID 2017
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Other Uses of Registry Data
Proof of Concept
• Left ventricular assist devices
• Stent grafts
• Surgical mesh
Control arm for pivotal
clinical study
• Post-approval study of Thoracic Endovascular Aortic Repair (TEVAR) device using the Vascular Quality Initiative Registry enrolled patients twice as fast as expected
Post approval
study
• Pilot on using registry data in lieu of submitting Medical Device Reports for on-label adverse events
Adverse event
reporting
William H. Maisel, MD, MPH – RAPID 2017
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NEST – Next Steps
Awarded $3 Million FDA Grant to establish NEST
Coordinating Center
MDUFA IV User Fee Agreement
Pilot projects funded to determine the usability of RWE for:
• Expanded indications for use• New clearances/approvals• Improved malfunction
reporting
William H. Maisel, MD, MPH – RAPID 2017
RAPID
National Evaluation System for
health Technology (NEST)
William Maisel, MD, MPHDeputy Director for Science and Chief Scientist
Center for Devices and Radiological HealthU.S. Food and Drug Administration
www.fda.gov