directions with real world evidence for medical devices
TRANSCRIPT
Directions With Real World Evidence for Medical Devices
The Evolving Regulatory Universe: What’s happening now and
what’s coming next
Disclaimer
The views expressed in this presentation are those of the
presenter and do not represent the official policies of the FDA or
any other Federal Agencies.
I am a full time employee of the FDA.
I have no conflicts of interest to report
Disclosure
John Laschinger, MDChief Medical Officer Clinical Trials Program
FDA/CDRH/ODE
Evidence Generation and EvaluationActionable Insights for Informed Clinical and Regulatory Decisions
Evidence Sources For Regulatory Decisions
Pre-Clinical Testing
Clinical Trials
Post-Market(Potential PAS)
Pre-MarketApplication
Traditional Regulatory Pathway
Sufficient SafetySafe and Effective
Benefits Outweigh Risks
Totality of the Evidence
EFS Pivotal Trial(limited and rigidly constructed
conditions and population)PAS
Clinical Evidence:Trial Size and
Timing
Bases of Regulatory Decisions
Clinical ReportsMDRs
Pre-Market Clinical Evidence LimitationsSources of Clinical and Regulatory Uncertainty for Medical Devices
Limitations of Pre-Market Evidence:
PopulationConditions
UsersLimited f/u
Transportability (generalizability)
Users PatientsDisease
Post-Market Changes:
Populations of use
Conditions of use
Device Iterations
Long-term Durability:
DeviceDevice
Performance
Device Effects
Disease:
Long term response to Rx Anatomic and
physiologic progression
Reasonable Assurance
of safety and effectiveness at the time of device Approval/Clearance
=Residual
Uncertaintyregarding appropriate and safe device use
in the post-market
Determinants of Tolerable Uncertainty For Medical Device Regulatory Approvals
Importance of Evolving Evidence over TPLC*
*Total Product Life Cycle
Definitions from the Guidance
Real-World Data (RWD)Data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources
Real-World Evidence (RWE)Clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD
Criteria for Use Of Real World DataDeriving Meaning from Big Data
We are now in the Era of Big Data!
“In ancient times having power meant having access to data. Today having power means knowing what data to ignore.”
Y.N. Hurari. Homo Deus. A Brief History of Tomorrow
AvailableExisting or Acquirable
CredibleHigh Quality and Traceable
RelevantFit to Purpose
AnalyzableInterpretable
Harnessing RWE for Regulatory DecisionsAssessing Evolving Evidence Over the Total Product Life Cycle
Non-Regulatory Uses of Existing Real World Data
Informing the community on optimal care
Identifying needs and gaps
Market analysis Assessing quality of care
Laschinger JC
Current Regulatory Uses of Post-Market Real World Observational Evidence
Control arm for pivotal clinical
study
New indications for approved
devices
Studying new improvements
to devices
Replacing post approval study
Adverse event reporting
Shifts to pre-post-market
balance
Laschinger JC
Re-engineering the Clinical Trial EnterpriseIdeal Properties of Infrastructure for RWD
Priorities for Creating a Reusable
Infrastructure for Clinical Evidence
Generation
Re-engineering the Clinical Trial EnterpriseNext Steps – Premarket and TPLC
*patients, clinicians, researchers, purchasers, payers, industry, hospitals and health systems, policy makers, and training institutions
*
FDA – A Public Health Agency
• Mandate is to embrace the full continuum of evidence from all sources along the TPLC – Not just at points of regulatory decisions
• Informed decisions evolve along a continuum as evidence evolves
• Mosaic of device uses and data sources• Never forget the Patient - Patient involvement and
patient data on outcomes and preferences is essential
Thank You!
John Laschinger, MDChief Medical Officer
Clinical Trials ProgramFDA/CDRH/ODE
