Medical Device Development Tools: FDA CDRH Pilot Program

1. MDIC M&S Summit Washington, DC June 26, 2014 Medical Device Development Tools:FDA CDRH Pilot Program Kathryn O‘Callaghan Senior Science Health Advisor Office of the Center Director FDA CDRH Tina Morrison Regulatory Advisor, Computational Modeling Office of Device Evaluation FDA CDRH

2. Medical Device Development REAL-WORLD PRODUCT PERFORMANCE The Total Product Life Cycle NEXT GENERATION PRODUCT DEVELOPMENT CMS REIMBURSE DECISION More Predictable More Efficient CMS EVALUATION INVENTION + PROTOTYPING PRE-CLINICAL CMS REIMBURSEMENT POST MARKET MONITORING DISCOVERY + IDEATION FDA REGULATORY DECISION PRODUCT LAUNCH CLINICAL

3. Regulatory Science Efforts Device Development & Regulatory Evaluation

4. Proposed New Voluntary Program http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm374427.htm

5. MDDT Case-by-case… Why is FDA CDRH developing a qualification process? TODAY: Tools considered and evaluated on a case-by-case basis TOMORROW: Qualified for regulatory purposes, within a defined context of use MDDT MDDT MDDT

6. KEY CONCEPTS

7. Types of Tools (Categories)

8. What is “FDA Qualification”? • The results of an assessment can be relied upon for device development & evaluation, within a specified context of use • Device industry need not reconfirm the suitability of a qualified MDDT • Device industry users may need to demonstrate the tool is used according to the specified context of use • Context of use ≈boundaries within which evidence & justification supports tool use

9. Context of Use

10. Context of Use:Clinical Roles for MDDT

11. Context of Use:Non-Clinical Roles for MDDT

12. Strength of Evidence Needed Depends on Context of Use Stages of Product Development

13. Next Steps:Pilot Program

14. Considerations for CDRH Qualification

15. Threshold for CDRH Qualification Key Criteria: • Description of MDDT. Is the MDDT adequately described? • Context of use. Is the context of use adequately and appropriately defined? • Strength of evidence. Tool validity, scientific plausibility, extent of prediction & capture. • Advantages & disadvantages. The strength of evidence needed to support qualification depends largely on the context of use.

16. Computational Models • For computational models that are MDDTs, we will leverage the Credibility Strategy as part of the qualification process • Credibility: the quality to elicit belief or trust in predictions of the CM&S within a context of use • The strategy is a tool for systematic identification of ‘how much’ VVUQ is necessary to support the CM&S in a specific context of use.

17. Computational Models

18. Availability of MDDTs in the Pilot Program • Transparency: For qualified MDDT, FDA will make public the context of use, and a summary of evidence and basis of the qualification determination (analogous to summaries of approved devices). FDA will keep proprietary information confidential. • Accessible to public: Submitter must consent to make MDDT accessible to the public for use, and not restrict to certain private entities, such as a single manufacturer • Licensing/pricing/IP: FDA and the MDDT program places no requirements on licensing/cost/degree of access to IP associated with a tool, nor does it consider restrictions related to patent claims. An MDDT submitter may include and protect proprietary methods, as long as access to the tool is not restricted to certain private entities.

19. WHY PARTICIPATE?

20. Benefits to Medical Device Manufacturers & FDA • Goal – Qualification will facilitate: • More predictable product evaluation. Medical device industry can use qualified tools without the need to reconfirm validity in individual submissions to FDA, potentially reducing time and other resources needed to develop new products. • More efficient regulation. FDA’s efforts to qualify one MDDT could by surpassed by the time and resources saved when the MDDT is applied to several device submissions or device development programs.

21. Benefits to Tool Developers • Goal – Publicizing MDDT qualification determinations will foster: • Adoption. Encourage adoption of tools • Transparency. Allow the FDA to more quickly and clearly communicate about important advances in regulatory science • Collaboration. Facilitate collaboration in a pre-competitive setting to amplify evidence collection and reduce individual resource expenditure 21

22. MDDT Case-by-case… Bridging Advances in Regulatory Science into Regulatory Application TODAY: Tools considered and evaluated on a case-by-case basis TOMORROW: Qualified for regulatory purposes within a defined context of use MDDT MDDT MDDT

23. Upcoming Meetings • ASME V&V40 Subcommittee • July 29-30, 2014, FDA Campus • Medical Device Special Interest Group with the Biomedical Engineering Society • Annual Meeting: October 22-25, 2014, San Antonio, TX • SIG Session on 10/23 • BMES/FDA Modeling and Simulation Conference • Formerly known as the ASME/FDA Frontiers Conference • May 18-20, 2015, University of Maryland

24. Contact Information Tina Morrison tina.morrison@fda.hhs.gov (301) 796-6310 Katie O’Callaghan kathryn.ocallaghan@fda.hhs.gov (301) 796-6349

25. FAQ: Examples of Tools Eligible to be Qualified • PRO rating scales, such as those for pain, symptom relief, function, improved mobility, or health status (also commonly referred to as “quality of life”) • Clearly defined clinical outcomes based on subjective clinical decision-making if used as a measure of treatment benefit, such as heart-failure related hospitalization • Nonclinical Assessment Models developed to measure a parameter of interest or to substitute for another generally accepted test or measurement, such as: • Computer modeling to assess conditions typically evaluated through human, animal or bench testing • In vitro models to replace animal testing • Use of tissue and other material phantoms to evaluate imaging devices

26. FAQ: Proposals • Proposal streams: • An MDDT developer chooses to pursue qualification to allow for use of the tool across multiple device programs • FDA identifies an area of need or calls for development activity in a specific area • Need and interest in an area is determined by individual or consortia of stakeholders (e.g., MDIC) 26

27. FAQ: Factors for Prioritization • Public health impact • Context of use includes life-threatening AND/OR serious chronic disease/condition • No/poor alternatives or unmet scientific need • Benefit for innovation or efficiency • Device area includes novel or innovative technology with no established regulatory paradigm • Major efficiencies to be gained • Scope of impact: Potential for use by multiple product development programs or sponsors

28. FAQ: Voluntary Program • Opt-in policy. • Medical device manufacturers may use qualified MDDT but are not required to do so* • Tool developers may pursue FDA qualification but are not required to do so* • Medical device manufacturers may still elect to pursue confidential use of a tool within a specific application only* • *See preceding benefits slides!