FDA s Center for Devices and Radiological Health: Current Status and Future Direction

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1 FDA s Center for Devices and Radiological Health: Current Status and Future Direction Jeff Shuren, MD, JD Center for Devices and Radiological Health U.S. Food and Drug Administra@on 1

2 Who We Are CDRH is a team of over 1,700 dedicated, highly-skilled people, including: Physicians Biologists Chemists Physicists Engineers Sta5s5cians Epidemiologists Accountants Microbiologists Nurses A=orneys Pharmacologists Veterinarians Toxicologists Specialists in Public Health Educa5on and Communica5on 2

3 The products we regulate 3

4 Medical Device Manufacturing Companies by Number of Employees % 3% 9% 12% <10 20 to to to 499 >500.7% Source: Dun and Bradstreet, Inc. Date:

5 Interesting Facts 18, ,000 DIFFERENT DEVICES ON THE U.S. MARKET 25,303 MEDICAL DEVICE MANUFACTURURES of which 8,995 ARE U.S. BASED MEDICAL DEVICE FACILITIES OF WHICH 13,363 ARE IN THE U.S. 50% 22,000 OF IMPORTED FDA- REGULATED PRODUCTS ARE CDRH PRODUCTS PREMARKET SUBMISSIONS ARE RECEIVED BY CDRH ANNUALLY 5

6 Importation of FDA-Regulated Products by Fiscal Year 40,000,000 35,000,000 30,000,000 25,000,000 20,000,000 15,000,000 10,000,000 5,000, CVM CTP CFSAN 2012 CDRH CDER 2015 CBER 6

7 We face a critical public health challenge The U.S. regulatory standard for market approval protects pa@ents by secng a high public health bar but imposes costs that make the U.S. marketplace less adrac@ve for innovators thereby delaying pa@ent 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 effec@veness standard Vision Patients in the U.S. have access to highquality, safe and effective medical devices of public health importance first in the world.

8 CDRH Strategic Priorities Strengthen the Clinical Trial Enterprise Establish a NaFonal EvaluaFon System for Medical Devices (NEST) Strike the Right Balance Between Premarket and Postmarket Data CollecFon Partner with PaFents Provide Excellent Customer Service Promote a Culture of Quality and OrganizaFonal Excellence 8

9 Strategic Priorities Noteworthy Results Established a Clinical Trials Program Implemented a flexible benefit- risk paradigm Increased the number of early feasibility studies (EFS) conducted in the US; Angel Catheter is first device in the EFS Program approved by the FDA Reduced to IDE full approval by over 1 year to 30 days In CY 2015, highest number of IDEs approved in first cycle since 2002 Established the Expedited Access Pathway (EAP) Program for breakthrough devices 47 requests for EAP designa@on received between program incep@on in April 2015 and July 2016; 21 requests granted, 11 IDEs already received Trained all CDRH staff on customer service; monitored and taken steps to improve service through survey results Improved customer sa@sfac@on to 84% overall; 90% among external customers In CY 2015, approved 70 novel devices; highest number since the beginning of the Medical Device User Fee Program 9

10 Flexible Regulatory Paradigms Applied Across the Total Product Life Cycle CDRH Vision Patient-Centered, TPLC Benefit-Risk Tradeoffs PMA, De Novo Benefit- Risk DeterminaFon Framework Guidance (2012) Postmarket Benefit- Risk DraY Guidance (2016) Evidence Generation Clinical Trials Regulatory Science MDDT DraY Guidance (2015) RWE DraY Guidance (2016) IDE Benefit- Risk DeterminaFon Framework DraY Guidance (2015) MDDT Pilot Program Unique Device IdenFficaFon Final Rule (2013) NEST Science of Patient Input Expedited Access Pathway Program (2015) PaFent Preference InformaFon Guidance (2016) Real-World Evidence Early Feasibility Study Paradigm Guidance (2013) PremarketPostmarket Balance Balancing Premarket and Postmarket Data CollecFon Guidance (2015) MDIC Medical Device InnovaFon ConsorFum (MDIC) PaFent Centered Benefit- Risk Project 10

11 National System Paradigm Shift Passive Surveillance Challenging to find right pre/post market balance without confidence in post- market data Ac5ve Surveillance to be=er protect pa5ents Leverage RWE to support regulatory decisions throughout TPLC Current Na5onal System Parallel track to clinical prac5ce Inefficient one- off studies Embedded in Health Care System (collect data during rou5ne clinical care) Shared system to inform the en5re ecosystem (pa5ents, clinicians, providers, payers, FDA, device firms) 11

12 Strengthening Our National System Taking the Next Steps 12

13 FDA s Vision for a National System Of, By, and For the Ecosystem Develops and communicates an evolving understanding of device benefits and risks throughout their marketed life using high- quality, linked electronic health informa@on Facilitates clearance and approval of new devices or new uses of exis@ng devices Iden@fies poten@al safety signals in near from a variety of privacy- protected data sources serving as a safety net Reduces burdens and costs of medical device postmarket surveillance 13

14 The Value Proposition would have access to safer, more devices and beder to make decisions Clinicians would have beder about the use of a given device in prac@ce Hospitals, clinical prac@ces, and integrated health systems would benefit from improved quality, reliable assurances of safety, and, possibly, relief from mul@ple repor@ng requirements Payers would benefit from access to high- quality evidence on device performance in clinical prac@ce, either alone or compared with other therapies 14

15 The Value Proposition Device manufacturers would be able to provide high- quality evidence at lower cost and in to support premarket approval, clearance, and payer coverage, coverage with evidence development and reimbursement decisions, to enable informed decisions about when devices should be used in and how to risk across the device s lifecycle, and to meet or reduce the need for postmarket study and adverse event repor@ng requirements In cases where the poten@al public health value of the device is high, some data that would otherwise be collected in the premarket secng could be responsibly collected aner market entry instead, owing to strong assurances that addi@onal postmarket data would be generated The system may obviate the need for FDA premarket review of some device modifica@ons because and informa@ve evalua@ons of the impact of those changes would occur in the course of rou@ne data collec@on In fact, the FDA has already taken some of these steps for a handful of device types 15

16 Key Features of NEST Purpose: To drive down the 5me and cost and increase the value and use of real- world data to meet the needs of medical device ecosystem stakeholders through a market- driven, collec5ve buying power approach and using a neural network data model: Independent Coordina5ng Center responsible for driving standardizafon of core data elements, data quality, use of common definifons, linkages between data sources, development of advanced analyfcs, and creafng data use agreements as condifons of data sources parfcipafng in NEST Overseen by a Governing Board comprised of representafves of ecosystem stakeholders that is responsible for establishing NEST s policies and procedures, seang strategic direcfon and priorifes, and direcfng investments by the CoordinaFng Center Business Model: Base funding from private and public sector sources NEST users pay for access to and analysis of data from parfcipafng NEST data sources Long- term sustainability and growth through administrafve fees for use of the system Success requires demonstrafng ROI, e.g., less expensive, more Fmely to use NEST 16

17 Recent Accomplishments Selected Center; FDA to award $3 million grant to the Center, $1 million for pilots Issued Use of Real- World Evidence Dran Guidance Establishing data use agreements with 14 registries to access 22 million records; more agreements in the works MDUFA 4 17

18 Partner with Patients Recent Accomplishments Issued Preference Guidance 51% of PMA, de novo, and HDE decision include a public summary of relevant pa@ent perspec@ve data considered Provided opportuni@es for CDRH employees to engage with pa@ents to obtain informa@on informa@ve to their work As of July 31, 2016, 50% of CDRH employees have interacted with pa@ents as part of their job du@es MDUFA 4 18

19 Adopt a Culture of Quality Case for Quality Create an environment that fosters market compe@@on around medical device quality and safety, not just effec@veness Collabora@ve effort, primarily through MDIC Establish a common framework for value- based quality among industry, FDA, purchasers, and users Iden@fy, collect, and share quality best prac@ces Create a Maturity Model Develop and pilot metrics for quality system performance, product quality, and corporate culture that align with Maturity Model Develop a strategy for a voluntary program to recognize independent evalua@on of product and manufacturing quality Explore regulatory and market incen@ves 19

20 Adopt a Culture of Quality CDRH Quality Management Program Issued and began implemen@ng a Quality Management Framework and stood up a QM Program in 2013 Oversaw implementa@on of MDUFA 3 independent assessment recommenda@ons in 2015 FEEDBACK!CDRH system for repor@ng and addressing sugges@ons and nonconformi@es implemented in 2014/2015 Customer Service efforts incorporated into the QM program in 2015 Management Review procedures implemented in 2016 ASQ Enterprise Membership and Training in 2016 Over 150 staff trained (Quality 101; Auditor) Document Control system and procedures in place, implementa@on to be completed in FY2017 Developing audit program, planning to expand quality 101 training, and to conduct execu@ve training in FY2017 MDUFA 4 20

21 Precision Medicine Flexible, streamlined, voluntary, approach with the clinical community using crowdsourcing, expert forums, and levels of evidence for next- sequencing (NGS) for germline diseases Scalable to other NGS and other tests Conformity with consensus standards developed by the clinical community to support validity Evidence generated from and interpreted by FDA- recognized, public databases to support clinical validity Recent taken Use of Standards for NGS Dran Guidance Use of Databases for NGS Dran Guidance PrecisionFDA 21

22 Other Noteworthy Policies Issued in (k) Dran Guidance Sonware Dran Guidance General Wellness Guidance Premarket Submissions for Interoperable Devices Dran Guidance Postmarket Management of Cybersecurity Dran Guidance Third Party Review Dran Guidance Use of Symbols in Labeling Final Rule of Specific from Devices Dran Guidance 22

23 Other Noteworthy Policies Issued in 2016 for Pediatric Guidance Human Factors Guidance and of Age, Race, and Ethnicity Data in Device Clinical Studies Dran Guidance Emerging Signals Dran Guidance Manufacturing (3D Dran Guidance Clinical Trials Guidance of IDEs for CMS Coverage Decisions Dran Guidance 23

24 MDIC Project Initiative Clinical Trial & Reform Goal: Improve the efficiency and cost- of medical device clinical trials by developing a framework for clinical trials simplifica@on Applica@on of Clinical Trial Prac@ces Goal: Based on prior clinical evidence, demonstrate it is possible to significantly reduce prospec@ve clinical evidence collec@on and pa@ent burden while maintaining safety and efficacy rigor by applying a MDIC developed model Pa@ent- Centered Benefit- Risk Assessment Goal: Advance the science of pa@ent preference assessment and improve our ability to include pa@ent perspec@ves in the development, pre- market approval and post- market evalua@on of medical devices 24

25 MDIC Project Initiatives Case for Quality Goal: Develop new tools, methods and metrics for innovators, manufacturers, regulators, and providers that improve product quality and experience Clinical Goal: Foster and test improvements by developing new tools and methods that will improve efficiency and cost- of tests Computer Modeling & Goal: Increase confidence in safety and efficacy, reduce clinical trial size and accelerate device review through regulatory grade computer models & 25

26 Streamlining the Pathway from Approval to Payment Parallel Review with CMS Medicine s Founda@onOne, incorpora@ng mul@ple companion diagnos@cs for cancer Private Payers Opportunity to par@cipate in pre- submission mee@ngs 26

27 Agency Level Activities Products Oncology Center of Excellence 27

28 International Medical Device Regulators Forum Unique Device Competent Authority Report Exchange Program Regulated Products Submission Adverse Event Device Registries Sonware as a Medical Device Medical Device Single Audit Program Premarket Review Meaningful harmoniza@on would likely require the voluntary reliance on safety and performance principles or standards for 510(k)s and the voluntary par@cipa@on in a progressive approval pathway for PMAs and de novos 28

29 Flexible Regulatory Paradigms Applied Across the Total Product Life Cycle CDRH Vision Patient-Centered, TPLC Benefit-Risk Tradeoffs PMA, De Novo Benefit- Risk DeterminaFon Framework Guidance (2012) Postmarket Benefit- Risk DraY Guidance (2016) Evidence Generation Clinical Trials Regulatory Science MDDT DraY Guidance (2015) RWE DraY Guidance (2016) IDE Benefit- Risk DeterminaFon Framework DraY Guidance (2015) MDDT Pilot Program Unique Device IdenFficaFon Final Rule (2013) NEST Science of Patient Input Expedited Access Pathway Program (2015) PaFent Preference InformaFon Guidance (2016) Real-World Evidence Early Feasibility Study Paradigm Guidance (2013) PremarketPostmarket Balance Balancing Premarket and Postmarket Data CollecFon Guidance (2015) MDIC Medical Device InnovaFon ConsorFum (MDIC) PaFent Centered Benefit- Risk Project 29

30 Learning Medical Device Ecosystem Total Product Life Cycle (TPLC) Framework INFORMATION EVOLUTION OFFLOW BENEFIT RISK EVIDENCE Premarket Pa5ent Access Review Benefit -Risk Benefit-Risk Premarket Decision Progressive Approval, Expedited Safety and Access Performance Pathway NEST Real-World Evidence National Postmarket Evaluation Surveillance System (NEST) Clinical Research Safety Net Incorporated Into Routine Clinical Practice INTERNATIONAL TIME TO MARKETHARMONIZATION 30

31 USER FEES: M4 31

32 Medical Device User Fee Act 4: Tentative Agreement Agreement in principle among FDA, AdvaMed, MDMA, MITA, ACLA Establish a dedicated premarket Quality Management team Conduct an Independent Assessment of M3 and M4 Improve: Consistency through enhanced supervisory oversight and rou@ne quality audits Quality and consistency of Addi@onal Informa@on and Major Deficiency leders Third Party Review Program Tracking and repor@ng of performance commitments; Time repor@ng repor@ng by end of FY2022 Recruitment and reten@on of FDA employees through a more effec@ve strategy and incen@ve pay 32

33 Medical Device User Fee Act 4: Tentative Agreement Improved Performance: Reduce average to decision to 108 days for 510(k)s and to 290 days for PMAs by the end of FY2022 Ramp up to of 70% of De Novo submissions within 150 days by the end of FY2022 Ramp up to providing wriden feedback on at least 1,950 Pre- Submissions within 70 days or 5 calendar days prior to the scheduled mee@ng, whichever comes sooner, by the end of FY2022 FDA will improve the CLIA waiver by applica@on process Enhance IT infrastructure to collect and report on structured data, allow sponsors to view individual submission status in near and develop structured electronic submission templates as a tool to guide Industry s prepara@on of premarket submissions 33

34 Medical Device User Fee Act New Efforts: 4: Tentative Agreement Digital Health: Improve consistency in review of sonware, streamline and align FDA review processes with sonware lifecycles Engagement: Develop internal to support the increased use of preference and reported outcomes in premarket submissions Real- World Evidence: Enhance internal and support establishment and pilots conducted by NEST Center to enable greater use of RWE in the premarket secng Seek authority to establish a conformance assessment program for cer@fied tes@ng laboratories that evaluate medical devices according to certain FDA- recognized standards Seek authority to permit FDA to keep over- collec@ons and eliminate 5th year offset Treat laboratory developed tests no less favorably than other device subject to MDUFA performance goals and report on performance Total MDUFA fee revenue target of $999.5 million over 5 years, to be adjusted for infla@on 34

35 CHALLENGES 35

36 Current CDRH IT Systems 36

37 Our Current Situation OIMT Bill (Initial FY16 Allocation) $36,599,417 CDRH IT O&M (Program Support) $14,000,000 CDRH IT Enhancements (Add l Needs) $12,500,000 $63M in FY16 37

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40 Thank You 40