2021 Annual Public Forum (Virtual)

June 1-2, 2021, Virtual

Welcome Remarks – Day 1

Pamela Goldberg, MBA, MDIC President and CEO, welcomed attendees to the MDIC 2021 virtual Annual Public Forum and set the stage for the two-day forum. Goldberg noted that MDIC was able to continue working with stakeholders to advance regulatory science for patient benefit despite the COVID-19 pandemic. Forum sessions highlight progress in 2020, which is also covered in the 2020 annual report.

Speaker:

Pamela Goldberg, MBA, MDIC President and CEO

Read the 2020 annual report.

Fireside Chat with Acting FDA Commissioner

Acting FDA Commissioner Janet Woodcock, MD, shared highlights of her experience at FDA and discussed key topics related to regulatory science and devices with MDIC President and CEO Pamela Goldberg during this fireside chat. Among her key accomplishments at FDA, Dr. Woodcock established patient-focused drug development, led the Critical Path Initiative to evolve regulatory review to align with scientific innovation, and led Operation Warp Speed to develop therapeutics for COVID-19.

Dr. Woodcock believes that the future of medical devices is bright. She expects to see a fusion of devices and drugs and biologics and more importance placed on diagnostics, which should be a foundation of medicine. During Operation Warp Speed, FDA staff stepped up to the increased workload to make a difference, while continuing to approve the usual number of devices. As a result of the pandemic, FDA will be different, with more work done remotely. Dr. Woodcock is also focusing on bringing modern, enterprise-wide technology to the agency.

Clinical Diagnostics Fingerstick Blueprint

Point of care testing (POCT) is performed at or near the site of a patient and provides test results in real time, enabling faster decision-making. Collection of capillary whole blood by fingerstick is one method of sample collection for POCT devices. As the demand for POCT devices that use fingerstick specimens increases, so does the need for scientifically sound and practical study design recommendations for evaluating their analytical performance characteristics.

MDIC’s new resource, Study Design Blueprint for Evaluating Analytical Performance Characteristics of Point of Care In Vitro Diagnostic Devices with Capillary Whole Blood (Fingerstick) Specimens, also known as the Fingerstick Blueprint, highlights design considerations for analytical validity studies of POCT devices that use fingerstick specimens including:

Study designs for candidate device clearance/approval as a non-waived test
Study designs for CLIA Waiver Application for the candidate device
Minimum sample size requirements for study execution
Use of surrogate samples
Data analysis for method comparison and precision studies, and more.

In this session, MDIC officially launched the Fingerstick Blueprint. Panelists discussed the Blueprint’s contents and participated in a panel discussion on how the Blueprint will benefit IVD manufacturers, FDA, CDC, and the Centers for Medicare & Medicaid Services (CMS).

Panelists noted that the Blueprint will be “enormously” helpful in POCT product development. In point of care settings, where operator experience and training requirements can vary, panelists expect the Blueprint to result in increased availability of high-quality robust assays that control for these variations.

Speakers:

Carolyn Hiller, MBA, Program Director, Clinical Diagnostics, MDIC
Shridhara Alva, PhD, Senior Director, Clinical Affairs, Abbott Diabetes Care (moderator)
Timothy Stenzel, MD, PhD, Director of Office of In Vitro Diagnostics and Radiological Health, OHT7, FDA CDRH
Corinne Fantz, PhD, Chief Medical Partner for Core Lab and Point of Care, Roche Diagnostics
Rex Astles, PhD, Lead, Data Science Team, Division of Laboratory Systems, CDC
Daralyn W. Hassan, MS, MT(ASCP), Clinical Laboratory Scientist, Division of Clinical Laboratory Improvement and Quality, CMS

NESTcc: Evolving the Future and Setting the Standards for Real-World Evidence

MDIC’s NESTcc is facilitating broader use of real-world evidence (RWE) in medical devices across the total product life cycle (TPLC) to accelerate access to safe medical technologies and inform clinical, regulatory, and reimbursement decisions. This session at MDIC’s 2021 Annual Public Forum provided an update on NESTcc’s progress, followed by a panel discussion on key topics related to the use of RWE in device development.

Through 21 Test-Cases NESTcc is exploring the feasibility of using real-world data and identifying areas where NESTcc could help create efficiencies. RAND is conducting an independent assessment of the Test Cases, 18 of which should be completed by September 2021.

NESTcc opened for sponsored research in June 2020 and already has several contracts. The research methods and data quality frameworks, released in 2020, are being transformed into RWE standards for sponsors, FDA reviewers, and other stakeholders.

Panelists noted that the use of RWE in early-stage device development is not one size fits all. Devices vary and RWE may fit well for some early-stage products but not others. As a neutral hub, NEST can facilitate and accelerate the more efficient development of trustworthy evidence and bring together collaborators to develop common data models. Panelists also discussed NESTcc’s role in unique device identifiers.

Speakers:

Sandra Siami, MPH, Senior Vice President, NESTcc
Adrian Hernandez, MD, MHS, Professor of Medicine at Duke School of Medicine, Vice Dean and Executive Director of the Duke Clinical Research Institute (DCRI)
Felipe Aguel, PhD, Deputy Office Director, OCEA, FDA CDRH
David Maggs, MD, Vice President, Medical Affairs, BD

Patient Perspectives: COVID-19, Access to Data and Care, and Engaging Patients through Digital Health

The COVID-19 pandemic accelerated the shift to telemedicine and digital health. In this panel discussion, four patient advocates shared their experiences with telemedicine and digital health, as well as opportunities for medical device manufacturers and industry stakeholders to expand access to devices and work with patients to enhance patient outcomes and safety.

Topics covered and key insights include:

Using digital health tools to improve healthcare for underserved and diverse communities:
- Ensure that these communities know about existing technology and enhance the accessibility and affordability of the technology.
Information and digital health solutions that would be useful to patients:
- Help patients learn about their diseases or conditions and give them access to the data they want from their devices.
How digital health and telemedicine can improve patient engagement in clinical trials:
- Patients have a voice in designing clinical trials that collect information that’s useful to clinicians and patients.
- Trial participants can virtually submit data and perspectives in the context of living their lives.
How to communicate effectively about clinical trials and reach audiences:
- Provide less information; Stress the meaning, not the details.
- Work with patients to develop understandable and meaningful summaries of clinical trial results.

Speakers:

Jijo James, MD, Chief Medical Officer, Johnson & Johnson Medical Devices Companies (moderator)
Heidi Dohse, Patient Advocate
Brian Walsh, Patient Advocate
Stephen Mikita, Patient Advocate
Cherise Shockley, Patient Advocate

Access our Science of Patient Input resources:

MDIC’s Patient Resources During COVID-19

MDIC’s Patient Engagement Forum

CDRH Town Hall

Leaders of FDA’s Center for Devices and Radiological Health (CDRH) highlighted the direction of the regulatory environment for medical technologies and the center’s partnership with MDIC. Each leader provided a brief overview of their work and then participated in a panel discussion.

Highlights of CDRH’s work with MDIC include:

MDIC’s Science of Patient Input initiative and CDRH’s Patient Science & Engagement Program are doing complimentary work to include the patient perspective in medical device development and evaluation.
High-quality, real-world data have increased the potential to generate robust real-world evidence for regulatory decision-making and new surveillance capabilities led by the National Evaluation System for health Technology Coordinating Center (NESTcc).
Early Feasibility Study (EFS) program advances, coupled with resources developed by MDIC, have helped the U.S. become a favorable location for conducting early phase medical device studies.
Steps to voluntarily improve device quality and the Medical Device Information Analysis & Sharing (MDIAS) initiative support FDA’s efforts to advance medical device quality in the shared Case for Quality initiative.
CDRH’s partnership with MDIC on cybersecurity focuses on disseminating knowledge to support the medical device ecosystem, including on threat modeling.

Topics covered and key insights from the panel discussion include:

Future role of CDRH’s partnership with MDIC in helping to shape the regulatory landscape for medical device innovations:
- Work products developed by MDIC are playing a critical role in CDRH’s programs and are increasingly being used by external stakeholders.
- MDIC’s positive impact on medical device innovation and safety will continue to expand, however, the extent of the impact will depend upon the ability to increase resources to support the work.
Lessons learned during the COVID-19 pandemic and how they will impact FDA’s future efforts:
- Lessons learned and activities begun or modified in response to the pandemic can be expanded to non-COVID activities.
- The pandemic illustrated the importance of public-private partnerships like MDIC in responding quickly and solving problems.

Speakers:

Nadim Yared, President and CEO, CVRx, and MDIC Board Chair (moderator)
Jeff Shuren, MD, JD, Director, FDA CDRH
Maureen Dreher, PhD, Assistant Director, Division of Clinical Science & Quality/OC, FDA CDRH
Andrew Farb, MD, Chief Medical Officer, Division for Cardiovascular Devices, FDA CDRH
Michelle Tarver, MD, PhD, Deputy Director, Office of Strategic Partnerships & Technology Innovation, FDA CDRH
Kevin Fu, PhD, Acting Director, Medical Device Cybersecurity, Program Director for Cybersecurity, Digital Health Center of Excellence, FDA CDRH
Francisco “Cisco” Vicenty, Program Manager, Case for Quality, FDA CDRH
Timothy Stenzel, MD, PhD, Director, Office of In Vitro Diagnostics and Radiological Health, FDA CDRH
Sean Tunis, MD MSc, Senior Advisor, Office of the Commissioner, FDA
Daniel Caños, PhD, MPH, Director, OCEA, FDA CDRH

Closing Remarks – Day 1

Pamela Goldberg, MBA, MDIC President and CEO, thanked attendees for participating in the first day of the MDIC 2021 virtual Annual Public Forum. Goldberg announced highlights of Day 2: including sessions on collaborative communities with FDA’s participation and, for the first time, a CMS Town Hall. She also noted additional on-demand only sessions that attendees can watch.

Speaker:

Pamela Goldberg, MBA, MDIC President and CEO

Welcome Remarks – Day 2

Pamela Goldberg, MBA, MDIC President and CEO, welcomed attendees to Day 2 of the MDIC 2021 virtual Annual Public Forum, which is providing updates from MDIC initiatives, programs, and partners. Goldberg noted highlights of Day 2, including a CMS Town Hall and a topic that’s been featured prominently in the news and is critical for patient safety: cybersecurity.

Speaker:

Pamela Goldberg, MBA, MDIC President and CEO

Medical Device Cybersecurity

Medical device cybersecurity covers the security of the physical device, as well as all of the steps in the total product life cycle (TPLC) such as design, manufacturing, distribution, patient use, monitoring, and the end of the product life cycle. The broad scope of medical device cybersecurity requires a collaborative effort among all stakeholders.

In this session, representatives of FDA, the Cybersecurity Health Sector Coordinating Council (HSCC), MDIC, and industry discussed their complimentary efforts to address cybersecurity challenges and strengthen cybersecurity in the healthcare sector. Panelists noted that information sharing is key and discussed resources and activities to help organizations of all sizes develop their cybersecurity plans, including:

Available Now:

HSCC Medical Device and Health IT Joint Security Plan: A voluntary framework of baseline standards to improve cybersecurity throughout the TPLC for organizations of all sizes and cybersecurity maturity.
FDA Medical Device Safety Action Plan: ontains FDA actions to advance medical device cybersecurity and, in general, to improve medical device safety.
Cybersecurity Best Practices and Recommendations: Published by the HSCC Joint Cybersecurity Working Group.

Coming Soon:

Model Contract Language: An HSCC working group is developing model cybersecurity contract language between medical device manufacturers and healthcare delivery organizations.
Updated FDA Draft Guidance: In 2021, FDA plans to release an update to its draft guidance: Content of Premarket Submissions for Management of Cybersecurity in Medical Devices.
Penetration Testing White Papers: MDIC’s new penetration testing (PT) working group will be releasing white papers on the role of software verification and validation in PT and best practices in PT.
Cybersecurity Benchmarking Survey and Report: MDIC has partnered with Booz Allen Hamilton to establish an industry benchmark to help organizations assess their cybersecurity maturity, develop improvement plans, and understand where they stand compared to their industry peers. By completing an anonymous survey, participating organizations will receive their own data and the aggregated data of all participants. Survey results will help determine areas of opportunity to improve cybersecurity practices for the ecosystem. Learn more and sign up for the Cybersecurity Benchmarking Initiative.

Panelists also discussed takeaways from the 2021 Colonial Pipeline attack, the 2021 Presidential Executive Order on healthcare cybersecurity efforts, and how to get involved with HSCC and MDIC cybersecurity initiatives.

Speakers:

Jithesh Veetil, PhD, Program Director, Data Science & Technology, MDIC
Suzanne Schwartz, MD, MBA, Director of the Office of Strategic Partnerships & Technology Innovation, FDA CDRH
Greg Garcia, Executive Director, Cyber Security Health Sector Coordinating Council
Rob Suarez, VP, Chief Information Security Officer, BD
Chris Tyberg, Division Vice President, Product Security, Abbott
Ken Hoyme, MS, Senior Fellow, Product Security, Boston Scientific

Access our Cybersecurity resources.

Case for Quality Collaborative Community

Representatives of FDA, MDIC, and industry provided updates on projects within MDIC’s Case for Quality Collaborative Community and participated in a panel discussion. Highlights of project updates include:

Voluntary Improvement Program:
- Results from 68 facilities and 29 appraisals are positive.
- The transition from a pilot to a program is a major step toward making voluntary improvement a mainstream practice.
CAPA Process Improvement Pilot Study:
- 22 pilot sites shifted their focus from compliance to problem solving.
- This risk-based approach to CAPA enables organizations to improve in months, not years.
Medical Device Information Analysis & Sharing (MDIAS):
- Industry, providers, and FDA are collaborating to access and analyze data and drive improvements.
- Goals are improved device quality, better predictive analysis, and improved patient outcomes.
Advanced Manufacturing Clearinghouse:
- This new MDIC and FDA project will engage with industry to enable people, processes, and technologies in manufacturing and across the total product life cycle.
- FDA wants to be part of solutions that enable technology to optimize organizational capability to better achieve goals.

The panel discussion included expectations for the Case for Quality Collaborative Community:

Build on previous work, expand into new areas that would benefit from collaboration, and identify synergies between the programs
More true collaboration to continue to solve common problems and create change
More solutions based on industry’s willingness to suggest new ideas instead of focusing on what FDA wants.

Collaborative Communities: Putting the Community in the Driver’s Seat to Navigate Healthcare Challenges

Collaborative communities, a new approach to public health, bring key stakeholders together to address shared challenges, achieve shared outcomes, and develop shared solutions. CDRH is one of many equal members of 10 collaborative communities and has developed a toolkit to assist stakeholders in developing new collaborative communities. This session provided an overview of collaborative communities and highlighted three collaborative communities convened by MDIC.

The National Evaluation System for health Technology Collaborative Community (NEST Collaborative Community) advances medical device innovation by leveraging real-world evidence, largely through studies conducted by its research network. Eleven collaborators have conducted 11 demonstration projects and 21 Test-Cases. NEST is also creating a roadmap to help move Unique Device Identifiers forward.

Visit NEST’s website.

The Case for Quality Collaborative Community (CfQcc) focuses on enhancing device quality and patient safety. The Voluntary Improvement Pilot Program has transitioned to a full program and led to the Accelerate Sustainable Capability project, which helps companies that want to improve. This collaborative community completed the Leadership Engagement Playbook to help develop a culture of quality. Members are working on projects to collect more consistent data, identify trends, and fast-track the CAPA process. Future plans include increasing engagement with patients, payers, providers, and small and mid-size companies.

Visit CfQcc’s website.

The Pathology Innovation Collaborative Community (PIcc), established in 2020, focuses on modernizing the clinical practice of pathology, including through digital pathology, machine-learning, artificial intelligence, imaging informatics, engineering, and computational and data science. This collaborative community aims to develop key performance indicators on pre-competitive regulatory science in pathology. Accomplishments include publication of three peer-reviewed articles.

Visit PIcc’s website.

In a brief panel discussion, speakers highlighted how stakeholders can become part of MDIC’s collaborative communities, what they would most like the collaborative communities to accomplish over the next 12 months, and more.

Speakers:

Jeff Shuren, MD, JD, Director, FDA CDRH (moderator)
Michelle Tarver, MD, PhD, Deputy Director, Office of Strategic Partnerships & Technology Innovation, FDA CDRH
Joseph Sapiente, AVP, Case for Quality, MDIC
Jithesh Veetil, PhD, Program Director, Data Science & Technology, MDIC
Robert Giffin, PhD, Director of Research, NESTcc, MDIC

CMS Town Hall

Leaders of the Coverage and Analysis Group and the Technology Coding and Pricing Group of the Centers for Medicare & Medicaid Services (CMS) provided updates on their work and offered clarification and advice on coverage and coding for device manufacturers introducing new technologies. Session highlights include:

The new Technology Coding and Pricing Group focuses on payment and coding issues related to new products and can assist device manufacturers pursuing Medicare coverage and payment.
CMS wants to work with stakeholders to answer their questions and find pathways for coverage and coding.
The Medicare Coverage for Innovative Technologies pathway opportunity has been delayed until December 15, 2021. Current pathways for coverage with evidence development for breakthrough technologies include claim-by-claim adjudication, local coverage determination, and requests for national coverage determination.
Artificial intelligence (AI) is new to Medicare and to healthcare. Although CMS is still learning about AI, analysis always begins with the device’s benefit category.
CMS recognizes that manufacturers face many challenges bringing a new technology to market and that beneficiaries want access to new technology. They are working on identifying opportunities to decrease regulatory complexity and innovator burden.
Many questions exist about real-world evidence (RWE), such as acceptable data sources, linkages and privacy, and analysis methods. CMS and FDA have been collaborating to try to reach a common approach to RWE.

Speakers:

Jo Carol Hiatt, MD, MBA, Vice President, Health Economics and Patient Value, MDIC (moderator)
Tamara Syrek Jensen, JD, Director, Coverage and Analysis Group, CMS
Steven Farmer, MD, PhD, Chief Strategy Officer, Coverage and Analysis Group, CMS
Jason Bennett, Acting Director, Technology Coding and Pricing Group, Center for Medicare, CMS

Access Health Economics and Patient Value and CMS resources:

Closing Remarks – Day 2

Pamela Goldberg, MBA, MDIC President and CEO, thanked attendees for participating in MDIC’s 2021 virtual Annual Public Forum. She noted that MDIC looks forward to applying the knowledge from the sessions to future work to provide critical tools so patients can have access to innovative, safe, and effective medical technologies. MDIC’s ability to meet industry needs is only possible because of the collaboration and expertise of partners across industry, academia, government, health care systems, and patient advocacy groups.

Annual Public Forum participants who would like to get involved with an MDIC initiative or program, or learn more about membership, can:

Visit MDIC’s Website: https://mdic.org

Email APF@mdic.org.

Speaker:

Pamela Goldberg, MBA, MDIC President and CEO

Artificial Intelligence Powered Cancer Diagnostics: How Regulatory Science Can Re-shape Digital Diagnostics

Digital pathology diagnostics for cancer that incorporate artificial intelligence or machine learning can increase accuracy and efficiency for pathologists and to identify new digital biomarkers and insights to help better diagnose, manage, and treat patients. But significant challenges must be overcome before these technologies become part of routine clinical practice.

Experts from the MDIC-convened Pathology Innovation Collaborative Community (PIcc) representing FDA, industry, academia, patient groups, and the physician pathologist community shared their perspectives on what it will take to make digital pathology cancer diagnostics part of routine clinical practice.

Panelists noted challenges in:

Evaluating model performance
Interoperability between a model and all of the systems involved in image acquisition, management, and display
Transitioning a model developed in a research setting for clinical use.

From a regulatory perspective, the scientific methods for evaluating safety and effectiveness of rapidly evolving technologies like digital pathology diagnostics often lag behind the technology itself. From a reimbursement perspective, the added value of digital pathology is unclear and current CPT codes do not account for all of the costs and the value that digital pathology provides.

Although there are many challenges, panelists were optimistic that the efforts of PIcc are bringing all stakeholders together for an “all-hands-on-deck” approach to developing standards for cancer digital pathology diagnostics. This approach includes incorporating the perspectives of patients, who have more access to their data and are more involved in their care than ever, as early as possible in the development process and conducting more patient-centered outcome studies that focus on demonstrating the added value of digital pathology diagnostics.

Speakers:

Joe Lennerz, MD, PhD, Medical Director, Center for Integrated Diagnostics, Massachusetts General Hospital
Esther Abels, MSc, Chief Clinical and Regulatory Officer, Visiopharm
Matthew G Hanna, MD, Director, Digital Pathology Informatics, Memorial Sloan Kettering Cancer Center
Edward Margerrison, PhD, Director Office of Science and Engineering, FDA CDRH
Laura Lasiter, PhD, Sr. Director, Policy and Research, PhRMA (previously Director, Health Policy, Friends of Cancer Research)
Markus Herrmann, MD, PhD, Assistant Professor of Pathology, Harvard Medical School

Building the Foundation for Active Surveillance

With funding from FDA, NESTcc is establishing a medical device active surveillance system and developing a roadmap to conduct timely, software-based real-world data analysis of medical devices to identify and refine safety signals for postmarket monitoring. This session highlighted NESTcc’s progress and the FDA perspective on NESTcc Active Surveillance, followed by a panel discussion.

NESTcc is building a cloud-based infrastructure using federated analytics to collect and analyze real-world data for medical device safety signal detection and assessment. NESTcc collaborators, representing 161 million patients and multiple types of real-world evidence, will participate in the study. The NESTcc cloud is a foundational infrastructure supporting FDA’s strategy to advance the use of real-world evidence through a multi-stakeholder, national system that employs active surveillance.

Topics covered and key insights from the panel discussion included:

The importance of active surveillance:
- Rigorous evaluation of issues and limiting the number of patients potentially affected
- Better serving patients through an ongoing relationship between industry, providers, regulators, and patients
Challenges in developing, implementing, and adopting active surveillance:
- Identifying the medical devices (unique device identifiers or UDIs) and obtaining the data
- Determining which outcomes to measure
Difference between NESTcc Active Surveillance and other approaches:
- NESTcc Active Surveillance is complementary to FDA’s mandated studies. It provides a timelier, ongoing approach across the device space in more diverse populations and settings.

Speakers:

Kathleen Blake, MD, MPH, Vice President, Healthcare Quality, AMA
April Lavender, MS, RAC, Senior Vice President Government Affairs, Cook Group
Rachele Hendricks-Sturrup, DHSc, MA, MS, Research Director, Future of Privacy Forum
Stephen Mikita, JD, Patient Advocate
Jacqueline Major, PhD, MS, Supervisory Epidemiologist, FDA CDRH
Paul Coplan, ScD, MBA, FISPE, Vice President and Global Head of Medical Device Epidemiology and Real-World Data Sciences, Johnson & Johnson
Alexandra ‘Zan’ Cha, PhD, MS, Chief Scientist, Booz Allen Hamilton
Omar Badawi, PharmD, MPH, FCCM, Director of Medical Device Safety, MDIC

Access our resources:

COVID Diagnostics Heroes

During the COVID-19 pandemic, MDIC members in industry and at FDA worked behind the scenes to make, develop, and regulate COVID-19 diagnostic tests. This session highlighted the stories of some of the heroes at FDA, Beckman Coulter, and Roche and how they innovated and compressed previous development and regulatory timelines to safely and quickly get COVID-19 diagnostic tests into the clinical setting.

Highlights of FDA’s work include:

Beginning at the start of the pandemic to develop data standards to harmonize and standardize diagnostic data in as close to real time as possible
Spurring innovation in diagnostics, particularly in point-of-care or home testing, to meet national public health data needs through a design-a-thon and a technology sprint
Preparing for the next pandemic by building future pipelines and a stronger public health information technology infrastructure.

Beckman Coulter developed serology and antigen assays for COVID-19 and Roche developed COVID-19 molecular assays, an antibody test, an antigen test, and a point-of-care test. The keys to their success in doing this quickly and safely were:

People who were motivated by patient needs
Putting the right people on the project teams
Ability to collaborate and coordinate
Strong involvement of leadership
Regulatory agility.

Speakers:

Carolyn Hiller, MBA, Program Director, Clinical Diagnostics, MDIC
Bryan Johnson, Staff Quality Assurance Scientist, Beckman Coulter
Courtney Walton, Director, Quality Assurance, Beckman Coulter
Erika Bladholm, Staff Clinical Studies Scientist, Beckman Coulter
Rachel Davison, Vice President, QRA, Beckman Coulter
Stephen Gaston, Vice President, Research, Beckman Coulter
Rita Hoady, MS RAC, Senior Manager of Regulatory Affairs, Roche
Sara Brenner, PhD, MD, MPH, Chief Medical Officer, FDA CDRH

MDIC External Evidence Methods (EEM) Framework for Leveraging External Data in Regulatory Decision-Making

Data collected outside of prospective clinical trials (external data) can potentially reduce the length of clinical trials, provide insights into device performance, and support regulatory decisions, including approval or clearance of a new device and indication expansion for a marketed device. MDIC’s External Evidence Methods (EEM) Framework is a practical guide to navigating through the nuts and bolts of leveraging external data. Developed in collaboration with biostatisticians from FDA and industry, the Framework:

Categorizes different sources of external data
Catalogs some of the traditional and novel statistical methods (Frequentist and Bayesian) for generating external evidence
Outlines considerations for use of external data for regulatory decision-making
Provides examples of past studies in which external data supported the approval or clearance of medical devices or expanded indications.

In this session, panelists:

Discussed the Framework’s contents
Highlighted an example of how external data were used to support indication changes for a drug-coated balloon catheter to treat peripheral artery disease
Described plans to use the virtual patient model, one type of external data, in a prospective clinical trial for diabetes.

Panelists also highlighted:

How the Framework provides a way to think about external data beyond real-world data and real-world evidence
How to ensure scientific validity and interpretability of study results when clinical outcomes associated with external data are already available
How industry statisticians can begin to consider novel statistical methods for generating external evidence
The biggest challenges in using external data and evidence in regulatory decisions.

MDIC released the EEM Framework for public comment in January 2021 and plans to release the final Framework in Fall 2021.

Speakers:

Randall L Schiestl, PMP, Vice President, Research & Development, Boston Scientific
Lilly Yue, PhD, Division Director, FDA CDRH
Ted Lystig, PhD, SVP Chief Analytics Officer, BridgeBio
Vandana Bhatia, PhD, Director Biostatistics, FDA CDRH

Medical Extended Reality (MXR): Toward Best Evaluation Practices for Virtual and Augmented Reality in Medicine

Medical extended reality (MXR), which includes augmented reality and virtual reality, is an emerging and rapidly changing field. MXR may improve efficiency and outcomes in surgery and interventional procedures, diagnostics, and therapeutics. But there are no rules for evaluating these technologies from the benefit-risk and outcomes perspectives.

Experts from MDIC’s newly launched Medical Extended Reality Device Working Group discussed how shaping the field from the ground up makes for an exciting, but also challenging task. Challenges include:

Understanding how to evaluate and adapt technology that’s often originally intended for entertainment for use in medical applications
Identifying and collecting the data necessary to demonstrate that MXR technologies will positively impact patients
Managing the rapid evolution of future generations of MXR technologies from the regulatory and reimbursement perspectives.

Panelists also noted:

Lessons of what did and did not work in evaluating technologies like wearables and 3-D printing can be applied to help move MXR forward.
A focus on MXR technology-specific outcomes instead of broader outcomes is key to explicitly demonstrating the benefit of MXR technologies.

MDIC is Seeking Working Group Members: Panelists encouraged MXR stakeholders to join the MDIC Medical Extended Reality Device Working Group and help shape the future of MXR. Membership in MDIC is not required. Email mxr@mdic.org to join the working group or to learn more.

Speakers:

Jithesh Veetil, PhD, Program Director, Data Science and Technology, MDIC
Ryan Beams, PhD, Physicist, FDA CDRH
Anindita Saha, Assistant Director, Digital Health Center of Excellence, FDA CDRH
Robert Fine, MS, Executive Director, International Virtual Reality and Healthcare Association
Jennifer N Avari Silva, MD, FHRS, FACC, FAHA, Co-Founder, Chief Medical Officer, SentiAR, Inc
Heather Benz, PhD, RAC, Associate Director, Clinical Research for Digital Surgery, Ethicon, Inc.

Science of Patient Input: Patient Involvement in Clinical Trials

Maximizing patient input in the design and development of medical device clinical trials has many benefits. After highlighting how to use a patient advisory board to improve trial enrollment and aspects of protocols and design, this session presents acase study of how a device company used a patient advisory board to identify barriers to recruitment.

The company developed a patient advisory board for a clinical trial for a new application of an existing therapy and met with patients via Zoom to gain their perspectives. Outcomes of involving patients in this trial were:

Deeper understanding of and appreciation for the patient’s experience by the project team
Improving/streamlining the informed consent document
Replacing a biopsy with imaging
Adding a quality-of-life patient-reported-outcome
Replacing the patient diary requirement with a passive actigraphy system to collect functional status and sleep outcomes.

The session ended with a panel discussion with FDA CDRH patient engagement leaders on how patient input can help lead to better trials and be used in regulatory discussions. Topics covered and key insights include:

How device companies can introduce patient perspectives into clinical trials in ways that are helpful for regulatory decision-making:
- Patients can help determine whether the right questions are being asked and help design trials that are practical to implement and complete
- In communicating about the trial, patients ensure that other patients will understand the risks, informed consent form, and recruitment materials.
FDA guidance on incorporating patient input in trials:
- Device companies should read Patient Engagement in the Design and Conduct of Medical Device Clinical Investigations: Draft Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders, September 2019.
- Device companies are welcome to contact FDA CDRH for advice about incorporating the patient perspective in clinical trials.
How CDRH engages patients:
- CDRH obtains patient input through patient conversations, aggregated responses from patient advocacy organizations, and in some scientific meetings.
- Patient input is used when evaluating specific medical devices and in policymaking.

Speakers:

Barry Liden, Vice President Patient Engagement, Edwards Lifesciences
Michelle Tarver, MD, PhD, Deputy Director, Office of Strategic Partnerships and Technology Innovation, FDA CDRH
Owen Faris, PhD, Principal Deputy Director, FDA CDRH

Access our resources:

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