Understanding Medical Device Users Through PPI: Webinar

Randy Horton

Published on: January 7th, 2025

Patient Preference Information (PPI): How Understanding Your Medical Device's Users Can Support Your Regulatory Submission

In today’s design-centered world, the most successful products are those designed with a deep understanding of their users’ lives. While MedTech has employed human factors to help design safe devices for our intended users, there’s more we could do to capture our user’s interest so that they receive the maximum clinical value of our medical devices.

Supporting the shift towards patient-centered design, the U.S. Food and Drug Administration (FDA) is actively encouraging medical device manufacturers to gather Patient Preference Information (PPI). PPI provides manufacturers with valuable data on the desires and limitations of their target patient audience. Additionally, the FDA has made it clear that, while voluntary, this information can influence regulatory decisions such as a medical device’s labeled indications for use. Put together, PPI could help manufacturers build a more clinically and commercially successful device.

Orthogonal and MATTER Chicago hosted a webinar on November 20th, 2024 at 2 PM CDT discussing PPI with Dr. Cynthia Grossman, who is leading the effort at the FDA in her role as Director, Division of Patient-Centered Development of the Center for Devices and Radiological Health (CDRH). She was joined by Olufemi Babalola, Health Economist, Division of Patient-Centered Development of the CDRH, who has been instrumental in advancing the FDA’s work on PPI, Todd Snell, Founder, Leadership Coach and Advisor at Camber Advisors, LLC, Christine Poulos, Senior Economist at RTI International and Vanessa DeBruin, Sr. Clinical Research Director, Coronary and Renal Denervation at Medtronic.

Webinar Summary

The Essence of Patient Preference Information (PPI)

PPI is a systematic approach to integrating patients’ voices into medical device development and regulatory processes. It involves quantitative and qualitative assessments of patients’ preferences regarding different treatment options, outcomes, and attributes.

Key Points:

Patient-Centered Approach: The FDA emphasizes placing patients at the heart of regulatory decision-making, focusing on patient experience data, patient engagement, and the science of patient input.
Total Product Lifecycle: PPI is applicable across all phases—from device development and clinical trial design to benefit-risk assessment and post-market activities.
Regulatory Guidance: The FDA has issued guidance documents to assist manufacturers in collecting and submitting PPI that meets regulatory standards.

Applications of PPI in Medical Device Development

Device Development and Clinical Trial Design: PPI helps manufacturers understand which features patients value most, informing target treatment profiles and performance targets for clinical trials. It ensures that clinical endpoints are meaningful to patients.
Benefit-Risk Assessment: In regulatory submissions, PPI provides context to clinical data by illustrating how patients weigh benefits against risks. This information can influence FDA approval decisions.
Post-Market Phase: PPI supports labeling expansions and informs post-market surveillance by capturing patient experiences with the device in real-world settings.

Case Studies Highlighting the Impact of PPI

Case Study 1: Ear Tube Delivery System for Children

Company: Tusker Medical
Challenge: Developing an in-office procedure for inserting ear tubes in children under local anesthesia had a lower success rate than the traditional operating room procedure under general anesthesia.
PPI Approach: A threshold technique study was conducted to determine the minimum acceptable success rate parents would accept for the in-office procedure.
Findings:
- Parents preferred the in-office procedure if the success rate was at least 68%.
- The PPI data informed the performance goals for the pivotal clinical trial.
Outcome: The device received FDA approval in 2019, demonstrating how PPI can influence clinical trial design and regulatory approval.

Case Study 2: Renal Denervation for Hypertension

Company: Medtronic
Challenge: Given multiple existing treatment options, understanding patient preferences for an interventional procedure (renal denervation) to treat hypertension.
PPI Approach: A discrete choice experiment involving 400 adults with uncontrolled hypertension was used to assess preferences among various treatment attributes.
Findings:
- Blood pressure reduction was the most important attribute for patients.
- Up to 30% of patients would choose an interventional treatment like renal denervation.
Outcome: The PPI data were included in the FDA submission, contributing to the device’s approval.

Case Study 3: Solo Home Hemodialysis

Company: NxStage Medical (part of Fresenius Medical Care)
Challenge: Removing the requirement for a trained partner during home hemodialysis treatments, which limited patient adoption.
PPI Approach: Conducted a direct preference assessment and a discrete choice model to identify patients’ willingness to accept risks associated with solo treatment.
Findings:
- 65% of patients were willing to perform dialysis solo despite the risks.
- Established risk thresholds that patients were willing to accept.
Outcome: The FDA approved an expanded indication allowing for solo home hemodialysis, increasing technology adoption and improving patient quality of life.

Challenges and Considerations in Implementing PPI

Resource Constraints:

Time and Cost: Conducting PPI studies requires significant resources, which may be challenging for smaller companies.
Expertise Required: Specialized knowledge of health economics, social sciences, and statistical analysis is necessary.

Strategies:

Early Planning: Incorporate PPI early in development to allocate resources effectively.
External Partnerships: Collaborate with organizations specializing in preference research to leverage their expertise.

Methodological Complexity:

Scientific Rigor: Ensuring methodological rigor is crucial for PPI studies to be considered valid scientific evidence.
Complex Methods: Techniques like threshold methods and discrete choice experiments require survey design and statistical analysis expertise.

Processes:

Cross-Functional Teams: Involve clinical experts, regulatory affairs specialists, and health economists to design and execute PPI studies.
FDA Engagement: Consult with the FDA early to receive guidance on study design and methodology, ensuring alignment with regulatory expectations.

Regulatory Uncertainty:

Impact on Submissions: Manufacturers may be concerned about how PPI will affect regulatory submissions, especially if results are unfavorable.
Complementary Data: PPI complements clinical data and does not replace evidentiary standards.

Clarifications:

Voluntary Submission: While PPI submissions are voluntary, they provide valuable context for regulatory decisions.
Transparency: Clear communication with the FDA can help mitigate uncertainties regarding using PPI.

Expanding the Use of PPI

The webinar highlighted that while PPI adoption is growing, there is significant potential for broader utilization, particularly in:

Early Development Phases: Integrating PPI during device design can lead to products that better meet patient needs and preferences.
Post-Market Activities: Using PPI in post-market surveillance can inform labeling updates and ongoing benefit-risk assessments.

Advancements Needed:

Methodological Innovation: Developing new PPI methodologies that are both rigorous and cost-effective.
Accessibility: Making PPI studies more accessible to companies of all sizes without compromising scientific integrity.

Key Takeaways

PPI is Essential: Incorporating patient preferences enhances medical device development and regulatory processes.
Growing Adoption: There is a positive trend in using PPI, but many opportunities remain untapped.
Collaborative Efforts: Successful PPI implementation requires collaboration among manufacturers, patients, and regulators.
Methodological Rigor: Ensuring scientific rigor is vital for PPI studies to be considered valid evidence.
Patient Benefits: Ultimately, integrating PPI leads to devices that better meet patient needs and improve health outcomes.

Conclusion

The integration of Patient Preference Information represents a paradigm shift toward patient-centered healthcare in the medical device industry. By systematically incorporating patients’ voices into device development and regulatory decisions, manufacturers can create products that align more closely with patient needs and improve health outcomes.

Future Directions:

Methodological Advancements: Continued innovation in PPI methodologies will make studies more efficient and accessible.
Industry Adoption: As more success stories emerge, PPI’s anticipated to become a standard practice in medical device development.
Regulatory Collaboration: Ongoing engagement with the FDA will help manufacturers navigate the regulatory landscape effectively.

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Speakers

Cynthia (Cyndi) Grossman, Ph.D., Director, Division of Patient-Centered Development, FDA

Dr. Cyndi Grossman is Division Director for Patient-Centered Development (DPCD) at the Center for Devices and Radiological Health (CDRH) at FDA. Cyndi came to FDA most recently from Industry, having also worked at NIH and in non-profit health policy. She is a social and behavioral scientist by training, and the throughline of her career has been leading efforts to bring the lived experience of patients, caregivers, and communities into medical product development and implementation. She has a doctoral degree in Clinical Psychology from the University of Vermont and completed post-doctoral training at Brown University.

Olufemi Babalola, MSc., MHS., PhD., Health Economist, Division of Patient-Centered Development (DPCD), FDA

Olufemi (Femi) Babalola, PhD is a health economist focusing on stated preference methods applied within a health care context. Specifically, how stated preference methods can advance regulatory science to inform benefit risk evaluations, clinical trial design and inform ordering of patient priorities. Dr. Babalola has worked at the FDA since 2016, and currently works in the Division of Patient-Centered Development (DCPD, formerly Patient Science and Engagement) within FDA’s Center for Devices and Radiological Health. Femi completed his Doctoral program in Health Economics at Thomas Jefferson University. He earlier received a Masters in Health Economics and Economics & Financial Economics respectively from John Hopkins University and the University of Nottingham, England.

Todd Snell, Founder, Leadership Coach and Advisor, Camber Advisors, LLC

Todd is an experienced leader, regulatory affairs and risk executive, strategic consultant, and coach who works with medical technology leaders and teams to improve performance and results. Prior to coaching and consulting, Todd was a Senior Vice President, Quality Assurance, Regulatory and Clinical Affairs at Fresenius Medical Care, Inc. (formerly NxStage Medical, Inc.), the world’s leading provider of products and services for individuals with renal diseases. Prior to Fresenius, Todd served in executive roles across Covidien, Genzyme, and General Electric Healthcare overseeing quality assurance, regulatory affairs and compliance for renal disease systems, parenteral fluids, vascular care products, rare disease treatments, genetic testing, and other therapeutic areas.

Todd received his BS in Systems Engineering from the United States Military Academy (West Point, NY) and his MBA in Operations Management from University of Colorado (Colorado Springs). Todd holds a current Regulatory Affairs Certification (RAC) and an Associate Certified Coach (ACC) certification from the International Coaching Federation.

Christine Poulos, Senior Economist, RTI International

Dr. Poulos is Senior Economist and Vice President of Health Preference Assessment at RTI HS. She has more than 25 years of academic, consulting, and government experience in stated preference research in health and environmental economics applications. She is an expert in the design of stated preference surveys and applying stated preference methods to measure the benefit-risk preferences of patients and other health care decision-makers. Her most recent work has applied these studies to support regulatory benefit-risk assessment of devices and biologics by the Food and Drug Administration, ACIP’s Evidence to Recommendation framework, structured benefit-risk assessment, and decision making throughout the clinical product development cycle for renal denervation, vaccines (including vaccines against influenza, coronavirus-19, tick-borne encephalitis, pneumococcal), severe emphysema, Alzheimers’ disease, multiple sclerosis, endometriosis, and diabetes, among others.

Vanessa DeBruin, Sr. Clinical Research Director, Coronary and Renal Denervation, Medtronic

With over 20 years of experience in the medical device industry, Vanessa DeBruin is a seasoned expert in clinical strategy from start-up to evidence generation. Holding a Master’s degree in Cellular and Integrative Physiology, she has a deep understanding of the scientific and clinical aspects of medical device development.

Vanessa has led and contributed to numerous global clinical trials across a wide range of therapeutic areas, including renal denervation, coronary artery disease, heart failure, neurostimulation, and arrhythmia management. She has extensive experience designing and executing complex clinical trial strategies, including innovative methods for patient recruitment. She has played a pivotal role in FDA Advisory Committee panel track trials and in developing evidence packages for regulatory submissions.

A key area of her recent work includes conducting a patient preference study for a renal denervation product, providing critical insights to support its FDA submission.

Moderator

Randy Horton, Chief Solutions Officer, Orthogonal

Randy Horton is Chief Solutions Officer at Orthogonal, a software consulting firm that improves patient outcomes faster by helping MedTech firms accelerate their development pipelines for Software as a Medical Device (SaMD), digital therapeutics (DTx) and connected medical device systems. Orthogonal makes that acceleration happen by fusing modern software engineering and product management tools and techniques (e.g., Agile, Lean Startup, User-Centered Design and Systems Thinking) with the regulated focus on device safety and effectiveness that is at the heart of MedTech.

Horton serves as Co-Chair for AAMI’s Cloud Computing Working Group, as well as AAMI CR:510(2021) and the in-process Technical Information Report #115, all of which address how to safely move medical device computing functions into the cloud. He is a frequent speaker at conferences and webinars, including events hosted by AdvaMed, AAMI, HLTH, RAPS and the Human Factors and Ergonomics Society (HFES).