Medtech Patient Engagement Tool Kit

Framing the Value of Patient Engagement for Medtech Product Developers, Milken Institute, 2026

1. Outlines key decision points across the total product life cycle (TPLC) of medtech product development where patient experience data can provide a meaningful impact on research and development (R&D) decisions and identifies how patient engagement can support the information and evidence they generate for a broad range of stakeholders.

Defining and Demonstrating the Value of Patient Engagement in Medtech Research and Product Development, Milken Institute, 2024

2. Examines the barriers and challenges to increased patient engagement in medtech R&D. It also identifies potential benefits and opportunities to expand patient engagement in medtech, proposes metrics to measure its impact, and offers recommendations to advance the state of medtech patient engagement.

Development and Application of a Patient Group Engagement Prioritization Tool for Use in Medical Product Development, Brian Perry et al., Therapeutic Innovation & Regulatory Science, 2021

3. Outlines a practical framework and prioritization tool to help medical product developers and patient groups identify valuable patient engagement activities across the R&D life cycle. The tool helps medtech teams evaluate engagement opportunities based on expected benefit; required investment; and potential impact on trial design, recruitment, retention, regulatory alignment, and patient-centered innovation.

Capturing the Value of Patient Engagement: Summary of Results of the 2016 Study of Patient-Centric Initiatives in Drug Development, Drug Information Association (DIA), 2017

4. Presents the findings of a study on patient-centric drug development and includes case examples and detailed metrics to describe the relative costs, ease of conduct, and impact of various patient-centric initiatives that may be helpful in determining which approaches to consider among different patient engagement options.

Planning to Engage: A Holistic Approach to Patient Inclusion, IQVIA

5. Provides a framework with four realms in which patient engagement can produce positive benefits, including clinical benefit, well-being, practicalities (e.g., access to care, support, and learning), and engagement and contribution. The framework provides an understanding of the different ways to engage patients related to their needs and preferences, while creating a shared language that supports communication among developers, providers, patients, and regulators.

Framing the Value of Patient Engagement for Medtech Product Developers, Milken Institute, 2026

1. Outlines key decision points across the total product life cycle (TPLC) of medtech product development where patient experience data can provide a meaningful impact on research and development (R&D) decisions, potential sources and methods for patient engagement, and how patient engagement can support the information and evidence they generate for a broad range of stakeholders.

Device Development Blueprint: Guidance for Early and Systematic Patient Involvement, Boehringer Ingelheim, Savvy Cooperative, 2023

2. Provides a step-by-step framework for integrating patient and stakeholder input in the early product development phase, from patient burden of disease and user-needs research through prototype testing, packaging, education materials, and post-release feedback. It helps medtech developers identify who to engage, when to engage with them, and which engagement methods to use at each phase of R&D to ensure devices are more usable, accessible, patient-centered, and aligned with real-world needs and preferences.

Practical How-To Guides for Patient Engagement, Patient Focused Medicines Development (PFMD)

3. Provides step-by-step guidance to help stakeholders plan, implement, and improve patient engagement activities across the medicine development life cycle. There are four separate guides on the following topics: early discovery and preclinical engagement, clinical outcomes assessment development, clinical trial design, and development and dissemination of peer-reviewed publications.

Guide to Patient Involvement in Rare Disease Therapy Development, EveryLife Foundation for Rare Diseases, 2022

4. Compiles resources, case studies, and best practices to help patient organizations and developers document patient experience, define unmet needs, identify meaningful outcomes, prepare for clinical trials, and demonstrate value in the rare disease context.

CTTI Recommendations: Patient Group Engagement, Clinical Trials Transformation Initiative (CTTI), 2015

5. Provides recommendations for effective collaboration between patient groups and research sponsors throughout the TPLC of R&D activities. It includes a depiction of potential patient engagement activities across different stages of the TPLC.

Patient Engagement Estimation Tool and User Guide, Medical Device Innovation Consortium (MDIC), 2025

1. Helps medtech teams determine the level and type of patient engagement needed in early development. It includes an interactive assessment, scoring framework, relevant planning questions, and guidance for using patient input during clinical discovery and product design.

Science of Patient Input (SPI) Resource Library, MDIC

2. Has reports, webinars, videos, and practical tools specifically designed to help medical device developers incorporate patient engagement across the total product life cycle. The resource organizes materials by development stage, making it easier for medtech teams to identify actionable approaches, best practices, and evidence-generation strategies relevant to their R&D and product development activities.

Patient Engagement Management Suite, PFMD

3. Brings together practical tools, guides, training, and good practices to support patient engagement across the product development life cycle. It provides step-by-step how-to guides, measurement and evaluation tools, training resources, and real-world examples to support consistent, high-quality, and impactful patient engagement in research and development.

Planning Patient Engagement, PARADIGM, 2020

4. Provides guidance on identifying the appropriate patient representatives for a given engagement activity. This document outlines what to consider when selecting patient organization partners and which competencies are helpful for patient engagement activities.

The National Health Council Rubric to Capture the Patient Voice, NHC, 2025

5. Provides a scorable rubric for incorporating patient perspectives systematically across the health ecosystem. It also provides definitions and a structured rubric to help organizations assess and strengthen how they can include the patient voice in decision-making.

Patient-Focused Drug Development (PFDD) Glossary, US Food and Drug Administration (FDA)

1. Defines key terms used in the agency’s PFDD guidance series and broader patient-focused medical product development work.

Glossary of Patient Engagement Terms, National Health Council (NHC)

2. Provides shared definitions to support clearer communication and more consistent patient engagement across the biomedical R&D ecosystem.

Patient Engagement Training for Medical Devices, Patient Focused Medicines Development (PFMD)

1. Introduces medtech professionals to the fundamentals of meaningful patient engagement. The training combines practical tools, examples, and exercises to help teams apply patient engagement across their work.

Patient Engagement Certification for Life Sciences Professionals, Alira Health

2. A five-hour, self-paced training designed to help life science professionals integrate the patient voice across the product life cycle. This fee-for-service course covers practical ways to embed patient engagement; US/EU regulatory information; legal and compliance concerns; and strategies to improve outcomes, access, and adoption. (Note: There is a cost associated with this program.)

AdvaMed® Patient Engagement Initiative, AdvaMed

3. This AdvaMed member patient engagement initiative provides training, coaching, peer exchanges, and tools to help medtech companies strengthen their patient engagement efforts. (Note: AdvaMed membership is required to access program.)

Patients as Advisors in Medical Device Clinical Studies, FDA Center for Devices and Radiological Health (CDRH), 2026

4. A series of courses designed to inform industry, caregivers and patients, patient organizations, and other health-care stakeholders about the role of patient engagement throughout the medical device life cycle, as well as about how patients can more effectively engage with the FDA and medical device developers.

FasterCures The Research Acceleration and Innovation Network (TRAIN) Participating Organizations, Milken Institute

1. Lists the 200-plus patient organizations involved in FasterCures’ TRAIN, an affinity network that is focused on building the capacity of patient organizations to fund and advance biomedical R&D.

Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience, FDA

2. Compiles patient-focused product development meeting reports and materials that capture patients’ experiences, perspectives, and priorities related to specific diseases and conditions. The meeting listing includes patient organizations that hosted or supported these meetings.

How TAP Facilitates Engagement with Non-FDA Parties, FDA, 2026

3. Provides a guide for how engagement with non-FDA parties (i.e., patients, providers, and payers) can support device development, clinical trials, and planning for market adoption and patient access. The website also provides a listing of non-FDA parties, including many patient organizations and provider societies that provide Total Product Lifecycle Advisory Program (TAP) innovators with strategic input on device research and development efforts.

CDRH Patient and Caregiver Connection, FDA, 2026

4. This is an FDA-led network of patient and caregiver organizations that helps connect the medical device community with groups representing people living with specific conditions and device experiences. The website lists partner organizations (including many patient organizations) that have engaged with the FDA Center for Devices and Radiological Health (CDRH) to provide patient-lived and device-related experience information.

Planning Patient Engagement, PARADIGM, 2020

5. Provides guidance on identifying the appropriate patient representatives for a given engagement activity. This document outlines what to consider when selecting patient organization partners and what competencies are helpful for patient engagement activities.

Considerations Guide to Implementing Patient-Centric Initiatives in Health Care Product Development, Drug Information Association (DIA)

6. Provides guidance to launch or strengthen patient-centric initiatives across the product development life cycle. Walks organizations through key stages such as setting objectives, preparing internal teams, selecting expert patients or patient groups, incorporating input effectively, and measuring success.

Patient Engagement Estimation Tool and User Guide, Medical Device Innovation Consortium (MDIC), 2025

1. Helps medtech teams determine the level and type of patient engagement needed in early development. It includes an interactive assessment, scoring framework, relevant planning questions, and guidance for using patient input during clinical discovery and product design.

Device Development Blueprint: Guidance for Early and Systematic Patient Involvement, Boehringer Ingelheim, Savvy Cooperative, 2023

2. Provides a step-by-step framework, including guidance, planning tools, and implementation considerations for integrating patient and stakeholder input throughout early medical device development, from disease and user-needs research through prototype testing, packaging, educational materials, and post-release feedback. It helps medtech developers determine who to engage; when to engage them; and which engagement methods, activities, and decision points to use at each stage of R&D, making it a useful resource for planning and implementing structured, patient-centered engagement activities across the product life cycle.

Patient Engagement Quality Guidance for the Medical Device Industry, Patient Focused Medicines Development (PFMD)

3. This practical guide for planning, developing, and assessing the quality of patient engagement activities introduces seven core quality criteria—shared purpose, respect and accessibility, representativeness of stakeholders, clearly defined roles and responsibilities, capacity for engagement, transparency in communication, and continuity and sustainability—that can be applied across medtech and other research projects.

A Playbook for Patient Engagement in MedTech in the US, Alira Health

4. Offers practical planning guidance for incorporating patient engagement into medtech development in the US context. It provides key patient engagement pain points, opportunities, value propositions, and activities across the life cycle of medtech R&D activities.

Patient-Centric Protocol Risk Assessment Tool, ICON

5. This interactive questionnaire is designed to help sponsors rapidly identify potential protocol-related, patient-focused risks to patient enrollment and retention and identify risk assessment and mitigation strategies. Importantly, the tool reframes patient engagement as a core risk-reduction strategy, highlighting how the proactive incorporation of patient perspectives can mitigate downstream product development risks and improve trial feasibility and success. (Note: Users will need to provide contact information to download resources.)

Engagement Plan Template, Patient-Centered Outcomes Research Institute (PCORI), 2025

6. Provides a structured framework for translating a study’s engagement strategy into an operational plan covering roles, activities, and stakeholder involvement over time. It is meant to help research teams implement meaningful engagement and document how those activities influence study outcomes.

Practical How-To Guides for Patient Engagement, PFMD

7. Offers step-by-step guidance to help stakeholders plan, implement, and improve patient engagement activities across the medicine development life cycle. There are four separate guides on the following topics: early discovery and preclinical engagement, clinical outcomes assessment development, clinical trial design, and development and dissemination of peer-reviewed publications.

Legal and Contractual Tools, PFMD

8. Provides guiding principles, reference agreements, and other tools that help to set up legal arrangements. It also points users to country-based guidelines and compliance resources, so engagements align with relevant codes of conduct and regulations.

Patient Engagement Remuneration and Fair Market Value, PFMD

9. Provides harmonized global standards and cocreated tools to support fair, transparent remuneration for patient engagement with the pharmaceutical industry. Its resources include global principles, activity and expertise frameworks, and a planner that helps define engagement activities and the expertise needed to determine appropriate compensation.

Fair-Market Value Calculator, National Health Council (NHC)

10. Provides companies with guidance on fair compensation and reimbursement for patients, caregivers, and patient representatives participating in engagement activities. It includes supporting materials such as a user guide, an hourly rate methodology, a results interpretation guide, and a glossary.

CTTI Prioritization Tool for Sponsors and Patient Groups,  Clinical Trials Transformation Initiative (CTTI)

11. Helps sponsors and patient groups identify and prioritize high-value opportunities to collaborate in clinical research. It uses a benefit-vs.-investment approach so both sides can compare ratings, discuss trade-offs, and decide which engagement opportunities to pursue together. (Note: This resource is hosted on an external website that does not appear to use secure encryption [HTTPS]. FasterCures does not control the security of third-party sites and recommends that users do not input sensitive or personally identifiable information.)

Engagement Tool and Resource Repository, PCORI

12. A collection of engagement-related tools and resources that allows users to search and filter results by topic, resource format, stakeholder type, health condition, and resource type.

Defining and Demonstrating the Value of Patient Engagement in Medtech Research and Product Development, Milken Institute, 2024

1. Examines the barriers and challenges to increased patient engagement in medtech research and development (R&D). It also identifies potential benefits and opportunities to expand patient engagement in medtech, proposes metrics to measure its impact, and offers recommendations to advance the state of medtech patient engagement.

Evaluating the “Return on Patient Engagement Initiatives” in Medicines Research and Development: A Literature Review, Lidewij Eva Vat et al., Health Expectations, 2019

2. Examines how patient engagement in medicines R&D has been evaluated across research priority settings, clinical trial designs, and early dialogue with regulators. It contains a list of benefits and costs stratified across a variety of stakeholders (e.g., patients, industry, regulators, health technology assessment [HTA] bodies) and related metrics for evaluation associated with those benefits and costs.

Expansion of Expected Net Present Value Framework for Evaluating Patient Engagement Methods, Clinical Trials Transformation Initiative (CTTI)

3. Adapts an expected net present value approach so organizations can model the financial and strategic value of different patient engagement methods in clinical trials. It offers recommended variables, example inputs, and data sources that companies can use to estimate how engagement may affect timelines, recruitment, retention, development risk, and overall profitability.

Capturing the Value of Patient Engagement: Summary of Results of the 2016 Study of Patient-Centric Initiatives in Drug Development, Drug Information Association (DIA), 2017

4. Presents findings of a study on patient-centricity in drug development and includes background information, case examples, and detailed metrics to describe the costs, ease of conducting, and impact of various patient engagement activities.

Considerations Guide to Implementing Patient-Centric Initiatives in Health Care Product Development, Drug Information Association (DIA)

1. Provides guidance to launch or strengthen patient-centric initiatives across the product development life cycle. It walks organizations through key stages such as setting objectives, preparing internal teams, selecting expert patients or patient groups, incorporating input effectively, and measuring success.

Capturing the Value of Patient Engagement: Summary of Results of the 2016 Study of Patient-Centric Initiatives in Drug Development, DIA, 2017

2. Provides concrete evidence, case examples, and metrics that demonstrate the operational and strategic value of patient-centric approaches in product development. It highlights measurable benefits such as improved recruitment and retention, fewer protocol amendments, and more patient-relevant end points, making it useful for building the business case for investment in patient engagement activities across an organization.

Development and Application of a Patient Group Engagement Prioritization Tool for Use in Medical Product Development, Brian Perry et al., Therapeutic Innovation & Regulatory Science, 2021

3. Provides a practical framework for identifying and prioritizing engagement activities based on their expected benefits, required investments, and strategic value. The resource offers evidence that patient engagement can improve trial quality, recruitment, retention, and alignment with regulator and payer expectations, helping teams make a clearer business and operational case for patient engagement across the product development life cycle.

Condition-Specific Meeting Reports and Other Information Related to Patients’ Experience, FDA, 2026

1. Compiles patient-focused product development meeting reports and materials that capture patients’ experiences, perspectives, and priorities related to specific diseases and conditions. The meeting listing includes any patient organizations that hosted or supported these meetings.

Human-Centered Design Toolkit for Treating Kidney Failure, Kidney Health Initiative

2. Designed for preclinical innovators and early-stage start-ups developing kidney replacement therapies, using human-centered design to help them better understand patient needs and translate those insights into product requirements. The approach could be applicable to other therapies and provides metrics that align with patients’ needs.

Guide to Patient Involvement in Rare Disease Therapy Development, EveryLife Foundation for Rare Diseases

3. Compiles resources, case studies, and best practices to help patient organizations and developers document patient experience, define unmet needs, identify meaningful outcomes, prepare for clinical trials, and demonstrate value in the rare disease context.

Patient Engagement Estimation Tool and User Guide, Medical Device Innovation Consortium (MDIC), 2025

4. Helps medtech teams determine the level and type of patient engagement needed in early development. It includes an interactive assessment; scoring framework; relevant planning questions, including those that dive into patient needs with respect to available treatments and treatment access; and guidance for using patient input during clinical discovery and product design.

Human-Centered Design Toolkit for Treating Kidney Failure, Kidney Health Initiative

1. Designed for preclinical innovators and early-stage start-ups developing kidney replacement therapies, using human-centered design to help them better understand patient needs and translate those insights into product requirements. The approach could be applicable to other therapies and also provides metrics that align with patients’ needs.

Maturity Model Assessment: Development of Digital Health Technologies, Medical Device Innovation Consortium (MDIC), 2025

2. Provides a structured benchmarking framework to help developers assess and improve digital health technologies with a focus on sustained adoption, user experience, shared clinical decision-making, and patient-centered product design. The resource helps teams evaluate how well their technologies meet the needs of patients, caregivers, and providers through an 80-question self-assessment tool; visual scoring outputs; real-world case examples; and curated best practices related to usability, accessibility, patient empowerment, workflow integration, and long-term engagement.

Human-Centered Design and Development in Digital Health: Approaches, Challenges, and Emerging Trends, Katerina D. Tzimourta, Cureus Journal of Medical Science, 2025

3. Examines how human-centered design can improve the usability, acceptability, and effectiveness of digital health technologies by embedding the needs of patients, clinicians, and caregivers throughout the development process. It reviews key design approaches, emerging trends, and common implementation challenges, including workflow integration and stakeholder alignment.

Insights and Best Practices for Planning and Implementing Patient Advisory Boards, Drug Information Association (DIA), 2017

1. Outlines best practices for advisory board structure, format, and implementation across drug development activities. It highlights how organizations use these boards to gather patient feedback on areas such as protocol design, consent materials, technology solutions, and trial communications and provides practical lessons alongside metrics on adoption and impact. retain for now and we can delete if they are not operational by the time we publish.

Rapid Participation Burden Survey Tool, ICON

1. Provides guidance to develop tailored surveys for patients and caregivers to assess participation burden in rare disease clinical trials. It contains a sample listing of questions and response options that can be used to assess clinical trial burden. (Note: Users will need to provide contact information to download resources.)

Patient Protocol Engagement Toolkit, TransCelerate Biopharma Inc.

2. Provides considerations for engaging patients and caregivers during protocol development with the goal of improving patient experience and reducing study burden. It includes a user guide, sample questions, visual aids, templates, and case examples to support early, structured conversations that help teams design more patient-centric clinical studies.

Patient Experience Mapping Toolbox, National Health Council (NHC)

1. Helps researchers capture and document patient experiences from diagnosis to living with a condition. It includes project planning and data collection tools such as interview guides, consent templates, interviewer training materials, and patient-facing visual aids to support more structured collection of patient experience data.

Conceptual Foundations for Measuring Disease Burden, Unmet Need, and Investment in Therapeutic Development in the United States, S. Schumm et al., eds., National Academies of Sciences, Engineering, and Medicine, 2025

1. This book chapter provides a foundational understanding of the burden of disease, including measures and approaches to estimating burden. The publication also calls out specific opportunities to integrate patient voices in the “Measures of Disease Burden on Individuals” section and in box 2-1.

Natural History Study Toolkit, National Center for Advancing Translational Sciences

1. Discusses the types of information collected in natural history studies, including how they can support therapy development and inform research priorities, and explains the possible advantages and challenges of each. It also includes a listing of resources on patient registries, natural history studies, grant programs, and patient-focused product development.

Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions, FDA Center for Devices and Radiological Health (CDRH), 2016

1. Outlines how the FDA and industry professionals can use a patient-focused benefit-risk framework when making decisions about medical device availability, compliance, and enforcement. It explains the factors that may shape actions such as recalls, shortages, and responses to quality or regulatory issues while emphasizing the importance of patient perspectives, available alternatives, and the potential public health consequences of limiting access to needed devices.

Patient Centered Benefit-Risk (PCBR) Framework, Medical Device Innovation Consortium (MDIC), 2024

2. Explains how patient-preference information can be incorporated into benefit-risk assessments for medical devices across the product life cycle. It outlines the value of including patient perspectives in regulatory submissions, discusses when and how such information can be collected, and offers considerations for selecting methods to evaluate patient preferences.

Best Practices for Communicating Benefit, Risk, and Uncertainty for Medical Devices, MDIC, 2024

3. Presents evidence-based guidance to help manufacturers, regulators, clinicians, and patients communicate the benefits, risks, and uncertainty of medical devices more clearly and effectively.

Quantifying Benefit-Risk Preferences for Heart Failure Devices: A Stated-Preference Study, Shelby D. Reed et al., Circulation: Heart Failure, 2022

4. Examines how people living with heart failure weigh the potential benefits and risks of implantable devices, using a stated-preference survey to measure the trade-offs patients are willing to make. It provides an example and approaches for leveraging quantitative evidence on patient risk tolerance and treatment priorities to inform product development, regulatory review, and clinical decision-making.

Patient Preference Information (PPI) in Medical Device Decision Making, FDA Center for Devices and Radiological Health (CDRH), 2026

1. Provides information on how PPI can be used to understand what patients value in the features of medical devices, including the benefits, risks, and trade-offs they are willing to accept. It highlights how this information can inform device design, clinical study development, and regulatory decision-making across the product life cycle.

Measuring Patient Preferences at the FDA Center for Devices and Radiological Health: Reflections and Projections, Christina M. Webber et al., Value in Health, 2021

2. Reviews how the FDA’s CDRH has advanced the use of PPI in medical device evaluation over the past decade through guidance, collaborations, and public engagement. It reflects on the agency’s experience incorporating patient voice into regulatory science, provides examples of how patient-preference studies have been used, and discusses future opportunities to apply preference data in areas such as emerging technologies and preference-sensitive decisions.

Adopting Patient Preference in Device Development Project, Medical Device Innovation Consortium (MDIC)

3. Compiles frameworks, guides, working groups, and practical examples to help manufacturers and other stakeholders collect and apply patient-preference data more effectively across the total product life cycle of medtech research and development.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force, John F. P. Bridges et al., Value in Health, 2023

4. Presents a good-practices road map for designing, conducting, and disseminating patient-preference studies so they are more useful and influential in health care decision-making. It organizes its recommendations around five key elements: context, purpose, population, method, and impact.

Attribute Library for Health Preference Studies, MDIC

5. A centralized database designed to improve the accessibility, comparability, and harmonization of patient-preference research by allowing users to search, compare, and analyze attributes from prior studies. It is intended to help researchers, industry teams, and regulators build on existing evidence and streamline the design of future preference studies.

Health Preference Study and Technology Registry, HPSTR.org

6. HPSTR.org is a web-based resource that provides patients, their family members, health care professionals, researchers, and the general population with easy access to information on publicly and privately supported health preference studies and technologies on a wide range of diseases and conditions.

Clinical Outcome Assessments (COAs) in Medical Device Decision Making, FDA Center for Devices and Radiological Health (CDRH), 2025

1. Defines the four types of COAs and describes how they can support benefit-risk assessment, eligibility criteria, safety, effectiveness evaluation, and labeling. Also provides case studies and related resources for regulatory use.

FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making, FDA, 2025

2. Provides systematic approaches to collect and use robust and meaningful patient and caregiver input that can better inform medical product development and regulatory decision-making. Guidance 3 discusses approaches for selecting, modifying, developing, and evaluating COAs to measure outcomes of importance to patients in clinical trials, and guidance 4 discusses the collection, capture, storage, and analysis of COA data and methods to better incorporate COAs into end points that are considered significantly robust for regulatory decision-making.

”How-To” Guide on Patient Engagement in the Development of a Clinical Outcome Assessment (COA) Strategy, Patient Focused Medicines Development (PFMD)

3. Provides a framework for more meaningful and systematic patient engagement in the development of a COA strategy within clinical trials. It is organized around eight steps, from setting up partnerships and identifying relevant disease concepts to implementing, interpreting, and communicating COA data.

New Medicines, Novel Insights: Achieving Patient-Guided Drug Development, Parexel, 2023

4. Provides definitions for four types of patient-centered COA factors that relate to quality of life on a treatment as well as considerations for COA development. It emphasizes sustained and systematic patient involvement, with particular attention paid to using patient-relevant COAs and patient-reported data to ensure products better reflect patient needs and priorities.

Inclusion of Patient-Reported Outcome Instruments in US FDA Medical Device Marketing Authorizations, Sophia T. Matts et al., Journal of Patient-Reported Outcomes, 2022

5. Reviews how patient-reported outcome (PRO) instruments were used in FDA medical device marketing authorizations over a six-year period, including the types of devices and regulatory decisions that incorporated patient-reported data. This resource can be used to better understand how PROs are being applied in device evaluation and to identify opportunities to incorporate patient-centered outcome measures into clinical studies, evidence generation, and regulatory submissions.

A 10-Step Guide to Standardising Patient-Reported Outcomes Data Collection in Healthcare: Insights from the Health Outcomes Observatory (H2O) Project on Overcoming Implementation Barriers, Gemma Galan et al., Journal of Patient-Reported Outcomes, 2025

6. Provides an example of how patient‑centered COAs can be developed by grounding measures in patients’ lived experiences, using PROs and observer‑reported outcomes that reflect what matters most to patients. It highlights the importance of stakeholder engagement, conceptual models of patient experience, and the adaptation of existing instruments to ensure COAs are meaningful, sensitive, and fit for use in clinical research.

Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices, FDA, 2025

1. Explains how the agency evaluates real-world data (RWD) to determine whether they are of sufficient quality to generate real-world evidence (RWE) that can be used in FDA regulatory decision-making for medical devices.

Examples of Real-World Evidence Used in Medical Device Regulatory Decisions, FDA, 2026

2. Compiles concrete examples of how RWD and RWE have been used to support medical device regulatory decisions across the product life cycle from 2020 to 2025. It serves as a practical reference for understanding acceptable sources (e.g., medical records, registries, literature) and how the agency has evaluated their use.

Elevating Your RWE Strategy with Direct Patient Input,  Medical Device Innovation Consortium (MDIC)

3. Designed to help the medical device community incorporate direct patient input into RWD and evidence generation across the product life cycle. It outlines practical approaches for collecting and standardizing patient perspectives, improving data quality and relevance, and using patient-informed evidence to strengthen clinical trial design, post-market surveillance, and regulatory decision-making.

Global Patient Experience Data Navigator, Patient Focused Medicines Development (PFMD)

4. Supports better evidence generation and health care decision-making by integrating patient engagement with patient experience data. It is designed to help stakeholders navigate how patient experience data can be identified, organized, and used more meaningfully in research, development, and broader health system decision-making.

Device Development Blueprint: Guidance for Early and Systematic Patient Involvement, Boehringer Ingelheim, Savvy Cooperative, 2023

1. Provides a detailed road map for integrating patient and stakeholder input into prototype design, usability testing, and iterative refinement throughout device development. The resource helps medtech developers plan and conduct experience testing activities—including usability and handling tests, human factors evaluations, ethnography, interviews, and focus groups—to refine device functionality, accessibility, ease of use, packaging, and educational materials based on real-world user needs and preferences.

Patient Engagement in the Design and Conduct of Medical Device Clinical Studies: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders, FDA Center for Devices and Radiological Health (CDRH), 2022

1. Explains how sponsors can voluntarily use patient engagement to elicit experience, perspectives, and other relevant information from patient advisors to improve the design and conduct of medical device clinical studies. It highlights the value of engaging patients early to help improve recruitment, retention, protocol feasibility, data relevance, and study execution while also clarifying which patient engagement activities do not constitute research and are not subject to FDA’s regulations.

Maximizing Patient Input in the Design and Development of Medical Device Clinical Trials, Medical Device Innovation Consortium (MDIC), 2021

2. Examines where patient input can add the most value in medical device clinical trials and provides a framework for deciding why, when, and how to incorporate that input. It focuses on device-specific trial features such as invasiveness, outcomes assessment, enrollment, and trial design and shows how patient perspectives can help define unmet needs, clarify preferences, and shape study elements that matter to participants.

Patient-Centric Protocol Risk Assessment Tool, ICON

3. This interactive questionnaire is designed to help sponsors rapidly identify potential protocol-related, patient-centric risks to patient enrollment and retention and identify risk assessment and mitigation strategies. The tool reframes patient engagement as a critical element for reducing product development risks and helps users identify, mitigate, and address potential risks. (Note: Users will need to provide contact information to download resources.)

How-To Guide on Patient Engagement in Clinical Trial Protocol Design, Patient Focused Medicines Development (PFMD)

4. Provides a practical framework for involving patients in clinical trial protocol design through a structured four-step process: preparing for collaboration, building effective partnerships, co-developing protocol elements with patient partners, and following up on feedback.

Patient Protocol Engagement Toolkit, TransCelerate Biopharma Inc.

5. Helps sponsors and other stakeholders engage patients and caregivers during protocol development to improve patient experience and reduce study burden. It includes sample questions, visual aids, templates, use cases, and follow-up resources that support structured conversations from early study design through the application of patient feedback.

Patient Technology Implementation Framework, TransCelerate Biopharma Inc.

1. Provides a structured approach for sponsors to adopt patient-facing technologies within their organizations in a more strategic, scalable, and patient-centered way. The resource is flexible for a broad range of applications of patient technologies and walks teams through stages such as use case ideation, proof of concept, pilot design, strategy development, and expansion.

New Medicines, Novel Insights: Achieving Patient-Guided Drug Development, Parexel, 2023

1. Synthesizes best practices for companies to incorporate the patient voice during product development through three key areas: integration with the patient journey, patient-relevant clinical outcome assessments, and patient-reported outcomes data.

AstraZeneca Human Factors Engineer Integrates the Patient Experience into Improving Drug Device User Experience, Andrew Goldstein, Fierce Pharma, 2024

2. Illustrates how patient input directly informs the design and improvement of drug-device products through the lens of an industry human factors engineer, highlighting key factors that influence the user experience—such as real-world use behaviors and usability challenges—and ways companies can proactively address them.

Medtech, Don’t Dismiss the Patient’s Role in Driving Commercial Success, Sudhanshu Bhatnagar, ZS, 2021

3. Explains two “broken links” that have hindered the medtech industry’s progress in patient engagement—dismissing its potential value and having a lack of knowledge about where to start—and discusses how considering the array of potential benefits can demonstrate its value. It outlines foundational steps companies can take to position themselves to invest in their patient-centric capabilities.

Division of Patient-Centered Development, FDA Center for Devices and Radiological Health (CDRH), 2024

1. Provides information and links to document how CDRH engages with patients, understands their perspectives, and proactively integrates their perspectives into the TPLC.

Total Product Life Cycle Advisory Program (TAP), FDA CDRH, 2026

2. Provides early, frequent, and strategic communications with the FDA for program participants and facilitates engagement with other key parties involved in the development of devices that impact public health. The website includes information on enrollment criteria, an engagement guide for working with non-FDA parties (i.e., patient organizations, providers, and payers), and a listing of non-FDA parties that engage with the program.

Clinical Outcome Assessments (COAs) in Medical Device Decision Making, FDA CDRH, 2025

3. Defines the four types of COAs and describes how they can support benefit-risk assessment, eligibility criteria, safety, effectiveness evaluation, and labeling. It also provides case studies and related resources for regulatory use.

Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices, FDA, 2025

4. Explains how the agency evaluates real-world data (RWD) to determine whether they are of sufficient quality to generate real-world evidence (RWE) that can be used in FDA regulatory decision-making for medical devices.

Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions, FDA CDRH, 2016

5. Outlines how the FDA and industry professionals can use a patient-focused benefit-risk framework when making decisions about medical device availability, compliance, and enforcement. It explains the factors that may shape actions such as recalls, shortages, and responses to quality or regulatory issues while emphasizing the importance of patient perspectives, available alternatives, and the potential public health consequences of limiting access to needed devices.

Patient Engagement in the Design and Conduct of Medical Device Clinical Studies: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders, FDA CDRH, 2022

6. Explains how sponsors can voluntarily use patient engagement to elicit experience, perspectives, and other relevant information from patient advisors to improve the design and conduct of medical device clinical studies. It highlights the value of engaging patients early to help improve recruitment, retention, protocol feasibility, data relevance, and study execution while also clarifying which patient engagement activities do not constitute research or an activity subject to FDA’s regulations.

Inclusion of Patient-Reported Outcome Instruments in US FDA Medical Device Marketing Authorizations, Sophia T. Matts et al., Journal of Patient-Reported Outcomes, 2022

7. Examines how patient-reported outcome instruments were incorporated into US FDA medical device marketing authorizations over a six-year period. It finds that PROs appeared in more than half of the authorizations reviewed and argues that expanding collaborative development and refinement of these instruments could strengthen the inclusion of patient voice in device evaluation.

Incorporating Patient-Preference Evidence Into Regulatory Decision Making, Martin P. Ho et al., Surgical Endoscopy, 2015

8. Describes the findings from a study on utilizing quantitative patient-preference evidence to inform regulatory approval decisions made by the FDA CDRH. The example demonstrates how structured patient input can be incorporated directly into regulatory evaluation.

How Patient Preferences Contribute to Regulatory Decisions for Medical Devices, Jeffrey Shuren et al., FDA Voice, 2017

1. Explains how patient preference information can be used as scientific evidence in regulatory decision-making for medical devices. It illustrates how structured patient input helps regulators evaluate benefit-risk information and make more patient-centered approval decisions, particularly in preference-sensitive contexts.

What is Meaningful Patient Involvement?, European Patients’ Academy on Therapeutic Innovation

10. Provides a framework through which patients can be engaged across the life cycle of medicines R&D and in specific European Medicines Agency activities, guidance for industry-led patient engagement, and links to relevant European regulatory processes and documents that describe patient engagement opportunities.

How Can Patient Preference Information Be Used in Payer Coverage Decisions and Health Technology Assessment?, Medical Device Innovation Consortium (MDIC), 2024

1. This white paper synthesizes perspectives on the patient information that matters for coverage and value decisions and highlights how structured PPI can be combined with clinical and economic evidence to inform assessments of value, unmet need, and likelihood of real-world adoption.

The Use of Real-World Evidence (RWE) Among Healthcare Payers: A Scoping Review, Lisa Masucci et al., International Journal of Technology Assessment in Health Care, 2025

2. Provides a literature review of the ways RWE is used by payers, including sources of real-world data such as patient experience data, clinical outcomes, and benefit-risk information, as well as barriers to its use, and its role in funding agreements between manufacturers and payers. The review highlights how RWE complements randomized controlled trial data by informing payer decisions on coverage, reimbursement, and formulary placement through insights on comparative effectiveness, cost analyses, treatment adherence, and patient-reported outcomes.

Assessment Framework for Digital Health Technologies, Institute for Clinical and Economic Review (ICER), Peterson Health Technology Institute, 2023

3. Outlines the user (i.e., patient and provider) information that informs health technology assessors (HTAs) and payer decisions, including patient-reported outcomes, usability, burden, and real-world use. It integrates these patient-relevant insights with clinical effectiveness, economic, and safety evidence to rigorously assess the adoption potential, affordability, and real-world value of digital health technologies.

ICER Patient Portal, ICER

4. Provides an overview of the purpose behind obtaining patient input and its relevance to policymakers. It also explains the opportunities for patients to contribute to efforts to address access and affordability, including through public meetings and reports.

Use of Patient Preference Studies in HTA Decision Making: A NICE Perspective, Jacoline C. Bouvy et al., The Patient, 2020

5. Identifies three primary uses for patient-preference studies along a technology pathway, from development to clinical use: to guide the selection of appropriate end points, to inform benefit-risk assessments, and to inform reimbursement decisions. It considers the role of patient input in evaluating non-health benefits and complementing clinical and economic evidence to assess overall value.

Seat Elevation Systems as an Accessory to Power Wheelchairs, Centers for Medicare & Medicaid Services (CMS)

6. Provides a real-life example of how patient information was considered alongside clinical evidence when determining CMS coverage. It reflects how payers could use patient-centered outcomes and patient experience data to determine whether a technology is likely, necessary, and reasonable to deliver value in care.

Planning to Engage: A Holistic Approach to Patient Inclusion, IQVIA, 2023

1. Explains how patient engagement can affect health-care provider-led support systems and sets forth a patient engagement framework in the development and positioning of therapies, establishing four realms where patient engagement can produce positive benefits in the provider and health-care context, including clinical benefit, well-being, practicalities (e.g., access to care, support, and learning), and engagement and contribution.

Revolutionizing Healthcare: Igniting Change through Patient Engagement, Gilde Healthcare, 2025

1. Identifies the essential elements for effective patient engagement from the investor perspective: culture, examples of practices and tools, measurement, and meaningful involvement. It addresses how investors use patient-derived evidence to assess market demand, scalability, and the likelihood that a technology will achieve sustainable adoption and returns.

Investment, Sustainability, and Impact Pivot Tool, Patient Focused Medicines Development (PFMD)

2. Provides resources that translate patient engagement data into investment-relevant insights. It helps users understand investor motivations, demonstrate the financial value of patient engagement, and embed it across the investment life cycle.

Patient Engagement Metrics Selector, Patient Focused Medicines Development (PFMD)

1. Provides a compendium of 115 metrics that can be used to capture and monitor the process, outcomes, and impact of patient engagement activities. The tool allows users to filter by six categories of metrics, such as stakeholder type and life cycle stage.

Evaluating the “Return on Patient Engagement Initiatives” in Medicines Research and Development, Lidewij Eva Vat et al., Health Expectations, 2019

2. Examines how patient engagement in medicines research and development has been evaluated across research priority settings, clinical trial designs, and early dialogue with regulators. It contains a listing of benefits and costs stratified across a variety of stakeholders (e.g., patients, industry, regulators, health technology assessment bodies) and metrics for evaluation associated with those benefits and costs.

Considerations Guide to Implementing Patient-Centric Initiatives in Health Care Product Development, Drug Information Association (DIA), 2017

3. Walks organizations through key stages of building and advancing patient-centricity in an organization, such as setting objectives, preparing internal teams, selecting expert patients or patient groups, incorporating input effectively, and measuring success through a series of questions that prompt users to consider quantitative and qualitative impact measures and how lessons learned from engagement are applied and communicated.

Medtech Patient Engagement Case Study Library, Milken Institute

1. Provides a repository of case examples that show the impact of patient engagement across the total product life cycle (TPLC) of medical device, diagnostic, and digital health (i.e., medtech) research and development activities. The library contains a range of patient engagement case examples covering different product types, applications, and purposes.

Patient Engagement Book of Good Practices, Patient Focused Medicines Development (PFMD), 2020

2. Provides a curated collection of real-world examples of effective patient engagement initiatives across the TPLC. These examples can be leveraged by developers to understand concrete patient engagement approaches and lessons learned and as resources to showcase the impact of meaningful patient engagement.

Made with Patients Awards, PFMD

3. Showcases real-world examples of effective patient engagement by recognizing projects that demonstrate high-quality, meaningful partnerships with patients and seeking to encourage broader adoption of these practices across the health sector.

Patient-Focused Medical Product Development Case Examples, National Health Council (NHC)

4. Provides concrete, practice-based examples that illustrate effective patient engagement approaches and the resulting improvements in trial design, decision-making, and product relevance.

Measuring Patient Preferences at the FDA Center for Devices and Radiological Health: Reflections and Projections, Christina M. Webber et al., Value in Health, 2021

5. Synthesizes published examples of patient engagement in medtech R&D, highlighting concrete approaches and outcomes reported across studies. The authors also identify patterns of effective patient engagement, including what worked in practice, the types of impacts achieved, and future avenues for growth.

Total Product Life Cycle Advisory Program (TAP), FDA Center for Devices and Radiological Health (CDRH), 2026

1. Provides early, frequent, and strategic communications with the FDA for program participants and facilitates engagement with other key parties involved in the development of devices that impact public health. The website includes information on enrollment criteria, metrics, processes, as well as additional information and resources on participating in the program.

Innovators Circles, MDIC

2. Convenes clinicians, medtech companies, patient organizations, and regulators to exchange experiences and ideas as well as to collaborate on priority topics such as patient engagement. They provide structured peer-to-peer discussion to share lessons learned and shape best practices across the medtech ecosystem. (Note: MDIC membership is required in order to access the program.)

AdvaMed® Patient Engagement Initiative, AdvaMed

3. Provides training, coaching, peer exchanges, and tools to help medtech companies strengthen their patient engagement efforts. (Note: AdvaMed membership is required in order to access the program.)

Medtech Synapse, Patient Focused Medicines Development (PFMD)

4. Provides a digital community and knowledge-sharing platform to discover, discuss, and share patient engagement events, initiatives, and resources. By mapping active organizations and showcasing real-world patient engagement, it enables peer-to-peer learning across the medtech ecosystem.

Collaborative Community, MedTech Color

5. Convenes diverse stakeholders to produce thought leadership addressing major medtech challenges, such as patient-centered design. Through best practices, educational resources, and virtual meetings, they aim to produce effective strategies to enhance medtech product development.

Patient Engagement Collaborative, Clinical Trials Transformation Initiative (CTTI), FDA

6. Convenes patient representatives, regulators, and other stakeholders to jointly discuss how to strengthen communications, education, and patient engagement in medical product regulation.

HTAi Interest Group for Patient and Citizen Involvement in HTA, Health Technology Assessment international

7. Focuses on creating practical resources, developing methodologies, and sharing best practices for systematically incorporating citizen and patient perspectives into health technology assessments.