Meeting Summary

2024 SCIENCE OF PATIENT ENGAGEMENT SYMPOSIUM

“PATIENT EXPERIENCES THROUGH THEIR LIFESPAN”

The National Health Council’s (NHC’s) Science of Patient Engagement Symposium was held on May 8 and 9, 2024, at the historic National Press Club in Washington, DC. This year’s Symposium brought together more than 150 leaders from patient organizations, non-profit groups, industry, academia, and government to discuss the meaningful engagement of patients and caregivers at different stages of the lifespan – through the lenses of pediatric, adolescent, young adult, adult, and aging populations.

The following are highlights from the two-day event.

DAY ONE

WELCOME AND INTRODUCTIONS

Randall Rutta, MA, CEO, National Health Council
Omar A. Escontrías, DrPH, MPH, Senior Vice President of Equity, Research, & Programs, National Health Council

In their opening remarks, Mr. Rutta and Dr. Escontrías welcomed attendees, thanked the event sponsors and panelists, and laid out the four key objectives of this year’s Symposium: 1) to understand the applicability of patient engagement research and practice across the lifespan; 2) to illustrate the importance of meaningful patient engagement in diverse populations; 3) to identify research opportunities between scientists and today’s patient advocacy community; and 4) to engage in innovative and thought-provoking discussion.

They noted that the NHC’s focus on patient engagement and the science around it enables its members to speak from both lived experiences and accompanying patient-centered data and evidence that is critical for decision-makers. And they stressed that meaningful engagement is when patients are recognized as equal partners and integrated from the very beginning in the development phases of scientific studies, research, drug and device design, regulation, value assessment, care delivery, and more – which is why it is never too early to fully engage them.

KEYNOTE REMARKS: HOW TO SEE INVISIBLE DATA: THE PurpLE HEALTH STORY

Moderator:

Kathryn G. Schubert, MPP, CAE, President & CEO, Society for Women’s Health Research

Speaker:

Anita Ravi, MD, MPH, MSHP, FAAFP, CEO, Co-Founder, PurpLE Health Foundation

Dr. Ravi began her remarks by providing an overview of her medical background and professional and volunteer work experiences, which included working at a federally qualified health center after medical school and becoming a health volunteer at a women’s jail where she worked with survivors of domestic violence and human trafficking. She then explained how those experiences caused her to challenge conventional thinking and start to look at providing quality care to patients in a different way. She conducted interviews to better understand from patients what would make them more comfortable getting care, and the learnings from those interviews lead her to form the PurpLE Clinic Project in 2015. Dr. Ravi noted the name means “purpose; listen and engage.”

The PurpLE Clinic Project’s model is lifelong trauma-informed primary care that spans the full part of the survivorship journey. The model does not use insurance, it functions beyond a free clinic because the full cost is covered (medication, labs, imaging, specialists), and it provides multidisciplinary care (primary care, therapists, a health care navigator, community partners). In 2019, the PurpLE Clinic Project became the PurpLE Health Foundation, the first health care center of excellence for survivors of gender-based violence.

Dr. Ravi believes that today’s health care systems still rely on conventional patterns and behaviors when it comes to what counts as impact, which may be insufficient to patients. This is why the PurpLE Health Foundation is working to create better conditions for trust and understanding between patients and caregivers. Those conditions – often the result of small tweaks that can be made to help the patient – include collecting practice-based evidence (e.g., collecting receipts from pharmacies that can be refunded); un-“invisibilizing” data (including trauma-informed billing, more customized explanations of benefits); and unlearning “vital” signs (e.g., optional weighing of a patient at the end of an appointment). She concluded her remarks by noting that the “win” for both the caregiver and the patient isn’t necessarily delivering the care, it is the learning from that delivery.

SESSION 1: IT’S NEVER TOO EARLY: ENGAGEMENT WITH PEDIATRICS AND CAREGIVERS

Moderator:

Elle Cole, Author, Podcaster, and Advocate

Panel:

Leanne West, President, International Children Advisory Network (iCAN) and Chief Engineer of Pediatric Technology, Georgia Institute of Technology
Colonel Steven L. Coffee, Chief of Staff, National Security Agency, Co-Founder, for Patient Safety U.S.

The session opened with the panelists sharing their individual backgrounds and experience in pediatric engagement in research.

Ms. Cole is an author, a mother to two, a podcaster, and a caregiver to a daughter living with sickle cell anemia and Type 1 Diabetes.

Colonel Coffee founded Patients for Patient Safety US in 2012 after his son, who was born with Galactosemia was misdiagnosed. Based on his experiences as a parent and caregiver – when his concerns were dismissed, and he felt marginalized – he started to look at the prevalent gaps and seams in the health care ecosystem and decided to take action. Patients for Patient Safety US has a broad network that includes patient safety champions around the country and strategic alliances with governmental institutions, civic organizations, industry, and health care facilities and demonstrates the power of their collective voices. The organization’s three priorities are: transparency, accountability and oversight, and patient and family engagement.

Ms. West is the head of iCAN, the International Children’s Advisory Network, which is committed to empowering every child, everywhere, and working to improve pediatric health, medicine, research, and innovation by sharing children’s voices. iCAN does this through a dedicated platform for children and families to give input and feedback into study designs, treatment plans, and educational materials of its industry partners; through 35 KIDS Chapters working at the local and international level to empower youth voices globally; and as a partner of the FDA Patient and Caregiver Connection Partner program and the Total Product Life Cycle Advisory Program.

The panelists discussed how best to communicate information to children, with the common thread being “human first.” Colonel Coffee stressed that trust and transparency between providers and patients are paramount and must be the building blocks to better and safer care. Ms. West noted that children want to be recognized as people, and their parents just want providers to be kind.

With regard to best practices for engagement with children and improving trust with caregivers, the key takeaways were:

Children are not tiny adults, and there should be a different way to engage with them at every stage of their development.
Children need to be heard and listened to better understand what they are actually going through.
Empower children by including them in conversations and allow them to have some say in what the outcome of their treatment should be.
Manage expectations on what success looks like – in more than just the clinical and research sense – so that pediatric patients and their caregivers feel success too.
Looking at pediatric patients as a resource all the way through the process will help change the course of medical research for the better.

SESSION 2: Future GOATs: ENGAGING WITH THE ADOLESCENT COMMUNITIY #IYKYK

Moderator:

Annie Kennedy, Chief of Policy, Advocacy & Patient Engagement, EveryLife Foundation for Rare Diseases

Panel:

Jonathan Shepherd, MD, Child, Adolescent & Adult Psychiatrist, Chief Clinical Officer, District of Columbia, Department of Behavioral Health
Elsa Kendall, Patient Advocate, EveryLife Foundation for Rare Diseases
Teressa Jackson, Caregiver, Hydrocephalus Association

The panelists began the discussion by sharing their unique perspectives on how to advance research and clinical care in ways that better address the needs of the adolescent population.

Ms. Kendall’s patient journey began when she was diagnosed with hemophilia at age 6. At age 9, she was given her first opportunity to advocate in front of the Virginia General Assembly and the U.S. Congress, and it was then that she began thinking about her own care and about how she was treated at her doctor’s office. As she started to find her own voice, she realized there was no dedicated, permanent space for women with bleeding disorders, and that led her to found the Red Ink Project – a nonprofit dedicated to rewriting the narratives of women and girls with bleeding disorders through education and raising awareness about issues uniquely affecting women and girls with bleeding disorders, including improving newborn screening protocols, unequal representation in research, and the period tax.

Ms. Jackson, the parent of a son who was diagnosed with Hydrocephalus at two months, has been forced to think every day as a caregiver about her son’s health and how his condition will progress over time. Now that her son is a teenager, she recognizes that he will soon start to have to speak for himself and learn how to navigate the health care system on his own. She shared the difficulty caregivers face in trying to find the right information for their children and how to transition them to be in charge of their own care.

Dr. Shepherd, who specializes in treating attention deficit hyperactivity disorder (ADHD), mood disorders, anxiety disorders, obsessive-compulsive disorders, and post traumatic stress disorders in adolescents, noted that teenagers are special, and their brains are not fully formed, so their care approach is very different than that of adults. His mission is to improve the well-being and mental health outcomes of children and adults by providing evidence-based, culturally responsive, and trauma informed care. He believes health care providers must consider that impulsivity, risk-taking, sensation seeking and trusting peers over parents are all normal functions of the teen psyche.

The panelists then discussed their experiences with adolescent patient-provider interactions and why direct communication is so important. Dr. Shepherd encourages parents to come into his office so they can experience his interaction with the adolescent patient; he also works with the parents to help them learn how to talk to each other and resolve minor conflicts. Ms. Jackson noted that while she and her son are not yet ready for separate appointments, the doctors do focus on and speak directly to her son, which she believes will help him learn how to speak on his own. Ms. Kendall admitted she feels lucky because her parents started preparing her from a very young age to be able to answer questions and advocate for herself. By the time she was 14, providers were talking directly to her, and she grew comfortable with that experience.

When asked about their experiences with and perspectives on clinical trials, the panelists all agreed that adolescents are underrepresented – particularly when it comes to females and historically excluded communities – and that age sub-populations must be considered. Ms. Jackson said her son participates in registries, but to date there have been no trials he has been able to participate in. Ms. Kendall spoke candidly of how excluding women and girls from trials can affect patients in their everyday lives. Once, when she tried a new medication and ended up in the emergency room she had no way of knowing if it was because of the new medication or something else, because only one woman had been part of that medication’s clinical trial.

The session concluded with the panelists stressing the need to put the adolescent patient at the center of treatment, while also ensuring the parents and/or caregivers get the information, guidance, and customized care they need to help their adolescent child navigate their health care as they transition to young adulthood.

SESSION 3: TAKING OWNERSHIP IN THE TRANSITION OF CARE FROM PEDIATRICS TO YOUNG ADULT

Moderator:

Sneha Dave, Founder & CEO, Generation Patient

Panel:

Taylor Hoffman, MS, Patient Advocate & Medical Student, University of Arizona
Maanya Tarnal, Patient Advocate, Epilepsy Foundation of America

This session’s panelists brought to life the challenges and opportunities facing young adult patients.

Ms. Dave, who was diagnosed with ulcerative colitis when she was six years old, serves as the Executive Director at Generation Patient, a young adult patient-led organization that represents young adults suffering from chronic and rare health conditions and focuses on enabling young adults to be able to lead conversations and drive health policy.

Ms. Hoffman was diagnosed with Type 1 Diabetes at age one, misdiagnosed with cystic fibrosis at age five (later correctly diagnosed as Celiac Disease), diagnosed with Hashimoto’s Thyroiditis at age 12, and diagnosed with rheumatoid arthritis at age 24. Now a third-year medical student at the University of Arizona Medical College-Phoenix, she advocates for the American Diabetes Association and uses her extensive lived experience to influence policy, pass legislation, and share insights on patient advocacy and access to care.

Ms. Tarnal, a sophomore at the University of Michigan, was diagnosed with epilepsy at age 16. As an Epilepsy Foundation intern in 2023 she helped conduct the Michigan Epilepsy Needs Assessment for the Epilepsy Foundation of America, which was designed for people with epilepsy, their families, and their friends to share their experiences and assess their medical care and quality of life.

The panelists agreed that an overarching challenge is defining who a young adult patient is and how the lack of clarification of this definition within the health care system adversely affects young adults living with chronic illnesses. There seem to be no clear-cut milestones, procedures, or practices to follow for young adult patients and their families navigating the system. As an example, Ms. Tarnal recounted how when she turned 18, with no warning, her parents no longer received notifications from her pharmacy about her prescription refills, even though her parents were paying for her care through their insurance. When pharmacies and care organizations do not have transition plans for young adults it can cause medication or care gaps.

The transition from young adult to adult presents additional challenges for the parent-child and doctor-patient relationship as well. The transition for a young adult patient from a pediatrician to an adult doctor can be very hard, as the patient is forced to develop a whole new relationship of trust with a provider who may seem more clinical and less caring, while learning to take charge of their own health care at the same time.

The panelists all agreed that, based on their own experiences, the current system makes managing chronic health conditions more difficult. They stressed the need for more personalized communication between providers, young adult patients, and their parents and caregivers at every stage – so they can cope with the challenges of medication management, doctor’s appointments, and navigating insurance together.

The panelists concluded the session by emphasizing that, while it is crucial to equip young adults with the skills necessary to manage their own health and advocate for themselves, it is unreasonable and impractical to expect young adults to have to devote huge portions of their daily lives doing so, spending hours on end dealing with insurance companies for example, especially while they are working and in school. Many patients – and not just young adults – are not properly equipped to understand how to “play the game.” It should be a right that every patient has an amazing experience with their health care providers. Sadly, today they view it still as just a privilege.

CLOSING REMARKS

Leah Howard, JD, President and CEO, National Psoriasis Foundation

Ms. Howard thanked the Symposium’s speakers and panelists for sharing their expertise and personal journeys. She noted that each of the day’s sessions provided key learnings for everyone involved in patient advocacy to take away – from how best to interact with patients and victims who have experienced sex trafficking and gender-based violence; to best practices for caregivers, families, and pediatric populations who may be struggling with new diagnoses for their children; to the special understanding needed when engaging with adolescents and their caregivers during the crucial transition from adolescent to young adult health care.

Reception Poster Presentations

Ten NHC member organizations presented posters, which focused on their organization’s patient engagement activities, during an evening reception. View PDFs of the posters below.

1) Patient Advocate Foundation
2) Prevent Blindness
3) Susan G. Komen
4) Alliance for Aging Research
5) Parkinson’s Foundation
6) Epilepsy Foundation
7) Asthma and Allergy Foundation of America
8) GBS CIDP
9) Bone Health and Osteoporosis Foundation
10) Foundation for the National Institutes of Health

DAY TWO

KEYNOTE: AI AND PATIENT ENGAGEMENT ACROSS THE LIFESPAN

Moderator:

Devin Jopp, EdD – President and CEO, Association for Professionals in Infection Control and Epidemiology (APIC)

Speaker:

Kapil Parakh, MD, MPH, PhD – Author & Senior Medical Lead, Google

Dr. Parakh began his remarks by noting that Artificial Intelligence (AI) is poised to make a significant impact in health care in a number of ways, and he offered the following examples: 1) AI products are being developed to synthesize complex medical information; 2) research organizations are interested in leveraging AI to support drug development; 3) AI-powered predictive models and analytics can help detect health trends and outcomes; and 4) AI software is being created to support provider education and improve patient care.

He continued by outlining the ways Google Health is working to deploy AI to help billions of people be healthier. Google’s efforts focus on three areas: consumers, caregivers, and the community. For consumers through Google applications such as YouTube, Health Connect, Fitbit, Pixel, and advertisements. For caregivers and care teams, through the Cloud, Fitbit health solutions, and Pixel. And for the community through grants and investments, health equity research, Open AI, investing in next generation start-ups, public health threat forecasting, and mobile sensing. He also stressed that Google’s focus on health equity underpins the company’s work across each of these areas.

Google has confidence in the promise of AI to power new forms of technology to reduce clinician burnout and boost efficiencies, to elevate consumer and patient centricity, and to advance life sciences research and development. To illustrate this point, he shared several examples of Google’s current work.

Deploying AI tools that record patient conversations with physicians which provide summaries almost in real time, leaving more time for the physician to apply clinical expertise and communicate with the patient.
Launching a series of ways for users to connect with emotionally supportive personal stories from creators on YouTube to help to reduce the stigma around mental and physical health topics.
Using Large Language Models to help shorten the time it takes to digest and understand complex health research.
Enabling developers and users to share and store health and fitness data across android devices via Health Connect and enhancing health sensing and personalization by investing in AI for Fitbit.
Conducting research focused on using AI to improve the screening and diagnoses of diseases.

SESSION 4: DISRUPTING THE PARADIOGM: REALITIES OF ADULT-BASED CARE FOR WOMEN’S RESEARCH

Moderator:

Tina Aswani-Omprakash, MPH, Senior Director, Strategic Partnerships, Sommer Consulting, & Patient Advocate

Panel:

Elizabeth Franklin, PhD, MSW, Head, U.S. Public Affairs and Patient Advocacy, Oncology, Sanofi
Ushma Patel, MSPH, PMP, Director of Patient Engagement, Innovation and Value Initiative (IVI)
Barbara L. Collura, MA, President & CEO, RESOLVE: The National Infertility Association

The panelists opened the session by sharing how they were each drawn to their professional and advocacy work in large part because of their personal health journeys and the dismissals they faced due to their gender. They began with personal stories before moving on to advice for researchers.

Ms. Collura was the recipient of RESOLVE’S infertility services, decided to volunteer at the organization, and then joined the staff.

Dr. Franklin, who suffers from fibroids, has known her entire life that she doesn’t want children, and was repeatedly refused a hysterectomy even though it would improve her condition. This is a telling example, she noted, of a woman not being listened to by medical staff.

Ms. Patel has worked at several nonprofits, specializing in patient engagement before joining IVI, but it wasn’t until she became a patient herself – with preeclampsia so severe she suffered a stroke five days after giving birth – that she gained a new perspective as a patient, and caregiver.

Ms. Omprekash went through her own difficult journey with Chrone’s disease. She was diagnosed at age 22 after pushing to get a colonoscopy, but she wasn’t taken seriously or given medications at the time, which caused her disease to fistulize. She has had nearly two dozen surgeries since.

The panelists touched on how infertility intersects with chronic illnesses such as cancer. As an example, Ms. Collura noted many clinical oncologists aren’t talking with their female patients about clinical guidelines for fertility preservation because they think their patients cannot afford them, or believe the conversation would be too emotionally difficult for the patient. And from an LBGTQ perspective, Dr. Franklin added that infertility and reproductive care must be much more inclusive of and culturally sensitive to all types of women.

The discussion then turned to how to lessen the burden carried by patients to advocate for their own health. Ms. Patel believes there is a huge need for increased awareness and education to empower women to speak up and trust their “gut,” and it needs to start at an early age. Women know when something is wrong. She also believes in increased and improved training for providers and researchers on how to better communicate and listen to their patients.

The discussion concluded with the panelists sharing thoughts and examples on how to improve patient engagement. Ms. Collura spoke about how RESOLVE deploys the power of their members to advocate at the federal level. They engage over 1,000 people annually to participate in their Federal Advocacy Day, and they train them on how to tell their stories in the most compelling ways. RESOLVE’s role is to inspire people to act, and that is where they have seen the most progress. Dr. Franklin offered that Sanofi takes feedback from its patient advocacy groups very seriously. The company has a “patient charter,” which articulates exactly what and how they are going to measure improvements, so they can put real data behind their commitments to programs that invest in clinical trial diversity. Ms. Patel sees a shift from person-focused engagement to a more system-wide, organizational level engagement, with patients getting involved in research and more opportunities – like the Symposium – to bring diverse voices together.

The panelists agreed that improved patient engagement means patients must be involved from the very beginning. That requires buy-in and commitment from leadership throughout the health care system, because these aren’t just women’s issues, they are human issues.

SESSION 5: FOUNDATIONAL EXPECTATIONS FOR PARTNERSHIPS IN RESEARCH

Speaker:

Kristin Carman, PhD, MA, Director, Public and Patient Engagement, Patient-Centered Outcomes Research Institute (PCORI)

Dr. Carman provided an overview of PCORI’s holistic approach to patient engagement. It begins with meaningful engagement, reflective of the PCORI community…to influence research to be patient-centered, relevant and useful…to make research and evidence understandable and accessible to the public…to establish trust and legitimacy for research findings…to foster successful uptake and use of research results.

She went on to describe PCORI’s journey to develop a new, more systematic and cohesive framework for partnerships and patient engagement in research. PCORI’s 10-plus years of practice-based experience led to the following findings:

Engagement influences the design and conduct of research studies.
Engagement improves study feasibility, acceptability, and quality as well as the relevance of findings.
Researchers have a better understanding of patients’ lives and of clinical processes and are committed to continuing engagement.
Partner involvement is dynamic, non-linear, and iterative, taking different forms over the course of the study.
Patients and other partners benefit from engagement by developing skills, contributing in a positive way, and possibly improving their own health.
With meaningful inclusion in decision making throughout the study, researchers and partners build trusting relationships that can benefit communities, community organizations, and academic institutions.

PCORI’s new guidance consists of three components:

An updated definition of meaningful engagement
Six Foundational Expectations
Information and Resources to Support Implementation of the Foundational Expectations

Dr. Carman concluded her presentation by stressing that engagement activities that consist solely of input (surveys and focus groups) are not meaningful, and that active engagement should include consultation, collaboration and shared leadership. The new Foundational Expectations for Partnerships can be found here.

SESSION 6: PEARLS OF WISDOM: NAVIGATING THE HEALTH SYSTEM IN THE FACE OF AGEISM

Moderator:

Adrianna Nava, PhD, MPA, MSN, RN, President, National Association of Hispanic Nurses and Research Scientist, NCQA

Panel:

Adriana Perez, PhD, ANP-BC, FAAN—Anthony Buividas Term Chair in Gerontology, Associate Professor of Nursing, University of Pennsylvania, School of Nursing
Carl V. Hill, PhD, MPH, Chief Diversity, Equity, and Inclusion Officer, Alzheimer’s Association

This session focused on how organizations can better acknowledge the needs of older patients and aging Americans, utilize new innovations, and assist in their transition and engagement.

The panelists first discussed the social determinants of health in older Americans and the structural barriers that often prohibit them from living their best lives. Dr. Perez described the interdisciplinary collaborative work that the National Association of Hispanic Nurses is conducting with Dr. Hill at the Alzheimer’s Association, which focuses on the barriers for inclusion of older Latinos in clinical trials. They have found that while there have been over 70,000 clinical trials focused on neurological disorders, and Alzheimer’s disease in particular, the patients who suffer more disproportionately from Alzheimer’s are not included – less than 2% are black, and less than 5% are Hispanic/LatinX.

Their work has examined these barriers at all levels. At the micro level: the individual and the family, at culture, and at language. At the meso level: the way clinical trials are designed, the makeup of principle investigators and the clinical trial staff that work on these trials – such as whether they are bilingual, bicultural, do they understand the challenges that potential research participants can have and how to engage that person. Most clinical trials require the participant to be English proficient, for example, and yet there is no scientific basis for that requirement. And at the macro level: policies and funding, where decisions are made, and whether the decision-makers are representative of the participants who should be included in clinical trials.

The panelists then went on to discuss how they are working to overcome these barriers. Dr. Perez described how her team in North Philadelphia is diverse sub-culturally – mostly representing Hispanics from Puerto Rico and the Dominican Republic – so they are learning how to design clinical trials in a community in ways that address this diversity. For example, they discovered early on, thanks to their staff, that some of the memory screenings they were using weren’t translating well. There is a question on a memory screen that asks the patient to list all the vegetables they can in one minute. The patient’s answer might be marked wrong if they said a platano – the Spanish word for both plantain and banana – was a fruit, but in some Spanish-speaking countries a platano is considered a vegetable. This highlights the need to have clinicians who understand culturally and linguistically where their patients are coming from.

Dr. Hill described how the Alzheimer’s Association is trying to get their resources to all communities and be more intentional about representation at the micro level. They are getting their partners to serve on their chapter boards, for example, and they brought all their partners together to advocate for the ENACT Act, which gives researchers at research centers the resources to better recruit and engage community members in Alzheimer’s and other dementia trials.

When it comes to navigating equity issues to overcome disparities and better include voices from historically underrepresented racial and ethnic communities, Dr. Hill shared his belief that the next frontier in disparities research is prioritizing intersectionality, because people often fit into more than one demographic category. He noted it is easy for an organization to prioritize certain groups because the government says it should, but to go deeper, to be effective, is to understand that people are more than one life domain. Understanding the influence of culture and religion that is sometimes above and beyond the racial or ethnic categories is very important.

Dr. Perez added that she would never go into the community and propose a study without understanding that community first. That approach guides her and helps her make sure that she is first looking at problems that are important to the community’s particular population.

The panelists then discussed how organizations can do a better job of building trust within these communities. Dr. Perez pointed out that most studies require a person to have a caregiver or a person with them that will be part of the study for the three or four years they are in the study, but that looks different for Latino families, because it is more normal to have several people helping to take care of someone, so they want more than one clinician to talk to. She urged the Symposium attendees to think about whether the procedures in place might exclude anyone, and most importantly, to make sure their actions match their words; otherwise, trust will be broken, and trust is the most important factor of all.

Dr. Hill described a study conducted by the Alzheimer’s association on race and ethnicity in 2021 which found that Latinos and Black Americans perceived or experienced discrimination in the dementia care system, and the top reason for their perceptions and experiences was because they felt as though they were not being heard. The Alzheimer’s Association has launched Project Brain Trust, which is organizing partners locally and putting together coalitions to hold community listening sessions to better understand how the lack of representation could differ in places like Chicago or Houston or Los Angeles. Their hope is to connect people in communities that are underrepresented to trials that are currently recruiting in those areas. By doing so, people in these communities will see themselves represented, will feel more comfortable that their participation will be appreciated and protected, and thus will have more overall trust in the process and the outcome.

SESSION 7: FINAL KEYNOTE REMARKS: ADVANCING HEALTH EQUITY THROUGH CLINICAL TRIAL DIVERSITY

Moderator:

Omar A. Escontrías, DrPH, MPH, Senior Vice President, Equity, Research & Programs, National Health Council

Speaker:

Namandjé Bumpus, PhD, Principal Deputy Commissioner, Food and Drug Administration (FDA)

Dr. Bumpus set the stage by giving the attendees some foundational considerations of biological science and pharmacology that inform why we need clinical trial diversity and why it is so important in advancing health equity. She noted that while there certainly is a moral imperative, there are some fundamental reasons why science itself and our understanding of basic biology is held back when we do not have clinical trial diversity.

She went on to explain the distinction between pharmacology (what a drug does to the body) and pharmacokinetics (what a body does to the drug and how the drug moves through the body), and how that can underly differences between people.

Dr. Bumpus then touched on pharmacogenetics, which is a key reason why diversity in clinical trials is so important. The differences between people can have a difference on the processing of drugs, and if a clinical trial is only looking at a small subset of the population it won’t enable a true understanding of the potential range of impact of individual genetics on a drug’s action or its ability to work. There is real value in understanding the pharmacology of a drug and communicating that to people when they are considering participating in a trial. This is foundational to clinical trial diversity.

She shared the FDA is doing that structurally – by providing recommendations on how to think about exclusion criteria in clinical trial study design, and by building a more inclusive workforce, with medical and public health professionals who reflect the patient populations they seek to reach.

Dr. Bumpus then touched on the work of the FDA’s Office of Pediatric Therapeutics to advance health equity in the context of research with children. They have formed a pediatric advisory committee, which provides advice around pediatric products, developing materials, and educating people about conducting pediatric clinical trials. With this increased focus around the inclusion of children, they have seen an increase not only in the number of pediatric clinical trials but also a new emphasis on how to utilize existing data to better understand their impact. The office has also issued draft guidance on the ethical framework for protecting children in clinical research. The framework is designed to make sure there are measures in place that make information and resources accessible for their caregivers and help them understand the benefits of and the science behind participation of children in these trials.

Dr. Bumpus shared some of the work focused on elderly adults that is currently being done in the FDA’s Office of Minority Health and Health Equity. She pointed to a recent study that looked at the perspectives of older adults with multiple morbidities on clinical trial participation.

In closing, Dr. Bumpus stressed that patient-centered engagement is the key to advancing health equity in clinical trials. She believes the better we understand the backgrounds of all patient populations and the more we communicate with them about the science behind clinical trials, the easier it is for them to feel connected to the work, to be more enthusiastic about engaging in some way, and to understand why it is so important.

CLOSING REMARKS

Omar A. Escontrías, DrPH, MPH, Senior Vice President, Equity, Research & Programs, National

In his brief closing remarks, Dr. Escontrías thanked the moderators, panelists, and attendees once again for participating. He concluded that the 2024 Symposium reinforced the importance of listening to patients throughout the course of their lives; the need to continue engaging with and learning about the needs and unique perspectives of specific age groups; and the collective obligation – of all the Symposium’s speakers, panelists and attendees – to work with the federal agencies and elected officials who drive the policies and regulatory processes that impact the NHC’s work every day.