Patient trust in health-care organizations and what it means for patient engagement
by Julien Rashid, Intern
The health-care world is filled with talk about data; patient records, clinical trial data, and patient-reported outcomes are used increasingly across the sector.
From a biomedical research and development (R&D) perspective, data is the key to understanding what patients truly need and the roadmap to direct where our work needs to focus.
Data leads to knowledge, and that knowledge is what empowers the discovery of cures for real people who are suffering from real diseases and disorders for which there are no current treatments.
But if patients feel that their touchpoints within the R&D pipeline are undependable or conducted with one-sided intent, they will not volunteer the data that is so precious to medical advancement.
Simply put, there is little hope for the discovery of cures without patient trust.
Across the R&D process, patients need to trust that all stakeholders value their perspectives and are partners in developing safe and effective treatments.
Many organizations have efforts to encourage and understand trustworthiness from a patient perspective. For example, the National Patient-Centered Clinical Research Network has dedicated events and forums to discuss this concept.
Generally, patients and caregivers are unlikely to engage beyond their care with entities they don’t trust. FasterCures set out to better understand patients’ and caregivers’ current perceptions of different stakeholders in medical research as part of the Health Data Basics project. Through a survey of over 700 patients and caregivers, we learned more about individuals’ perspectives on health data and how much they trust others with it. The survey results below indicate the spectrum of trust across different organizations.
According to the survey results, patients trust pharmaceutical companies/medical device manufacturers the least, with 42 percent claiming they do not trust them at all. Patients trust health-care providers the most, with 25 percent indicating they trust their health-care provider “a lot.” Only 3 percent do not trust their provider “at all.”
Patients moderately trust universities and hospitals. Approximately 80 percent of respondents are split between somewhat trusting and fairly trusting of both. Patients trust patient organizations and public health departments less, with many leaning towards “somewhat trust.”
While patients have at least some trust in most organizations, a plurality have no trust at all in insurance, pharmaceutical, or medical device companies. Two out of five patients do not trust pharmaceutical companies, and nearly half do not trust insurance companies. A scant 2 percent and 4 percent held “a lot” of trust in insurance companies and pharmaceutical companies, respectively.
What does trust have to do with data?
There is an evident pattern. In the survey, patients trust providers with whom they build relationships. The further the organization is removed from the patient, the lower the patient trust. Why? One likely cause is that patients don’t regularly interact with representatives from insurance companies, pharmaceutical companies, or public health departments, and most don’t belong to patient organizations. As a result, patients don’t have personal relationships with these organizations. To some patients, these organizations might feel less palpable, less human, and thus less trustworthy. Such a pattern parallels patterns of trust towards other large, “faceless” institutions. For example, Americans typically believe Congress, a “faceless” entity, is much more corrupt than their particular representatives.
Though patients mistrust insurance companies, these companies have access to big data and are well-equipped to improve individual-level health outcomes. Insurance companies have an incentive to keep their customers healthy and out of hospitals. With expansive access to behavioral and clinical health data, insurance companies are now trying to predict their customers’ health trajectories for more effective prevention. As data collection and analysis improve, these companies will use more insightful nudges. For example, a person with a sedentary lifestyle might be offered a reduced premium to join a gym. Insurance companies will need to improve patient trust to fulfill these initiatives and improve patient health.
Patients also distrust pharmaceutical and medical device companies, but these companies don’t have as much access to patients’ health data as insurance companies. Most identifiable health data that pharmaceutical and medical device companies have been collected directly from patients. Still, these companies have the potential to improve patient health through stronger trust.
Trust begins with transparency
It’s clear why greater trust is needed, but how can historically mistrusted organizations generate trust with patients and caregivers, and how can other organization move from a fair amount of trust to a lot? These organizations should increase data transparency and access for patients. Most patients are probably unaware of the depth of data these companies have. Giving patients insight and access to their data can help them improve their health, and with access to their data profiles, patients may even opt to change lifestyle or volunteer new data.
Patients are becoming increasingly independent, asking questions, joining patient organizations, and seeking to maximize their health care. This movement is reshaping health care into a more patient-centric system. This reshaping not only makes sense but is critical – patient data is precious. When the biomedical R&D system becomes untethered from patients’ lives, it loses focus on the very issues it exists to solve.
Many companies are now beginning to realize that building trust through transparency and patient engagement is beneficial for everyone. If these companies can help patients leverage their health data, patients will lead healthier lives, and, in that scenario, everyone wins.
Patient-Reported Outcomes: Design with the End in Mind
by Cynthia Grossman, Director, Science of Patient Input
There’s an exciting change underway in the biomedical research and development community.
Over the last several years, patient perspective data have become increasingly valued as evidence important to the way medical products are discovered, developed, and delivered. Rather than treating patients’ needs as an afterthought, patients’ priorities have begun to inform the products and discoveries that will best serve them. However, this paradigm change comes with its own set of challenges.
FasterCures recently published a white paper, “Patient-Reported Outcomes: Design with the End in Mind.” This publication was spurred by a workshop we organized to ask a multi-stakeholder group about the impact of patient-reported outcomes (PROs) and the challenges they face in gathering and utilizing them across the full spectrum, from early-stage research to care.
Our discussion with these experts made clear the benefits of PROs. Addressing unmet needs, facilitating better decision-making across every aspect of the R&D process, and aligning care priorities within diverse patient populations are all important objectives that PROs can help us reach more quickly.
To get there, we must overcome hurdles concerning the type of measures we are using, how we are gathering this data, and what resources we have to make sure they are reflected in decisions at the product level, the system level, and the level of the individual patient.
Our research on the issue has made clear that there is a lot of work to be done to ensure that PROs are utilized effectively and consistently throughout the entire R&D pipeline. I invite you to read our white paper and consider how you can help us push this important work forward.
Key Milestones in Progress Toward Patient Centricity
by Kim McCleary, Acting Executive Director, Managing Director
In 2012, digital strategist Leonard Kish declared, “If patient engagement were a drug, it would be the blockbuster drug of the century and malpractice not to use it,” a line seized by patient advocates and media alike to mark a new dawn in the patient-centricity movement. The advancements made that year through policy initiatives, collaborative efforts, and foundation-led programs opened the floodgates for the rush of activity that we are seeing in the patient-centricity movement today.
Our recent publication, From Aspiration to Application: 5 Years of Patient Centricity, provides stories of impact and milestones from the ongoing movement. This expanded timeline guides you through the events that laid the foundation for where we are today.
2012:
• The Food and Drug Administration Safety and Innovation Act (FDASIA) passes (including Prescription Drug User Fee Act [PDUFA]-V), which requires the Patient-Focused Drug Development initiative and structured benefit-risk assessment that incorporates patient perspectives.
• The Patient-Centered Outcomes Research Institute’s (PCORI) “Year of Engagement” establishes priorities and initiates funding for comparative effectiveness research that meaningfully incorporates patient perspectives on outcomes that matter to them.
• The Institute of Medicine assesses the National Institutes of Health’s (NIH) Clinical and Translational Science Award program and reinforces the “value of community engagement to shortening the time between discovery and application, and to improve the uptake of research findings by the public.
• The European Union’s Innovative Medicines Initiative commences support for an academy on therapeutic innovation to empower patients to work with regulators, health-care professionals, and industry to influence medicines development for patient benefit.
2013:
• Health Affairs dedicates its February issue to the growing body of evidence that when patients are involved in their health and health care, they tend to have better outcomes at lower cost.
• The Clinical Trials Transformation Initiative (CTTI), a consortium of life sciences companies, FDA, and patient organizations, establishes a Patient Groups and Clinical Trials project to develop models for better engaging patients as partners in clinical trials. It later popularizes a chevron diagram illustrating opportunities to engage patients across the full continuum of research.
• FDA’s Center for Drug Evaluation and Research hosts first of 24 Patient-Focused Drug Development meetings, each of which results in a detailed “Voice of the Patient” report.
• FDA’s Center for Devices and Radiological Health (CDRH) launches the Patient Preference Initiative with a two-day workshop to explore ways to better incorporate patient perspectives into its regulatory decisions.
2014:
• PCORI invests $250 million to create the National Patient-Centered Clinical Research Network (PCORnet), composed of Patient-Powered Research Networks and Clinical Data Research Networks, to support research that is faster, easier, less costly, and more relevant to making informed health-care decisions.
• National Health Council issues a stratification tool and guide to help patient organizations collect information about their communities that may be helpful to FDA in making benefit-risk assessments. This tool is one of the first created in response to opportunities enacted in FDASIA for more integration of patient perspectives.
• FasterCures convenes a “Benefit-Risk Boot Camp” to educate life sciences professionals, academics, and patient organizations about opportunities for patient perspectives to inform regulatory decisions about benefit-risk tradeoffs.
• Parent Project Muscular Dystrophy (PPMD) enlists more than 80 community members and stakeholders to draft the first-ever regulatory guidance submitted by a patient advocacy organization with the goal of accelerating development and regulatory review of applications for new medicines to treat Duchenne muscular dystrophy. The FDA issues formal guidance a year later, drawing heavily on PPMD’s draft.
2015:
• In his State of the Union address, President Obama outlines a new Precision Medicine Initiative to “empower patients, researchers, and provider to work together toward development of individualized care.”
• FDA approves the Maestro Rechargeable System for treatment of obesity, notable because the clinical study did not fully meet its primary endpoints but the device performed in line with patients’ stated preferences for benefit-risk tradeoffs.
• Medical Device Innovation Consortium issues a framework for integrating patient perspectives in the total product life cycle of medical devices, along with a catalog of methods for assessing patient preferences.
• CTTI publishes recommendations for effectively engaging patients in the development and conduct of clinical trials.
• CDER issues guidelines for FDA participation in externally led Patient-Focused Drug Development meetings to expand impact beyond 24 conditions selected under PDUFA-V.
2016:
• European Union Patient Academy for Therapeutic Innovation (EUPATI) releases the “Toolbox on Medicines R&D” in seven languages, attracting more than 110,000 visitors within the first year.
• CDRH names partnering with patients as one of three strategic priorities for 2016-2017 and issues final guidance on how FDA may consider patient preference information in the assessment of the benefit-risk profile of certain medical devices.
• 21st Century Cures Act (P.L. 114-255) passes the Senate on Dec. 7, 2016, and is signed into law on Dec. 13, expanding opportunities for patient input to be factored into regulatory decisions.
2017:
• Reauthorization of PDUFA and the Medical Drug User Fee Amendments (MDUFA) contain key provisions to enhance the science of patient input.
• FDA proposes the creation of a central Office of Patient Affairs to be housed within the Office of the Commissioner.
• FasterCures and Avalere release version 1.0 of the Patient Perspective Value Framework and methodology designed to assess the patient perspective on the value of medical products and health-care services.
• The People-Centered Research Foundation is launched to provide long-term sustainability for PCORnet and to pioneer novel methods for generating evidence that leads to better care and patient health.
• NIH launches the beta phase of the “All of Us” cohort of the Precision Medicine Initiative, enrolling the first group of individuals who will form the million-person research community of citizen scientists and academic researchers.
• The Food and Drug Administration Reauthorization Act, including PDUFA and MDUFA provisions enhancing the science of patient input, is signed into law on Aug. 18, 2017.
• FDA convenes the Patient Engagement Advisory Committee, the first committee dedicated to helping the agency increase integration of patient perspectives into regulatory decision-making.
Through FasterCures' work in our Patients Count program, we will strive to build on these accomplishments and foster patient-centric policies and practices that enable greater patient participation in decision-making.
Chordoma Foundation on the Creation of a Patient Navigation Service
Q&A with Josh Sommer, Executive Director
Q: Can you tell us briefly what the Chordoma Foundation’s Patient Navigation Service does?
The Chordoma Foundation’s Patient Navigation Service (PNS) helps individuals affected by chordoma overcome barriers to getting the best care possible, whether it’s finding the right doctors, understanding treatment options, or getting social and emotional support.
While CF has always served as a resource for patients and families in need of support, the launch of our PNS in April of 2015 allowed us to formalize and expand this offering, providing in-depth, personalized assistance to patients and caregivers around the world via e-mail, phone, and social media.
In the past 14 months, we have helped more than 450 individuals in 37 countries navigate the medical, emotional, and practical challenges posed by chordoma and connect with qualified care teams. Some examples of the assistance we provide include:
Answering questions and explaining important concepts about chordoma and treatment options.
Facilitating referrals to experienced physicians and treatment centers.
Identifying and providing information about clinical trials for which a patient could be eligible.
Recommending home health services, medical equipment suppliers, and other relevant vendors and service providers.
Providing information about programs and organizations that offer travel and lodging assistance, co-pay relief, and other benefits.
Supporting requests and appeals to insurance companies.
Q: What was the need – both in the chordoma patient community and at the Foundation – that the PNS was intended to address?
Chordoma is a rare disease with a lack of available information for patients, and the majority of people it touches aren’t prepared to address the many barriers that stand in the way of getting appropriate treatment. Too few providers are equipped to accurately diagnose and manage chordoma, and too many patients receive suboptimal care. Getting the right treatment from a trained care team can have a huge impact on patients’ outcomes, and can literally mean the difference between life and death.
That’s where we come in. From its start, CF has had a two-pronged mission: to lead the search for a cure and to improve the lives of those affected by chordoma. We view these objectives as inextricably linked, and recognize that if we can help patients overcome the barriers that exist in both care AND research, we can hopefully prevent them from getting suboptimal treatment and also improve their odds of beating the disease. Supporting the patient community through the dissemination of reliable information and the offering of practical and emotional support not only creates a better experience for everyone affected by chordoma, but it also enables us to build the relationships and trust needed to increase participation in clinical trials and advance research toward cures.
Q: What have been the biggest challenges in planning, launching, and sustaining this effort? What lessons have you learned?
The biggest hurdles we have had to overcome to create the Patient Navigation Service have been knowledge and training, information technology tools, and global population needs.
Because chordoma is such a rare and complex disease, and so few providers are adequately trained to address it, our staff needs to be armed with a significant amount of specialized knowledge to be able to respond and assist patients appropriately. There are many different scenarios and situations that patients face, and lots of different questions arise. Growing our knowledge base and providing the training needed to educate our staff about the nuances of chordoma has been a core focus in the first year of the service.
Even with the proper training and experience, however, there is still a huge amount of knowledge that has to be documented, requiring the development of a customized case management system to keep track of it all. From which doctors are experienced to how to contact them to the nuances of various health systems and care practices around the world, a breadth of information must be gathered and tracked on an ongoing basis to enable us to provide the best, most up-to-date guidance possible. Also, with multiple touch-points for every patient, we needed to be able to record the details of each interaction so that conversations could be picked up weeks to months later with no thread lost.
Finally, our global patient population presents a challenge as we must navigate cultural, political (e.g., health systems and processes), and language barriers in order to serve them, which requires a working knowledge of which doctors have chordoma expertise around the world and what the most reliable tools and resources are in dozens of countries.
As you can imagine, these all require both time and resources to get right, two things foundations like ours don’t tend to have a surplus of.
Q: What new capacities did your organization have to acquire or develop internally?
Before starting the PNS, we fielded calls from patients and family members looking for advice or assistance of various types. The demand outstripped our ability to keep up. Furthermore, given how much is at stake for patients, we recognized a need to provide a higher level of service from a skilled patient navigator.
To develop the PNS we first needed to hire the right person to run it. In 2014, thanks in part to a grant from Celgene, we hired our first Patient Services Manager, a social worker with over a decade of experience in education and social services. Early steps along the way to implementing the new service included creating a comprehensive knowledge management tool, developing a core set of principles to govern the service, establishing procedures to get feedback from our medical advisory board, and creating new educational materials (in multiple languages) to augment the messages delivered by our staff. More recently, we’ve been working on developing marketing materials and investing in outreach to the patient community to drive utilization of the service.
Q: Are there successes you can point to? Have there been benefits to the Foundation offering this service to patients?
As mentioned above, since our launch in 2015, we have helped more than 450 individuals in 37 countries find experienced doctors, make informed decisions, alleviate anxiety and concern, connect with peers, and avoid potentially devastating consequences of receiving inadequate, or just plain wrong, treatment. And the need for this service continues to grow. We anticipate serving another 500 affected individuals in the year ahead, and more beyond that.
Our PNS has also helped facilitate referrals to an ongoing phase II clinical trial at the National Cancer Institute and will begin assisting in patient education for three upcoming chordoma clinical trials set to begin later this fall. In the future, we expect that PNS will play a critical role in educating providers about clinical trials and connecting clinical research partners with our patient community.
For a personal example of a patient whose path was dramatically altered by her involvement with our PNS, read Monica’s story.
Q: What advice would you give other foundations that might be interested in undertaking a similar effort?
Plan to scale. Build the service not just for current demand, but for a multiple of that in the future.
Recognize that the size of your team, your standard operating procedures, your knowledge and case management technology, and potentially even your international capabilities will need to scale as demand grows.
Put in place a medical advisory board or some similar sounding board comprised of experts that can guide the development of your knowledge base, and provide advice and guidance to staff as questions arise.
Be prepared and willing to adapt “traditional” patient navigation practices to accommodate the geographic scope and unique needs of the patients you serve.
Patient-Reported Outcomes and the Elephant in the Conference Room
by Cynthia Grossman, Associate Director, Science of Patient Input
Patient-reported outcomes (PROs), defined as outcomes reported by patients without filtering or interpretation by others, are an important part of patient-centered research and care. The applications of PROs are varied and complex. They sit like an elephant in a room full of people, each of whom is touching his or her small part. No person can see beyond what is directly in front of him or her to the full picture.
At FasterCures, we see great potential in more effectively using PROs to advance the science of patient input. This year we are launching a new project under our Patients Count program to help provide a wider angle view of PROs and to reveal the big picture of their potential. The time is right for this approach.
While many agree in theory that PROs used in research and clinical care should be based on what patients report as being most important to their disease management, health and well-being, this has often not been adequately considered. For example, a PRO might capture whether a patient with arthritis can dress him or herself. In the patient’s experience, however, the time it takes to dress is a better measure of how well he or she is doing. This misalignment of measures hurts efforts to create patient-centered research and care.
Making PROs themselves truly patient-centered is one element of the challenge ahead. Another challenge is aligning various stakeholders’ interest in, and use of, PROs. As a starting point, we have identified the relevant parties and their interest or stake in PROs.
Patients
Patients and caregivers have the most “skin in the game.” There is no substitute for patients being able to base treatment decisions on outcomes that a) matter to them, b) are gathered from large numbers of patients like them and c) are specific to the treatment options available. Patients are eager for information collected from them to be used to improve and accelerate patient-centered discovery, design, development and delivery of medical products.
Drug and Device Companies
Companies want the PRO data they collect to be used in regulatory decision-making and the information they communicate to health-care providers, payers and patients about their medical products. Companies include PROs in clinical trials to help understand efficacy, safety, adverse events, tolerability and other potential benefits and harms. In spite of this investment, a recent study documented a steady decline in PRO data on labels for new drug applications. While companies are collecting PROs, the information about a product’s effect on these measures is not routinely making it to patients and providers for care decision-making.
Clinics
Hospital systems and provider groups use PROs and other tools to understand clinical care outcomes, or the way particular care approaches affect patient health and health-care utilization at the population level. As the health-care system as a whole moves toward value-based health care, there is a greater focus on engaging patients and measuring clinical outcomes that matter to them, rather than simply billing for services rendered.
Clinicians
Physicians and other health-care providers frequently use PROs to assess how well individual patients feel or function as a basis for shared decision-making about care options. And they use PROs to monitor how well an individual is doing on his or her course of therapy.
Payers
There is growing recognition that PROs can be useful in establishing the value and comparative effectiveness of medical interventions, both of which are important factors for payers determining medical coverage and reimbursement policies.
Researchers
Researchers apply their expertise to develop PROs that capture the domains of interest and meet scientific standards sufficient to be used in decision-making.
Regulators
The Food and Drug Administration (FDA) encourages sponsors to use PROs to help demonstrate the safety and efficacy of drugs and medical devices, both in clinical trials and after products are made available to patients. In spite of numerous efforts, including a 2009 guidance outlining a process to validate PROs, many companies feel the expected standards are too rigid and the process too lengthy and unpredictable. There is great interest in resetting expectations, and FDA has committed to revisiting its guidance by 2021.
Patient Organizations
Many patient organizations are contributing to efforts to create patient-centered PROs specific to their condition. They also assist in collecting PROs through patient registries and other means that can describe the natural history of the condition, inform research and regulatory decisions, and improve patient care and access.
With so many parties taking a stake in PRO development and utilization, and each from a different vantage point, it’s easy to understand why this particular elephant is tough to get in full view. Our objective at FasterCures will be to invite stakeholders to step back, take a broader look and approach the challenges from a more holistic perspective. From there we hope to identify opportunities to streamline the path to making PROs a stronger foundation for the science of patient input.
FasterCures to Further Accelerate Biomedical Innovation with $3 Million Grant from Helmsley Trust
The Leona M. and Harry B. Helmsley Charitable Trust has awarded FasterCures, a center of the Milken Institute, a three-year, $3 million grant to support its quest to improve medical research systems and accelerate the pace at which patients gain access to life-saving treatments and therapies. Specifically, the funding will support two of FasterCures’ key programs, Patients Count: The Science of Patient Input and Collaboration 2.0.
“Our efforts at Helmsley always seek to enhance the voice of the patient, and we believe that collaboration is the crucial key to yield great advances in the health field,” said Stephanie Cuskley, CEO of the Helmsley Charitable Trust. “Patients Count and Collaboration 2.0 are both outstanding opportunities to bring patients and all stakeholders together to accelerate the translation of scientific discoveries into innovative therapies that will improve lives. We are so excited to continue expanding our partnership with FasterCures through these important initiatives.”
“Since our founding, our goal has been to save lives by speeding up and improving the medical research system by focusing on the patient,” said Margaret Anderson, executive director of FasterCures. “We are honored that the Helmsley Charitable Trust chose to fund two of our pivotal programs.”
About Patients Count and Collaboration 2.0
Patients Count: The Science of Patient Input
As a greater number of stakeholders realize the importance of patient input to drug development and regulatory decision-making, they increasingly seek approaches that integrate the patient perspective into their work. Through Patients Count, FasterCures aims to:
Develop a training program to help funders better understand and navigate the regulatory aspects of the therapeutic development and approval processes;
Create an educational tool to help patients access and understand their health data, and thus increase engagement; and
Craft model legal provisions and principles to help reduce contractual, ethical, and regulatory challenges to foundation-industry partnerships.
Collaboration 2.0
The drug development process involves an ecosystem of diverse stakeholders, each with unique goals, viewpoints, and approaches. FasterCures’ Collaboration 2.0 program bridges these various parts of the ecosystem and supports cross-sector collaboration by:
Expanding the “Consortia-pedia Catalogue,” an online database of more than 400 R&D consortia, which encourages sharing of effective practices to bring partners together toward a shared goal;
Engaging with stakeholders to identify and promote solutions for sharing data and designing innovative clinical trials; and
Hosting Partnering for Cures, a summit that provides a dynamic opportunity for the biomedical research community to build connections and exchange information.
About the Helmsley Charitable Trust
The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting effective organizations in health, place-based initiatives, and education and human services. Since beginning active grantmaking in 2008, Helmsley has committed more than $1.8 billion for a wide range of charitable purposes. For more information on Helmsley and its programs, visit www.helmsleytrust.org.
About FasterCures
FasterCures, a DC-based Center of the Milken Institute, is driven by a singular goal: to save lives by speeding up and improving the medical research system. It focuses on cutting through the roadblocks that slow medical progress by spurring cross-sector collaboration, cultivating a culture of innovation and engaging patients as partners. FasterCures works across sectors and diseases to accelerate the process by which great advances in science and technology are turned into meaningful medical solutions for patients. www.fastercures.org | @fastercures
About the Milken Institute
The Milken Institute is a nonprofit, nonpartisan think tank determined to increase global prosperity by advancing collaborative solutions that widen access to capital, create jobs and improve health. It conducts data-driven research, convenes action-oriented meetings and promotes meaningful policy initiatives. www.milkeninstitute.org | @milkeninstitute
