How Can Patients & Researchers Realize Value from Real-Time Real World Data?
What does Real World Data look like?
What are the elements needed for a better study participant experience?
What is the Participant Digital Front Door™?
How can it empower study participants and researchers?
First, I provide an overview of all the hurdles that patients face in clinical trials. Next, I will introduce the front door and how it directly addresses each hurdle. Last, I examine the ways researchers also benefit from patient-consented Real World Data.
My Father’s Patient Journey
I have witnessed first-hand the wonders that can happen for patients by collecting and owning all their health data in one place. My father had a Cologuard® test and we were alarmed by his positive results. He had visited many different providers in recent years and had various lab work done as well. Through the Digital Patient Front Door, I was able to combine all of my fathers’ health data in one place for Kaiser Permanente’s top-tier oncologists and pathologists to run a virtual tumor board in six hours, not only accurately diagnosing his condition but also getting him to surgery within only a week. He prevented his colon cancer from spreading by having a 52mm tumor removed from his ascending colon. Real-time access to high quality data saved my father’s life.
“Health Data is Medicine.”
In this way, through the Digital Patient Front Door, my father was empowered to engage with his comprehensive health data and share it seamlessly with his physician and care team for fast and accurate diagnosis and treatment. Care is much more effective when it is provided before a health condition has advanced to a later stage. I realized right away that Health Data is Medicine.
Kaiser immediately understood the value of my father’s data. They arrived with consent papers as my father was recovering from his surgery. He was invited to participate in a clinical trial but he denied it because he did not understand its value or what they were trying to get him to sign to be a part of.
What if there was a Participant Digital Front Door that could empower all potential study participants like my father to consent to enroll in clinical trials, connect to all of their health data in a highly intuitive and engaging way, and provide researchers true Real World Data, in any format they choose? If my father had access to it, he would have definitely consented to enroll in the trial because it would have been clear to him that he would directly advance research and drug discovery.
Before we explore the Participant Digital Front Door™ and all of its defining elements, we first need to understand patient data challenges in clinical trials. That is always where I first focus: what are the patients’ needs?
Patient Data Challenges in Clinical Trials
The main challenge of clinical researchers is the same challenge faced by patients: Data.
Healthcare is already difficult to navigate when we are healthy, but it gets even more complicated when we get sick and become patients. Patients embark on a healthcare journey that includes doctor office visits, diagnostics, and maybe even genetic testing — all resulting in valuable health data that are siloed. This places an undue burden on the patient, their caregiver, and the healthcare system to painstakingly piece it all together. The situation is even worse for those who have visited multiple care facilities, which is common for oncology and rare disease patients. They likely have access to separate patient portals that are operated by at least 16 different EHR systems including Epic, Cerner, Allscripts, Athenahealth, Practice Fusion, and McKesson as well as various versions of those systems. The complexity is staggering and getting worse day-by-day.
Interoperability, Patient Access and Data Sharing
The data fragmentation in healthcare is a barrier to better patient health and effective treatments. What cannot be measured, cannot be managed. Siloed health data is not only bad for the patient, it is also a huge loss for society. One result is the inability to identify the right patients for recruitment to clinical research studies. Without important health data, accelerated drug discovery is not possible.
The need for simplicity is not reserved for just the patients who become trial participants. It also applies to the physicians and care teams, as well as the researchers who benefit from access to longitudinal patient health data. Physicians and care teams can provide faster, better diagnosis and treatment with more complete health information. Without it, manual data curation is often costly, time-consuming, and can delay onboarding. This can negatively affect health outcomes, especially in the case of advanced disease progression.
The informed consent process is just one area in clinical trials that can be further simplified.
Consent & Re-Consent
The participant informed consent process for clinical trial enrollment has traditionally been difficult and involves multiple steps. With the advent of electronic consent (eConsent), traditional paper-based consent forms have been replaced which is a step in the right direction but can also leave much to be desired when engaging with potential study participants.
Once an interested study participant consents and enrolls in a study, their data is often confined to that specific study. If there is an opportunity to engage them for a future study, the participant would have to go through the same time-consuming process again, and hence the likelihood of recapturing that participant data is low. There is a need for a streamlined process that enables the study participant to seamlessly consent to enroll in a clinical study, share their data, and join a future study when it is available.
The consent process involves much more than simply using an e-signature or signing a form. It includes a conversation with a research staff member either in person or through online telehealth. The eConsent method is most effective when it is easy to understand and use for both the participant and the research staff. Oftentimes they use multiple software suites during the study enrollment process causing frustration and wasted time.
Once the participant has consented to join a clinical trial, keeping them enrolled in the trial becomes the next challenging objective.
By not effectively engaging study participants, clinical studies and trials often experience high dropout rates. Retention is a very tough and critical hurdle to clinical trial success.
“Approximately 30% of patients drop out of clinical trials, resulting in heavy financial costs. On average, it costs $6,533 to recruit one patient to a clinical study, and the cost of replacing patients is even higher. The average cost to recruit a new patient if one is lost due to non-compliance is $19,533” (2020).
“When a patient is lost from a clinical study, it means a great deal of wasted time and resources. Studies have shown that 80% of trials are delayed by at least a month, causing potential losses of approximately $600,000 (and potentially as high as $8 million) per day. Trials can be set back by months, or even years, depending on the extent of the issues faced and how long it takes to get back on track” (2020).
Making the consent as well as the data collection processes simple for the study participant increases their likelihood of staying in the current study as well as their interest to participate in future studies. It also helps alleviate caregiver burden.
Caregiver burden is a common condition suffered by caregivers when taking care of their loved ones. Such burden is exacerbated depending on the condition suffered by the patient and the care needed, as is the case for oncology and rare-disease patients who require specific long-term care. Here are some interesting statistics on caregiver burden from The Family Caregiver Alliance website (2016).
While researchers have long known that caregiving can have harmful mental health effects for caregivers, research also shows that caregiving can have serious physical health consequences. Further supporting this is the fact that 17% of caregivers feel their health in general has gotten worse as a result of their caregiving responsibilities. [AARP Public Policy Institute. (2008). Valuing the Invaluable: The Economic Value of Family Caregiving.]
Toll of caregiving on caregiver health worsens over time. Caregivers reporting fair or poor health increases from 14% within the first year to 20% after 5 years or more of providing care. Those caring for a spouse are most likely to report fair or poor health (27% versus 15% for all other relationships). [National Alliance for Caregiving and AARP. (2015). Caregiving in the U.S.]
Research shows an estimated 17–35% of family caregivers view their health as fair to poor. Higher hour caregivers are even more likely to rate their health as fair or poor (22%) and less likely to report excellent or very good health (40%). [AARP Public Policy Institute. (2011). Valuing the Invaluable: The Economic Value of Family Caregiving.]
It can be concluded from the statistics above that the more hours spent giving care, the more likely the caregiver would rate their own health as poor.
One of the common burdens of caregivers is manual data collection, which can require considerable time, travel, and resources and can result in a very thick binder filled with charts and large raw files stored in CDs. Below is a real example of such a binder that includes medical records from oncology centers across the U.S., compiled by a determined caregiver taking care of her husband who suffered from a rare sarcoma (Price, 2020).
When electronically transferring and storing patient health data, data security and privacy become critical.
Patient Health Data Security & Privacy
Patient health information (PHI) is vulnerable to security breaches due to the vulnerability of legacy systems’ design, oftentimes utilizing a single encryption key for their entire patient database.
In 2021, 45 million individuals were affected by healthcare cyber attacks, up from 34 million in 2020. That number has tripled in just three years, growing from 14 million in 2018, according to the report, which analyzes breach data reported to the U.S. Department of Health and Human Services (HHS) by healthcare organizations (Landi, 2022).
The total number of individuals affected increased 32% over 2020, meaning that more records are exposed per breach each year. The total number of breaches only rose 2.4% from 663 in 2020 to 679 in 2021 but still hit historic highs (Landi, 2022).
Patient data privacy is protected by HIPAA and other laws such as the California Consumer Privacy Act (CCPA) of 2018. The Health Insurance Portability and Accountability Act (HIPAA) of 1996 sets the national standards to protect sensitive patient health information from being disclosed without the patient’s consent or knowledge.
How the Participant Digital Front Door™ Addresses the Patient Challenges in Clinical Trials
Interoperability, Patient Access and Data Sharing
The Participant Digital Front Door™ puts the participant at the center to connect to any health data source — EHRs, DNA/genomic labs, and remote monitoring devices- by engaging and empowering them to control, own, and share their health information with family/caregivers and physician/care teams. The disparate health data, residing in different EHR systems and versions exists in a variety of formats and categorizations.
The Digital Front Door automatically locates where the health data resides and accesses it, while standardizing, normalizing, and visualizing disparate health data through an intuitive longitudinal timeline. The data is displayed in a way that is unified and understandable for the patient. It unlocks true health data interoperability — foundational, structural, and semantic interoperability — seen as the Holy Grail of Digital Health. There is no need to login to individual portals or devices twice — a convenient auto-sync feature enables dynamic data connectivity.
The COVID-19 pandemic has made remote health data sharing not only convenient but critical. Health data sharing has the potential to accelerate research by providing researchers access to high quality and high fidelity data. The impact the Participant Digital Front Door has on research and drug development will be discussed later. Automated health data sharing accelerates clinical research.
A quick overview of the emergence of virtual and hybrid clinical trials is now necessary to better explain the Participant Digital Front Door’s function.
Emergence of Virtual and Hybrid Trials
In the traditional clinical trial model patients would have to have their own transportation to the research site which can present a challenge for some low-income demographics. In certain disease populations or age populations there is also the need for caregivers to coordinate transportation or coordinate care for their loved-ones presenting further challenges to study participation.
The COVID-19 pandemic forced the clinical research industry to embrace the use of new technology and telehealth tools, which is a step in the right direction for reaching more patients to become study participants without having to leave home.
“The shift of clinical-trial activities closer to patients has been enabled by a constellation of evolving technologies and services. Tools such as electronic consent, telehealthcare, remote patient monitoring, and electronic clinical-outcome assessments (eCOAs) allow investigators to maintain links to trial participants without in-person visits. Mobile and home healthcare, as well as alternative-care locations, enable more procedures to occur away from research sites (Agrawal et al., 2020 Exhibit 1).
Those diverse options allow for a broad spectrum of decentralized and hybrid clinical-trial designs. In the most complete articulation, a trial can be fully virtual, with enrollment and assessments taking place in a patient’s home, enabled by end-to-end digital tools and the self-administration of medicines. That fully virtual model is gradually migrating from smaller early-phase and postapproval studies toward larger pivotal trials. Nonetheless, in the near term, sponsors, investigators, and research-service providers expect fully virtual trials to remain limited to a narrow set of use cases, such as a well-characterized drug with few adverse events in a mild indication, with endpoints suited to remote measurement (Agrawal et al., 2020).
While most clinical trials are not likely to be entirely virtual, they will use one or more decentralization elements based on suitability for their end points, patient populations, and treatments” (Agrawal et al., 2020).
The advent of the smartphone has opened up more opportunities for engaging with low-income and/or minority populations who may not have access to traditional healthcare. Allowing the ubiquitous smartphone technology to serve as a bridge to study participation has huge advantages to clinical trial recruitment and diversity. The Participant Digital Front Door™ enables patients to become study participants with the click of a button.
eConsent & Re-Consent
The patient consent and re-consent processes are streamlined through the Participant Digital Front Door™. Patients are able to electronically consent to an agreement to join a clinical study as well as to collect their medical records and/or other important health data required for the study. The front door empowers the patient to have full visibility into the study they are participating in and engages them by putting their health data at the center. eConsent and re-consent help overcome the drop-off rates and other hurdles to effectively running clinical studies.
eConsent also benefits the researchers in streamlining the recording of consenting documents and e-signatures into an automated process that is fully auditable. Updated versions are available to study participants should re-consent be necessary during the course of a study.
The Participant Digital Front Door™ engages study participants by allowing them to seamlessly connect to and view their EHR + DNA/genomic tests + remote monitoring device data all in one longitudinal 360° view, as well as by empowering participants to share it with their physician/care team and caregiver/family. This provides continuous connectivity to key data as well as the participants’ circle of care.
Engagement is critical to participant retention for current studies as well as for future studies. The Participant Digital Front Door can also become a means of recruitment for future studies by a sponsor or institution as well as long-term follow up.
Having a dedicated concierge team adds the needed technology support for patients utilizing the Digital Front Door. Providers and health systems regularly update their EHR systems or migrate to new versions or systems. New laboratory tests and remote monitoring devices are introduced to the market daily.
The Participant Digital Front Door™ alleviates the caregiver burden by addressing one of their main traditional challenges: health data interoperability and access. I described earlier my experience helping my father get the right care at the right time. Caregivers can help patients enroll in clinical studies and connect their data through the Digital Front Door.
Patient Health Data Security & Privacy
The data breaches described earlier highlight how important patient health data security and privacy is, and the additional role of the Participant Digital Front Door as a safeguard protecting sensitive Patient Health Information (PHI) by making sure that key data identifying the patient is not kept in the same location as their respective health data. The Digital Front Door must be more secure than the health data sources with which it connects. Many legacy EHR systems have only one encryption key for their patient population database, leading to severe vulnerability. The Digital Front Door in essence is a heavily guarded vault with its master-key belonging to the patient and only those who are granted access through its automated data sharing function. The Participant Digital Front Door™ unsilos and combines comprehensive health data for the participant and also for researchers.
How the Participant Digital Front Door ™ Addresses the Researcher Challenges in Clinical Trials
Top 5 Value Drivers for the Researcher:
1. Speed/ Automating Data Curation
The Participant Digital Front Door™ automates data collection for data scientists and researchers. The manual collection of medical history data alone typically takes a minimum of 4–6 weeks, not even including the capital-intensive processes of having bioinformaticians and data scientists painstakingly stitch together the disparate data sources into a common form. Due to the time and capital intensive nature of manual data curation, it is not a sustainable model when scaling to a large number of study participants.
The Participant Digital Front Door™ standardizes, normalizes, and visualizes health information, without requiring any additional resources or staff. Researchers gain access to this retrospective data first and as a study continues, the data connection persists allowing prospective data collection as well. The ability to access high quality and high fidelity health data in real-time results in true Real World Data (RWD) that accelerates and improves clinical studies and the resulting RWE.
2. Study Design Flexibility and Control
Real-time RWD powered by the Participant Digital Front Door™ leads to design flexibility and control, beyond the traditional site-based clinical study setting. Sponsors and administrators will be able to conduct many aspects of the study remotely, and not require participants to have to travel long distances to the study site. As mentioned earlier, the logistical and resource burden is immediately lifted for participants and their caregivers, leading to improved study retention and health equity. The travel restrictions from the pandemic have accelerated the decentralization of different study phases, from recruitment to post-market surveillance. The Participant Digital Front Door™ provides flexibility and control for researchers that was previously not imaginable.
3. High Quality, High Fidelity Real World Data
Real World Data (RWD) has taken many different forms. Researchers have traditionally relied on aggregated, tokenized data sets that are often inaccurate and not patient-consented, for their Real World Evidence studies. They have also utilized expensive Electronic Data Capture (EDC) systems that are not interoperable with the aforementioned EHR systems in clinical trials. This all leads to manual data entry, possible transcription errors, work replication, and very resource intensive activities that may also not be very effective. Such manual work also requires specialized trained staff to monitor and help curate the data and even travel to and from research sites, further adding to the costs of research and development of new therapies. In many cases, patients are disintermediated from the data collection workflow altogether, and often are unaware how, when, and to whom their health data is being sold and utilized. On their own, tokenized datasets have very little value.
RWD has also been synonymous with claims data which is often at least 30 days old and does not always accurately reflect the patient’s condition for which they are being treated. On its own, claims data has very little value.
Yet others collect questionnaires and surveys from a large pool of respondents, who are often compensated for their time, and name this Real World Data. While questionnaires and surveys glean useful information, on their own, they cannot be referred to as Real World Data.
Electronic Health Record data provides the best source of patient health data as it is chain-of-custody data and is recorded by the physician or care team. Combined with the other traditional RWD sources listed earlier, EHR data that is captured from a patient’s visit, including physician notes, image scans, and laboratory tests, is extremely valuable.
However, the lack of interoperability, accessibility, and data sharing needed to translate EHR data into useful health information is not possible without a Participant Digital Front Door™.
4. Regulatory Compliance
Key Health Data Mandates — HIPAA, 21st Century Cures Act, CMS-ONC Interoperability and Patient Access Final Rule & 21 CFR Part 11
The Participant Digital Front Door™ complies with local, state and federal data sharing and privacy mandates listed above.
Companies that currently work with electronic patient data are required to abide by the Health Insurance Portability and Accountability Act of 1996 (HIPAA) , a federal law that requires the creation of national standards to protect sensitive patient health information from being disclosed without the patient’s consent or knowledge.
These companies must also provide patients access to their health data and are discouraged from data blocking, according to the 21st Century Cures Act.
The ONC Cures Act Final Rule implements interoperability requirements outlined in the Cures Act. Patients need more power in their health care, and access to information is key to making that happen. Putting the patient first in health technology enables the healthcare system to deliver:
- Transparency into the cost and outcomes of their care
- Competitive options in getting medical care
- Modern smartphone apps to provide them convenient access to their records
- An app economy that provides patients, physicians, hospitals, payers, and employers with innovation and choice
21 CFR Part 11 outlines regulatory guidelines for electronic documentation and signature, and its compliance is required for Pharma companies completing drug submissions.
5. Health Equity and Trial Diversity
The Participant Digital Front Door™ levels the playing field through data access, resulting in better health equity and improved trial diversity. The proliferation of virtual clinical studies helps to overcome traditional logistical challenges faced by the underserved communities, including minorities as mentioned earlier. However, data access is only one critical piece of the puzzle.
There are various reasons for the lack of diversity in clinical trials. According to the FDA, “barriers to participation among racial and ethnic groups may include mistrust of the clinical research system due to historical abuses, aspects of the trial design such as inadequate recruitment and retention efforts, frequency of study visits, time and resource constraints for participants, transportation and participation conflicting with caregiver or family responsibilities. In addition, language and cultural differences, health literacy, religion, limited access within the health care system and a lack of awareness and knowledge about what a clinical trial is and what it means to participate may impact clinical trial participation among racial and ethnic minority populations” (Armstrong, 2022).
“We just don’t have the studies” became a familiar refrain for Kirsten Bibbins-Domingo, M.D., Ph.D., chair of the Committee on Improving Representation of Women and Underrepresented Minorities in Clinical Trials and Research (Armstrong, 2022).
A new report from the committee shines a light on the “critical shortcoming” in clinical research conducted in the U.S.: a lack of representation in clinical trials. While diversity has become an increasingly important priority in clinical research, racial and ethnic minority populations continue to be left out. Progress has, however, been made when it comes to including a better balance of women to men in research. Other groups left out include members of the LGBTQIA+ community, older adults, pregnant and lactating individuals and people with disabilities (Armstrong, 2022).
FDA Commissioner Robert M. Califf, M.D. stated,
“The U.S. population has become increasingly diverse, and ensuring meaningful representation of racial and ethnic minorities in clinical trials for regulated medical products is fundamental to public health. Going forward, achieving greater diversity will be a key focus throughout the FDA to facilitate the development of better treatments and better ways to fight diseases that often disproportionately impact diverse communities. This guidance also further demonstrates how we support the Administration’s Cancer Moonshot goal of addressing inequities in cancer care, helping to ensure that every community in America has access to cutting-edge cancer diagnostics, therapeutics and clinical trials (2022, April 13).”
The FDA will likely soon mandate conditions to ensure trial diversity. It has recommended that sponsors of medical products develop and submit a Race and Ethnicity Diversity Plan to the agency early in clinical development, as outlined in its draft guidance “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Subgroups in Clinical Trials.” The Office of Minority Health and Health Equity created the “Diversity in Clinical Trials Initiative,” which includes an ongoing public education and outreach campaign to help address some of the barriers preventing diverse groups from participating in clinical trials (2022, April 13).
I can now share with you the most exciting news. The Participant Digital Front Door™ has already been created. Yes, you read it correctly. The SEQSTER Operating System for Patient Registries and Clinical Studies™ powers the Participant Digital Front Door™.
The Participant Digital Front Door™ solves a multitude of data challenges for patients enrolling in and researchers conducting clinical trials. For patients, the digital front door unlocks interoperability, patient access, and data sharing, simplifies the informed consent process, boosts participant engagement, alleviates caregiver burden, all while ensuring patient health data security and privacy.
For researchers conducting clinical trials, the Participant Digital Front Door™ drives speed through real-time health data collection (retrospective & prospective), study design flexibility and control, high quality and high fidelity Real World Data, regulatory compliance, and health equity & trial diversity which will be mandated by the FDA very soon.
For my father and those like him on a patient journey, the Participant Digital Front Door™ places them at the center of the trial and their health data to advance science through their contribution to research.
Works Cited & References
About ONC’s Cures Act Final Rule. The Office of the National Coordinator for Health Information Technology. (2020). https://www.healthit.gov/curesrule/overview/about-oncs-cures-act-final-rule
Agrawal, G., Xue, J., Moss, R., Raschke, R., & Wurzer, S. (2021, June 10). No place like home? Stepping up the decentralization of clinical trials. McKinsey & Company. https://www.mckinsey.com/industries/life-sciences/our-insights/no-place-like-home-stepping-up-the-decentralization-of-clinical-trials
Armstrong, A. (2022, May 18). ‘We just don’t have the studies’: New report details critical diversity shortcomings in US Clinical Research. Fierce Biotech. https://www.fiercebiotech.com/biotech/we-just-dont-have-studies-new-report-details-critical-diversity-shortcomings-us-clinical
CFR — Code of Federal Regulations Title 21. U.S. Food & Drug Administration. (1997, March 20). https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=11
HIPAA Privacy Rule. 45 CFR Part 160 and Subparts A and E of Part 164. US Department of Health & Human Services. (1999). https://www.hhs.gov/hipaa/for-professionals/privacy/index.html
H.R.34–21st Century Cures Act. (2015). 114th U.S. Congress. https://www.congress.gov/bill/114th-congress/house-bill/34
Landi, H. (2022, February 1). Healthcare data breaches hit all-time high in 2021, impacting 45M people. Fierce Biotech. https://www.fiercehealthcare.com/health-tech/healthcare-data-breaches-hit-all-time-high-2021-impacting-45m-people
Price, S. (2020, February 26). There’s now an easier way for your different doctors to see and share your medical records [Health News Report]. CBS8News San Diego. https://www.cbs8.com/article/news/health/theres-now-an-easier-way-for-different-doctors-to-share-your-medical-records-with-each-other/509-55ccf3ba-d0dc-4ac2-a971-c7c461a43a21
Medicare and Medicaid Programs; Patient Protection and Affordable Care Act; Interoperability and Patient Access for Medicare Advantage Organization and Medicaid Managed Care Plans, State Medicaid Agencies, CHIP Agencies and CHIP Managed Care Entities, Issuers of Qualified Health Plans on the Federally-Facilitated Exchanges, and Health Care Providers. Centers for Medicare & Medicaid Services. (2020, May 1). https://www.federalregister.gov/documents/2020/05/01/2020-05050/medicare-and-medicaid-programs-patient-protection-and-affordable-care-act-interoperability-and
(2016). Caregiver Statistics: Health, Technology, and Caregiving Resources. The Family Caregiver Alliance. https://www.caregiver.org/resource/caregiver-statistics-health-technology-and-caregiving-resources/
(2020, October 1). The True Cost of Patient Drop-Outs in Clinical Trials. Mdgroup. https://mdgroup.com/blog/the-true-cost-of-patient-drop-outs-in-clinical-trials/
(2022, April 13). FDA Takes Important Steps to Increase Racial and Ethnic Diversity in Clinical Trials. FDA News Release. https://www.fda.gov/news-events/press-announcements/fda-takes-important-steps-increase-racial-and-ethnic-diversity-clinical-trials