A session on operational challenges affecting patient data initiatives, and how these are evolving as patients gain increasing control of their health data, took place at the IQVIA Institute’s Patient Advocacy Summit (December 1, 2021). Participants represented patient organizations for chronic and rare diseases, large and small patient populations, and various stages of maturity and levels of resourcing. Key takeaways are provided below.

The COVID-19 pandemic has challenged patient organizations’ fundraising and patient support services. The pandemic also accelerated adoption of decentralized technologies and engagement and raised awareness of the role of data to clarify barriers to ideal health outcomes. Coupled with new patient-centric regulations in the United States, this has empowered patients to leverage their health information and become a more equal partner in their care. Patient organizations are building a greater role as clinical and observational research paradigms evolve.

Drivers of these opportunities include the passage of the 21st Century Cures Act. This authorized U.S. Department of Health and Human Services agencies to implement regulations enabling interoperability—the secure sharing of electronic health information between healthcare providers, payers, and patients—and to stop traditional data holders from “blocking” access to outside requesters.

In addition, the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) are increasingly open to using real-world data from patient registries in regulatory decision-making. This enables patient organizations to transition from being primarily funders of research to becoming active innovation drivers through patient health data initiatives.

A Transformational Shift in Patient Engagement

This transformational shift is generating opportunities for patient advocacy organizations and acceleration of development of new therapies. Historically, many patient organizations have kept a hands-off relationship with patient data, funding research at clinical sites. Organizations that are already comfortable with directly engaging patients are collecting data through case report forms and patient reported outcomes. However, the form-based technology underpinning even mature programs is not sufficient to leverage interoperability and patient access advancements, creating strategic investment decisions. Existing processes for managing patient consent and data governance become more complex when accessing raw health information from electronic health records, administrative claims, and wearable devices. This context framed panel discussions of opportunities for organizations to advance their missions and resulting challenges.

Moving Beyond Traditional Registries

With modern approaches, the registry concept can be expanded to capture a holistic, longitudinal view of the patient journey, without increasing patient or provider burden. This is achieved by replacing human data entry with patient-mediated access to automated data feeds where possible. Improvements may occur in several areas:

• Environmental and Social Determinants: Information on these can help segment disease populations. With patients now able to share their clinical data directly, the time saved can be used to capture information to inform novel endpoints or predict clinical trial matching success.
• Diagnosis/Early Risk: Modern registries make it feasible and cost effective to focus on pre-diagnosis and include at-risk patients who do not develop a disease.
• Partner Sites: Patients can automatically share structured information on every encounter, procedure, medication, and lab result from each care setting without the need to manually enter more data.
• External Providers: Patients can automatically share data from peripheral encounters with a healthcare provider or facility that is not participating in a particular research endeavor. Health data platforms can harmonize these data across locations and time.
• Specialists: As with other external providers, it can be challenging for traditional registries to capture data from specialists who see a patient only once; this is no longer a problem as patients can share information from each care team member with the same minimal effort.

Advancing from Tactical to Strategic Approaches

The panel discussed opportunities for broader mindsets and research initiatives using the increasing volumes of data that are available. Topics included:

• Returning value to the patient: Multiple organizations are seeking to replace the one-sided interactions common in traditional registries with interactions using patient-centric best practices and “give-get” principles. Examples included patient-facing dashboards and generation of pre-healthcare visit planning documents to facilitate productive conversations.
• Addressing health disparities: There is a renewed focus on identifying and addressing health disparities, coupled with potential for rapid impacts from patient-mediated data. Patient organizations are interested in exploring public/private partnerships to integrate and repurpose data.
• Establishing more sophisticated patient consent and data governance models: For most patient organizations, gaining access to patient data will require an overhaul of patient consent forms to adopt broader initial consent language and incremental follow-on. Data governance policies will need to be more sophisticated.

Panel Discussion Topics

Has the 21st Century Cures Act had any immediate effects on your operational models?

Patient centricity and data access are central to the 21st Century Cures Act and Cures 2.0. One speaker reported that Cures 2.0 was directly responsible for some data-related successes in 2021. The Crohn’s & Colitis Foundation benefited from the Cures Act, including gaining funding from the Patient-Centered Outcomes Research Institute and participating in an FDA real-world evidence project. This Foundation has a network that allows patients to vote on research priorities; topics chosen include pain, fatigue, and bowel urgency.

Has it been necessary to adapt operational models to better align with new sustainability paradigms?

Sustainability and operations are closely connected. The advent of cell and gene therapies is driving a need to follow clinical trial participants for 15 years or more. Patient organizations can leverage pre-existing, trust-based relationships with community members and modern data platforms to support company efforts to stay connected with these individuals. One registry was built with the aim of benefiting patients and industry partners based on a model that is intended to be sustained via fees from industry. Data initiatives may also offer Life Science companies access to candidates for clinical trials, starting with pilot studies to demonstrate value.

In the next decade, how will the role of patient organizations in data and research initiatives evolve?

The decade ahead will build on improvements in interoperability and access to data, seeing power move towards patients and patient organizations. A key goal for organizations will be to continue investing in transformative research and play a larger role as ‘neutral convener’ of stakeholders with shared interests to advance data use to benefit patients and science. Organizations will also need to ensure that patients understand the implications of sharing their data, driving patient empowerment.

Conclusion

Advances in modern data standards and patient-empowering regulations are shifting control of health data access from care settings to patients. This offers organizations expanded roles across education, self-care, clinical research, and care delivery—and allows research prioritization to include the patient voice. However, this potential is neither guaranteed nor automatically available even to mature programs. Strategic alignment and investment will be required in the next few years—or the opportunity may be lost to competing non-profit or for-profit organizations. Patient organizations that invest in data infrastructure, patient applications and research services may be able to improve health outcomes by advancing life changing therapies for their patient communities more sustainably than ever.