Data from and about patients is essential to advance biomedical research and development, but its collection and dissemination can be a significant investment of time and resources for patient organizations that choose to be the intermediary. Patient groups and their research collaborators must determine what data is needed, how to ethically and securely collect it, how it will be managed, what can be shared, and how these efforts will be funded and sustained long-term. During this webinar, leaders within the patient data and patient organization communities discussed modern operational challenges and opportunities for patient registries, and how to leverage the modern patient registry to generate real-world data and insights in support of patient advocacy. This webinar was a collaborative effort between FasterCures and IQVIA and is part of our continuing webinar series from The Research Acceleration and Innovation Network (TRAIN) program. 

Moderator 

Raymond Puerini 
Associate Director, FasterCures, a Center of the Milken Institute 

Speakers 

Ryan Fischer 
Chief Advocacy Officer, Parent Project Muscular Dystrophy 

Elise Hoover 
Senior Director of Research, PKD Foundation 

David Voccola 
Senior Director of Global Strategic Planning, Integrated Health Practice, IQVIA 

Alexandra Weiss 
Director of Strategic Operations, Patient Advocacy Organizations, IQVIA 

Summary 

Establishing and developing a data collection and management platform is a central activity for many patient foundations whose missions seek to advance biomedical research and development. Throughout the history of  The Research Acceleration and Innovation Network (TRAIN), FasterCures has provided resources and insights from the patient organization community to leverage innovation from those that have established effective patient data platforms to drive their research initiatives forward. As registries have grown in their capacity to collect complex longitudinal and real-world patient data, corresponding issues related to 1) the rapidly transforming role of patient organizations in healthcare and research; and 2) the operational challenges and opportunities to leverage real-world patient data and the patient voice to improve patient outcomes have emerged. To explore such issues, Raymond Puerini (Associate Director at FasterCures, a center of the Milken Institute) moderated a TRAIN webinar co-produced by FasterCures and IQVIA featuring Ryan Fischer (Chief Advocacy Officer at Parent Project Muscular Dystrophy), Elise Hoover (Senior Director of Research at the PKD Foundation), David Voccola (Senior Director of Global Strategic Planning at IQVIA) and Alexandra Weiss (Director of Strategic Operations at IQVIA) to share how their organizations continue to work toward empowering patients’ voices amidst the emergence of new data technologies and regulations. 

Led by David Voccola and Alexandra Weiss from IQVIA, the presentation outlined external factors affecting modern patient registries, with a focus on leveraging emerging best practices to ensure health information is collected, managed, and shared to wholly address the needs of patients, clinicians, and researchers operating in real-world settings. The presenters highlighted examples from patient advocacy organizations to provide considerations for establishing or re-evaluating their patient registry platforms in support of their research priorities. 

Modern Operational Challenges and Opportunities for Patient Registries 

Although patient organizations remain unified in their commitment to enhancing patients’ lives and access to potentially life-saving therapies, the potential of utilizing an ever-expanding pool of patient data to advance their research efforts has never been greater. Amidst a confluence of emerging technologies and regulations that focus on expanding patient data access and ensuring the high volume of patient data is utilized to effectively address real-world solutions for the diverse needs of patient populations, patient organizations are increasingly empowered to: 

• Fund awareness-building and policy advocacy that drives action and unlocks more money for research. 

• Drive research with data and infrastructure to accelerate science, research, and development, and engage with patient communities. 

• Deliver research services directly and expand capabilities across the continuum of research and care delivery. 

However, despite a more empowered patient community, Ryan Fischer, Chief Advocacy Officer at Parent Project Muscular Dystrophy (PPMD), noted that regulatory guidance is often written toward industry and is rarely patient-friendly. To overcome such barriers, Fischer shared that PPMD connected with trusted partners to sift through the often ‘intimidating’ language of the regulatory guidance, together crafting a different approach to the organization’s registry strategy – such as data standards, design, and utilization – and adapting how to best engage with stakeholders. The presenters agreed that ‘shoehorning’ new data types into old models will neither be sustainable nor effective and encouraged participating patient organizations to engage with their stakeholders to re-evaluate their program goals in tandem with their registry strategy. 

A New Paradigm for Providing Value 

As the patient registry experience transitions away from the simplicity of the form-based worldview, the potential for disparities in the user experience skyrockets. With this in mind, modern patient registries must perform at ‘consumer grade’ – meaning, a participant should have as simplistic and as seamless of an experience engaging with the organization’s data collection interface as any other third-party consumer interaction (i.e., Venmo, Zillow, et cetera) they have experienced in the past. Presenters shared that modern patient registries may achieve ‘consumer grade’ status through: 

• Device Integration: Enhance the integration of consumer wearables (i.e., FitBit and Apple Watch) and study device data. 

• Dynamic Patient Communication: Emulate call-center experiences to reduce lost-to-follow-up outcomes and support patient workflow problems via text reminders, email, and chat engagement. 

• Enrollment Activities: Deliver a streamlined recruitment experience via frictionless identity verification, informed consent, and self-service enrollment. 

• Multi-Modality Data Capture: Collect a rich data set from both active and passive data collection methodologies, namely by leveraging native phone capabilities to capture patient-reported outcomes.  

• Patient Retention: Support digital and gamified experiences to ease the overall data collection burden and encourage high user engagement. 

• Patient Reported Outcomes: Provide an interoperable format to include patient report data such as patient medication diaries, OTC narratives, and demographic and social data in an easy-to-use, plain-language framework. 

• Patient-Mediated Electronic Health Records and Claims: Support the direct integration of electronic medical records and claims data through patient-mediation. 

In building the next generation of modern patient registries, the IQVIA team noted that basic databases are no longer sufficient – instead, large commercial cloud networks will offer data combination analysis tools at both a scale and cost-point that basic databases cannot reasonably sustain. Such data combination analysis tools will allow modern patient registries the flexibility to handle, analyze, and share diverse quantities of data, leading to overall harmonization and enhanced value for patient organizations. In fact, exceptionally complex patient registry designs are readily doable in the present day, offering opportunities to explore the care journey process, complete patient-reported outcomes, facilitate specimen collection analysis, mediate the release(s) of clinical data, provide population-specific feedback, and ultimately capture data that can be regulatory-grade. 

Elise Hoover, Senior Director of Research at the PKD Foundation, chimed in to offer how her organization built a patient-powered registry, highlighting the importance of including outcome measures that center around patient-reported data, such as quality-of-life, pain, and COVID-19 outcomes. She reiterated the necessity of integrating electronic medical records and claims data through patient-mediation, noting that high-quality, in-depth data enables modern patient registries to proactively chart out and provide tailored recommendations aimed at improving individual patients’ overall health.  

Great Going Forward 

In building modern patient registries, the IQVIA team cautioned that it remains necessary to build tools that engage with patients where they are while continuing to advocate on their behalf. With this in mind, the presenters suggested standardizing and optimizing frameworks for bring-your-own-device experiences, as well as growing virtualization of the study experience and changes in patient data ownership. Not only does this drive patient empowerment, but also enhances inclusivity in the overall patient registry experience. The presenters opined that modern patient registries can provide the infrastructure necessary to engage with and empower patient communities that are traditionally under-served, resulting in access to patient data that is more representative of the broader patient experience and will encourage participation among under-represented patient communities. 

Acknowledging the inequities to access that some patient communities experience in accessing patient registries and patient data, the presenters outlined the following best practices for including a plain-language framework when engaging with patient communities: 

• Engage provider networks in a way that it is easy to share on behalf of their patient communities, keeping in mind barriers such as pre-paid data plans or lacking reliable access to a smartphone or internet network. 

• Identify partners that can provide flexibility within the patient registry to adjust and differentiate content based on ‘appropriate profiling’ to avoid inadvertently cutting certain patient communities out. 

• Utilizing plain language, as opposed to medical jargon, to ensure that registry content is neither biased against or misrepresentation of those individuals with low health literacy. 

Conclusion 

The IQVIA team concluded the presentation by reiterating the value of patient-mediated data that is provided by modern patient registries: it links data capturing the patient voice to data used for regulatory decision-making, bringing together much-needed validation and longitudinal value. The value of patient-mediated data not only holds the potential to change the cost curve of research but also dramatically shift the existing power dynamics of the policy and advocacy cycles in favor of patients and patient organizations. As patient organizations engage with their respective research and patient stakeholders to prioritize use cases for the data from their patient registries, it is imperative that all stakeholders contribute to building and sustaining platforms for collecting and sharing patient data to remain connected across their shared missions and goals to enhance patients’ lives and access to new, potentially life-saving therapies.