In this article I will discuss:
- The definition of excellence in patient advocacy in 2021
- Why data is so important
- Types of data used
- Pitfalls of advocacy without data
- Different approaches to generating, accessing and curating data
- The importance of partnering for data
- Recommendations for patient advocacy organizations
Patient advocacy is usually envisioned as people teary-eyed before Congress or shouting in front of government buildings, perhaps even chained to the gates. That activism and emotion are not unwarranted or misplaced, but are insufficient, particularly in the current age, to achieve the clinical care and policy changes that will ensure better therapies, access, and outcomes for patients.
Today excellence in patient advocacy requires access to data – epidemiological, clinical, patient experience, among other types – to inform credible, persuasive arguments to policymakers, payers, and other stakeholders. These data, along with human narrative and visuals become an undeniable force to fully describe the impact of a disease and the path towards a solution.
There are tens of thousands of 501(c)3 patient advocacy organizations in the United States alone. The vast majority are small, both from a revenue and staff perspective, community-based, with founders who were patients or parents of patients and want to give back or advance research for the condition that affected them. Few have the resources or expertise to hire epidemiologists, data scientists, or other professionals who can conduct research or set up platforms or registries for the organization. The consequence of this is an over-reliance on emotional stories, investigator-initiated research, gaps in publicly collected databases, and ultimately an imbalance in investment and support for conditions which should be based on evidence of prevalence, incidence, and impact on individuals, families, communities, and the workforce or even national security/preparedness. Additionally, lack of access to data, or failure to appropriately contextualize data to complement other advocacy tools undermines the credibility of the patient voice and risks set backs in patient advocate engagement in regulatory processes. (https://www.statnews.com/2021/07/23/patient-advocacy-organizations-lessons-from-aducanumab/)
Below I briefly describe three examples of patient advocacy organization approaches to data acquisition, curation, and use.
American Heart Association: In 2019 The American Heart Association’s Go Red for Women and Verily’s Project Baseline (part of Alphabet) announced a collaboration termed Research Goes Red that is the pinnacle of patient advocacy partnership with technology and provides a great example of the array of tools available to capture and curate the 360 degree patient experience – matching clinical trials, surveys, focus groups, and testing various mobile and digital technologies to help shape the future of disease management. Through this partnership AHA is able to access world-class scientists, physicians, and data scientists from major academic institutions like Stanford and Duke as well as the Alphabet companies with include Google. (Projectbaseline.com/gored)
Fox Insights: Another example of leveraging technology for advocacy is Fox Insights, from the Michael J. Fox Parkinson’s Foundation. The Fox Insights platform’s more than 50,000 participants shape research and programming by providing opportunities for answering surveys at 90 day intervals and participating in studies through a partnership with 23andMe or FoxTrialFinder. Deidentified data are made available to researchers. (Foxinsight.Michaeljfox.org)
PBC Foundation: Primary Biliary Cholangitis is a rare auto immune liver disease. The PBC Foundation has focused its technology resources on an app with a self-management focus, making available educational materials and community support. The richness of matching patient experience data with clinical data is why PBC Foundation deidenitified data has been referenced by the US FDA and the UK NHS for drug development and quality measurement development respectively. (Pbcfoundation.org.uk)
Each organization has taken a slightly different approach based on organizational strategy, culture, and financial resources, but they elucidate the trend that patient advocacy organizations are no longer ceding the collection, curation, and leveraging of data to others – whether CROs, health system EHRs, or government surveillance systems. Fully and more formally understanding the patient experience is the fuel for designing research that patients want to participate in, developing programs that solve problems that are relevant and optimally impactful, and advancing policy that resonates with all stakeholders.
These examples may seem unattainable to most organizations, but I would suggest that there are at least 3 ways that any patient advocacy organization can better position themselves to reap the benefits of data-driven advocacy:
- Make the most of qualitative information. Systematically coding elements of your patient stories to see the pattern can give important directional information or raise questions that expose the need for more data or research.
- Set the agenda: Patient insights into their lived experience paired with researcher expertise in scientific roundtables can inform and change the direction or prioritization of the research being conducted and add elements to the data being collected to better answer those questions.
- Use the existing data. The cost of building an app, database, or registry may be daunting, even with the proliferation of technology platforms and options, however, organizations ignore that fact that data about their patients, their diseases, exist already in thousands of silos. Enlisting a research fellow from a local university to identify the places and the potential queries on data in other registries, hospital EHRs, and payer systems may be more productive than building another silo and may answer more questions more quickly at a lower cost.
Becoming more familiar and active with data sources, generation, curation, and use is no longer optional for effective, credible patient advocacy today.
Donna R. Cryer, JD is the President and CEO of the Global Liver Institute. She is formerly a member of the HIT Policy Committee and an FDA Patient Representative. Currently Mrs. Cryer sits on the Boards of the Sibley Memorial Hospital/Johns Hopkins Medicine, the Council of Medical Specialty Societies, and the Innovation and Value Initiative. She is a graduate of Harvard and Georgetown University Law Center.