Contributed Commentary by Vincent Keunen, Andaman7 

May 10, 2024 | The life science industry is changing. There are growing calls for real-world evidence (RWE) and feedback from patients (PROs). Rare diseases are growing in importance, and there is increasing recognition of equity as an ethical and drug-efficacy necessity. 

Combined, these factors mean more-and-more data will be collected directly from patients. This patient-mediated research, which empowers people by giving them access to and control over their own health data, is set to disrupt how clinical research is conducted. 

Companies willing to invest in empowering patients now will reap the rewards of increased data quality and quantity and improved treatment outcomes. 

New Trends in Clinical Research 

One of the most significant recent trends in clinical research has been regulators calling for real-world evidence (RWE) in addition to clinical evidence. The FDA has identified electronic health records (EHRs), medical claims, product or disease registries, and digital health technologies as some of the potential sources for this data. 

Regulators have also been requesting more feedback from patients—patient reported outcomes (PROs)—and setting out guidance on how remote trial participation (decentralized clinical trials) can reduce participant burden and facilitate research on rare diseases.  

At the same time, equity in clinical research is becoming an ethical and efficacy necessity. Data must be generated by diverse populations if we are to understand how effective drugs or treatments are in the real-world and identify potential safety or efficacy outcomes, which occur more frequently in certain populations.  

Against this regulatory and ethical backdrop, patients themselves are becoming more aware of their right to their own data and willing to actively participate in treatment. They are becoming care consumers. While this can increase opportunities to make patients partners in research, it also means potential participants are becoming more discerning. They have more choices, expect responsive tools, and will shop around for what helps them most effectively manage their health. 

Additionally, privacy regulations have changed the data landscape forever. Regulations like GDPR in Europe give patients more rights to access and modify their own data, forbid its use, or move data to another company. 

Combined, these trends make the case for patient-mediated research. 

The Case for Patient-Mediated Research 

Patient-mediated research improves data quality and insights into the real-world impact of treatments and devices. It helps tackle privacy concerns and allows the life science industry to more effectively collect evidence for new treatments. 

Hospital EHRs are currently a significant source of RWD, however, at least half may contain an error. There is also a lack of EHRs which allow all participants, including patients themselves, to contribute. In contrast, personal health records (PHRs) collect data from multiple sources while still giving patients complete control. This increases engagement and allows patients to correct errors, improving data quality. With the right level of traceability, all stakeholders can also assign the right level of trust on different pieces of data, depending on who generated this data (patient, care provider, consumer, or medical grade device…). 

In a classic clinical trial, data might be collected by a healthcare professional over three consultations with the patient. This limits the scope of available information. In contrast, allowing patients to enter data at multiple points through a PHR, gives researchers a better view of, for example, the evolution of a disease and effects of treatment. Extended further, this can enable longitudinal studies and the feeding of continuous data to registries. 

In the rare disease space, efficient clinical research tools which utilise data already being shared by patients, helps meet the historic economic challenges of research. The ability to tap into thriving communities of empowered patients speeds up recruitment of traditionally hard-to-reach rare disease groups. 

Patient-centred approaches also help to tackle privacy concerns. For example, allowing patients to store data on their own smartphone rather than in the cloud means they have full control over how and when they share it for research. This empowers patients and encourages greater engagement. 

Embracing Disruption 

I believe patient-mediated research could be as important as disruptive innovations like generative AI, the smartphone, and the internet.  

There are still barriers to change. Patient access to data is limited by a lack of awareness and resistance to change from some stakeholders. When data is accessible, it is very often in unusable formats. However, there are solutions which will enable a paradigm shift. 

For patients, we can improve literacy by explaining complex documents in simple terms and we are currently working on a capability to summarize the full health record of a patient, using a hybrid AI and structured information approach.  

Patient-mediated research platforms are providing a novel way to collect evidence, not just for new treatments, but also for pharmacovigilance, very long-term studies, registry building and updating. We are also working to overcome the challenges of data access and usability. 

Forward-thinking organisations which adapt to this emerging patient-mediated research environment will strengthen their research data, increase their ethical value, and contribute to improving global health outcomes. It is time to embrace the disruption. 

Vincent Keunen is the founder and CEO of Andaman7.  Previously, he developed numerous medical IT systems (a secure data exchange platform used by more than 90% of Belgian hospitals and family doctors, and a prevention EHR, managing today close to 1 million patients).  Vincent was also the CIO of success story Lampiris and was awarded the CIO of the year prize for his work there.  When Vincent was diagnosed with leukaemia in 2007, and his 10-year-old son was diagnosed with bone cancer three months later, he decided to use his software engineering skills to provide all patients with a tool to manage their health information on their smartphone. That platform can now be used to improve medical research by collecting RWE (Real World Evidence) and PRO (Patient Reported Outcomes) while facilitating telemedicine, remote patient monitoring, and continuity of care. He can be contacted at vincent.keunen@andaman7.com. Find out more about Vincent Keunen’s personal story at http://bit.ly/a7vkblogen.