Rare diseases, diversity, data and other big issues facing patient recruitment and retention - an interview with the founder and CEO of Andaman7

Previously, Vincent developed numerous medical IT systems, that included MediBridge, meXi and the medical prevention dossier of Idewe, managing today close to one million patients.  Vincent was also the chief information officer (CIO) of success story Lampiris and was awarded the CIO of the year prize for his work there. Keen to provide more information, he answered a number of questions on his thoughts on the current situation with patient recruitment and retention and what more can be done to overcome existing hurdles. 

What are the key challenges faced by patient recruitment and patient retention service providers?​

If you take a helicopter view of drug development, it’s clear that medical research must focus more and more on rarer and rarer diseases - because most diseases with a high incidence are now correctly treated with existing drugs.  And some diseases (e.g. cancer) are actually a large number of different diseases requiring different treatments, so they can also be considered “rare diseases” individually. A second problem is diversity.  It’s now clear that diseases and drugs affect the various ethnicities of the human race in different manners. Finally, some diseases are endemic to certain geographical areas, making them somehow “rarer” also.  This is compounded by the fact that some of these geographical areas lack the medical / research infrastructure.

How can we boost the efficiency of patient identification for clinical research?​

We need to address the problem at various levels:

-          patients must be aware of research and the benefits for all of us, so we need to educate them, but also make it easier for them to contribute - possibly with mobile tools they will use on a daily basis;

-          when the research may be beneficial for a specific patient, because they are concerned by the disease, we can give them additional motivation by offering them tools like advanced PHRs like Andaman7 so that they can better control             and manage their health data - which will immediately benefit the research;

-          for serious diseases without efficient treatments, CRaaCO (Clinical Research as a Care Option) can become crucial for patients; mobile tools that can bridge the gap between patients - care providers - and research become essential;

-          finally, for regions that lack the medical / research infrastructure, lightweight mobile tools - that don’t require heavy cloud infrastructure - also become critical.

The volume, depth, and quality of data available to researchers embarking on a trial or real-world evidence study is often limited. Often, they rely on hospital electronic health records (EHRs), which, in around 30% of cases, contain errors. Additionally, information in hospital EHRs is also 70% composed of documents, not readily useful for computer processes. This can hamper participant identification efforts and data quality. Solutions include tools such as app-based, patient-held personal health records (PHR) that encompass information from multiple sources, including hospital electronic health records (EHRs), laboratory findings, family doctor records, data from connected devices and patients’ input - via surveys and ePROs. They put patients in a position of power, making them more willing to consent to their information being shared to contribute to medical research. Importantly, this cross fertilization of information enables the building of rich, accurate, high-quality datasets that provide researchers with all the information they need to make informed selection decisions.

What constitutes effective recruitment and retention strategies – what’s needed – what’s involved – how can that impact the patient?​

Giving people access to their own health data, and ultimate power over how to share it, could be extremely powerful in the patient recruitment space. This kind of empowerment makes people more willing to contribute to medical research. It also creates a long lasting relationship between the patients and the providers of these tools, giving pharma the capability to run long term studies (longitudinal aspect) or multiple studies with the same patients, without breaking the privacy and anonymity.

We can also see breakthroughs coming from EU funded projects like Andaman7.  The PHR that patients use to collect all their health data locally on their smartphone can be, if patients consent, searched for matching inclusion / exclusion criteria.  Since the criteria are downloaded to consenting patients’ phones, no data is shared by patients, augmenting the privacy / anonymity.  Only when criteria for a matching study are met, are the patients notified and proposed to contribute to the specific study. Of course, an additional consent is requested before they are enrolled in the study.

This opens the door to a new revolutionary approach that could be named: “large scale patient recruiting without them sharing data”...

As technologies integrate, improved usability is the operative characteristic. It will not only improve productivity, but also increase engagement to the benefit of retention. Central to this approach is drawing from patients’ experiences and perspectives to develop next-generation tools.

At a development level, PHRs can be used by pharma companies and manufacturers which need to run clinical and device trials via electronic questionnaires. There are a number of advantages to this patient-centric approach. Consent information and the trial itself can be adapted and provided in different formats to meet diverse learning styles and levels of health literacy.  Data quality is improved as patients take the study more seriously and manage the quality of their responses more accurately because they don’t use ‘a simple clinical trial app’ which is of limited benefit to them, but they use their full PHR. Dropout rates and canceled trials are consequently reduced as patients feel more engaged through their control of what is shared. The logical next step here is strong support for Patient Engagement Platforms (PEPs).

What blockers do you see still impacting recruitment and retention?​

Many companies are focusing a lot of energy on collecting as much data as possible. This is creating a  temptation to “force” users to share data or to collect data without obtaining explicit consent which destroys trust in the recruitment and retention process.

Additionally, focusing on the needs of the sponsor to collect data and forgetting the interests of patients will also reduce the retention.  Patients must feel empowered and have a real (short term) interest: giving them a powerful PHR to manage their health data before, during and after the study can do a lot for trust, retention and the genuine interest of patients to aggregate their health data in both a qualitative and quantitative manner.

Obtaining quality data for research is an ongoing challenge, especially from more difficult-to-reach sources such as over-the-counter delivery, patient-generated data and real-world drug usage. In fact, real world data is challenging on several fronts: both generating the quality and quantity of the data and securing the inclusion of patients while respecting their right to privacy.

In research , pharma focus on a very narrow area of interest and can have a blinkered view, looking for tools, solutions, and methodologies that serve the needs of that niche. Patients are global citizens and equally have a narrow focus: interest in the health of themselves and their families. These two views can be at odds with each other. To have global patient appeal and utility, the pharma industry must deploy solutions and broaden processes to truly serve the health interests of all citizens: they also need to avoid tunnel vision and think about solutions that serve those needs today, tomorrow, and the next day: not those that serve the needs of a single clinical study at a single time.

Stakeholder Management for a seamless solution​

The key to successful relationships and partnerships is clear agreements that accurately delineate responsibilities and areas of operations for all stakeholders. Also, paramount are efficient tools for communication and project management which can anticipate aspects such as costs, regulatory considerations, reporting, tools, and licenses, as well as a clearly defined budget for unplanned items. That said, it is also important to leave room for innovation, improvements, and discoveries during the journey.

Innovating for the Future: What do you see as the paradigm shift we can expect for R&D innovation, access and personalized medicine moving forward?​

Industry should embrace the innovation potential of patient-mediated research. People are collecting their own data on their smartphones and have the power to share it with institutions, researchers, and clinicians. This information can guide innovation by helping us to understand patient priorities, how diseases affect different people over time, ways to improve care, processes, speed of interaction, and all the complex interactions that influence health in the real world. Rich data and structured data that is FAIR (Findable, Accessible, Interoperable and Reusable) is becoming the reality and machine learning and AI can drill into this to deliver real insight.

Patient-mediated research will be a key trend as we increasingly recognize the need to place people with lived experience as central players in the healthcare ecosystem. Going through the patient is also significantly better aligned with new regulations: these push forward the control of their own data by patients/citizens and add more constraints on those using the data of patients…  We have already seen large CROs recruit patients and caregivers in more active roles. We are also seeing a rise in ‘patient entrepreneurs’ developing tools and technologies, carrying out research, consulting and speaking at conferences. That is how Andaman7 started. I was a patient and software engineer caring for my sick son. I built the solution I wanted to see, and I believe future innovations will be driven by others like me.