Early Access to Medicines Scheme: Supporting Patients and Life Sciences with Advanced Technology

The journey for medicines seeking full regulatory approval is a long one – a timeline I would argue is most keenly felt by the very patients waiting at the end of the pipeline, often without many, if any, viable alternatives. For many of these patients, daily quality of life is severely impacted by the intense challenges brought about by their disease.

In the case of chronic, debilitating, and even life-threatening diseases, ensuring patients can access novel and emerging treatments earlier in the approvals process in an otherwise sparse landscape can make an incredible difference to their all-round wellbeing. Herein lies the foundation of pre-launch programmes such as the Early Access to Medicines Scheme (EAMS), supporting not only those patients with direct access to potentially life changing medicines through the scheme, but also the broader patient population through accelerated and more in-depth safety and efficacy evidence generation to support approvals.

Patients and pharmaceutical organisations alike may in turn see boosts to wider awareness across public, clinical, and research communities, driving the development of a powerful position that strengthens future uptake of those treatments that deliver true benefit to patients upon receiving regulatory approval for commercial launch.

Our work with leading life science partners, particularly in rare conditions and those that have seen little therapeutic innovation for decades, has highlighted the growing importance of programmes such as EAMS in ensuring that the treatments that really work reach the patients that need them most. Time and time again, we have seen just how critical powerful real-world evidence – beyond traditional follow-up periods and endpoints – are to driving regulatory approvals.

The impact of these therapies day-to-day at a quality of life, physiological, and real-time level, are crucial to monitoring and demonstrating the prospective benefits and risks to patients – building upon early clinical trial risk-benefit balances, to create a fuller picture of a treatment’s potential.

How Can Technology Help?

Advancements in technology over recent years has seen significant strides in improving the quality of data collected for clinical trials, with significant potential across a wide range of domains. Already, our work in real-time monitoring for patients with Sickle Cell Disease (SCD) has unlocked the ability to change patient lives through daily disease management and predictive insights, at their fingertips. This is supported by FDA cleared and CE marked wearable devices that allow for automated and remote monitoring of key biometrics linked to health and wellbeing – sleep quality, activity, heart rate, blood oxygen saturation, and ECGs.

This approach has seen incredible improvements to patient engagement and retention, the resulting live data creating new sources of day-to-day information and more granular endpoints, without additional burden to patients. This real-world evidence base holds the key to demonstrating therapeutic efficacy and safety at a level not typically seen, and is merged alongside base medical records, depth of diagnosis episodic data, as well as patient-reported outcomes such as EQ-5D and daily pain scores – derived from our dedicated patient portal.

Supporting the Fight Against COVID

Our work in SCD has seen a great deal of focus around vaccination in what is an extremely high-risk patient group, severe forms of infection and mortality much more prevalent in those with the disease. However, public uncertainty around vaccine safety is an issue that has by no means bypassed even the most high risk of patients, many with specific conditions voicing concerns around their safety and impact in the context of diseases such as SCD.

As such, our recent work has tracked this impact in real-time, applying both our patient-reported outcomes portal and remote monitoring wearable technology to track the real-time impacts of COVID and influenza vaccinations. Following daily patient journeys in terms of pain, psychological wellbeing, symptoms, as well as automated sleep and activity biometrics, our advanced analytics platform has captured real-world evidence for each vaccination’s effect, and any variation from patient baselines.

Indeed, this work has highlighted that any effects seen remain well in line with those of traditional vaccinations, such as flu, and may even have a lesser impact across the second and booster COVID vaccinations. Our goal has been to help patients make informed decisions around their health, supporting the improvement of patient confidence and vaccine uptake in light of the ongoing risks of the pandemic through real-world evidence.

As new therapeutics and vaccinations continue to emerge, it is this very ethos of demonstrable safety and efficacy that we work to apply to strategic partnerships centred around EAMS programmes.

“Data science and AI solutions offer unique opportunities for monitoring patient outcomes, mapping this against specific treatments for better understanding of their real-world impact. I truly believe that Eleven’s ability to monitor live patient biometrics, alongside the integration of a real-time patient-reported outcomes portal, will be vital in supporting a deeper depth of data collection and analytic insight as part of EAMS.”

Consultant Psychologist, Confidential NHS Trust

Mapping Patient Quality of Life Against Key Events and Metrics

Patient-reported outcomes are a crucial part of understanding patient experiences and quality of life, as they embark on their treatment journeys. Through Eleven’s platform, this data can be easily input by patients as frequently as needed, far beyond what is currently recorded in limited clinic visits. With more frequent datapoints therefore comes the ability to map these against specific measurements and events — building a full picture of the impact of sleep upon quality of life.

As such, this provides the ability to apply live, remote monitoring methods to track and identify correlations between both automated validated, patient-reported, mood-based measures, such as the EQ-5D, and treatments or clinical events.

With the creation of a daily timeline of patient-reported outcomes both across the cohort and at a patient-level, this allows us to pinpoint the impact of key events upon the associated quality of life experienced by patients.

Our research and deep platform insights, married with discussions with patients and clinicians, have additionally flagged sleep as an important player in monitoring wellbeing and predicting the potential onset of complications, with Eleven’s work to date highlighting key correlations between sleep and pain – supported by past publications that highlight the prevalence of sleep disorders in patients with SCD and the impact on both quality of life and physiological markers of health.

Our data and analytics therefore work to create a longitudinal dataset of live biometrics and quality of life measures against metrics such as sleep, with a timeline along which additional factors such as disease events, healthcare utilisation, and the impact of treatment pathways can be mapped. Through this, we build greater understanding of the disease in order to identify ways in which to improve outcomes for patients.

And while much of our initial work has focused upon patients with SCD, it is an approach with translatability across the continuum of chronic and rare diseases – expansion into oncology and nephrology already in the early stages of development. The biomarkers, records, and patient-reported outcomes we use are ones that have significant utility in a range of conditions, with quality of life monitoring an essential part of following the patient journey and treatment pathway, universally.  

The Patient Voice

We believe that the patient voice is a vital part of advancing patient care, better outcomes, and new medicines. To that end, a core part of Eleven focuses upon driving ongoing engagement with our patient community through a dedicated team who ensure all feedback is heard – be this about Eleven itself, our technology, their health care and experiences, or the issues that affect them. As we continue to learn more about patient experiences each day, this helps us to build a picture of what does and does not work well in terms of their care and treatments, in addition to helping to drive improvements to their interactions with Eleven.

As we apply our ecosystem to supporting life science partners in bringing their innovations one step closer to regulatory approval, a critical part of the success of this form of patient-driven, real-time data work is continued patient engagement and satisfaction. Already, our patient community has expressed significant benefits to their day-to-day life and overall wellbeing – helping to cement ongoing retention throughout the development of deep data insights and real-world evidence to drive improvements in their care and available therapeutic options. As a member of our powerful patient community says:

“I feel like I am contributing to the understanding of Sickle Cell Disease. Using my data to measure and potentially improve outcomes is really valuable for me and patients like me. Being able to potentially predict a crisis or VOC will really change things for Sickle Disease patients and I am excited to be part of this programme.”