Why Pharma, Providers and Patients Need ePROs

Commentary

Hanna Phelan

It will come as no surprise that cancer patients experience highly challenging symptoms related to their illness and treatment - both physically and emotionally. While clinical guidelines in Europe and the US have since the 1980’s recommended the close monitoring of patient reported outcomes and symptoms throughout their treatment pathway, to date, this has not been the reality beyond self reported recall during brick and mortar visits.

But, all of that seems to be changing with electronic patient reported outcomes (ePROs) which have recently piqued significant interest for providers and pharma alike. In the past decade, we have seen continuous interest and investment in patient outcomes, patient engagement, remote monitoring and patient centricity as highlighted by the HealthXL Platform data below showing all digital health investments in these areas from 2010-2018. This data also highlighted cancer as the leading therapeutic area of investment in terms of patient outcome measures.

How do we know? Well, while we cannot vouch for each ambitious start-up in the space, the good intentions of each partnership and accept that we don’t have a window into the future. One thing is for sure, patient empowerment and the democratization of healthcare sits at the very core of digital health reality.

This week, we take a look at the key implications, partnerships and evidence associated with integrating ePROs - patients’ own accounting of their symptoms, quality of life and functional status - into clinical trials, healthcare provision, and patients own day-to-day.

What’s in It For Pharma?

For pharma, ePROs are particularly interesting faced with the ever maturing realities of value based care (VBC) reimbursement models. As the healthcare community dabble with VBC, Pharma have discovered their edge with ePROs - remote monitoring of patients and collection of real world data (RWD) that allows them to assess their products in action. This is a very appealing proposition for Pharma as demonstrated by the increasing depth and frequency of Pharma and ePRO company collaborations - uMotif and Merck, BMS and Noona, Voluntis and Roche; the list goes on.

While future gazing AI has generated substantial excitement and some ill-informed hype in Pharma circles in terms of speeding up drug discovery, drug re-purposing and other R&D areas, ePROs offer a much more accessible way to optimize drug trials and a way in which Pharma can move from their traditional perch as a drug manufacturer to a place of greater consumer understanding. This is something that could potentially be useful for Pharma and Life Sciences across the continuum, from R&D all the way through to commercialization.

A number of additional regulatory factors have influenced the Pharma community to engage with digitally enabled patient reported outcomes and digital bio-markers including the 21st Century Cures Act in the US and the European Medicines Agency in the EU who are now pushing for a greater presence of patient generated data. When it comes to Pharma's actual activity with ePROs, there are partnerships aplenty. Novartis recently announced a partnership with UK based uMotif, IQVIA and The Christie NHS Foundation Trust in tackling care pathway inconsistencies and gaining a more meaningful view of the patient pathways for breast cancer patients. uMotif have gained significant interest from Pharma groups since their inception in 2012 boasting partnerships with 8 of the top 20 pharma, including AstraZeneca, Merck. And uMotif are not alone in their Pharma engagement traction. French HQ’d Voluntis have gained the confidence of Roche, AZ and Sanofi for their products such as Theraxium Oncology that is aimed at empowering patients to self-manage their symptoms while receiving cancer treatments, in coordination with their care teams and guidance through their digital therapeutic.

Notable ePRO and Pharma Partnerships

As we saw last week, clinical trials are starting to undergo a digital re-imagination - ePROs are central to the optimizing the ease of data collection and ability to gain a holistic insight. For Pharma, late stage oncology trials traditionally focus on getting patients to fill out multiple forms and require regular travel to and from trial sites for check-ins often resulting in drop-offs. There was already a mechanism in place to collect these data points. ePROs are simply offering an improvement in the quality of trial data and the efficiency with which it is collected.

Clinical Considerations for ePROs and Cancer Care

When it comes to healthcare providers, the opportunity to remotely monitor symptoms in order to streamline the clinical decision making process flags a chance for deeper empathy and more holistic and timely understanding of the patient experience. The key consideration here is if the integration of ePROs is a worthwhile investment in terms of proven clinical efficacy and improved outcomes and how can this fit into a clinical workflow seamlessly.

Today, Cancer is the second leading cause of death after cardiovascular disease. However, similar to the situation we find ourselves in with unprecedented populations living well beyond 65 years of age, cancer is also becoming a much longer term condition than ever before requiring prolonged self-management and HCP touch-points. Paired with the multidisciplinary care teams involved in cancer care and the ever increasing burden of clinician workloads (physician burnout now regarded as being at epidemic levels of prevalence as captured by this Medscape survey), an opportunity to streamline communication between care teams and patients seems like it would be a match made in heaven.

It has been suggested by a plethora of studies that an increase in contact and support with a care team and more effective communication dynamics can improve patient outcomes. Patient Engagement and ePRO companies are now offering a way in which this continuum can be optimized, closing the gap between brick and mortar visits and putting the patient at the center for their care. Relying solely on clinic visits to capture symptoms can often lead to poorly controlled or reported symptoms and experiences.

“Although smartphones have achieved near ubiquity, patients and physicians remain largely unfamiliar with their use in healthcare. As a result I expect some reluctance to adopt a tool of this sort.”Dr Andrew Norden

It has been highlighted in multiple studies (a handful highlighted below), that integrating ePROs into routine cancer care has been shown to improve patient-clinician communication, patient satisfaction, symptom management and control, and quality of life. Recent data have found reductions in ER visits, longer tolerability of chemotherapy, and improved survival.

Undeniably, we are far from ePROs being standard of care and having resistance free buy-in from the clinician community. But, there are a number of promising partnerships between hospitals and ePRO solution providers. The Kaiku Platform, for example, is being used by over 30 European hospitals and clinics to better monitor their patients including Attendo Occupational Health, Docrates Cancer Center, Felicitas Mehiläinen, Hirslanden Clinic Zurich and Turku University Hospital for both cancer and non-cancer focused interventions. Noona, specifically focused on breast cancer, has also partnered with Stanford Medicine and the Helsinki University Cancer Center in its native Finland with whom they completed an RCT involving 885 breast cancer patients. In cancer care and beyond, PROs have gradually revealed their potential:

A multi-centre RCT of Parkinson's Patients published in Nature shows that uMotif effectively engages patients to track and manage their condition over time - resulting in significant clinical impact

A RCT study in MSK (Basch, 2016) with 766 oncology patients found that Patients receiving intervention had improved HRQL in comparison to usual care (34% b 18%), were less frequently admitted to the ER (34% v 41%) and remained on chemotherapy longer.

In a follow up from the above RCT published in JAMA, it was found that 516 out of 766 patients had died, but there was a 5 month increase in overall survival in the PRO group

In another RCT with 358 patients published in 2017 similar improvements in symptom severity and duration were achieved using a telephone-based symptom-reporting system. The IC resulted in a 43% reduction in symptom severity

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The Bottom Line - Implications for Cancer Patients

For patients, it’s a chance to be heard. Digital tools that empower the patient voice are a key element of true patient centricity. Unfortunately, this phase has been used liberally to-date in line with marketing trends. So, evidence of true implementation and evidence of outcomes will be crucial if ePROs are to be genuinely adopted in healthcare provision and clinical trials of the future. While there are logistical considerations around how Providers and Pharma may interpret and leverage these patient reported data points, the appropriate levels of customization is also crucial when it comes to the data insights the patients have access to on their side of the applications - how do we make this actionable and insightful for a patient without overburdening or inciting fear? Each condition, each patient cohort and each individual context will require some level of customization in order to optimize the utility of these solutions for patients.

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“The right amount of customization is key. On one hand, you will want to provide flexibility to the doctor as to what data might he see , but over customization may lead to missing out key info. Patients, when given too much raw data may have difficulty in interpretation and may get misinformed.”Dr Faiq Shaikh

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While ePROs demonstrate a chance for patients to access increased support and continuity from their care teams personalized to their own needs, there may also be understandable patient concerns around usability of these solutions. ePROs should lay minimum burden and provide maximum insight and efficacy for the patient.

There is also a strong need for validation in each patient cohort. With many variables at play (therapy, condition, demographic) - something that works for colorectal cancer patients may not be as efficacious or applicable for those going through breast cancer treatment.

The efficacy of automated symptom monitoring is also dependent on provision of timely feedback from care teams. Collecting more data without an appropriately enabled feedback loop to action the insights is pointless.

The Future of Cancer Care and ePROs - Nice-to-Have or Must-Have?

So, in theory, the integration of ePROs and digital bio-markers in medical care and clinical trials seems to make sense. But when push comes to shove, does the patient really know best? How much value does yet another data point make in health outcomes and actionable insights? Do patients feel like big brother is watching? What is the future value of true patient engagement with the help of ePROs?

In the hands of the patient as a part of the standard clinical pathway for cancer treatments, this is an chance to catch non-responsiveness and relapses before it is too late. For health systems and payers, it is a chance to reduce costs of ineffective treatments. For Pharma R&D, the systematic integration of patient reported data rings in an era of the creation of better targeted and better tolerated treatments. But at its core, as fluffy as it may sound, it is really about patient empowerment.

With all that said, how do we actually make it succeed? According to uMotif CEO Bruce Hellman “don’t make it about the ePRO” - if you make it about the ePRO it’s just investing more resources and energy into the R&D dimensions already sapping huge volumes of capital. Make it about the patient - how can we provide the most value to patients and their care teams, what is the most valuable and actionable data that we can feed back to the patient to help them self manage?

Is this the future of routine cancer care? We think so. Given the robust clinical validation, the continued acceptance of value based payment models, ePROs and remote symptom monitoring systems may be expected to become commonplace. Hopefully, we will get to a point in the coming years that clinical trial leads, healthcare practitioners, patient and caregivers marvel at the blind spots we once accepted as the ordinary.

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Data sourced from HealthXL, which includes various data resources, including crunchbase.