Patient Engagement in the Era of CAR-T



Rendering CGT Genuinely Patient Centric

In recent years, the much-discussed concept of “patient centricity” has been top-of-mind across all corners of the pharma industry. This is especially pertinent now that many innovative drug makers are seeking to become much more than just purveyors of pills and expand along the life science continuum, providing positive healthcare outcomes and wellness solutions in place of mere units of product.

In recent years, the much-discussed concept of “patient centricity” has been top-of-mind across all corners of the pharma industry. This is especially pertinent now that many innovative drug makers are seeking to become much more than just purveyors of pills and expand along the life science continuum, providing positive healthcare outcomes and wellness solutions in place of mere units of product.


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Perhaps nowhere, though, has real patient centricity been more in evidence than with the new generation of cell and gene-based therapies.

This is largely because with state-of-the-art, biology-based, personalised medicines — such as cell, gene and neoantigen therapies — the traditional boundaries separating the patient, the process, and the product are very much blurred. “What really distinguishes treatments such as chimeric antigen receptor T cell therapies is the patient is not just the recipient of a drug product. The patient is the product,” explains Marc Boutin, global head patient engagement at Novartis and former CEO of the US National Health Council. “Patients are truly central to each and every activity as the patient’s own cells are the starting material for all manufacturing processed and, after genetic modification, form the essence of the final material,” he emphasizes.

More than a Buzzword

Due to the inherent nature of these therapies Boutin believes that there is a particular onus on the pharma industry to pursue a holistic and consistent engagement of patients across the entire lifecycle of the medicine: whether that be in co-creating patient relevant endpoints or in co-designing the clinical trial protocols directly with the community.

Melanie Croce-Galis, director of US patient engagement at Novartis Oncology very much agrees. “There is a growing recognition, both by industry and elite regulators such as the US FDA, that patients are experts in their own illnesses, that they are increasingly well educated about treatments, and that they want a say about what is being studied and what kind of treatments are available. This holds particularly true for cell and gene therapies given that different patients may exhibit different sets of symptoms and the overall patient experience tends to be highly individualized,” she notes.

What, though, does true patient centricity look like in concrete terms in such instances? “We work together with the medical community – that is both the pharma industry and medical practitioners and clinicians – to help them better understand the patient experience from the point of view of the person being treated,” explains Natacha Bolaños, global alliances manager and regional manager for Europe at the Lymphoma Coalition, a worldwide network of patient groups.

She lists examples of some priority day-to-day concerns for real life patients that otherwise might not feature prominently on the agenda of a pharma company fixated on clinical end outcomes. These include addressing the ever-pervasive fear of relapse and symptoms like fatigue that might at first glance appear comparatively minor but can nonetheless have a heavy impact on quality of life.


Systematic Internalisation

“At root, effective patient engagement and patient advocacy is about talking and listening to the patients so that you can get their unique insights and then start to properly apply them,” observes Claire Saxton, vice president for patient experience at the Cancer Support Community, the largest professionally led non-profit network of cancer support worldwide. “These insights should go well beyond just the collection of data and are as much about internalising patient perspectives and factoring them into all design, processes and decisions,” she insists.

For this to happen, there needs to be a high degree of formal collaboration and a strong effort to ensure that each and every stage of the therapy discovery, development, and execution processes are conducted in an inclusive, participatory manner. “Patient advocacy groups are the natural locus where the interests of these patients are aggregated, analysed and translated into community positions… For that reason, it is essential that we engage with these groups at the local, national, and international levels,” insists Karin Blumer, director of global patient engagement at Novartis Oncology. “In the same vein, we also take care to interact with patient-driven foundations which invest in research; a model born in the US, but which is now rapidly proliferating around the globe,” she adds.

Moreover, Blumer notes that such forms of collaboration are becoming ever more effective over time as many patient organisations start to raise their game courtesy of increasing sophistication and maturity. “Many of the professional patient organisations nowadays are extremely well versed in the nitty gritty of the science, which means they are able to contribute real value even in technical spheres such as when reviewing our clinical trial protocols,” she confides.

“Sponsors certainly shouldn’t feel hesitant to reach out to patient organizations and fully integrate them from the very outset of the research and development process. Many of us are well versed in the science and have a great deal to contribute in terms of helping render clinical trials a much smoother more positive experience for the patients,” argues João de Bragança, Childhood Cancer International, is the largest parent-led international organization supporting children with cancer

“After all, real patient engagement is always a two-way street,” reasons Melanie Croce-Galis. “Advocacy groups in advanced western markets such as the United States tend to be highly effective in educating their members and raising awareness about the latest treatments, alongside their typical function of patient support.” “The time-honoured dictum that, if you want to go fast, then go alone; but, if you want to go far, go together, tends to hold true in this instance,” she confirms.

High-Touch Operating Models

Meanwhile, the distinctive characteristics of cell and gene therapy are helping to take the concept of patient centricity in all manner of exciting new directions. Karin Blumer, for example, notes that massive efforts are presently underway to develop a brand new, high-touch operating model that can successfully bring together sponsors, physicians and drug makers specifically to guide patients through the treatment process; one that can ultimately orchestrate monitoring, support and therapy into a positive patient experience.

“The CAR-T patient journey presents particular issues around time, travel, cost, and emotional strain that elevates the importance of strong patient centricity and enhanced collaboration. We’re talking about a patient treatment pathway that is decidedly more complex than that of patients who are prescribed a pill by their physician that they then pick up in a pharmacy and take orally. This is because regulations are set nationally or regionally, but patients have to be treated locally,” she explains.

“While we know that the journey can be a comparatively smooth experience for patients who live in densely populated areas in mature markets with nearby treatment centres, this is certainly not the case for those in rural areas or who need to travel to another country to receive the treatment, which actually tends to be the more typical scenario. These patients generally require a complex and multi-faceted support system to navigate both logistical and emotional challenges, and yet many may not even speak the local language hence the need for a new paradigm of intensive engagement and collaboration,” she continues.


Psychological Implications

Claire Saxton agrees that the logistical and process complexities place an additional psychological burden on patients that should not be underestimated, and which requires a special kind of mentoring.

“Much like bone marrow transplants, CAR-T does not involve multiple rounds of the same therapy. Each step of the process is profoundly different. Reaching step one does not mean that the patient knows what step two looks like. Especially in a vast country like the US where a lot of CAR-T happens at a distance from where the patients actually live, this presents an unprecedented level of upheaval from the patient experience perspective,” she notes.

“The idea of somebody having to move for their treatment for four to 10 weeks along with their caregiver means that lots of logistical and practical information and resources are needed. And because of the overwhelming amount of information to communicate, it can be hard for patients and their caregivers to retain it so there’s a need to really reconsider how we go about it and identify a better model,” she warns.

A New Breed of Patient Centricity

CAR-T sponsors have therefore been working hard to figure out the key pain points in specific healthcare centres or geographies and to collaborate with local ecosystems in new ways in the quest for tailored solutions. In some instances, this has meant assisting hospitals to establish dedicated stem cell coordinators tasked with helping guide patients through the stem cell transplants process. In others, it has meant mobilizing support organizations for paediatric patients such as Ronald McDonald Houses. Both efforts – in tandem with enhanced direct engagement with patients and caregivers themselves – have so far been considered instrumental in the ongoing push to identify a new breed of patient-centric solutions.

“Traditionally, the industry has always focused on the hard medical data, which is where our truth strength lies. However, key benefits of a medicine may not be visible at first glance but will nonetheless carry huge implications for the patient. For example, in paediatric oncology the difference between a child spending six months in a hospital versus the same child getting treatment in an outpatient setting can make the world of difference. The COVID lockdown experience has shown us all what it means for a child to be not able to go to school or play with their friends for six months. An outpatient treatment option means that paediatric patients can lead a more normal life, even during cancer therapy. That’s why we need to get insights from empowered patients who speak up to give us the true picture of what their daily life looks like and how disease and therapy affects it,” argues Blumer. Meanwhile the intrinsically intrusive nature of cell and gene treatments forces the industry and practitioner community to confront these issues head on.

A Paragon for its Peers

“In my opinion, the advent of cell and gene therapy is definitely helping to shift the needle and get people thinking about patient centricity in a new light… We have been contributing to this discourse in our own way by bringing in data from our global patient survey of 12,000 lymphoma patients and caregivers and covering all the different stages of the patient experience according to subtype,” affirms Lorna Warwick, CEO of the Lymphoma Coalition.

“While we understand that industry and the regulators look primarily at drugs’ safety profile and efficacy, they also need to be more aware of patient experience data pre- and post-treatment and how drugs may resolve certain issues but remain problematic in others and this is what we are bringing to the table and trying to cement as a new kind of standard,” she concludes. Her hope, like that of many others, is that the sort of first-of-a-kind patient centric initiatives currently being tested and trialled in the cell and gene space may eventually gain traction in other treatment paradigms as well.

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