The rise of patient 2.0

‘Patient engagement’, as a concept, has evolved considerably since I joined the industry over two decades ago. As the end users of our products, patients have always been at the heart of this industry, but back then they didn’t hold the same ‘key stakeholder’ status they do today. Innovation guided by patient insights was the exception, rather than the rule.

These days we are more likely to develop products and services for patients, with patients – recognising that when it comes to understanding the physical and emotional impact of a disease, or therapeutic needs, it is the patients, their carers and often their families who are truly the experts.

Today we recognise the benefit of patient involvement throughout the product life cycle, from the early stages of drug development to patent expiry and beyond. Indeed, there is almost no part of a medicine’s life cycle that doesn’t benefit from the incorporation of patients’ perspectives.

As an industry, we are more willing to partner with patients to amplify their voices on disease-related issues and co-create innovative ‘beyond the pill’ solutions to help them manage their disease effectively.

Game changer

There are many reasons why change has occurred, but if I had to choose one in particular, I’d say it was the evolution of digital communications.

When I first started out, most people wanting to find out about a disease and its treatments had limited options: healthcare professionals, patient organisations (where any existed) or even the local library – on reflection, the potential for shame and embarrassment with this option doesn’t bear thinking about now.

Digital communication, however, means more than just access to disease information; it also means that people living with a disease can find others like them, create networks, share experiences, provide each other with emotional support and organise themselves into campaigning groups to advocate for better recognition, public support or access to treatment.

That said, free-flowing information through digital channels also comes with some obvious downsides. Plenty of information out there about diseases and treatments is misleading, or just plain wrong, not to mention dangerous. A prime example of this being the misinformation surrounding COVID-19.

This sets a challenge to everyone in our industry, professional health bodies, public health organisations and the media – to do as much as we can to direct people to evidence-based disease information.

Peak practice

Empowered patients have arrived and many national health systems are shifting to a model where patients are encouraged to have a greater say in treatment decision-making.

The phrase, ‘no decision about me without me,’ which emerged in the UK over ten years ago, is still relevant today. While shared decision-making isn’t for everybody, those patients who are able to take an active role in their disease journey can experience better health outcomes.

A good example of this – and one which Janssen is proud to have been part of – was the development of a consistent information resource to support patients and patient group input through Health Technology Assessment (HTA) decision-making.

We worked with the UK reimbursement body, NICE, to create a plain language summary of the medicine being assessed. This approach can be used as best practice by other organisations when submitting evidence dossiers to HTA agencies across the globe.

Group therapy

In recent years, the influence of patient organisations has increased significantly. Along with patients, they have become regular partners and collaborators with Regulatory and Heath Technology bodies, where their knowledge, expertise and experience are routinely sought to guide decision-making.

Digital innovation has also played a role in increasing the influence of patient organisations. This creates opportunities for patients and patient organisations to reach a wider audience, creating global communities. This is particularly important for people with rare diseases where those affected are unlikely to personally know anyone else with the condition.

While pharma has long recognised the importance of patient organisations, regularly collaborating with them on a range of issues, this has not always been the case. Historically, we’ve tended towards more transactional rather than continuous relationships with these organisations, only engaging with them in specific instances when their help is required.

Likewise, financial support for these organisations has previously been provided only as long as a company is actively involved in the respective disease area. Recognising that this isn’t the way to build trusting relationships, a forward-looking company should seek continuous engagement with patient organisations, overseen by a single point of contact.

Patients please

Patient involvement in clinical trials is pivotal. Regulatory agencies, such as The European Medicines Agency (EMA) and the Food and Drug Administration (FDA), actively encourage pharmaceutical research companies to involve patients in trial design. They place particular emphasis on taking a structured and systematic approach to this engagement, so that replicable expertise will build up over time.

Taking the time to seek perspectives from patients or carers for the creation of a more patient-friendly clinical trial can be beneficial in the long term. It can speed up recruitment, improve patient retention, deliver results faster, and in some cases even save money by cutting out unnecessary steps or overly burdensome procedures.

Patients can also provide valuable insights in terms of identifying the most appropriate trial endpoints; for example, when investigating chronic diseases certain symptoms impact quality of life far more than others (and they may not be the obvious ones). The ability to demonstrate improvement in areas that matter most to patients is not just a moral and ethical imperative, but also pragmatic commercial practice.

The feedback gathered from patients via Janssen’s Patient Voice in Clinical Trial programme has made a significant impact on both trial design and implementation. Since its launch in 2018, operational changes have been made to 100% of the trials within the programme and protocol modifications made to over half of the trials. Importantly, these insights are made available to other research teams so the learnings can be applied to other studies as appropriate.

Question time

Engagement with Patient 2.0 is now enabling the patient community, healthcare providers, government, policymakers and pharma to actively collaborate in tailoring healthcare solutions to create exactly what the patient needs, so where else should we focus our efforts?

Just as digital communication has brought transparency of healthcare and treatment information, we should also be mindful that transparency has a role in our engagement work. Can we clearly and meaningfully demonstrate how patient engagement has influenced or changed decisions in policy or development?

Furthermore, are we monitoring the extent and effectiveness of patient engagement, and are we able to show the value it brings? Also, are we continuously reviewing how such new methods can supplement and enhance our work with patients?

Common, underlying principles of patient engagement are increasingly being shared across public and private organisations, and further development of standards and systems on how industry should partner with patients will help to bring more consistency to objectives and approaches.

To me, creating an environment which enables mutually beneficial partnerships between these communities is something we should all actively strive to achieve. I feel this is the most efficient way to deliver medicines and services that patients and health systems truly value.

Emily Bone is Patient Engagement Excellence Lead, Europe, Middle East and Africa at Janssen.
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