Patient centricity: From buzzword to business impact

Patient centricity is not new. Since the early 2000s, pharmaceutical companies and regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have increasingly recognised the importance of patient perspectives and the need to engage patients in all stages of drug development.

While patient engagement has become more common, the approaches to doing so and the levels of intensity vary wildly.

Often, they are focused primarily on the later stages of the drug development process, impacting uptake, adherence, and usage.

An example referred to at this year’s UK Galien Forum, in a session called ‘Improving global health outcomes through clinical innovation’, was the case of a new drug for people with multiple myeloma, a type of bone cancer. In the real-world setting, many patients stopped taking it, in part due to life-impacting insomnia which is a commonly occurring side effect.

Dr Hilary Marston, the FDA’s Chief Medical Officer, considered whether this could have been addressed during the development process if consideration had been given to the impact that a lack of sleep would have on patients and whether that could affect their willingness to continue taking the drug. The challenges of adherence with this drug suggests that, for many, living with disease progression is better than not being able to sleep and the associated impact on quality of life.

Dr Marston argued that incorporating patients’ voices at every stage in the development process can help ensure that a drug will work for those who really need it in the way that matters most to them.

Patient-centred R&D: Is it time for a bolder vision?

Knowing that patient-centric research and development (R&D), especially using patient perspectives to inform strategic decision-making, is critical to taking health and care to the next level is just the start. How to optimise its potential, integrate it into systems and processes, and ensure that patients are stakeholders in the journey from bench to bedside is an evolving discipline and is being actively discussed.

In the Galien Forum discussion, Dr Marston discussed how many recommendations in current clinical guidelines are lacking strong evidence to support optimal care and that more investment is needed in research to improve health outcomes.

“Fundamentally, I think that the evidence generation system works well for Phases 1-3 but the issue comes in the post-approval space, where evidence generation is not filling the gaps most critical for clinical research.” Dr Hilary Marston

Regardless of the route, the goal is the same: to help ensure drugs that reach the market make a meaningful difference to patients’ lives, get approved sooner, and have more impact. And to do so whilst also reducing late-stage failures, improving trial recruitment and retention, and increasing approval success rates.

Learning from the regulatory landscape

The journey that regulators have been on has reflected the increasing belief that integrating the patient voice can support better drug development. Consequently, they have increasingly integrated the patient perspective in a more systematic way.

From the earliest days of the EMA, almost three decades ago, patients recognised the value of working with regulators. And there has been an ongoing dialogue and commitment to learning from interactions with patients ever since.

At the FDA, patient involvement started with meetings held with patients to inform the FDA’s benefit-risk assessments. What came out of those early meetings was the realisation that endpoints didn’t necessarily match what mattered to patients. It was also made clear that clinical trial designs were overly burdensome and kept patients from being able to actively participate or be retained in studies.

Structures and processes have been integrated to ensure the patient perspective is built into regulatory decision-making. The FDA launched the Patient-Focused Drug Development (PFDD) Initiative in 2012, whilst in 2024, the EMA built on the Patients’ and Consumers’ Working Party (PCWP), established in the early 2000s, with a more formalised framework for the interaction between the EMA and patients.

Regulators want the patients’ perspectives to now be incorporated earlier and throughout the process rather than at the very end. According to Dr Marston, the FDA’s approach to patient engagement involves:

-          Listening to patient and caregiver perspectives that can inform regulatory work

-          Partnering and collaborating to support patient engagement

-          Enhancing bidirectional communication through online tools, education, and events

-          Leading patient engagement activities across medical product centres

Embracing the patient voice: ‘No decision for me without me’

As panellist Dr Sarah Gheuens, Chief Medical Officer, Agios, speaking on the panel on ‘Putting patients at the heart of drug development’, chaired by Dr Mel Walker, eloquently demonstrated, the industry is rapidly evolving its mindset.

The pyramid with doctors at the top and patients and patient advocacy groups at the base where patients are engaged in drug development only at the end, after all core decisions are already taken, leaving space only for input on implementation issues, is being challenged. Patients are starting to be seen as equal stakeholders and companies are looking to them to contribute their opinions and perspectives earlier and earlier, based on their day-to-day experience of living with a condition. And for good reason.

“..[at Agios] we have a slogan ‘fuelled by connections’ … we are trying to engage with all the different stakeholders that are important to drug development … the most important stakeholder is the patient.” Dr Sarah Gheuens

Patients and their carers can describe the impact of the condition, what’s important to them, and what they need from new treatments and care. They can explain what would motivate them to participate in clinical trials and stay on them. They can share what they know and what they need to know.

Importantly, patients want and expect to get involved.

The call to action ‘no decision for me without me’ rings true. First coined by the HIV community in the 90s, when patients stood up and demanded to be listened to, patients today want to be heard, to be ‘thought leaders’, and to contribute to research and evidence generation.

As companies embrace this viewpoint, the business impact of greater involvement of patients and consideration of the patient perspective is becoming clear.

Patient-centred trials are a case in point. The rate of positive results reported in such trials is 20% higher than in traditional trials (87% v 68% (Ref: Economist piece 2020)). There is also data showing that when companies have tracked products developed with patient involvement from bench to bedside, against those without such heavy involvement, the medicines with consistent patient input performed better than those without.

Where can patients help with R&D?

The perceived wisdom (Ref: Zvonerareva et al. Research involvement and Engagement: a systematic scoping review 2022) indicates that the earlier engagement takes place the more impact on drug development can be expected. A framework has been described with three stages where the patient and carer voice and perspective can be of value:

R&D program set up

-          Participate in defining research agenda and priorities

-          Inform choice of specific directions of inquiry to pursue

-          Identify barriers

-          Provide insight into preferred outcomes and drug characteristics in terms of balance between risks and benefits

-          Contribute to designing drug development programmes including ensuring meaningful representation of diverse patient populations

Designing studies

-          Deciding what outcomes to consider

-          Recommending amendments in trial design and organisation to reduce barriers to trial participation and improve enrolment and retention

-          Contribute to the development of tools for trial oversight

-          Evaluate reporting process

-          Flag ethical issues such as access

Fine-tuning stage of drug development

-          Inform patient recruitment strategies

-          Evaluate and contribute to the development of information and education materials, informed consent forms and tools e.g., decision aids

-          Considering the point highlighted by Dr Marston at the UK Galien Forum that more efforts are required in the post-approval stages, it could be argued that a fourth stage exists:

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Development and post-approval stages

-          Inform real-world evidence strategies

-          Evaluate and contribute to the development of support initiatives and materials to further improve patient carers

As well as the stages at which patient engagement takes place, the way in which patients are involved is also critical to their impact. Different levels of intensity of engagement have been described:

Consultation (low intensity of engagement) in which patients are asked for their views but the company is under no obligation to act on these views. In many cases, there is limited feedback and patients may not learn whether or how their insights have been integrated.

Involvement (mid-level intensity of engagement) in which companies create a dialogue and interaction with patients where there is a degree of mutual influence and accountability. The relationships can be short, mid, or long-term but typically there is feedback and active participation by patients and carers.

Partnership (high-level intensity of engagement) in which there is an active, ongoing, and equal collaboration between the company, patients, and often carers. These are almost always long-term relationships where feedback and building trust is an important part of the engagement.

Patient leadership (highest level of intensity of patient engagement) where patients drive drug development and decide who else and when to invite them into the process. This is rare but not unknown, especially in the rare disease space where patients become frustrated with the lack of progress.

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Recent research, and our experience with clients, is that most patient engagement initiatives initiated across the pharmaceutical industry still take the form of consultation and involvement.

Increasingly, however, at FleishmanHillard we are working with companies who have a bolder vision, one in which long-term partnerships are being developed and solutions are being co-created. These remain mostly in the mid to late stages of drug development, but this too is starting to change. We are helping companies unearth data-led insights of disease-specific patient experiences, and working together to build and establish partnerships across all stakeholders with patients front and centre, and included much earlier in the R&D cycle.

For more information on how FleishmanHillard helps companies draw insights from patient perspectives and integrate patient engagement in strategic planning and implementation contact:

Jacqui Sanders, Senior Partner and Managing Director, Health and Life Sciences

Laura Musgrave, Partner and Senior Advocacy Specialist, Health and Life Sciences at helping_health_companies_put_patients_first@fleishman.com