Regulators are getting serious about incorporating patient experiences and preferences into their decision-making. As definitions and expectations become clearer, momentum is building behind harmonized approaches to collecting and applying PED.

When an idea’s time has come, its uptake accelerates until it becomes so widespread that observers wonder why this new way of doing things wasn’t always the norm. When it comes to incorporating patient experience data (PED) into healthcare decision-making, the momentum feels unstoppable. 

It wasn’t always the case. For years, advocates pushed regulators, payers and healthcare providers to find ways to include PED in their work. With some exceptions, their calls often went unheard. However, recent years have seen growing interest in this field, with regulators now producing guidance that will catalyse the collection and use of patient experience and preferences in key decisions about drugs and devices. 

At the Patient Engagement Open Forum 2021, a diverse group of experts shared their takes on the state of play and what needs to be done to build on the progress reported by stakeholders. 

‘There are exciting things happening right now,’ said Eleanor Perfetto, Executive Vice President Strategic Initiatives, National Health Council (NHC). ‘We’re at a tipping point: PED is not just being talked about, it’s really starting to happen.’

Brett Hauber, Senior Director, Patient Preference Elicitation at Pfizer said the field was developing a critical mass of experience and activity: ‘It feels like things are about to start breaking loose, and that’s very exciting.’

The push for global alignment 

Nicholas Brooke, PFMD Executive Director at The Synergist, looked at key milestones in the patient engagement and PED landscape over the past 24 months. ‘It is accelerating and broadening,’ he said, pointing to increasingly explicit guidance from regulators and public health authorities. For example, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) now requires applicants to show evidence of patient involvement activities when they apply for marketing authorisation. 

Mr Brooke said global alignment would further advance the widespread use of PED by key decision-making bodies, adding that the ICH is working to harmonise regulatory approaches across the world. ‘What used to be a wish is turning into a real expectation,’ he said. 

The US Food & Drug Administration (FDA) is a key player globally and has led the way in defining and encouraging the use of PED in product development. Over 90% of submissions from companies to the FDA now include PED. 

Michelle Tarver, Deputy Director of the FDA’s Office of Strategic Partnership and Technology Innovation at the Center for Devices & Radiological Health (CDRH), offered insights on how patient input can shape decisions on medical devices. 

‘In healthcare’ we have been in paternalistic mode, historically, where clinicians drove how healthcare was delivered,’ Dr Tarver said. ‘We have seen studies showing that patients’ priorities and values can differ from what physicians might have expected. Addressing these priorities improves patients’ lives.’

The CDRH has stepped up its use of patient input since it’s 2016-2017 Strategic Priorities set the agency two key goals: promoting meaningful engagement with patients and increasing the use of patient input as evidence in the Center’s decision-making. Since then, 24 industry-sponsored PPI studies have been completed or are underway as part of the regulatory process. These have helped to inform regulatory decisions on a new obesity device and deepened understanding of the acceptable level of risk for patients and carers performing home dialysis. 

Patient input can be collected in the form of a Patient Reported Outcome Measure (PROM), Patient Preference Information (PPI), or Patient-Generated Health Data (PGHD) from wearables, registries and online discussion. ‘Information should be collected in a well-defined and structured way that can be used as scientific evidence,’ Dr Tarver said. ‘It should also be done throughout the process rather than at one time or one stage of development.’

Regulators signposting the path ahead 

Brett Hauber said the most valuable element of the CDRH guidance is that it defines patient preference studies and sets out what is required to generate valid scientific evidence. ‘I see these as guideposts which provide a great foundation from which the field can evolve,’ he said. 

Fuelled by this guidance, the industry has been gaining experience of how to elicit patient preferences and use these data in regulatory filings. Dr Hauber said there is also movement in Europe where the European Medicines Agency is currently looking at how to expand its use of patient preference information and the IMI Prefer project is bringing stakeholders together to develop guidance and a checklist for performing studies. 

Real-world examples are helping to show industry and regulators how this area of research can improve products and patient experiences. For example, a study looking at the administration of cancer medication revealed a strong preference for subcutaneous injection over intravenous treatment. This was reflected in the product label, which was a milestone event in the brief history of PED. 

Companies continue to refine how they generate PED in response to guidance from regulators and specific feedback on methodological issues raised by decision-making bodies. However, working through this exciting phase brings rewards. ‘When the pieces come together and everything clicks, it’s like magic,’ said Dr Hauber. ‘That’s because we’ve aligned the patient’s perspective with the decision at hand.’

Beyond clinical outcomes

Dr Eleanor Perfetto described a project which aims to develop Disease-Specific Patient-Centred Core Impact Sets (PC-CIS). The goal is to capture all the potential impacts a disease or treatment can have on people’s lives, their family or their care partners. ‘By engaging with patients and carers in a rigorous and structured way, we can narrow down what is important to them,’ she explained. ‘From there, we can whittle it down to the most important disease-specific impacts.’ 

Those factors include clinical outcomes but also social and financial issues, the impact on caregivers on patients’ capacity for work. The multi-stakeholder initiative will create a blueprint and toolkit that the patient community and others can use to develop a PC-CIS for a specific disease or population. 

‘A PC-CIS can be used for many things, including in clinical trials, real-world studies, product development, audit, quality measurement, value-assessment and clinical diseaseion support,’ Dr Perfetto said. ‘Our goal is to begin with patients and ensure that they drive the process throughout, ensuring that a whole range of things that affect their lives are captured.’

The event concluded with an interactive co-creation session using GroupMap to explore the most important aspects of PED, how they could be used in decision-making and barriers to their wider use in healthcare. 

While there is still much work to do, the sense of momentum ensured that attendees left with a strong sense that PED is on the cusp of going mainstream.