Increasingly, industry, patient organizations, academics, regulators and payers are interested in the generation and collection of data from patients’ experiences across all aspects of their lives (commonly referred to as Patient Experience Data or PED). The development of PED models is equally important and is emerging as a critical aspect in research and healthcare to accurately define evidence-based unmet patient needs, health outcomes and impact. This presents a new opportunity to design a truly patient-centered model, one that is streamlined and more efficient and serves all stakeholders.
This PEOF 2021 session provided a forum for exploring the vision and possible approaches for more effective patient engagement in the design, generation, analysis and decision-making of Patient Experience Data. This session also encouraged discussion among attendees and sought to identify some of the common ground, opportunities and challenges when talking about Patient Experience Data.
Chaired by PFMD co-founder Tony Hoos, the panel represented the multi-stakeholder interest in the patient experience, with speakers including Geraldine Reilly, Global Patient Engagement Lead with Gilead Sciences, Kevin Moody of EATG, Nathalie Bere from the European Medicines Agency and Richard Harding of the Cecily Saunders Institute. Mr Hoos noted that working together and engaging in discourse such as this is the best way to serve patients and wider society.
Ms Reilly began by outlining the Gilead experience of informing PED and PRO strategy via an expert patient panel. With the PE team at Gilead formed only a year ago, there has nonetheless been significant progress and she also described their ambitious plans going forward. With a strong legacy of creating transformative therapies, most notably within Hepatitis C and HIV, Gilead has partnered with the patient community on many levels. But now the focus is on making PE systematic so that co-creation becomes routine. Having joined PFMD 15 months ago, Reilly noted that the “open collaborative forum” has allowed the Gilead team to consider how they interact with patients and how they can apply the quality criteria to their work with patients. She called PFMD “an incredible movement”.
The particular project she outlined was a real-world evidence study that was actively recruiting 12 months ago and being expanded. This study had six different PROs being captured and was running in 14 countries around the world with around 1,000 patients recruited. In relation to the PROs, they wanted to know what was the purpose of capturing them, and the objectives they wished to achieve. “Did they capture the real feelings, needs and wants of patients i.e. Patient Experience Data?”
Patient experience data is data that captures what is important to patients based on their experience of disease and or treatment, whereas a PRO is a tool to support and capture this PED. If the tool is not co-created or designed to capture those elements of the patient experience that is important to the patient then it simply is not capturing PED,” she explained.
After much back and forth with the study organisers, the Gilead PE team finally got approval to pilot the patient engagement initiative alongside the Phase IV real-world study. The co-creation of plain language summaries for the participants and a review of the PROs were carried out. The group of patients have been “magnificent and really insightful – brutally honest at times”, but Gilead is grateful for this, Reilly noted. The study organisers are in amazement at what has been achieved, and the study has had an immediate impact, she added. The learnings will now be applied across many other studies, and at a much earlier stage; Gilead will now begin including patients at the very beginning of the process.
International consultant with EATG Kevin Moody then described how EATG has been working for years on PROMs, and is seeking to find out how they are being used in medicines development. PROMs are increasingly being used in practice, research and medicines discovery, and can be used as endpoints in clinical trials if they embody the characteristics of typical endpoints and are valid and reliable. HIV-specific PROMs are important because they address issues relevant to people living with HIV. PROMs in R&D have a number of challenges, namely around timing and homogeneity of the patient population in HIV. “PROMs are by their nature subjective, and difficult to attribute to treatment arms in short timeframes.”
The patient survey received 96 responses in total, and what they learned was that patients wanted better treatment and that they had a range of side effects from their current treatment. They were also asked to outline their wish list for future treatments, such as whether they would like to hide their treatment. Even the term PROMs was not well known to patients, showing the need for literacy in this area.
“There is no magic bullet for identifying new products for patients. Every patient experiences their treatment differently.” Moody added that they also learned it is important to incentivise patient involvement in PROMs; “patients do not want to fill out survey after survey so we need to find a better way to get them involved.” Involvement in PROMs is best when patients feel they are the driving force or true co-creators, he added.
Moody concluded by saying that PROMs are an important and increasingly popular way to gather patient experience data, and can add value to improving the quality of life of people living with chronic diseases by preventing, identifying and treating co-morbidities. PROMs for HIV R&D are currently useful for secondary and exploratory endpoints and more research is needed to make PROMs more useful as primary endpoints under R&D conditions. “Investments in patient organisations should be made to increase their capacity to co-produce and implement PROMs and to lead the advocacy for engagement in these Processes.”
Nathalie Bere of the EMA described how PE has become an integral element of activities at the Agency. Dialogue with patients was initiated back in 1996 after the agency’s establishment but it has progressed over time so that today there is systematic patient input in the medicines regulatory lifecycle. Following development with patients, they have put in place opportunities for them to contribute to the regulatory discussions, from early dialogue to evaluation to safety monitoring. “Their added value is well acknowledged and it means we have a comprehensive data set.” There are mutual benefits across the board, she added. Patients contribute directly to the scientific discussions, while their role is different it is no less important than the scientific experts.
Bere outlined some learnings for optimal engagement, such as creating a diverse group of stakeholders to consult and the need to test and implement various engagement methodologies, as well as providing the appropriate support and training. Transparency is also critical – sharing relevant information and outcomes with participants is key.
While PE is established at the EMA, there is “still a lot of room to optimise”, Bere noted. A new framework of interaction will develop new methodologies and “enhance the generation and use of patient experience data and provide guidance”.
Harding began his talk by asking “what does quality care look like?” While there are numerous definitions, the common denominator is that it is focused on the patient and is about more than just the disease. As well as the physical problems, the spiritual, emotional and social problems of illness should be reflected in the care a patient receives.
Patient Reported Outcome Measures (PROMs) ensure that care is good quality, accessible, and reflect what matters to the patient, he noted. “It’s not just about doing things, it’s about doing things that make a difference for people living with any number of conditions,” Harding stated.
His work focuses on getting the measure right – “there is the risk of measuring the wrong things and not measuring what matters or you measure the right things but do it so badly that you can’t show that change,” he explained. A complex and evolving science, how we understand measures and outcomes is changing the whole time. Any tool must be valid and reliable, responsive to changing circumstances, and importantly accessible and interpretable. When devising PROMs to improve care, “patient involvement is absolutely key”, he said, echoing earlier speakers. “Patients should have the same status as anyone else on the team.”
The panel then teased out the difference between Patient Experience Data (PED) and Patient Reported Outcomes (PROs) or Outcome Measures (PROMs). It was noted that these nuances may not be fully understood by the patient community, who may also not know what the acronyms themselves mean. “We need to be thinking about what these really mean to patients as there is a lot of confusion out there.” Mr Hoos agreed saying a common language and approach is needed.
Co-creation session
A hallmark of the PEOF sessions is their interactivity and this one was no different as the audience provided their thoughts on three distinct questions:
- Why do we need to understand the patient experience?
Audience feedback suggested that patient needs are to be clearly understood in order to design, collect and analyze meaningful data. This process is not stakeholder exclusive – there must be a common understanding across stakeholders, and also within organizations. Evidence based unmet needs, designed WITH patients, should be addressed and the goal should be to empower patients.
- What can stakeholders learn from patient experiences and how can they act on them?
Stakeholders throughout the healthcare system have important roles to play in patient experiences, and there are significant opportunities for multi-stakeholder involvement and collaboration. Audience members also suggested efforts should be made to increase awareness and education for patients to better understand opportunities for involvement and how, when, why, their experience is used and the resulting impact.
- What should be considered to ensure that data effectively captures the patient experience?
The audience agreed that data should be gathered from multiple sources involving collaboration and validation from multiple stakeholders, while data collection methods are made publicly available and endorsed by multiple stakeholders, starting with the patients.
The design, consolidation and communication of the analysis should meet the needs of several stakeholders, they added.
