ISPOR 2022: Plenary Session 2 – Real-World Patient-Centered Research: Is It Possible Across Countries?



Real World Data is being increasingly used across the globe to support medical-product regulatory decision-making, which fosters the generation and use of Patient Experience Data. This discussion, moderated by Tara Symonds, Clinical Outcomes Assessment (COA) strategic lead & chief science officer at Clinical Outcomes Solutions, explored patient-centred research.

The panellists contributed their own key learnings and insights from current initiatives into capturing patient-centric data and its use in evaluating new medicines to inform future thinking. Topics covered included: the DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative, examples of Patient Reported Outcomes Measurement (PROMs) programme and their learnings and Core Outcomes Sets (COS).

Harmonisation across borders requires a transparent approach and common understanding

For multi-stakeholder dialogue, a common, robust and meaningful understanding of Patient Experience Data (PED) is required. At a recent European Medicines Agency (EMA) workshop, PED was defined as data collected via a variety of patient engagement activities and methodologies to collect patients’ experience of their health status, symptoms, disease course, treatment preferences, quality of life and impact of health care. Patient Reported Outcomes (PROs), Patient Preferences (PPs) and Patient Engagement (PE) are all examples of PED. Common terminology is fundamental to enhancing global discussion.

The workshop also reinforced the relevance of robust methodology to capture and analyse PED to optimise medicines development, regulatory decision-making and HTA assessments. This will require alignment between regulatory and HTA bodies to ensure that data generated at early stages can be used to inform companies’ submissions to HTA agencies later.

The DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative exemplifies the opportunity for digital clinical data generation and analysis. Institutions working as part of DARWIN have access to healthcare data in a real-world setting. It is expected that by 2025 the initiative will deliver 150 studies to provide and analyse data to support HTA agencies in their assessment of medical technologies. Arguably, this volume of studies generated provides great clinical data resources however the impact of these studies must be considered, are the outcomes meaningful for the patient?

Reinforcing patient relevance in evidence generation is a key priority for the network. Guidance is critical, beyond that of published documents. Support is required to implement collection of PED, such as establishing a coordination centre. Collaboration centres within Networks is important to harmonise and optimise the data collection process to ensure difficulties are addressed along the way. One example where harmonisation is potentially needed is between national and local levels in countries where discrepancies in data management and safety may exist.

The decisions that the data will inform should be considered when establishing PROMs

Data collection is just the starting point. HTA requires a ‘whole-system’ approach to evaluate new technologies which considers whether the patient benefit justifies the cost. This requires data on costs and outcomes, giving rise to the establishment of Patient Reported Outcomes Measurement (PROMs) programmes. These are considered as large-scale, routine measurement in real-world settings; implemented as system wide. Many countries have now implemented their own examples of PROM initiatives, UK’s private HealthCare Information Network (PHIN), Southern Cross NZ and Sweden’s National Patient Quality Registers are just a few examples, to help understand strengthen patient choice, monitor service effectiveness, and improve value for money and evaluate pathways of care, respectively.

Capturing such large quantities of data across borders is only of value if it is clear how the evidence will drive healthcare improvements for patients. Careful stakeholder engagement is required to inform the decision of instrument choice and to provide analyses that are ‘fit for purpose’ and meaningful to patients. Methodological challenges should also be considered, such as case-mix adjustment is crucial to the credibility and validity of the data.

For instance, if between-provider comparisons are a goal, appropriate case-mix adjustment is essential. Also, we need to think beyond feasibility in data collection. We know it is possible to collect data at scale, but we need to ensure good response rates. Approaches can include both incentives that could arguably promote a more genuine belief in the data, but lower responses versus mandatory regulations which are not always so well received but could lead to higher response rates.

Finally, to capture the essence behind patient-centred research, the evidence must be provided in a way that is meaningful to patients. Dissemination of information must be addressed appropriately.

Standardising patient-centred research can relieve burden but must not lose relevance

Patient-centred research must be informed by patients to be important and meaningful. So, what is meaningful to patients? In the US, the development of a framework has begun (Figure 1) and has been run in pilots. The patient-centred core impact set (PC-CIS) is a patient-derived and prioritised list of impacts on a patient’s health and daily life and that of their family and caregivers. It incorporates this with information from other stakeholders, which is combined through a ‘prioritisation process’. However, the process ensures the patient voice remains intact and is not outvoted with the other information.

Figure 1

Implementing a framework such as the PC-CIS could help streamline data collection, preventing the need for it to be collected repeatedly. In pursuit of efficiency, we must ensure that relevance is not lost, and that the tool is able to effectively answer the situation-specific question being asked and inform relevant decisions.

A Core Outcome Set (COS) is an agreed-upon, standard list of outcomes that should be measured and reported, as a minimum in all clinical trials. A method to assist with ensuring the COS is patient-centred and can contribute to meaningful change is the impact to endpoint pathway.

Figure 2

Figure 2 demonstrates this using an Osteoarthritis (OA) PRO Example. These frameworks are recommended as ‘blueprints’ to allow for adaptation to other diseases, or made country or region specific, as examples. In the US, it is envisioned that access to such blueprints can be facilitated through patient communities sharing to their own website to make publicly available.

The idea of a central depositary is also being explored which perhaps could be more viable to assess the potential of these frameworks in Europe. The bottom-line vision is that such frameworks should be publicly available, to empower patients and utilise data to improve and reinforce relevant change.

3 Takeaways

  1. Patient-centred versus patient-informed: Data collected about patients must be meaningful to inform decisions that will have benefits for the patient.
  2. It’s all in the endgame…if you get it right at the start: Time should be taken at the start to understand what is important for the patient, this will guide the correct data to be captured ensuring the potential for vast amounts of data to be collected has a purpose.
  3. We learn from experience: Current PROMs should be evaluated to ensure they are being implemented in a way that benefits the patient. Ongoing maintenance will be required to inform instruments or frameworks to make data country or region specific.

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