Patient-Centered Research: Challenges in Gathering and Analyzing Patient Experience Data in a Real-World Setting

Leadership for Change

Leadership for Change


The FDA and EMA are committed to gathering more patient experience data in healthcare, particularly for drug approvals and capturing real-world evidence for effectiveness evaluation. This post reviews ISPOR’s second plenary panel discussion in Vienna 2022 on patient-centered research and its challenges in gathering and analyzing patient experience data in a real-world setting, particularly within the EU. Below we present the importance of rigor in choosing, implementing, and analyzing patient experience data in non-randomized, uncontrolled study designs with the potential for missing data.

Panelists include:

Tara Symonds (Clinical Outcomes Solutions Ltd, Folkestone, Kent, United Kingdom) moderated the discussion.

Dr. Burgos discusses Darwin EU initiative (distributed network of real-world data) and a recent workshop on patient experience data and how to integrate this in the real-world setting.

Professor Nancy Devlin discusses the implementation of patient-reported outcome data capture and challenges of data analysis in real-world setting, as well as the PROMS initiative in the UK, Canada, and New Zealand.

Dr. Eleanor Perfetto discusses the Core Impact initiative (National Health Council, USA) for standardizing patient experience data collection and the use of core outcome sets.

Juan Garcia Burgos

  • Reported on a recent multi-stakeholder workshop on patient experience data in healthcare.
  • Main objectives: common understanding of patient experience data, gap analysis and inventory of current methodologies, discussion of priorities to enhance patient data collection in medicines development and regulatory assessment.
  • Definition of patient experience data as data collected through various patient engagement activities or methodologies to collect patient experiences on health status, symptoms, treatment preferences, quality of life, and impact on healthcare.
  • Darwin EU initiative is a federated data network providing EMA and the European Commission with access to real-world healthcare data for use in medicines assessment.
  • Patient experience data is a top priority for the EU regulatory network and the issuance of regulatory strategy, together with all stakeholders.
  • There’s a need for guidance to facilitate generation and collection of patient experience data, and a future reflection paper from the EMA will set out the best approach.
  • Challenges: terminology, data quality, data integration and harmonization, stakeholder engagement and capacity building.
  • Next steps: further development of guidance and tools, collaboration with stakeholders to advance patient experience data collection and use in regulatory decision-making and medicines development.

How is the DARWIN network progressing to help implement the joint clinical assessment to start in 2025? How will EU countries and data partners be encouraged to contribute data?

  • DARWIN EU initiative progressing well with establishment of Coordination Center and first 10 data partners. Data partners are public and private institutions with access to real-world healthcare data.
  • DARWIN initiating three studies: rare blood cancer prevalence, vaccine consumption, antimicrobial resistance.
  • Call for expression of interest for new partners to join the network by end-of-year.
  • Target of 150 studies per year by 2025 to provide data for regulatory decision-making and medicines development through an analysis of real-world healthcare data.
  • Importance of coordination among EU countries and regions for patient experience data collection and use.
  • Need for dialogue with all stakeholders to ensure understanding and agreement on data protection and sharing.
  • Good experiences from initial studies and collaborations can be shared within the network to facilitate future initiatives and overcome difficulties.
  • Incentivizing versus mandating data collection, there are pros and cons of each approach. The use of incentivizing can lead to genuine belief in the value of data but may not achieve high response rates. Mandating can achieve high response rates but may not be well received by healthcare providers.

How will the EMA facilitate a common approach to patient experience data across all EU countries?

  • Evaluation of patient experience data will be done centrally at the European Medicines Agency (EMA). All member states participate in this process, facilitating a common approach and discussion of different opinions.
  • It is important to have a common understanding of patient experience data in healthcare not only within the EU but also globally.
  • Transparency is important in the assessment and incorporation of patient experience data into decision making and medicine labeling.
  • There is a need for consistency in how patient experience data is valued and used.
  • Encouraging developers to seek scientific advice on their development plans, including the collection of patient experience data, can streamline the process and improve the quality of the data generated.

Nancy Devlin

  • Health technology appraisal focuses on evaluating new technologies, not older treatments and services where cost effectiveness evidence is lacking to facilitate disinvestment decisions. Disinvestment enables investments in new technologies.
  • Entire system approaches to health technology appraisal and health economics and outcomes research are needed to address the bulk of spending in healthcare systems.
  • System-wide use of patient reported outcome measures (PROMs) involves large scale, routine collection of patient outcomes data in real world settings.
  • PROMs data can be used to:
    • Monitor and evaluate effectiveness of healthcare
    • Compare cost effectiveness of services
    • Understand pathways of care and what works best for different patient types
    • Monitor performance of healthcare providers
    • Improve patient choice
    • Understand health inequalities beyond mortality
  • Examples of PROMs initiatives include:
    • English NHS PROMs program implemented 2010 for elective surgeries (high volume, high cost, hip replacements, knee replacements). Data from PROMS before and after surgery helps with monitoring variation and provider performance linked to reimbursement.
    • UK private health care information network collecting patient reported outcomes.
    • Alberta Health Services and Alberta Health Quality Council collecting patient reported outcomes to monitor effectiveness and cost effectiveness.
    • Sweden has national patient quality registers collecting generic and condition-specific patient reported outcome measures.
    • Southern Cross in New Zealand uses patient reported outcomes to inform funded care packages.
    • The New Zealand government is also beginning to collect patient reported outcomes.
  • Methodological Challenges:
    • Methodological challenges include choosing appropriate instruments and interpreting variation in data.
    • Case mix adjustment is important for credibility and validity when comparing between healthcare providers.
    • Many data sets are limited to simple before and after comparisons, which may miss relevant long-term differences and rely on assumptions about counterfactuals.
    • It is important to go beyond single elective procedures and consider complex pathways of care with multiple interventions when analyzing outcomes and care delivery.
  • Key Learnings:
    • It is important to think beyond feasibility and use the data to inform decisions
    • Be clear on the decisions the data will inform and how it will be analyzed and reported
    • The data and analysis should be fit for purpose and defensible to inform decisions
    • Case mix adjustment is important for understanding variations and performance
    • Patient choice can be an important goal, and the evidence should be provided in a way that is meaningful to patients
    • Clinical leadership and strong outcome champions are crucial for success
    • Data collection should lead to positive actions and impact on patients

Eleanor Perfetto

  • Patient centered core impacts are a patient-derived, patient-prioritized list of the most important effects of disease and treatment on a patient’s health and daily life, and that of their family and caregivers.
  • Patient experience data includes potential aspects of patient experience, gathered through patient engagement activities, such as qualitative research, focus groups, and interviews.
  • Patient-reported information on most important impacts is combined with other stakeholder information and literature through a prioritization process to create the core impact set, in which patients’ voices are preserved.
  • Patient centered core impact sets are recommended as a tool to ensure that patient voices are heard and carried through identifying the most important impacts of disease and treatment on patients’ health and daily life.
  • The patient community, in partnership with researchers, should drive the creation of the core impact sets to ensure their relevance and meaningfulness to patients.
  • The core impact sets should be maintained and kept up to date and may need to be specific to certain regions or countries.
  • The core impact sets can inform the development and selection of patient-reported outcome measures, as well as other data collection efforts.
  • Currently, data on the most important impacts of disease and treatment on patients’ lives is not being collected, and the use of core impact sets can help gather this information.

Patient Experience Data in Healthcare

Development and selection of patient-reported outcome measures.

Will there be a central repository, and will it be licensed?

  • There will be no licensing issues for patient centered core impact sets, as they will be created by patient communities for the purpose of sharing information.
  • Good documentation of the core impact sets will allow potential users to determine their relevance and potential for use in their own populations.
  • Each patient community that develops a core impact set will post it publicly on their own website, and a repository may be created at the National Health Council or another organization.
  • Pilot studies with patient advocacy organizations will be conducted to gather more information on the creation and use of core impact sets.
  • The core impact sets will be publicly available for anyone to use and leverage.

How can DARWIN be used for reimbursement decisions (HTA) as well as regulatory submissions (EMA)? Will other stakeholders be involved in furnishing that data, or using it as well to support reimbursement decisions?

  • Data generated should be used across the whole lifecycle, including for regulatory purposes and Health Technology Assessment (HTA).
  • Collaboration is necessary to ensure that data meets all needs and can support various decisions, including cost-effectiveness.
  • Efforts should be made to align policies and strategies with HTA bodies to improve the use and usefulness of data in the future.

Key Takeaways

  • Collecting data from individual patients can be costly, resource-intensive, and burdensome. It is important to ensure that the data being collected is relevant and meaningful to patients. A patient-centered approach to data collection can lead to a vast amount of useful data that can improve the lives of patients and their families.
  • Patient experience data in healthcare is becoming essential for optimizing medicine development and regulatory decision making. Patient experience data is closely linked with real world evidence and real-world data. There is a need to foster the generation and use of this data in regulatory decision making. It is also important to facilitate the incorporation of this data into daily activities.
  • Collect data that are meaningful to patients and ensure that data is used to make decisions that benefit patients. Evaluate implementation of PROMs to ensure benefit to patients justifies the cost of collecting data.