Guidance on the use of patient experience data in real world studies – EU perspectives

By Julia Lakeland
Chief Product Officer 

 

Patient insights are expected to become increasingly instrumental in optimising medicine development and regulatory decision making. Gathering patient insights and understanding the perspectives of living with and treating specific medical treatments and medical conditions provides invaluable insights to the EMA and EU regulatory network, as well as technology and pharma companies. Questions remain however, as to what data should be collected, how it should be collected, analysed, and ultimately used to support medicines development. As such, patient insights are not systematically captured and used in medication development and regulatory decision making. The outcomes from a workshop held by the EMA with input from patients, consumers, healthcare professionals, academia, regulators, HTA bodies and industry held in late September 2022 looked to define and address some of the key challenges associated with this patient insights to support medicine development and regulatory decision-making.

The concept of patient experience data, as defined in the EU broadly covers five distinct areas:

• Patient Experience Data is data collected from a variety of patient engagement activities and methodologies to collect patient experience of their health status, symptoms, disease course, treatment preferences, QOL and impact of health.
• Patient Experience Evidence is scientifically validated evidence captured.
• Patient Engagement refers to all activities involving interaction with patients to gather their experience on disease, preferences, outcomes, and treatments.
• Patient Preferences is how desirable or acceptable a patient is to a given alternative or choice of outcomes of a given medicine.
• Patient Reported Outcomes refers to health or treatment outcomes reported directly by the patient without interpretation of a third person (clinician or other).

Defining methodologies with patient input

Protocol creation outlines the methodologies which are fit for purpose and produce reliable, scientifically validated data to capture research. With the increased interest in capturing digital real-world patient insights, there is an increased interest in determining appropriate patient focused patient reported outcome (PRO) methodologies to support the relevant information required for Product Information and the Summary of Product Characteristics (SmPC).

Establishing a core team of multi stakeholders early in the study design which includes the voice of the patient alongside scientific and technology experts helps define new methodologies which measure both the effects of medicine as well as capturing supplemental patient experience data and ensures outcomes are aligned to the protocol requirements.

Digitisation of data capture

Digital transformation of data capture offers huge benefits to the collection and analysis of patient experience and real-world data. Consumer grade applications and interfaces, with incorporated patient reminders, educational content, real-time monitoring, and remote patient data capture have made a significant difference in how life science companies can interact with patient populations.

Compliance to data security and data control requirements, such as including GDPR, HIPAA and safe harbour are pivotal requirements across the EU and the wider global community as trials strive to meet the demands of different local country regulatory requirements. System integration activities led by the European Health Data Space are underway and in future will lead to optimised data linkage and standardisation ready for data analysis.

Regulatory acceptance of patient experience data

Most challenges associated with gathering patient experience data can be addressed through effective research design, collaboration, and communication between different patient focused stakeholder groups. One reason often outlined as to why patient experience data is not actively included in medicines development is because of a belief that the data will not be accepted by regulators or is not considered of value in their decision making. Whilst it is recognised that there continues to be a need for further guidance from regulators on what criteria and minimum patient experience data is needed for regulatory decision-making, and ultimately to ensure medicines are fit-for-purpose, EU Regulators have said that they actively welcome patient insights, namely COAs (PerfOs, ClinROs and ObsROs) as part of medicines development and agree that this type of information should be part of marketing authorisation applications. The existing framework developed by the EMA for establishing the qualification of new methodologies, regulatory/HTA parallel scientific advice and the EMA’s Innovation Task Force (ITF) can pave the way to ensuring future international adoption with international regulatory decision makers.

Summary

Gathering direct patient insights as defined by patient experience data is considered as a new scientific discipline that has great potential to help medicines development and regulatory decision-making. As with the adoption of any new concept, challenges exist but with continued input and guidance from stakeholders – developers, patients and regulatory – comes acceptance. Technology vendors should focus on simplifying required study tasks and patient focused activities and encompass personalized workflows with user-friendly interfaces and BYOD modalities. eCOA providers should work closely with patient focused stakeholder groups as well as gaining insights from regulatory decision makers to ensure that COA requirements fully align with scientific outcomes and patient experience data outcomes. By combining ePRO data with other real-world patient experience data such as wearables and EHRs, a full and holistic picture of the patient’s health and lifestyle is created, which not only supports the medicine development and regulatory decision-making but also gives a true indication in to the patient’s wellbeing.