Patient-generated evidence to improve outcomes, support decision making, and accelerate innovation

The amount of health data generated by citizens themselves is rapidly increasing. Such data includes patient-reported outcome measures (PROMs), patient preference information (PPI), and patient-reported experience measures (PREMs), as well as other digital health data/digital biomarkers. While the potential for these data to be harnessed to improve individual healthcare is enormous, these data are often fragmented among multiple providers, so that neither the citizen, nor the healthcare ecosystem have a comprehensive overview, and therefore it is very challenging to fully use these data to provide reliable evidence for decision-makers, and to improve health outcomes.

Research and innovation (R&I) actions to be supported under this topic will aim to address this challenge by:

• Developing a framework to integrate patient input and patient-generated data for use in decision making (regulatory, health economic evaluation, reimbursement, healthcare programme design, tailored prescription of therapies, and technology development), benefit-risk evaluation and value assessment of integrated healthcare solutions.¹ Applicants should build on existing frameworks where appropriate and appropriately address ethics considerations.
• Implement several use cases to support and demonstrate the use of the framework, focusing on using patient input and patient-generated evidence to address challenges that are not adequately addressed by other initiatives. These use cases should demonstrate the value of using patient input (PROMs, PPI, PREMs) and patient generated data (digital health data/digital biomarkers) along the healthcare continuum, including showcasing improvements to data interoperability, healthcare workflows and processes, disease prevention, and care, including home-based care. These use cases should also act as examples of best practice for future use of the framework.
• Facilitating multi-stakeholder access to patient inputs and patient-generated health data such that actionable harmonised data can be used for quality decision making.
• Comparing/contrasting the properties of the three types of patient input (PROMs, PPI, PREMs), identify differences and opportunities for integrated/complementary use.
• Developing an approach or approaches to integrating PROMs, PPI, and PREMs data into the design of core outcomes sets, end-to-end patient treatment pathways, clinical decision support systems, and treatment guidelines. The core outcome sets used within the project should be made available more widely where possible.

Applicants are expected to seek engagement with regulators where relevant (e.g. through the EMA Innovation Task Force, scientific advice) and consider allocating appropriate resources to explore synergies with other relevant initiatives and projects.

The following impacts are expected:

• Enable the added value of people-centred integrated healthcare solutions¹ to be assessed according to criteria that matter to patients and citizens, using patient-reported outcome measures (PROMs), patient preference information (PPI), and patient-reported experience measures (PREMs).
• Facilitate the development and implementation of integrated healthcare solutions based on patient input including PROMs, PPI, and PREMs. These solutions should better respond to the needs and preferences of patients and citizens and support an inclusive approach.
• Enable the smart use of patient input and patient-generated evidence to facilitate the faster market entry of patient-centric and cost-effective advanced integrated healthcare solutions¹, and also spur further innovation by improving return on research and innovation investments.
• Use patient input gathered via m-health, e-health and other technologies to gain improved insights into the real-life behaviour of, and challenges faced by, patients of all ages with complex, chronic diseases and co-morbidities.

¹ _{Integrated healthcare solutions are innovative solutions integrating various technologies, coupled with complementary tools and services.}