UNIFIED

Today, a lot of health research, innovation, and decision-making takes place with little to no patient input. This has a range of consequences, including sub-optimal care and an inefficient use of resources as patients turn away from innovations that don’t fully meet their needs, for example.

The good news is that there are now more ways than ever to gather different types of input from patients. For instance, patient preference information (PPI) captures what matters to patients, how much it matters, and what trade-offs patients are willing to make. Clinical outcome assessments (COAs) measure how a patient feels and functions and include patient-reported outcome (PRO), clinician-reported outcome (ClinRO), observer-reported outcome (ObsRO), and performance outcome (PerfO) measures. Finally, digital health technologies (DHTs) can provide (direct) measures of patients’ health status, feelings, and function, and offer opportunities to collect, analyse, and communicate patient data on a scale previously unimaginable.

Because these different measures are complementary, the most accurate insights into the benefits of a new treatment for patients will likely come from combining them, but doing this in practice is far from easy.

Enter UNIFIED, which aims to generate a robust, unified framework for obtaining a holistic view of a health intervention’s benefits to patients by integrating information from PPIs, COAs, and DHTs. The project will establish evidence-based good practices for using these measures in combination to demonstrate the importance to patients of what is being measured by DHT-derived clinical-study endpoints. The project will also help to determine what patients consider the minimum clinically important difference (MCID) in the context of a clinical trial. When designing the framework, the UNIFIED team will seek to address the use of these patient-centred measures at all stages of the medical product lifecycle, from clinical development right through regulatory review, health technology assessment (HTA), reimbursement, and clinical practice.

The framework and the project’s recommendations for its use will be supported by the results of use cases in five medical domains: paediatric radiation oncology, lung cancer, Parkinson’s disease, obesity, and juvenile idiopathic arthritis. The use cases were chosen because of their diversity in terms of disease areas and the ages and profiles of patients affected.

UNIFIED plans to obtain acceptance and adoption of the UNIFIED framework from a range of stakeholders, including patient groups, regulators, HTA bodies, and healthcare providers.

Ultimately, by ensuring that decisions regarding treatments are made based on patient-centric measures, UNIFIED hopes to improve decision-making at all levels so that patients can benefit from treatments that genuinely meet their needs.