Value, Access and Regulatory Strategy Workshop

Patient relevant endpoints (PRE), patient-reported outcomes (PRO) and patient-centred outcomes (PCO) are frequently used interchangeably, even if their definitions and roles differ considerably. PRE are clinical endpoints that reflect how a patient feels, functions and survives; they cover broadly morbidity (due to a disease or its treatment), mortality and health-related quality of life and may be assessed both by patients and by physicians; quite often, PRO and PRE are identical. The simultaneous assessment of all PRE versus adequate comparator is a hallmark of relative effectiveness assessment in Europe. PRO are outcomes that can be reported only by patients themselves due to their subjective nature; they cover simple measures such as symptoms, and more complex measures such as symptom rating scales and multidimensional questionnaires of health-related quality of life. Patient-centered outcomes and related research (PCOR) are aimed at developing structured patient engagement and centeredness in the comparative effectiveness field as well as in the shared decision making in clinical practice. This panel will discuss PRO, PRE and PCOR, and their use to support marketing authorisation and reimbursement decisions in Europe. Collaboration between patients and decision makers (regulatory, HTA and payers) will be described and discussed. The need for patients and patient representatives well informed and trained in the area of medicines development will be highlighted, and ongoing training initiatives, e.g. the European Patient Academy (EUPATI) will be discussed as relevant actions to allow the informed patient engagement in the medicines development process and clinical use.

• Patient’s role in regulatory and HTA process
• Patients as experts: role, training, committees
• Suitability of data from new sources (eHealth, mobile phone apps) - How to include it into the decision making process