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PSI Conference 2023: ​​Importance of patient-reported outcomes in clinical studies: methodological challenges and future strategies

12 June 2023

Konstantina Skaltsa; Karl Karu; Khadija Rantell; Christoph Schürman; Lee Aiyegbusi.

In this session, the speakers talk about PROs from drug development through to market access. Konstantina Skaltsa (IQVIA) opens the session with an introduction on the importance of PROs in drug development. Karl Karu (IQVIA) follows with a presentation on specific challenges when analysing PRO data. Khadija Rantell (MHRA) provides the regulatory perspective and Christoph Schürmann (IQWiG) a Health Technology Assessment (HTA) angle. The session concludes with a panel discussion, in which the speakers are joined by Lee Aiyegbusi (University of Birmingham). 

Patient reported outcomes (PROs) provide unique information on the impact of a medical condition and its treatment from the patients’ perspective. Incorporation of PRO measures in clinical studies is one way of establishing a more patient-centric approach to trial design. Several guidelines and discussion documents have been published by different stakeholders with respect to measurement and analysis of PROs. The FDA published a guidance on PROs in 2009, while currently, a series of methodological guidance documents on the collection of patient experience data are being developed through Patient-Focused Drug Development. EMA published a guidance on PRO measures in 2005 and a specific one for oncology studies in 2016. EUnetHTA includes HRQoL measures as one of the main three clinical endpoints used in a relative effectiveness assessment. SISAQOL-IMI (Setting International Standards in Analysing PROs and Quality of Life Endpoints in Cancer Clinical Trials–IMI) international consortium aims to generate recommendations to standardize the use, analysis, and interpretation of PRO data in oncology trials. Despite the volume of guidance documents and ongoing initiatives, significant challenges remain to ensure PRO data are formally and systematically incorporated into appraisal discussions and decisions. In this session, four speakers talk about PROs from drug development through to market access. Konstantina Skaltsa (IQVIA) opens the session with an introduction on the importance of PROs in drug development. Karl Karu (IQVIA) follows with a presentation on specific challenges when analysing PRO data. Khadija Rantell (MHRA) provides the regulatory perspective and Christoph Schürmann (IQWiG) a Health Technology Assessment (HTA) angle. The session concludes with a panel discussion, in which the speakers are joined by Lee Aiyegbusi (University of Birmingham).

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