Background/objectives: Treatment guidelines and strategies are often based on data from randomized controlled trials and observational clinical studies. These sources drive treatment decisions, yet the data they provide may have limited relevance to the wider population in real-world clinical practice due to the narrow selection criteria applied to patients in trials. Information used to inform clinical practice and improve patient outcomes can, therefore, be unreflective of real-world clinical situations. The purpose of this article is to assess the value of Adelphi Disease Specific Programmes (DSPs) as sources of real world data.
Methods: DSPs are large, multinational, observational studies of clinical practice for a range of common chronic diseases. Treatment practice data are collected by physicians (n = 700) who are asked to provide information for the next 10 patients consulting for a specific condition. These patients (n = 7000) are also invited to fill out a self-completion form providing their own assessment of symptoms, expectations and quality of life.
Analyses: This article provides examples of the statistical techniques that have been employed to analyse the data in terms of cost/burden of illness, quality of life, disease severity and progression, compliance and adherence to therapy, impact of treatment guidelines and analyses of unmet need.
Conclusions: DSPs can support clinical understanding of how diseases are managed including rationale for doctor decision-making and patient attitudes to their condition. Comparisons with other data sources and limitations of the programmes are discussed (including the fact that, unlike claims databases and registries, the DSPs are cross-sectional and not longitudinal).