11 March 2025, 17:00-17:30 Clinical Efficacy vs. Effectiveness: Ideal Results vs. Real-World Impact

Abstract

Advanced targeted therapies have revolutionised treatment options for patients living with immune-mediated inflammatory diseases (IMIDs). Randomised controlled trials (RCTs) are widely accepted trial designs that evaluate the effect of the treatment compared to standard therapies or placebo and are an essential step for gaining regulatory approval. RCTs often have stricter eligibility criteria but are not always reflective of real-world usage of medication and therefore medication effectiveness. This webinar will summarise efficacy and clinical effectiveness, their similarities and how they differ in their context and scope with examples from the field of rheumatology.

Dr James Bluett (MBBS, MRes, MSc, PhD; Orchid ID: https://orcid.org/0000-0001-5062-5779) is a Senior Clinical Lecturer and Honorary Consultant in Rheumatology at the University of Manchester. In 2016 he completed his PhD developing and validating a mass spectrometry assay for the detection of methotrexate which has been applied in clinical trials. His research interests encompass a wide range of topics, including pharmacogenomics, treatment optimisation and adherence.

James is dedicated to training the next generation of scientists and clinicians. He supervises PhD, MSc and medical students and has served as an examiner for PhD research degrees.

In addition to his academic pursuits James is actively involved in a number of working groups and leadership positions. He is a member of the British Psoriatic Arthritis ConsorTium (Brit-PACT) steering committee and holds leadership roles including the Integrated Clinical Academic Training Rheumatology Academic Programme Lead and is the National Specialty Lead of the NIHR Research Delivery Network (RDN) for Musculoskeletal and Orthopaedics.

James’ research efforts have been supported by prestigious grants and collaboration with industry. He has secured substantial funding from organisations including the National Institute for Health and Care Research.