Leeds Biomedical
Research Centre
PMR and GCA research: what’s next?
Dr Sarah Mackie, rheumatologistAssociate Professor, University of Leeds
Honorary Consultant, Leeds Teaching Hospitals NHS Trust
Disclaimer
The views expressed are those of the
presenter and not necessarily those of the
NHS, the NIHR or the Department of
Health and Social Care.
• Experimental approach – clinical trials
– “evidence based medicine”
• Observational approach – “basic science”
– understanding of the biological basis of
disease (”lab science”)
– using “big data” to understand patterns of
disease in populations (epidemiology)
• Approaches drawn from social science:
qualitative, mixed methods appraoches
Types of research
Name (company) Country Intervention n
GiAnT France Anakinra vs placebo 70
UGCA US Ustekinumab (single arm only) 20
tbc (Sanofi) worldwide Sarilumab vs placebo 360
(Lilly) US Bariticinib (single arm only) 15
(Kiniksa) worldwide Mavrilimumab vs placebo 60
GCA
All information in public domain and taken from: • clinicaltrials.gov• EudraCT
searches last updated 7 Sept 2018
Name (company) Country Intervention n
SEMAPHORE France TCZ vs placebo 100
PMR-SPARE Austria TCZ vs placebo 32
PMRLEFRCT Netherlands LEF vs placebo 94
tbc (Sanofi) worldwide Sarilumab vs placebo 280
ALORS France Abatacept vs placebo 34
PMR
All information in public domain and taken from: • clinicaltrials.gov• EudraCT
searches last updated 7 Sept 2018
• Clinical trials
– Drug trials
– Trials of non-drug approaches (exercise, diet)
• Lab science
– New technologies for identifying molecules,
cells, processes involved in the diseases
• Epidemiology
– Routinely-collected healthcare data
• Qualitative / social science
More than just drug trials