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

Hierarchy of evidence

tocilizumab

Horizon scanning

• clinicaltrials.gov• EudraCT

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

• Thank you for listening

• Any questions?