Lupus community panel proposals for optimising clinical trials: 2018.

Formidable impediments stand in the way of treatment development for lupus. These include the unwieldy size of current trials, international competition for scarce patients, complex outcome measures and a poor understanding of these outcomes in the world at large. The heterogeneity of the disease itself coupled to superimposition of variegated background polypharmacy has created enough immunological noise to virtually ensure the failure of lupus treatment trials, leaving an understandable suspicion that at least some of the results in testing failed drugs over the years may not have been negative, but merely uninterpretable. The authors have consulted with many clinical trial investigators, biopharmaceutical developers and stakeholders from government and voluntary sectors. This paper examines the available evidence that supports workable trial designs and proposes approaches to improve the odds of completing interpretable treatment development programs for lupus.
Competing Interests: Competing interests: JTM, MD Consultant: Anthera, Amgen, Baxalta, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb Company, Celgene Corporation, Eisai, EMD Serono; Exagen Diagnostics, Genentech, GSK, Gilead, ImmuPharma PLC, Horizon, Incyte, Janssen Pharmaceuticals; Mallinckrodt Pharmaceuticals, MedImmune/AstraZeneca, Neovacs SA, Pfizer, ReAlta, RRD International, Sanofi, Takeda Pharmaceuticals USA; UCB. VPW, MD Consultant: Resolve, Eli Lilly, Biogen, Idera, Medimmune, Genentech, BMS, Amgen, Penn own the copywright for the CLASI. IB MD, FRCP Research Support: Roche, GSK and Genzyme, and Sanofi Consulting/Speaker: Eli Lilly, AstraZeneca, GSK and UCB. IB is a National Institute for Health Research (NIHR) Senior Investigator and is funded by Arthritis Research UK, the Medical Research Council and the NIHR Manchester Biomedical Research Centre. BR MD Consultant: Alexion, Aurinia, Biogen, Biomarin, Bristol-Myers Squibb, EMB Serono, Fraizer, Genentech, Gilead, Eli Lilly, Lupus Foundation of America, Mallinckrodt, Pfizer, Pharmalink, Retrophin, Rigel, Trinity, Trinity Partners. JM MD, MMSc Consultant/Advisor: GSK and Biogen IDEC in Lupus indications. EG MD, MPH Consultant: Ablynx/PPDI Data Monitoring Board: Janssen Clinical Trial Investigator: Aurina, BMS, Genentech, GSK. CA MD Consultant: GSK Research Support: GlaxoSmithKline, EMD Serrono, Xencor, Takeda Pharmaceuticals and Janssen. BC MD Investigator: Daavin Corporation and Biogen. MD’E MD Consultant: Kezar Abbvie Independent Data Monitoring Committee: Janssen Genentech Biogen. KK MD Consultant: Amgen, Eli Lilly, Genentech, Roche, BMS, Medimmune, AstraZeneca, Janssen, Xencor, Anthera, Exagen, Baxalta, Biogen Idec, Sorrento Therapeutics Grants/Contracts: NIH, Lupus Research Alliance, Sanford Regenerative Medicine Consortium, Pfizer, Ablynx, Resolve, Celgene, Takeda, Questcor, Proximagen, UCB, Gilead, Ampel, Kirin. KC MD MPH Consultant: AstraZeneca GSK Merck Research Collaborations: AstraZeneca GSK Merck. Research Support: NIH. SM MD, MPH Consultant: Exagen Diagnotics, Inc; GSK, UCB Advisory Board, Lupus Foundation of America (Medical Director), AstraZeneca (Advisory Board).