Lupus community panel proposals for optimising clinical trials: 2018

Area of Science:

• Rheumatology
• Clinical Trial Design
• Systemic Lupus Erythematosus

Background:

• Developing effective treatments for systemic lupus erythematosus (SLE) faces significant hurdles.
• Current clinical trials are often too large, suffer from international patient recruitment competition, and utilize complex outcome measures.
• Disease heterogeneity and background polypharmacy contribute to "immunological noise," potentially rendering trial results uninterpretable.

Purpose of the Study:

• To examine evidence supporting workable clinical trial designs for lupus.
• To propose strategies for enhancing the interpretability and success rate of lupus treatment development programs.
• To address challenges in lupus clinical research by consulting with key stakeholders.

Main Methods:

• Review of existing evidence on clinical trial methodologies in lupus.
• Consultation with clinical trial investigators, biopharmaceutical developers, and government/voluntary sector stakeholders.
• Analysis of factors contributing to trial failures and uninterpretable results.

Main Results:

• Identified formidable impediments including trial size, patient scarcity, and complex/poorly understood outcome measures.
• Highlighted the impact of SLE heterogeneity and polypharmacy on trial data interpretability.
• Gathered insights from diverse stakeholders on improving trial design and execution.

Conclusions:

• Significant challenges impede lupus treatment development, often leading to uninterpretable results.
• Workable trial designs and improved approaches are crucial for advancing lupus therapeutics.
• Further research and strategic planning are needed to overcome current obstacles in lupus clinical trials.