Mar. 22, 2018

Leading Lupus Experts Propose Actionable Solutions to Improve Clinical Trials

Washington, D.C., March 23, 2018 – A white paper published today by leaders in clinical research for lupus provides actionable proposals for improving lupus treatment trials and advances persuasive evidence for the U.S. Food and Drug Administration (FDA) to consider in support of new approaches. The white paper “Lupus Community Panel Proposals for Optimizing Clinical Trials: 2018,” was developed to address critical issues that have stymied the lupus drug development process for decades.

Lupus is a misunderstood autoimmune disease that can ravage different parts of the body in an unpredictable manner. It is difficult to diagnose, hard to live with and challenging to treat. There is only one treatment approved by the FDA created specifically for lupus – Belimumab. No treatment works for all patients during a lifelong chronic illness, and the white paper establishes that the current standard of disease control is unacceptable, identifying specific problems with the design of lupus clinical trials that are getting in the way of developing effective treatments.

The recommendations in the white paper highlight five key solutions for optimizing lupus clinical trials, which include making trials available to people with all types of lupus, decreasing the size of trials, using recent scientific breakthroughs to select patients most likely to benefit from a given treatment, improving choices in the background medications used, and improving the methods by which the symptoms of people with lupus are tracked and graded.

“I’ve been involved with more than thirty failed clinical trials for lupus since the 1990’s,” said Joan T. Merrill, M.D., Chief Advisor of Clinical Development at the Lupus Foundation of America and member of the Oklahoma Medical Research Foundation, and the lead author of the paper. “The heroes of our field are those who patiently re-evaluated the data from each disappointing study. Now, finally, we can apply those lessons learned to give promising new treatments for lupus a fair test.”

The white paper builds upon the Lupus Foundation of America’s 2009 Lewin Report, which issued recommendations on ways to overcome the barriers that have obstructed lupus drug development. The white paper is also an outgrowth of the Foundation’s ongoing leadership in working with the FDA to advance clinical trials in lupus.

Dr. Merrill along with Susan Manzi, M.D., M.P.H., Chair of the Medicine Institute at Allegheny Health Network and Medical Director of the Lupus Foundation of America, and Victoria P. Werth, M.D., Professor of Dermatology & Medicine at the University of Pennsylvania, School of Medicine drafted the paper. As part of the research for composing the paper, they received input and guidance from nearly three dozen opinion leaders in the field of lupus including lengthy consultations with key clinical scientists from a number of biopharmaceutical companies. This represents the first time the lupus research community has come together to submit recommendations to the FDA for improving clinical trials.

“The breadth of input in this white paper clearly demonstrates that a community of leaders in the field has agreed upon a plan for improving lupus clinical trial design,” said Sandra C. Raymond, CEO of the Lupus Foundation of America. “This is a first of its kind collaboration among lupus experts that may shift the paradigm for how lupus clinical trials are conducted so that more effective treatments for this devastating disease can be developed.”

To view the “Lupus Community Panel Proposals for Optimizing Clinical Trials: 2018” white paper, visit lupus.bmj.com/content/lupusscimed/5/1/e000258.full.pdf.