Sep. 28, 2018

Lupus Experts Present Consensus Proposals to FDA for Advancing Clinical Trials and Health Outcomes

A Lupus Foundation of America-led delegation, including several world-renowned lupus experts and senior leaders from the Foundation, met with more than a dozen top decision makers at the U.S. Food and Drug Administration (FDA) to present recommendations for improving lupus clinical trial design and to discuss actionable steps toward advancing these solutions.

The recommendations are part of a white paper “Lupus Community Panel Proposals for Optimizing Clinical Trials: 2018,” that was recently published in Lupus Science & Medicine and submitted to the FDA prior to the meeting with the Foundation-led delegation. During the meeting, the delegation highlighted five key solutions for optimizing lupus clinical trials. These recommendations from the white paper would allow for smaller, more efficient trials; use of recent scientific breakthroughs to select patients; new approaches to involve the use of background medications; trials to be made available to people with all types of lupus; and improving the tools by which the symptoms of people with lupus are measured.

“For the past few decades, we have seen issues related to the heterogeneity of lupus and challenges associated with clinical trial design get in the way of developing effective lupus treatments that are so desperately needed for people battling this disease,” said Sandra C. Raymond, CEO of the Lupus Foundation of America. “The white paper’s recommendations were developed by a consensus among lupus opinion leaders aimed at removing barriers that exist in clinical trial design. The proposals provide a path forward for industry to develop new, safe and effective medications for individuals with lupus, and opened a productive dialogue between the FDA and lupus community, which will continue.”

Susan Manzi, M.D., M.P.H., medical director of the Lupus Foundation of America and chair of the Medicine Institute at Allegheny Health Network, began the lupus stakeholders’ presentations by providing an overview of the Foundation’s longstanding efforts to address the barriers of lupus clinical trial design, as well as a brief recap of past discussions with FDA representatives about improving trials. Several lupus experts within the delegation explained the white paper’s recommendations in further detail before opening up the meeting for a two-way discussion with the FDA to get their input and feedback, as well as to discuss next steps.

The FDA acknowledged the value of consensus recommendations, and indicated that it would refer to the recommendations in future discussions with sponsors of lupus clinical trials.

The Agency also shared the opportunity for sponsor companies to invite Foundation representatives to join them at meetings with the FDA (e.g., Pre-IND, Pre-NDA) in order to provide the patient perspective. The FDA also stated its support for members of the academic research community to attend such meetings to provide additional stakeholder perspectives.

“We’re grateful for the opportunity to have met with the FDA to discuss well-established advances in lupus clinical trial design. This is a critical step forward as we continue to advance lupus drug development,” said Dr. Manzi. “I’m pleased to see that the FDA recognizes the urgent need for continued collaboration and to begin to implement creative solutions like those outlined in the white paper.”