Focusing on rare kidney diseases with unique collaborative efforts

Focusing on rare kidney diseases with unique collaborative efforts

Rare disease research faces a common challenge: how do you study new treatments and advance research when there aren't a lot of people living with the rare disease? And how do you incentivize drug development sponsors to invest in these important studies and encourage patients to participate in clinical trials?

To help address these challenges in Immunoglobulin A Nephropathy (IgAN), a rare kidney disease, Barbara Gillespie, MD, FASN, Vice President and Therapeutic Head of Nephrology at Fortrea, joined a workgroup with the Kidney Health Initiative (KHI) and U.S. FDA. At the time, there were no FDA-approved therapies for IgAN, even though IgAN was first recognized in 1968.

Fast forward from 2016 to today: patients living with IgAN now have three FDA-approved options and drug development sponsors have an accelerated approval pathway to help get new treatments to patients earlier.

Accelerating clinical trials with a surrogate endpoint

Dr. Gillespie recently discussed how the drug development landscape for IgAN shifted as a result of this unique collaboration. She reflected on powerful data sharing efforts that gathered data from many trials to evaluate proteinuria, a widely known biomarker of IgAN. The results of this large-scale effort were published in a peer-reviewed publication that increased sponsors' confidence to study new therapies for IgAN. Their analysis demonstrated that proteinuria is a reasonable likely surrogate endpoint for IgAN clinical trials.

"Our efforts involved the FDA, academia and sponsors. It was an important first step to enable the accelerated pathway for IgAN clinical development," explained Dr. Gillespie.

“While many drugs were being tested in various glomerular diseases, the number of IgAN trials subsequently increased because our paper endorsed a clear path toward accelerated approval, thereby incentivizing sponsors to complete the trials, thus getting drugs into the hands of patients earlier,” said Dr. Gillespie.

Ongoing collaborations to advance rare kidney disease research 

Based on the initial success of the first IgAN workgroup, new collaborations have developed to include another rare kidney disease: focal segmental glomerulosclerosis (FSGS). Dr. Gillespie has joined a team of nephrology key opinion leaders and FDA leaders with the hope of assessing endpoints to better define approval pathways and achieve new drug approvals for FSGS patients.

Get the full story

In our article, Dr. Gillespie shares her involvement in the latest initiatives, recent outreach to patient advocacy groups and her hopes for improving nephrology research and making a difference for people living with kidney disease. Read the article.

Join Dr. Gillespie and our renal/hepatic impairment trial experts for an informative and engaging Renal/Hepatic impairment webinar on May 9, 2024: “Get your study enrolled – What to avoid putting in your impairment study protocol that slows recruitment.”

In addition, Dr. Gillespie co-moderated a recent Kidney Health Initiative webinar: “KHI Listening Session on Data Sharing to Advance Clinical Development of IgAN Therapies” which is hosted on youtube.