Clearing a Path for OA Drugs

By Jill Tyrer

The development of drugs to treat osteoarthritis will be the focus of a workshop June 22 presented by the U.S. Food and Drug Administration (FDA) in partnership with the Arthritis Foundation.

The Arthritis Foundation has long been committed to finding more options to treat osteoarthritis (OA), the most common form of arthritis. It affects an estimated 32.5 million Americans and costing hundreds of billions of dollars annually in direct and indirect costs in the U.S. Yet the only medications available to treat it are designed to relieve pain and other symptoms. There are none to slow or alter the progression of the disease, as there are for other forms of arthritis.

This workshop builds on previous meetings with the FDA, most recently in 2017, when the Foundation brought together patients living with OA to share their stories and describe to FDA officials the day-to-day challenges they face from arthritis. In June, this event will bring researchers, clinicians, academics, drug manufacturers and patients together with rheumatologists, regulatory scientists, statisticians and others to seek better ways to measure the long-term effectiveness of drugs for OA. The participants will discuss the challenges of development and receiving approval of drugs intended to treat the underlying disease of OA, not just the symptoms, and consider ways to overcome those obstacles.

Endpoint Obstacles

At this point, there still isn’t consensus on how to determine whether a drug is providing long-term benefit for OA. In order for the FDA to approve a drug, it must be shown to achieve a certain goal, or “endpoint” — an outcome that can be reliably measured to determine whether it is providing any benefit.

With osteoarthritis, the FDA has relied primarily on measurements of patients’ pain and function over a few months to approve drugs. However, assessing the benefits of drugs intended to treat the underlying disease has proven challenging. While measuring the space in a joint (“joint narrowing” as cartilage wears away) using X-ray or MRI imaging, may show progression of the joint changes, the disease of OA is very complex. What is currently not know is whether slowing these changes in the joint will result in long-term clinical benefit to patients with OA. The drug might eventually show benefits over a much longer time, years or decades, making it difficult to study these drugs.

That’s why it’s critical to identify endpoints that the FDA can use to measure the long-term benefits of a drug to slow, halt or even reverse the progression of osteoarthritis and reduce its long-term effects on function and pain.

Measuring Success

People living with osteoarthritis do have some effective treatment options, including regular physical activity and weight loss, ice and heat therapy and medications that reduce inflammation and pain. None, however, treats the disease itself; joint replacement surgery is typically the ultimate option for those with end-stage OA.

Other effective medications and therapies are sorely needed to slow, stop or prevent joint deterioration that occurs in osteoarthritis and allow patients to be as functional as possible with limited or no pain in the long haul. But without endpoints that the FDA finds acceptable, researchers and drug makers have been stymied in efforts to pursue affordable options.

Stakeholders are considering various options for endpoints, including how changes in biomarkers in the blood or the use of more sensitive imaging technology, like MRI, can reliably predict the long-term benefit.

In addition, the FDA has proposed endpoints based on the amount of pain and disability a patient experiences that necessitate a joint replacement. This will also be discussed during the workshop.

This workshop is a significant step toward identifying the knowledge gaps with those endpoints and paving the way toward more nonsurgical treatment options for the millions of people living with OA today and in the future.

The day-long virtual workshop will begin at 10 a.m. ET Tuesday, June 22. Learn more and register today.