Lack of treatment-naive patients makes recruiting tough, but a good partner can help uncover hidden pockets of these patients to spur the research process along.
The Challenge with IBD
Inflammatory Bowel Disease (IBD) affects millions of patients around the world, including a million people in the US and 2.5 million people in Europe. Yet efforts to combat these diseases have been frustrated in the past due to the complexity and cost associated with running clinical trials.
IBD covers a group of disorders, primarily including ulcerative colitis (UC) and Crohn's Disease (CD), in which all or part of the patient’s digestive tract becomes inflamed. These conditions periodically “flare up” causing the patient severe diarrhea, pain, fatigue and/or weight loss, with the most severe cases leading to life-threatening complications. The lack of new treatments for these diseases has left a significant unmet medical need among IBD patients around the world.
For decades, physicians treated IBD flare-ups with steroids to trigger remission, but steroids have multiple negative side effects. Then in the late 90s, infliximab, a genetically engineered monoclonal antibody was approved to treat CD and later other IBD conditions. Today, in addition to other TNF α-antagonists, they are common first-line treatment option for patients with moderate/severe disease, though it can cost more than $20,000 per year with approximately one third of patients not responding to treatment and then one third of patients that do respond, losing the response within 1 year.
Infliximab continues to be the treatment of choice; however, a number of clinical research efforts have helped improve our understanding of these conditions with hopes of using this information to develop new and better treatment options. Over the past 10 years, we have seen significant progress in identifying genes for CD and UC to more proactively diagnose these conditions, technology for DNA sequencing has improved, and researchers have identified species and groups of bacteria that may be involved in IBD. We are also seeing several Phase II and Phase III trials underway to develop oral monoclonal antibody as well as oral non biologic small molecules that will make the treatment more convenient, and potentially safer as TNF α-antagonists have potential side effects such as infections and possible Lymphoma.
IBD also has a number of powerful patient advocacy groups, including The Crohn’s & Colitis Foundation of America (CCFA), that are helping to drive this research forward by partnering with industry leaders and investing millions of dollars in projects.
Searching the globe for patients
The growing interest in developing new drugs for IBD is also making these trials more challenging to deliver. It is a hugely competitive landscape, with many competing studies actively recruiting. Adding to the challenge are the limited number of treatment-naive patients with IBD (patients who have never undergone treatment for their condition), because biologics such as infliximab, adalimumab and vedolizumab are readily available first-line treatment option that are usually reimbursed, making it an easy choice for physicians and patients. That means even when researchers find treatment-naive patients, they may be unwilling to participate in a study that may land them in a placebo group.
Unfortunately, there are no simple answers to this situation. The best hope for many trial sponsors is to work with a partner that has deep expertise in and specific experience addressing IBD research, and the industry data to identify which trial sites around the world are most likely to have access to these patient populations. Even if a trial is targeting a specific country or region, a good partner can help them identify the local thought leaders, physicians, and patient advocates who are linked to these patient populations, and may be able to identify those who are interested in clinical research as a treatment option.
We have also found success working with advocacy groups to build online registries for their members to educate them about their disease and allow them to register their interest in taking part in a trial. This not only helps the sponsor engage with the local patient community, it also acts as a prescreening tool to focus recruiting efforts on patients who have already shown an interest in the process.
When choosing these partners, sponsors should be sure their team has expertise in the regulatory environment related to IBD and understand how best to capture real-world data through registries and observational studies to support their research and build a strong case for the safety and efficacy of their treatments.
Even with a great partner, recruiting for these trials will continue to be a challenge, but those sponsors who are able to overcome these obstacles have an opportunity to address a significant healthcare gap. IBD patients around the world are in need of innovative new treatments to better manage or eliminate their symptoms and improve their quality of life. The companies that are able to bring drugs to market to address this need promise to impact millions of lives worldwide.