Written by Bailey Arman
Recently, a group of researchers published their recommendations for standardizing celiac disease clinical trials. While on the whole we agree with the content, we were dismayed to learn that no patients or patient organizations had been consulted to develop these guidelines. At Beyond Celiac, we firmly believe that the patient voice is not only a valuable addition to conversations about research and potential treatments, but a necessary one.
We are proud to say that our response, a letter to the editor authored by Dr. Debra Silberg, Chief Scientific Officer, Dr. Jordan Dubow, Chief Medical Officer, and Alice Bast, CEO, was recently published in Gastroenterology.
A group of researchers wrote an article titled “Standardizing Randomized Controlled Trials in Celiac Disease: An International Multidisciplinary Appropriateness Study.” They offered potential guidelines that all researchers can use in clinical trials to determine whether or not a drug for celiac disease is effective or not.
These guidelines focused heavily on “histological endpoints.” In other words, they want any potential treatment to show that it can heal the lining of the intestine. If it doesn’t do this, it’s not worth the effort.
There are aspects of the guidelines that we agree with, and aspects that we argue would benefit from patient insight, so we wrote a letter to the editor in response.
We fully agree that clinical trials for celiac disease should be standardized. If there is agreement on what it means for a medication to successfully treat celiac disease, we can better compare the benefits, drawbacks and overall success of various drugs.
We were also pleased that the researchers commented on pediatric trials. Parents and caregivers want the best for their children, and they often ask us about when their child could potentially have a treatment that is more effective and less restrictive than the gluten-free diet. In a 2019 Beyond Celiac survey of 487 parents of children diagnosed with CD, 57.3% indicated they would allow their child to participate in research that requires blood tests. As a sensitive class, children are usually only allowed into trials after the drug has been approved or found safe and effective in adult populations, but we appreciate the researchers being proactive.
Based on our data from patients, the recommendations over-rely on intestinal healing to measure the success of a drug. There are two major issues with this: one, intestinal healing is difficult to achieve and requires multiple endoscopies, and two, symptom relief is worth working toward, too.
While intestinal healing is absolutely something we should work toward, improvement to the damage of the intestine can take weeks or even months to show, and healing can take even longer. Clinical trials aren’t always long enough to show healing.
Furthermore, few patients want to intentionally damage their intestine without a guarantee that they will get better. Although 57.3% of caregivers indicated they would allow their child to participate in research that requires blood tests, that number dropped to 19.9% if an endoscopy with biopsy was required and dropped even further to 7.6% if a long-term gluten challenge with 4-6 weeks of gluten exposure was required.
We won’t have a treatment beyond the gluten-free diet if no one is willing to sign up for clinical trials, so it is worth discussing whether some of these elements are truly necessary to measure success.
There was little mention of tracking symptoms in the recommendations. However, when we look at our data, it’s clear that our patients want relief from symptoms caused by accidental exposure. Symptoms can be debilitating, and patients are interested in a medication that would bring them relief after a glutening.
We also encourage researchers to consider non-GI symptoms as valid measures. Many patients with celiac disease have non-GI symptoms—in fact, some only have non-GI symptoms. These can be as inconvenient or painful as GI symptoms, and are worth tracking.
Again, while we agree on aspects of the recommendations, we were dismayed to learn that there was no input from patients or patient-centered organizations. To rectify this, we provided data we previously collected from patients in our letter to the editor, and encouraged researchers, academics and doctors to talk with their patients on this topic in the future.
We are proud and humbled to be able to represent you, our community, and Beyond Celiac will continue to keep the patient perspective at the forefront of our work as we navigate questions around funding research, providing resources, and deciding which organizations to partner with. Your insight matters to us! Please continue to share your opinions and stories with us so we can better serve you; comment on social media, take our annual community survey, and join our patient registry Go Beyond Celiac to share your thoughts.
If you’d like to read our full letter to the editor, visit the Gastroenterology website.