The FDA published proposed guidelines for clinical trials for celiac disease treatments. Since we talk a lot about clinical trials, I feel like it is important for us to discuss these changes and what it might mean for clinical trials moving forward. This is going to be a series of posts because this is VERY important moving forward. Today – endoscopy….

Current FDA guidance does not require endoscopy to be performed at diagnosis nor at any point during the trial. Proposed guidance suggests that celiac must be diagnosed via endoscopy prior to entering the trial. Additionally, a endoscopy must be performed prior to entering the trial with significant damage to the small intestine to justify celiac diagnosis.

What is interesting here is that the FDA does not take into account that many are diagnosed via triple positive blood tests and that they are requiring significant damage to the small intestine prior to entering a clinical trial. Triple positive is a pretty good indicator of celiac disease and many, many studies have confirmed that triple positive is sufficient. It also eliminates any human error in the diagnosis of celiac – no doctor mistaking a sample or pathologist misreading a slide. Second, endoscopies are invasive, expensive, and not totally risk-free procedures for the patient.

On the flip side, I have always said that clinical trials without an endoscopy at the beginning and end are not very useful. Celiac blood tests are not sensitive enough to detect minor gluten indiscretions. Celiac blood tests were never meant to monitor celiac disease but were meant to diagnose celiac disease. The FDA is trying to make certain that the symptoms someone is experiencing are related to celiac and not something else, which is commendable. I think, though, with this requirement the FDA has gone a little overboard.

I get what the FDA is trying to do here. However, what this indicates is a major hole in celiac disease monitoring. First, we need better tools that help monitor gluten ingestion. If you are 100% gluten free, then celiac symptoms should go away and the small intestine could heal. But because most people don’t grown their own food, raise and process for food their own animals, we are at the mercy of food manufacturers. The FDA has failed repeatedly to enforce their own food labeling requirements and maybe better enforcement might be something for them to consider. Second, we need better monitoring for intestinal healing. Right now, the only way to monitor intestinal healing is invasive, expensive, and potentially dangerous. A tool to easily and efficiently monitor intestinal healing in those with celiac disease is required.

I see what the FDA is trying to do and can respect it. But it feels like the pharmaceutical industry has skipped over a few steps. I want a cure as much as the next person, but making sure we get a good cure is equally as important. I think the endoscopy requirement is not a bad thing.

This is the first of a few days of posts…..here is a link to the complete requirements.

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