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Great trial design question
- Thread starter asadmom
- Start date
Because it's much easier and more relevant to prove whether a drug works better than nothing (placebo) vs works better than an anti-TNF.
Imagine new drug X that isn't perfect but works pretty well. It induces remission in say 45% of IBD patients - modestly better than current anti-TNF Y that works in say 40% of patients and way better than placebo Z for which say 15% randomly achieve remission. It will take a clinical trial of a few hundred patients to convincingly show that new drug X is of value because it works better than placebo Z, but it will take thousands of patients and many more millions of dollars to show that it works modestly better than anti-TNF Y.
After drug X is FDA approved based on the placebo study, the company can do the head-to-head comparison with drug Y if there is a clamoring need to. But beneficial new drugs (which means new treatment options for us) should not be held to a higher standard of superiority to existing drugs and held off the market because those trials have not yet been done (and may never be done - see the phrase " many more millions of dollars" above).
Imagine new drug X that isn't perfect but works pretty well. It induces remission in say 45% of IBD patients - modestly better than current anti-TNF Y that works in say 40% of patients and way better than placebo Z for which say 15% randomly achieve remission. It will take a clinical trial of a few hundred patients to convincingly show that new drug X is of value because it works better than placebo Z, but it will take thousands of patients and many more millions of dollars to show that it works modestly better than anti-TNF Y.
After drug X is FDA approved based on the placebo study, the company can do the head-to-head comparison with drug Y if there is a clamoring need to. But beneficial new drugs (which means new treatment options for us) should not be held to a higher standard of superiority to existing drugs and held off the market because those trials have not yet been done (and may never be done - see the phrase " many more millions of dollars" above).
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Think of it another way: if this trial design had already been a requirement, today we wouldn't have any new IBD drugs beyond Remicade. Because it would have been very difficult and ruinously expensive for the drug companies to prove that Humira, Entyvio, Stelara, Skyrizi, etc. and the JAK inhibitors work a lot better than Remicade instead of a lot better than placebo.
So for us that would mean Remicade or nothing. If Remicade didn't work for you or it lost effectiveness over time, you would be left with only the therapeutic weak tea options that we had 25 years ago before the advent of the biologics era.
So for us that would mean Remicade or nothing. If Remicade didn't work for you or it lost effectiveness over time, you would be left with only the therapeutic weak tea options that we had 25 years ago before the advent of the biologics era.