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Specifics of clinical trials in rare diseases

MedExpress Team

Published July 10, 2024 10:00

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Interview with Prof. Zbigniew Żuber, pediatrician, rheumatologist, head of the Department of Pediatrics and Rheumatology at St. Louis Hospital in Krakow.

Today we are going to talk about clinical trials in rare diseases. We have 7,000 rare diseases described and only 140 registered orphan drugs (please correct me if these figures are a little different). And rare diseases in clinical trials have their own difficult specificity, so let's tell you what it is.

The specificity is that we have rare or ultra-rare diseases, that is, diseases that are rare in the population. That is, we have such disease entities that we are even treating at the moment, such as Charcot-Marie disease in five patients. In the population, the incidence of just such disease entities is once in several million - one birth in several million births. And I want to emphasize that in order to do a clinical trial, we should have at least a few dozen to a few hundred or a few thousand patients. We can imagine that if we have in the Polish population, which is very roughly 10 percent of the population of the European Union (actually about 7.5 percent), we should have in European countries - a few hundred such patients at most. That is, we need to have a large worldwide study, if we have a few or a few hundred patients in general, to organize and conduct this study reliably.

But this research is going on in rare diseases.

Of course, only these are generally multicenter, international studies, that is, many countries have to participate. It is a matter of coordination and the right approach. But we must remember that we have different regulations, different insurance systems. That is, it is not easy. It is much easier to conduct a study with similar conditions, because clinical trials must be s...

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