Clinical trials and research

The regi​stry supports research into rare diseases and the development of orphan drugs. For example, patients with very specific rare diseases are sought for the development of new treatments. Recruitment for these clinical trials is therefore often a very slow process. The registry will help to speed up this search, thus promoting the development of new treatments.

What is a clinical trial?
A clinical trial is part of the scientific research  to develop new drugs. Extensive studies need to show that a new product or treatment is effective and safe before they can be used. More specifically, a clinical trial is a study where a potential drug is tested in humans: first in healthy subjects, and later in patients with the disease for which the drug is being developed.
Extensive research will always take place in the laboratory and the drug will first be tested on laboratory animals before a clinical trial is undertaken in humans.

Why is it worthwhile taking part in a clinical trial?
Without clinical trials there would be no progress in medicine. Patients' participation in clinical trials therefore contributes towards this progress.
What's more, by taking part patients may in some cases benefit from gaining access to certain new drugs that are not yet available on the market. This could be particularly important if there is no other treatment available.
Patients undergo close medical monitoring while taking part in clinical trials.

What role does the registry play in this?
The Central Registry of Rare Diseases facilitates the search for suitable patients for taking part in clinical trials.
When patients with a particular rare disease are sought for a clinical trial, it is often difficult for researchers to find patients to take part in these trials due to the rare nature of the disease. To locate these individuals the researchers look for specialists who have knowledge of a particular rare disease, and who therefore treat patients with this rare disease.
The registry can simplify this search, by making it easier to find out where patients are treated for a particular diagnosis.
The registry makes it possible for more patients to be informed about clinical trials. Faster recruitment for clinical trials will help to advance research into treatment of rare diseases.

Will the registry or a researcher contact a patient directly?
No. The registry does not contain any recognisable identification details or contact details, so patients will never be contacted directly via the registry. Contact will always take place via the physician.
The registry can act as an intermediary by contacting a specific physician with the message that a specific patient may be eligible for a particular clinical trial, based on the data available in the registry (diagnosis, gender, age etc.). During the secure transmission of this message, the encoded identification details available to the researchers are decoded. This means that only the physician can see the identity of the patient in question.
The normal procedure will then always be followed, as is also the case without the intervention of the registry. The physician will look into whether the patients are eligible to take part in the clinical trial and, if so, contact them. The patient will then receive extensive information about the clinical trial and decide whether or not to take part.