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Clinical trials for rare diseases

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Published - 14.Dec.2015
Clinical trials registry eliminates any risks of possible selective reporting

When we compare clinical trials for rare diseases and clinical trials for non-rare diseases we can see that the participation of people in the first type has been said to be extremely low. A recent survey shows that out of 24,088 clinical trials, 2,759 (11, 5 %) of them were defined as rare disease trials in which under 50 individuals are indicated to demonstrate interest in taking part in one of them. In this regard, recruiters experience major difficulties in finding not only proper but enough number of volunteers for their studies. Why is that so? Affected by factors such as cost, time and pressure, fewer people express desire and readiness to dedicate part of their routines to lengthy and expensive research procedures that require traveling from one point to another.

In order to handle this lack of inclination and predisposition and to turn things around for the advantage of clinical studies, researchers and sponsors try to find alternative methods in conducting their projects. One such idea which has the goal to transform into a magnet for more people to take part in clinical trials for rare diseases and conditions is the performance of in-house clinical researches. The positive aspect of it would be the fact that patients could stay at their homes while clinical nurses would visit them to run simple medical procedures essential for the proper implementation of the trial. Offering the comfort of saving time and money is a beneficial undertaking for both participants and researchers – the first ones will be given an opportunity to contribute for the development of new drug at lower costs and shorter time while the second ones will receive more candidates.

Something else that will also help clinical organizations and institutions to solve the problem of not having enough participants is to search for patient support groups. Such groups can provide with locations of possible volunteers and can also help find clinical or research professionals as well as other current treatment centres.

 To summarize, a good clinical trial design is the one that is able to “attract” as many participants as possible. When it comes to studies for rare diseases, the number of people who are inclined to take part in one research or another is dropping each year. Attempting to increase the rates of interest and to make the participation of individuals more beneficial, researchers are pursuing new techniques and schemes. 

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