The challenges faced by suffers of the rare diseases can be explained. The non availability of the standard treatment or the availability of expensive treatments is making the sufferers undergo great hassle. Pharmaceutical companies and many government agencies have now opened the doors for sponsoring the rare diseases clinical trial which is not an easy task even the investigators will find is very difficult to make the way for the research and FDA approval as there is limited availability of resources, data and expert staff. The only away to bring out the desired success in the clinical trial is to Work with treating physicians, patients associations and patients’ families and brings the required patients on to the clinical trial.
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