Ed. note: This is the final post by Dr. Kim Hunter-Schaedle recapping the Conference on Clinical Research for Rare Diseases to see the first post please click here.
A major consideration for a rare disease clinical trial is that it may take a very long time to recruit all of the patients you need. During the time it takes to do a trial, you run the risk of the trial losing momentum; staff turnover, which is inevitable in any clinical research center; or depletion of your own eligible patient population. This is one reason why rare disease initiatives will often include a number of centers, so that this increases the chances of successful recruitment and advancing the trial more rapidly. However, adding more centers can also dilute out the expertise.
Another implication to be kept in mind from the outset is that if the trial is being supported in full or part by industry, they will have a focus on getting an outcome measure (does drug work or not?) as soon as possible. The funder should be made aware from the outset of the vagaries and potential delays of a rare disease clinical trial.
Finally – a word about conflicts of interest. The NIH began regulating conflicts of interest in1995, and federal interest in this area has increased since 2005. For rare diseases, conflicts can arise if an investigator has multiple roles in one trial –such as screening, selection and consenting of patients; and evaluation of data. In particular evaluating data must be done independently of other trial related activities. Today, most rare disease patients are heavily educated on their condition. Nevertheless, consenting patients for a trial is a complex issue as the clinician often has to go through a fairly complex form with the patient in a limited time frame.
