Innovative Trial Design to Speed Up FDA Approval
​[Posted on: Thursday, July 13, 2017]

Traditional clinical trials are designed to evaluate one drug in the treatment of one indication. Many patients are screened to find the few that perfectly match the inclusion criteria of the trial and rest are rejected. Screen failures could account for as high as half of the patients who initially showed interest in participating in a given trial.  What if you could design a study that matches practically every single patient who shows up to be screened for a trial, to a sub-study, each sub-study being based on a specific marker or other criteria, so that you not only include all possible patients in the trial but also evaluate multiple treatment options within the same trial. Such a Master protocol will maximize the recruitment and screening resources and allow testing of multiple smaller subset of a given disease or multiple indications for a given drug, thereby increasing the chances of finding specific treatment for every sub-set of the patient population. A given drug could treat multiple diseases for example a given cancer drug may be effective against multiple kinds of cancers. Similarly, there may be multiple drugs to treat the same disease each targeting a sub-type of the said disease such as once cancer having multiple treatment options each most effective against one sub-type compared to other subtypes. Master clinical protocols that can test a single drug for treating multiple diseases, or test multiple drugs to treat one disease with multiple drugs are increasing in popularity particularly for diseases with multiple presentations such as cancers and neurological diseases. And there is a strong support from FDA for such protocols. However, these protocols are far more complex and expensive compared to traditional trials, and require addressing conflict of interest issues if the multiple drugs being tested are owned by different companies. That is why most of the master clinical trials so far have been conducted with NIH or other government or non-profit sponsors as the lead.