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Prior to initiating a trial with ED sites, three factors must be considered in order to achieve success.
Conducting clinical trials in the emergency department (ED) setting presents unique challenges for all companies, but especially for smaller biotechs feeling the constraints of funding and time. This might partly explain why the current standard of care to treat acute exacerbations of asthma (AEA), for example, hasn't changed in more than 20 years. Prior to initiating a trial with ED sites, three factors must be thoroughly considered in order to achieve success: investigators' unique needs, study timing, and enrollment patterns.
Yuichi Iwaki, MD, PhD
When a patient's life is on the line, the luxury of time is not available for an elaborate evaluation. ED physicians want to be especially comfortable with the safety and efficacy of a therapy since there are limited follow-up visits compared to a trial in the typical clinical setting. Therefore it is vital to conduct early stage studies to accumulate as much data as possible.
In addition to safety and efficacy data, it is also beneficial to show other pharmacoeconomic endpoints, such as reduction in hospital stays, which help make a compelling case for encouraging patient enrollment in the trial.
In addition to ensuring that ED physicians are comfortable with the investigational therapy, the timeframe for treatment should closely resemble a real-world scenario. For example, if an endpoint is reduction in hospitalizations after five hours, but the ED physicians typically decide to admit much earlier, then this might inhibit a clinically beneficial improvement outcome.
To maximize success, design the trial to ensure that the trial endpoints match the real-world decision points. Following this match rule will not only help get to the next stage of clinical development, but also, if everything else falls into place, commercialization.
Finally, when gauging enrollment patterns, it is important to understand the underlying patient population and other factors, such as seasonality. If a condition, such as AEA, is more prevalent during the winter, for example, then sites need to be prepared to be busier during the cold months—and have patience if enrollment is not as high as expected during the warmer months.
MediciNova recently completed enrollment in a Phase II clinical trial evaluating the safety and efficacy of a drug for treatment of AEA. A total of 176 patients suffering from AEA in the ED who were not promptly responding to standard pharmacotherapy were enrolled. In addition to the inherent challenges of the ED setting, another challenge presented itself: How to measure AEA patient progress in an organized way, as this was not standardized. Through careful advance planning, the company developed a measurement and validated it, so it could evaluate the predicted change in lung function from baseline.
The smooth operation of this trial has hinged in part on careful attention to the considerations such as those noted above, and will help MediciNova plan its Phase III clinical program, in which it will determine if the inclusion/exclusion criteria can be broadened to support the goal of commercialization.
Considering these factors could benefit every company, especially smaller ones, that wish to test their therapies in the emergency setting while minimizing financial expense and time.
Yuichi Iwaki, MD, PhD, Founder, President and Chief Executive Officer MediciNova, Inc. E-mail: firstname.lastname@example.org.