Panel: Weighing the Risks of Randomized Controlled Trials and Alternatives
On June 21, 2017, Executive Director Holly Fernandez Lynch participated in a panel discussion on "Weighing the Risks of Randomized Controlled Trials and Alternatives," which was part of the conference "The Need to Accelerate Therapeutic Development - Must Randomized Controlled Trials Give Way?" hosted by the New York Academy of Sciences. From the conference page:
Randomized controlled trials (RCT) are frequently used as part of the clinical trial process, and are generally considered the gold standard for determining the efficacy and safety of an investigational product. As such, they have become a crucial part of the regulatory pathway whereby a new treatment can gain access to the market. While randomized controlled trials are widely considered valuable for their ability to present unbiased and consistent results, ethical concerns have been raised in recent years. For example, questions have surfaced regarding the ethics of placebo usage, especially if there are viable standards of care available; the length of time to complete development programs using traditional RCT paradigms, specifically when the patient population is small or the patient need for a treatment is immediate (e.g. the Ebola outbreak); or the potential of providing a treatment to the experimental group that may prove to be inferior than the standard treatment. In many situations, a case could be made for the need for alternatives to RCTs to speed development and bring treatments to patients in need more rapidly without compromising establishment of appropriate levels of efficacy and safety for registration.
To address these important issues, the NYU School of Medicine and the New York Academy of Sciences will present the two-day colloquium, The Need to Accelerate Therapeutic Development — Must Randomized Controlled Trials Give Way?, which will convene thought leaders who — in a public-facing dialogue — will discuss the ethical, legal, medical, scientific, safety, and economic issues pertaining to the design of clinical trials. Session topics will include the history and future of randomized controlled trials, the role of alternative trials, the ethical and scientific issues associated the randomized controlled trials and alternatives, a case study of a modified randomized controlled trial, and risk and liability associated with accelerated trials. Pitting randomized controlled trials against alternatives will inevitably result in a highly nuanced, ethically ambiguous discussion, and it could have profound effects on public health and the regulation of pharmaceuticals.
Watch video of the event here (panel begins at 52:00).bioethics clinical research health law policy holly fernandez lynch human subjects research pharmaceuticals public health regulation