ASCO Connection

July 2017

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Daniel F. Hayes, MD, FACP, FASCO University of Michigan Comprehensive Cancer Center @hoosierdfh Except From Adverse Events: A Clinical Trialist's Thoughts on Reporting and Right-to-Try …One piece in USA Today titled "Reports of drugs' side effects soar" caught my attention. At first, I thought this report, written by Matthew Wynn and John Fauber, would be about some dangerous drug making a headline. In reality, it was an objectively written story of the improved adverse event (AE) reporting system instituted by the U.S. Food and Drug Administration (FDA) that permits better surveillance of AEs in the post-marketing environment. Some might lean on this report to raise the oft-repeated criticism that the FDA fails to protect the safety of our population by approving drugs sooner and faster than they should. Rather, in my opinion, it highlights the very narrow tightrope that FDA must walk, working as quickly as it can to get new therapeutics out to us, while ensuring that they are both safe and effective before they enter the marketplace. In many respects, the agency is damned if they do, and damned if they don't. Let's dig a bit deeper into this conundrum. Led by ASCO member Dr. Richard Pazdur, the Office of Hematology and Oncology Products, which has morphed into the Oncology Center of Excellence, has worked hard to streamline both the approval and the AE reporting systems. Indeed, over the last 3 years, FDA approved more than 40 new drugs or new indications for marketed drugs for cancer care. In each case, these drugs were proven to have exceeded a relatively high bar of efficacy, usually (although not always) demonstrated in well-designed prospective randomized trials. Like- wise, in each case, at least the major and most common toxicities of the new drug had been evaluated in these same trials. Further, as highlighted in Wynn and Fauber's article, FDA has worked hard to make post-marketing AE reporting easier and faster. Is this system perfect? No —but as Winston Churchill famously stated, "Democracy is the worst form of government, except for all the others." Indeed, ASCO has provided its fair share of advice, and continues to do so. For example, in 2014, led by Dr. Lee Ellis and a team of ASCO volunteers, the Society published a statement that reflected consensus expert opinion on the magnitude of benefit that should be demonstrated in cancer clinical trials to declare a statistically significant result to be clinically meaning- ful. One can argue that this bar was either too high or low, but at least it provided some guidance to clinical trialists on what level of activity we as clinicians felt was meaningful to patients. Others have argued that efficacy doesn't need to be demonstrated at all before drugs should be made available to patients with no standard Everyone Has a Story to Tell—Share Yours With ASCO Connection ASCO Connection welcomes guest blog posts authored by ASCO members on all aspects of oncology practice, research, and training. Contact editorial staff at to tell us your story and learn more about the submission process. 14 I 07.2017 Connected continued

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