ASCO Connection

July 2017

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treatment options. Dating back a decade and fostered by the Abigail Alliance, the Goldwater Institute, and other advocacy groups, many have called for a "right-to-try" policy. Such a policy would allow patients who have incurable cancer to have access to any drug that looks good in preclinical studies and that has gone through phase I clinical trials. Such an approach has broad emotional appeal, and right-to-try laws have now been passed in 33 states. Should emotion override science? Personally, I think not, but I can be accused of having a self-interest in such an opinion, since I have built a career as a clinical trialist. I do, in fact, hear and understand the argument of, "What do they have to lose?" Nonetheless, such laws concern me, and many of our colleagues, for several reasons. First, such a policy does, indeed, undercut the very nature of clinical trials that determine if the drug has sufficient evidence of activity to justify its being taken forward into larger, more definitive studies. In fact, the overall response rate in phase I trials, which is the circum- stance in which many of the right-to-try patients would find themselves, is typically less than 10% and most drugs that enter phase I testing never make it to mar- ket. In other words, these agents looked good but failed to deliver. Second, and more important, the toxicities identified in phase I trials, which typically include a small num- ber of carefully selected patients, are usually the most common and dramatic for a given drug. Less common adverse events, which may be either major or just annoying, are usually identified in later, larger trials and in post-marketing surveillance. Thus, in a right-to-try situation, a physician is offering a drug to a patient for whom quality of life is often the most important aspect of their circumstance, and about which the physician knows little or nothing of how it may affect them. Finally, who is to pay for these? Should we ask third- party payers to support the use of expensive drugs for which activity is unclear? Should we ask patients to pay out of pocket —a situation that will either lead to severe financial hardship or focus right-to-try drugs in a small group of wealthy individuals? Should we ask pharma- ceutical companies to provide them for free? None of these is a satisfactory solution. ASCO has supported FDA's efforts to streamline expanded access to investigational drugs, including requirements that an IRB review the circumstances and that the patient sign an IRB-approved consent form. Further, expanded access policies require that FDA has reviewed the request. Indeed, FDA has worked on streamlining this request process to make it as fast as possible, and it has approved over 99% of them. What's more, on a societal benefit level, FDA requires the physi- cian to submit a minimum amount of AE data from an expanded access program, so that future patients have an even better idea of what might be in store. ASCO released a Position Statement on Access to Investiga- tional Drugs emphasizing these principles. I recognize this train has left the station, but I fear for the unintended consequences of these laws and for our patients' well-being. I hope with time the expanded access program can be enhanced. In this case, patients who are justifiably desperate can receive exciting new drugs while we ensure their safety, and while we learn something for future individuals afflicted with cancer…. Find full post and references online at I 15

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