Last week I attended the semi-annual meeting of the Gynecologic Oncology Group, the cooperative group focused on trials for gynecologic cancers. Over two days I was engaged in updates of ongoing clinical trials and discussion of new trial concepts for ovarian, cervix, endometrial, and other gynecologic cancers. We learned that we are entering a new era in clinical trials—gone are the days of doing a trial in gynecologic cancer using the "next best drug" because it worked in ____ cancer. No—the onus on investigators is to design new trials based on science. As my mentor and friend, Dr. Carol Aghajanian (chair of the Developmental Therapeutics Committee) put it: "No tissue, no marker, no target—no trial."

During GOG I also found myself considering the endpoints of clinical trials: should it be the response rate? Should it be the proportion of patients who have no tumor growth at six months? Or should it be overall survival? What about quality of life—could this ever be a primary endpoint in clinical trials? What about toxicity as an endpoint? Obviously, the endpoints in a trial depend on the trial itself—phase I studies are geared towards toxicity evaluations, phase II towards effectiveness, and phase III towards survival.

But in the same way that we as investigators need to ask "smarter" questions in the hopes of delivering tailored (i.e., "personalized") therapies, I was left wondering if we should also take a step back and consider the endpoints of our studies. Maybe "overall survival" is not sufficient—maybe it should be clarified by use of quality of life or, in our world of limited resources, cost-effectiveness. Then it occurred to me—maybe the endpoints should not be determined solely by a group of investigators in a conference hall in San Diego. Maybe it's time to have this dialogue with the public—beyond the National Cancer Institute, beyond the American Cancer Society, beyond ASCO. Maybe we should engage the public into a discussion of what endpoint is "meaningful."

When I came home from GOG, I found myself in clinic with a patient who had been diagnosed with recurrent ovarian cancer. She and her son (neither of them in medicine) came to the visit with information pulled off the Web. We ended up having a thoughtful and engaging discussion about completed clinical trials—study designs, patient eligibility, objectives,  and results. Yet, even after reviewing all of this data, I could not answer the question that came next: 

Patient: "That's all great, but what does that all mean to me?" 
ME: "Well, I think you could benefit from treatment as was shown in this trial. It met the endpoint it was designed to address—progression-free survival."
Patient: "But, I want my life to be more than just 'progression-free survival.' I want my life to be about enjoying my family, planting in my garden, going to the beach, not suffering, and being who I am right now. I don't care how 'long' I live—I care about being able to live well."

At that moment I understood the challenge we face in oncology. We are not only being called on to design "smarter" trials. Our patients, living in an electronic age where information is at the fingertips, are calling on us to provide "smarter" answers. 

It's a daunting task, but fortunately, I am surrounded by colleagues locally, nationally, and internationally who are up to the challenge, as am I.

Oh—in case you are wondering how I responded to my patient above—I gave an answer rooted by my fellowship at Memorial Sloan-Kettering and reinforced in my continued commitment to clinical trials, GOG, and ASCO:

"Often no better treatment is available to patients than clinical trials. I'd encourage you to consider this one—"