Clinical Trials: Does One Size Really Fit All?

Margaret Tempero
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Clinical trials in oncology have been instrumental in shaping care. Hundreds of new drugs and other therapeutic strategies have been generated to improve the lives of our patients. Promoting and executing carefully planned and designed clinical trials has been the cornerstone for improving care and for determining the best care. Sometimes we have triumphed and sometimes we have not, but we have always learned something.

As a result, many advocacy groups and professional societies, in their public outreach, stress the importance of participating in clinical trials for patients with cancer. Because of this, some have suggested that if you are not participating in a clinical trial, perhaps you are not getting the best care. Historically, I held that view too. Realistically, of course, there can’t be a trial for every condition, although that would be ideal. Furthermore, now that we’ve entered the genomic era, I am starting to question this philosophy.

Don’t get me wrong: I am not questioning the value of clinical trials, only our approach to educating the public about them. First, if healthcare providers stress that the best care is always a clinical trial, then that automatically implies that “standard-of-care” treatment is inferior care. We know that is not true. Treatment that is based on level 1 evidence is, by definition, outstanding care.

In addition, many of my patients, having heard this message, request enrollment to a clinical trial, and although we want to accommodate that request, it is not always possible. For example, perhaps they don’t meet eligibility criteria. That is often devastating news for them. I try not to use the words: “You are not eligible to participate,” because I don’t want to leave the impression that they somehow don’t measure up. I try to say instead, “The trials we have open right now are not a good fit for you,” hoping to shift the blame to us. It’s a small thing, but I hope it helps.

Finally, now that we have entered the genomic era, many patients walk in the door with germline testing and somatic molecular profiling already completed. Often these results provide a clue that there is a preferred choice for them among the already established treatments for their condition. For example, I recently saw a new patient who had just learned that he came from a family with BRCA1 mutations. He underwent testing and found he was a carrier. Because there was pancreatic cancer in the family, he asked for a CT scan, which showed pancreatic cancer metastatic to liver. He asked if we had a clinical trial he could participate in. I explained that we did, but that the chemotherapy backbone of the trial was gemcitabine and albumin-bound paclitaxel. Given the BRCA1 mutation, he had a higher likelihood of experiencing a response to a platinum-containing regimen, and I advised against the trial. I think these conversations are being had regularly around the world and that these observations inform and improve our care.

So, to conclude, I agree that major progress comes from clinical trials and that all patients should have access to trials. Participation is highly recommended when the trials fit. But maybe we should soften our messaging to the public just a little and reassure them that they can get great care on or off a clinical trial.


Margaret Tempero, MD, is a Professor of Medicine and Director of the UCSF Pancreas Center and editor-in-chief of JNCCN. Her research career has focused on pancreatic ductal adenocarcinoma, especially in the area of investigational therapeutics. Dr. Tempero has served on the ASCO Board of Directors and as ASCO President. She currently serves on the ASCO Conquer Cancer Foundation Board. She codirected the AACR/ASCO Methods in Clinical Cancer Research and taught this course and similar courses in Europe and Australia. She was founding Chair of the NCI Clinical Oncology Study Section and served as a member and Chair of the NCI Board of Scientific Counselors Subcommittee A. She is a member of the Scientific Steering Committee and Chair of the Clinical and Translational Study Section for the Cancer Prevention & Research Institute of Texas. She is or has been on the Scientific Advisory Boards of the Lustgarten Foundation, the Pancreatic Cancer Action Network, the V Foundation, The Alberta Canada Cancer Board, and the EORTC. She served as a member of the Oncology Drug Advisory Committee for the FDA. She has served as Deputy Director and Interim Director for the UNMC Eppley Cancer Center. She is Chief Emeritus of the Division of Medical Oncology at UCSF. She served as the founding Deputy Director and was later Director of Research Programs at the UCSF Helen Diller Family Comprehensive Cancer Center.

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