Why RCTs? Part 5 continued

Learning and Changing in Response to RCTs… Challenges Remain

Mastectomies have become more and more openly discussed over the past decade, as celebrities have spoken about their experiences and more women are screened for breast cancer. During their lifetimes, 12% of women in the U.S. will develop invasive breast cancer, and the rate of women choosing mastectomies after a positive diagnosis rose from 2% in 1998 to 11% in 2011. Recently the number of women who have opted for double mastectomies has increased despite recent research showing that the procedure does not improve one’s chances of survival or quality of life.

This is the second installment of part 5 in a series called “Why RCTs?,” which explores experiences with and without the benefit of randomized study across disciplines . (See the first installment here). We can learn a lot from breakthroughs in the medical field, which underwent drastic change in the mid-twentieth century. During that period, clinical trials became more fundamental to the legitimacy and improvement of medicine in the United States. In the field of oncology alone, RCTs have transformed our knowledge base and decision-making. From Hormone Replacement Therapy (HRT) to mastectomies, new and reliable findings have enabled doctors to provide better and more complete information to their patients. However, challenges remain as the field builds on that progress.

A recent study published in the Journal of Clinical Oncology notes that patients who underwent mastectomies did not have experience outcomes than those opted against breast removal. Importantly, however, this study did not adopt an RCT methodology. Instead, it was in-depth analysis of 20 years of data on 100,000 women who had developed the earliest form of breast cancer (ductal carcinoma in situ, or DCIS), a majority of whom had lumpectomies or mastectomies. However, no large clinical trial has been conducted to compare the relative impact of different treatments. In fact, medical experts consider it unlikely that clinical trials will ever be done to study the effectiveness of mastectomies, due to: (1) the high survival rate for women with DCIS; (2) the length of time and case volume needed for randomization; and (3) ways that treatment might change during the study period.

There are always concerns about randomizing when a treatment potentially means the difference between life and death. Ethical issues are best addressed by how scarce the randomized resources is and whether we are in a state of equipoise regarding its benefits. Part 2 of this series explored how to address the scientific benefits of RCTs: how they can provide one might not even know is missing, and reveal critical issues with previous research methods. For example, the Women’s Health Initiative’s (WHI) RCT “changed abruptly” the use of hormone replacement therapy and is now not recommended as much for women as it used to be. Aren’t these types of situations where we most need evidence as to what works and what doesn’t? After a diagnosis of breast cancer, “nine times out of 10, the women are the ones who decide” what course of treatment they undergo. But how are women meant to make these choices if they don’t have the information to do so? And, to bring it back to the legal field, how are legal service providers meant to construct programs and provide legal aid if they do not have evidence showing what actually helps people?

Dr. Laura J. Esserman, a change maker in the field said of breast cancer screening and surgery, said of a breast-cancer screening RCT: “I’m asking everyone else to be randomized, so I’ll probably be randomized. I try to design trials that I would want to participate in.” The types of concerns we need to keep in mind when considering RCTs include how to construct an experiment ethically to find out much-needed answers to problems we care about. RCTs don’t have to be scary; in fact RCTs can open up a world of knowledge and shed light on incredibly important decision-making points. When diagnosed with breast cancer, should women automatically consider mastectomies the safest and best option? When constructing legal services, should providers spend money and resources on mailings focused on getting people to court to pair up with volunteer lawyers, or will online resources do the same job? With ever-scarce resources, we already impose artificial limits on who receives services. RCTs can provide a fairer way of distributing services, and one that will allow us to offer better services down the road.

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