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CRITical Thinking is a blog written by staff, directors, and friends of the Collaboration for Research Integrity and Transparency (CRIT), a joint program of Yale Law School, Yale School of Public Health, and Yale School of Medicine. CRIT's mission is to promote health by improving the integrity and transparency of biomedical and clinical research.

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Patient Advocacy Must Be Rooted in Evidence and Science Literacy to Make a Real Difference

August 31, 2017

In June 2017, Yale CRIT hosted an international conference titled “Ensuring Safety, Efficacy, and Access to Medical Products in the Age of Global Deregulation.” The following blogpost is the fifth installment of a blog series with commentaries from the conference participants. The views and opinions expressed in this blogpost are those of the author and do not necessarily reflect the position of Yale CRIT. For more blogposts related to this series, see here or click the tag “YaleCRIT17” below.

An informed patient advocacy perspective is vital to ensuring the integrity and transparency of the research enterprise. As a coalition of diverse grassroots groups from across the country, the National Breast Cancer Coalition (NBCC) helps fill that role. Its mission is to end breast cancer through action and advocacy. NBCC is about system change, not direct services or individual needs. While access to effective, safe therapies is key to saving lives, much effort is spent chasing interventions that are neither.

The statistics tell part of the story. An estimated 20–30% of patients diagnosed with early-stage disease will eventually have metastatic disease, for which there is no cure [1]. Metastatic patients have an approximate median survival of 3 years and at the current rate of progress, this will be 3.6 years by 2021 [2]. All patients, including those who will never get lethal cancer, are treated with interventions that are toxic and often life threatening.

This is the backdrop against which many demand access to unproven therapies. If the goal is to end breast cancer and death from the disease, it is important to be clear: the current drugs do not work. Clinical trials for cancer drugs are usually not designed to evaluate overall survival or quality of life, and most treatment options are approved on surrogate endpoints that do not correlate to overall survival [3]. Yet, millions of dollars are spent on marketing these drugs, hype is often based on nothing more than an industry or academic institution press release, and the prices of the drugs are extremely high. Rather than protest the situation, too often advocates disregard evidence and demand access, earlier and earlier in the evidence process.

In a world where patients are afraid or out of treatment options, it is easy to lose sight of the goal of obtaining effective, non-toxic, and affordable interventions that can prevent lethal disease or cure it. Expanded access or compassionate use policies undermine the clinical trials system and the principle of evidence-based medicine. These policies create a misleading atmosphere that these drugs are worth fighting for at any cost. We need to focus on clinical trials that may result in high-level evidence of real benefit for patients.

NBCC’s solution is to train and educate advocates that represent a constituency and demand that they be part of the investigative, scientific, and policy review processes, at every level. One NBCC program, Project LEAD, is an intensive science training course, giving advocates skills and knowledge needed to critically analyze research, collaborate with scientists and help set priorities. We help advocates understand the problems with access to investigational drugs outside of clinical trials, and we speak out against legislation that may compromise the research process, including Right-to-Try Laws. If there are expanded access programs, we work to make certain they are fair and require all data to be added to current scientific knowledge. In order for breast cancer patients to achieve real benefit, health authorities, the government, and the scientific community need to focus efforts on requiring overall survival for all new drug approvals moving forward.

Fran Visco is the president of the National Breast Cancer Coalition (NBCC).

  1. Harris JR, Lippman ME, Morrow M., et al. (Eds.). 2000. Diseases of the Breast, 2nd ed. Philadelphia: J.B. Lippincott, Williams & Wilkins.
  2. PHARMA: Decision Resources 2012 Oncology Patient Flow Model: Breast Cancer.
  3. Prasad V, Kim C, Burotto M, et al. The Strength of Association Between Surrogate End Points and Survival in Oncology. A Systematic Review of Trial-Level Meta-analyses. JAMA Intern Med. 2015;175(8):1389-1398.