Putting Patients First: My Journey in Advocacy
The most important lesson I have drawn in 2 decades is this: Although science remains the touchstone in our long fight together, each patient individually matters.— Ellen V. Sigal, PhD
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When I lost my only sister to breast cancer in 1986, patients like her had devastatingly few choices. Over the intervening decades, sustained commitment to biomedical research at the National Institutes of Health (NIH) and major technologic advances have led to transformative changes in cancer research and treatment. Advanced therapies and protocols have boosted survival rates. New drugs offer new hope. And, though more difficult to quantify, patient care and communication have also improved. Once treated as passive recipients of care, patients are now active participants.
The end of my sister’s fight with cancer was the start of mine. In 1996, joined by my long-time friend Marlene Malek, I founded Friends of Cancer Research (Friends), an advocacy organization, out of a personal sense of loss and urgency. I fully committed myself to overcoming obstacles to defeating cancer. I am deeply proud of the contributions we have made since then, including raising awareness of this disease and cementing partnerships to create a more open dialogue among both public and private sectors, driving legislative reform, and crafting public policy. What started as a modest one-person effort has grown into an institution that has positively impacted the trajectory of cancer research and demonstrated the power of patient voices.
And, as I have watched research and therapy evolve, so, too, have I observed shifts in advocacy. The most important lesson I have drawn in 2 decades is this: Although science remains the touchstone in our long fight together, each patient individually matters.
Launch of an Advocacy Movement
Friends initially focused on raising awareness and connecting with those affected by cancer the most. We hosted town halls across the country to learn what patients and practitioners dealing with cancer needed. In those early days, as I digested the stories of the amazing people I met, I resolved to always prioritize patients in my advocacy work.
As the organization expanded, so, too, did our mission. We identified and supported high-impact policy interventions, bringing together stakeholders to speed cutting-edge treatments to patients. To this end, we successfully helped to create two new models of collaboration: the Lung-MAP clinical trial model and the U.S. Food and Drug Administration’s (FDA’s) Breakthrough Therapy designation.
As many of you know, Lung-MAP is a collaborative clinical trial model that pairs patients with substudies of new treatments through genomic screening. The result is that patients receive access to treatments matched to the genetics of their tumors, researchers find the most appropriate subjects for subtrials, and providers and scientists share troves of information that otherwise would not be available to them.
The FDA’s Breakthrough Therapy designation, which expedites the development and review of drugs, is similarly promising. Working with bipartisan congressional leadership, as well as with partners in all sectors, Friends took “breakthrough therapies” from concept and bipartisan legislative solution to a tool in full use by the FDA to expedite the development of treatments that demonstrate a substantial improvement over existing therapies for serious or life-threatening diseases. The process removes barriers to development for the drugs that show the most potential at the earliest stages, allowing patients to receive their benefits more quickly. Rather than stymie innovation, regulation now serves as a catalyst.
We have continued to drive collaboration with regulators through opportunities like the White House Cancer Moonshot Blue Ribbon Panel, on which I have the privilege of serving. Friends recognized the need for research and development to transcend intra-agency boundaries through integrated planning, innovation, and decision-making. That’s why we teamed with other organizations to call on the FDA to streamline its cancer expertise by creating a consolidated Oncology Center of Excellence to house biologic, drug, and medical device discussions under the same roof. The FDA accepted our proposal, creating a center that brings together experts from various disciplines, speeds the review of products, and elevates the patient voice in investigating new therapies.
Impact of Patients in Innovating Research
Through these initiatives, I have witnessed how patient needs and concerns can shape the trajectory of research. I have learned that their judgment and input remain vital as we look ahead to a new era in science.
As Vice President Joe Biden observed, we stand today at an inflection point in the fight against cancer. As we move forward, we must remember that patients are equal partners in the quest to find cures for cancer and that they matter beyond medical outcomes—they matter as individuals and as human beings. We cannot beat cancer without their partnership.
Collaboration is the hallmark of the National Cancer Moonshot Initiative. To be successful, industry, regulators, doctors, and institutions must talk to each another, with gene sequences or anonymous patient data providing a common language. And they must partner with patients. Patient participation, choice, and dignity are, in parallel with scientific innovation, vital to overcoming the challenges of cancer.
So what does partnering with patients truly mean in practice? It means engaging and involving patients in their own care, because they deserve the opportunity to make informed decisions. Partnering with patients means recognizing that quality of life matters massively; illness should not deprive patients of the ability to make crucial decisions about how they live.
Above all, partnership means seeing each patient, no matter the prognosis, treatment, or institution, as a human being with unique needs and motivations, both physically and emotionally. We should engage patients beyond checklists and charts. We should engage them on a human level.
I will never forget my sister confronting her doctor during her treatment. He was a capable physician, but emotionally ungenerous. One day, she presented him with a photo of her then 4-year-old daughter, Jillian, imploring him to look closely at her photograph. My sister wanted him to recognize that she, his patient, was a human being—a mother with everything at stake. In that moment, she made explicit what all doctors fundamentally understand but sometimes forget: our common humanity.
As we look ahead to the next 20 years, I am encouraged by the great potential for rapid improvement in oncology care. But advancements must come through a renewed focus on patients. The promise of immunotherapy brightens the horizon as CRISPR (clustered regularly interspaced short palindromic repeats) gene editing and other technologies open new worlds of risks as well as durable benefits. Novel therapies also pose challenges, as understanding and communicating options becomes more difficult and the information volume grows overwhelming in this burgeoning age of precision medicine.
But such concerns should not temper the exuberance of hope. If the patient voice remains prominent throughout these next challenges and advancements, I am confident that doctors and patients can team up to meet the potential obstacles ahead and realize the tremendous potential of this new paradigm.
Standing Together to Conquer Cancer
I am deeply grateful to the physicians, nurses, and caregivers whose contributions we too often take for granted. You deserve praise for your indefatigable efforts, especially as you are stretched ever thinner. Thank you for being our bulwark against hopelessness and helplessness. As I gear up for the fight ahead, I often think of Vice President Biden’s reminder that, for cancer patients, every single day matters. This essential truth should not instill fear but inspire fortitude. Research, medicine, and advocacy can achieve so much through cooperation around a mutual vision. I look forward to pushing forward with the utmost urgency on the noble task at hand. ■
Disclosure: Dr. Sigal reported no potential conflicts of interest.