Working to Improve Cancer Care for Patients

Cancer, for so many, changes lives in a devastating way. Over 20 years ago, my sister lost her battle with cancer, leaving her family and children behind. Through mourning her, I became committed to doing anything I could to prevent another family from having to experience such a terrible loss.

From that point forward, it was time to take a stand. In 1996, with my colleague and dear friend Marlene Malek, I co-founded Friends of Cancer Research (Friends). Friends drives collaboration among partners from every healthcare sector to power advances in science, policy, and regulation that speed lifesaving treatments to patients.

We created the organization with the goal of helping patients gain access to the therapies they need, regardless of institutional barriers. After 20 years, I can say that we have made considerable progress, but there is still much more work to be done. National Cancer Survivors Day is an opportunity to reflect on how we can further improve cancer care.

The patient voice is one of the most important perspectives in drug development, yet it often goes unheard. While medical care and advances in oncology continue to provide new and exciting treatments, patients need more from the drug development process to ensure they are receiving the most appropriate and effective treatment. At Friends and PCORI, on whose Board of Governors I serve, we incorporate the patient voice in all of our work in order to improve the status quo and ensure that patients have access to the safe and effective therapies they need.

At Friends, through partnerships, science, and solutions, we have seen real outcomes for patients. During our annual meeting in 2011, we proposed an expedited regulatory pathway in cases where preliminary evidence for a new drug intended to treat a serious or life-threatening disease shows a substantial improvement over existing therapies. The next year, Congress approved legislation creating the Breakthrough Therapy Designation, which ushered in a new era of more quickly providing important treatments to patients. Now, four years later, the program has seen upwards of 300 requests, more than 100 designations granted, and more than 40 of those drugs approved by the FDA.

In addition, we revolutionized precision medicine in 2014 with a new model for clinical trials. Our ongoing Lung-MAP clinical trial is efficiently studying squamous non-small cell lung cancer. The trial is based on a multi-stakeholder partnership that includes five pharmaceutical companies, the Foundation for the National Institutes of Health, the National Cancer Institute, SWOG, and other partners. Lung-MAP, now open at more than 700 sites across the United States, offers patients straightforward access to a precision medicine–based clinical trial. Lung-MAP is focused on squamous cell lung cancer because it is common and hard to treat. There are few effective treatments, particularly for patients who don’t respond to chemotherapy. Lung-MAP’s innovative approach improves a patient’s likelihood of receiving a drug targeted at the genetic profile of his or her particular tumor, while allowing for new therapies in development to be added as the trial progresses.

Evidence to Guide Patient Choices

PCORI is also helping patients receive more-effective care, through funding research to give patients and those who care for them the evidence they need to make better-informed choices. Patients can have difficulties not only in accessing new therapies but also in making decisions about the available, often complex, tests and treatments. After all, patients, their families, and clinicians may face a wide range of difficult choices. Perhaps nowhere is this truer than in cancer with the continuing development of new diagnostic tests and treatments, such as immunotherapies, proton therapy, and combination therapies.

PCORI is addressing these concerns by funding important patient-centered clinical effectiveness research. In my role as a PCORI Board member and a member of its Science Oversight Committee, I, along with others, am working to identify and fund comparative effectiveness studies that will produce patient-centered, high-quality, timely, usable, and trustworthy results.

Despite the many healthcare innovations we hear about daily, the pace at which discoveries can be turned into widely available, potentially lifesaving therapies is far too slow for far too many patients. There is no doubt that more must be done to speed the development and delivery of evidence-based treatments for cancer and many other diseases—without compromising safety. At the same time, there are also too many gaps in the evidence needed to reliably help patients and clinicians make challenging decisions about care for the many conditions, including cancer, that impose a high burden on patients, their families, and the healthcare system.

PCORI and Friends both work to give patients the resources and opportunities to take advantage of today’s science, innovation, and collaborations. We have an opportunity as advocates, researchers, scientists, and clinicians to put patient concerns, values, safety, and outcomes first, and we will continue to work every day to ensure that this happens.