More Than Numbers
We are living in the midst of a revolution in cancer treatment. Over the past 20 years, we’ve seen significant advancements in cancer research, with medicines helping people live with cancers that were once death sentences. Today, doctors routinely use treatments such as targeted medicines and immunotherapies designed to address the underlying biology of cancer or activate the immune system in the fight against disease.
While we now know more about the science of cancer and have more cancer medicines than ever before, we still have little information about the treatment burden – how the side effects of a treatment can affect a patient’s overall quality of life.
"In cancer trials, physicians may underreport their patients’ side-effects by as much as half."
Traditionally, clinical trials are designed to collect data from tests and measures that are essential to understanding a medicine’s safety and efficacy profile. But they rarely ask patients for first-hand, comprehensive information about what it’s like to undergo treatment. In cancer trials, physicians may underreport their patients’ side-effects by as much as half.1 This is a tremendous lost opportunity. Who better to provide information about side effects and other adverse events related to their disease and treatment than the people receiving the treatments? The measure of these patient-reported outcomes, or PROs, could provide more comprehensive information about a medicine and should be printed on medicine labels and in other places where physicians and patients can use them to make better-informed treatment choices. Understanding these PROs and the potential treatment burden could help patients work with their doctors to make informed decisions about which medicine might be best for them and their families.
Patients and their doctors need access to this kind of patient-reported evidence. To get PROs in medicine labels and in front of doctors and patients, we must have a standardized way to record them. Such a tool exists: the National Cancer Institute’s (NCI) Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). This is essentially a questionnaire about 78 common adverse events that people with cancer are known to experience with various treatments. PRO-CTCAE poses its questions in patient-friendly language – for example, it asks about mouth sores and hives, not in technical terms like mucositis and urticaria.
The PRO-CTCAE is a valuable resource that has been embraced by many researchers and endorsed by former Vice President Joe Biden’s Cancer Moonshot. However, discussions at the November 2015 Clinical Cancer Meeting hosted by Friends of Cancer Research, the FDA and the Brookings Institute highlighted some major barriers to the broad use of PRO-CTCAE. One major obstacle: it is available only in English, Spanish, Danish, and German. Given that many pivotal cancer trials are conducted in non-English speaking countries, this tool is not included in many key clinical trials that could generate patient-relevant data and help inform oncologists and their patients.
To accelerate the collection and inclusion of these data in labeling, Genentech is working with others in the industry to translate the PRO-CTCAE into approximately 35 languages. As these translations are completed, they will be transferred to NCI and distributed globally. This could dramatically accelerate progress in the collection of patient-reported outcomes, so that in the future, patients will be able to look at a medication label and clearly understand the impact of potential side effects over the course of therapy, allowing patients and their doctors to make the most appropriate choice for them.
A successful clinical trial means more than collecting vital data. Understanding the treatment experience and impact on the quality of life of a person with cancer is as important as a survival statistic or how much a tumor shrinks. We need to make sure that we are collecting as much information as possible so that doctors can educate their patients with the most comprehensive view of a medicine and how it may affect them.
Genentech is currently utilizing the PRO-CTCAE tool in two clinical studies, which will give us data on more than 1,000 patients within four years. If cancer researchers around the world implement these translations of PRO-CTCAE into more clinical trials to assess patients’ experiences in a standardized way, together we can work to ensure that patients get all of the information they need to make one of the biggest decisions of their life.