By: US Head of Medical Affairs Oncology, Camille Hertzka and Head of US Advocacy and Alliance, Martha Orzechowski
Rapid scientific progress against cancer has introduced a new era of precision medicine and patient-centric care. To keep pace, the Food and Drug Administration (FDA) has actively engaged advocacy groups, professional medical organizations, and drug developers to integrate the patient voice into cancer clinical trials. Patient advocacy groups, such as the GO2 Foundation for Lung Cancer and LUNGevity Foundation, have partnered with companies like AstraZeneca, who have a long-standing history of innovation in clinical trial design, with more than 80 cancer trials employing patient-reported questionnaires to get a clearer picture of the patient experience.
As a bridge between cancer patients and the research community, patient advocacy groups are working together to advance the use of patient-reported outcome (PRO) data in cancer clinical trials. Research has shown that PRO results complement traditional cancer efficacy measures, such as a drug’s ability to decrease tumor size or prolong a patient’s life, and ultimately, support patient‐centered care.
Empowering Cancer Patients through Project Patient Voice
In an exciting development for the oncology community, the FDA launched a pilot website that provides, for the first time, public access to patient-reported adverse events (AEs) data from a cancer clinical trial.
The initiative, called Project Patient Voice, features a website with information about symptomatic AEs based on patient questionnaires. AstraZeneca is the inaugural partner to provide data for the program and was also the first company to use the National Cancer Institute’s new PRO-CTCAE™ questionnaire with participants in a Phase III clinical trial.
“Project Patient Voice is a fantastic first step in delivering information to patients, or their caregivers, to understand how they are going to feel and function when taking different therapeutic regimens. I applaud the FDA and AstraZeneca for their leadership in creating a public-facing portal that displays robust, consistent, and comparable data for patients and their healthcare providers to make informed decisions,” said Andrea Ferris, president and CEO, LUNGevity. “As it has been discussed at LUNGevity’s various Scientific and Clinical Roundtables, it is essential to distill information down to the most important elements that are relevant to patients, and display them in a way that can be understood and used for shared decision-making by patients and their treating physicians.
Project Patient Voice will be a valuable resource for cancer patients and physicians. Patients can provide important information about their experience with a medication during a cancer trial, such as the scope, intensity, and duration of symptoms over time. The questionnaire results are displayed visually on the website to make it more accessible for patients to see and understand. The data provide additional, complementary insights for healthcare providers to discuss options with their patients to help them make informed treatment decisions. This type of subjective data has rarely been included in a cancer drug’s product labeling, and therefore, was mostly unavailable in the public domain.
As a next step, the FDA plans to seek public feedback on the usefulness of the website’s content, design, and usability.
The PRO-CTCAE™ Questionnaire is a tool for measuring patient-reported adverse events (AEs) of cancer treatment during a clinical trial. It includes a library of 124 discrete items representing 78 AEs that are common in cancer clinical trials. Patients’ responses are scored on a scale from 0 to 4 (0=low, 4=high) to characterize the frequency, severity, and impact of treatment-related symptoms. The assessment of patient-reported symptomatic AEs can provide insights to complement clinician reporting of drug safety data included in the product labeling.
Putting Patients at the Center
With Project Patient Voice, the FDA’s Oncology Center of Excellence has developed a consistent source of patient-reported AE data collected during cancer trials. The program aligns with the missions of key patient advocacy groups such as the GO2 Foundation for Lung Cancer and LUNGevity Foundation: to put cancer patients at the heart of clinical research. Among one of their recent initiatives, the GO2 Foundation for Lung Cancer published a peer-reviewed article in “Health Expectations” on optimizing PRO trial design and implementation in cancer trials. Additionally, LUNGevity Foundation has partnered with the FDA to connect patients with public health experts for guidance on participating in cancer trials during the COVID-19 pandemic.
The key to advancing cancer treatments and improving patient outcomes lies within the patients themselves. Their input is more vital than ever with so many new treatments in the pipeline. I applaud the FDA and AstraZeneca for putting patients first as the key to accelerating discoveries,” said Bonnie J. Addario, lung cancer survivor and co-founder and chair, GO2 Foundation for Lung Cancer.
Harnessing the Power of PRO
All PRO stakeholders – from researchers to trial investigators to advocacy groups and drug-developers – are partners in improving the lives of cancer patients. As we continue toward this shared goal, let us remember to incorporate the patient’s voice and experience into every stage of cancer clinical research.