Reliable Patient-Reported Outcomes Data Informs Treatment Decisions

January 29, 2021, by Brittany Cordeiro, NCI-CONNECT Program Manager

Questionnaires that measure patients’ view of their health status or their perception of their experience while receiving care are called patient-reported outcomes. They play a significant role in helping health care providers make treatment decisions and researchers better understand brain and spine tumors. NCI-CONNECT uses a team of experts to design and develop questionnaires in web-based applications and then analyze the data collected.

“In order to have reliable, accurate, trustworthy results, you need reliable, accurate, and trustworthy data,” says Elizabeth Vera, mathematical statistician in the NCI Center for Cancer Research (CCR) Neuro-Oncology Branch. “While it is not the most exciting part of research, designing consistent, feasible data collection is important.”

Vera provides statistical analysis and guidance for data collection of patient-report outcomes. She works closely with programmer analysts who build and design the survey forms and software that captures clinical study data. 

Designing and Building Trustworthy Tools

Vera joined the Neuro-Oncology Branch in 2016 but has coordinated and managed data for brain and spine tumor patient studies for over 10 years. Her experience started with a study collecting outcomes data on a rare brain and spine tumor called ependymoma. The work led to the largest tissue repository of ependymoma—and at least 18 publications and further knowledge about this tumor type, including defining molecular subgroups. 

Vera’s experience makes her a key resource to help decide what questionnaires to provide to patients and which data to collect. Then, Yuti Gandhi, programmer analyst for CCR's Office of Information Technology, and her colleagues—led by Jason Levine, M.D., associate director of CCR's Office of Information Technology—design the data collection tools in the electronic capture system Scribe or in the database Labmatrix. They maintain both systems and modify them based on the needs of NCI-CONNECT studies.

“We test survey models to make sure they work based on these needs and requirements, and then we make them available to users,” says Gandhi. “The biggest accomplishment on this project is the development of an automated tool that allows researchers to perform further analysis on the clinical study data.”

This allows nurses to create and fill out a survey about the patient’s health status and experiences, and then sync the data with the Labmatrix database. Researchers are then able to perform further analysis on the data. Importantly, the analysis leads to more publications that provide patients and health care providers necessary information to manage care decisions.

“Gandhi and the rest of Dr. Levine’s team are very knowledgeable and creative. They help ensure our data collection is consistent, leading to a stronger statistical analysis and, ultimately, to trustworthy results,” says Vera.

Implications of Data Collection and Analysis

Patients with brain and spine tumors and health care providers who complete survey forms and questionnaires are describing symptoms, mood, general health status, and perceived cognitive symptoms. They also answer questions to help researchers understand the impact and risk factors associated with the disease. This information is used by doctors, along with the evaluation of the tumor tissue and clinical exam, to make recommendations related to treatment and clinical care. 

Good data collection and analysis also leads to publications that are trusted and utilized by neuro-oncology health care providers. These publications are especially important because NCI-CONNECT focuses on rare brain and spine tumors, which have limited treatment and care options. “The more options and educational information we can provide patients and health care professionals that is based on dependable data, the better,” says Vera.

Impact on the Patient  

NCI-CONNECT researchers and health care providers have learned a lot of valuable information from their study questionnaires. Topics like financial toxicity and employment have been prevalent for participants. People living with brain and spine tumors are often unable to return to work. The loss of income can be substantial for families and affect their care decisions.

Questionnaires also address symptoms and medical history. Knowing these types of data can help health care providers have appropriate conversations with patients, provide adequate resources, and provide the best treatment options.

Researchers are also studying risk factors for rare brain and spine tumors. Asking patients about their experiences, circumstances, and life prior to their diagnosis, such as environmental history and exposures, can guide new studies on which factors may lead to these rare diseases.

Questionnaires are also completed by physicians to address specifics about the patient’s tumor and treatment history. These questionnaires are paired with the tumor tissue analysis to help researchers study the tumors and design clinical trials.

“Any type of information that we can provide to patients with rare diseases can help them,” says Vera. “I am a data person. If I can use math to help someone feel better or feel more prepared for what they could experience when given a tumor diagnosis, then that makes me feel like I am helping in some small way.”