In collaboration with leaders in the field, Dr. Terri Armstrong published a paper outlining the importance of tracking patient outcomes in clinical trials.
By Neuro-Oncology Branch Staff
March 6, 2020
Clinical trials for brain and spine tumor patients usually test how well a drug is working to improve survival. Oftentimes, an increase in survival and tumor stability (or shrinkage) are considered positive results.
However, people with brain and spine tumors often struggle with many functional limitations. They can also experience symptoms based on the location of their tumor. Moreover, research has shown that symptoms that prevent people from performing their daily activities can negatively affect survival.
Because the goal of any new treatment is to benefit the patient, it is important to include parameters that measure their clinical outcomes—such as questionnaires on symptoms, function, and health-related quality of life. Many neuro-oncology practitioners understand the importance of tracking such measures during a clinical trial, but there has been a lack of consensus on the optimal information to include.
A new study in Lancet Oncology by Terri Armstrong, Ph.D., deputy chief of the NCI Center for Cancer Research's Neuro-Oncology Branch (NOB), investigated how including specific functional and symptom data in clinical trials can help determine the benefit of a trial.
To develop a standard list of recommended outcomes, Dr. Armstrong and her co-authors combined input from patients and their families, literature, and clinical groups.
Recommended Patient-Reported Outcome Questions
On a scale of 1 to 10, this questionnaire measures the extent of the following symptoms and functions that the patient might be experiencing:
- Pain: Reporting the scale and location of pain can help determine if the pain is due something other than expected side effects of the trial treatment, or newly developed pain that is unrelated to the new treatment.
- Difficulty Communicating: This can span many variables, such as spoken language, writing, gestures, or hearing.
- Cognitive Ability: This includes changes in memory or concentration, which is often recognized by the caregiver.
- Seizures: This is a common symptom reported by patients prior to diagnosis and provides crucial information on patient function.
- Symptomatic Adverse Events: These may partially overlap with disease symptoms and are important to track to ensure efficacy and maximal safety.
- Physical Function: Changes in performing tasks—such as walking or participation in daily activities—can indicate weakness, especially if the disease is suspected to have spread.
- Role Functioning: This refers to a person’s social, familial, and professional role. How a patient feels and functions can affect disease state and recovery.
Several of the patient-report outcomes also present as symptoms prior to diagnosis and can change as an individual undergoes surgery, treatment, and clinical trials. A formal record of these parameters can help better define quality of life and treatment burden.
Looking ahead, the authors outline that the next steps are to formally establish these outcomes in clinical trials. Incorporating patient outcomes data into all clinical trials will require significant cooperation from institutions, sponsors, physicians, and the patient themselves.