Tracking Patient Outcomes to Improve Clinical Trials
Clinical trials for brain and spine tumor patients usually test how well a drug is working to improve survival. Often, an increase in survival, as well as 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 impact survival.
Since the goal of any new treatment is to benefit the patient, it is important to include parameters that measure their clinical outcomes – 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 Deputy Chief Terri Armstrong, Ph.D., of NCI’s Center for Cancer Research, Neuro-Oncology Branch now investigates 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 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 scale and location of pain can be beneficial to determine if their pain is caused by something other than expected side effects of the trial treatment or newly developed pain that is unrelated to the new treatment.
- Difficulty Communicating - can span many variables, such as spoken language, writing, gestures, or hearing others.
- Cognitive Ability - can influence memory or concentration, changes in which are often recognized by the caregiver.
- Seizures - are a common symptom reported by patients prior to diagnosis and provide crucial information on patient function including details such as severity and frequency.
- Symptomatic Adverse Events - may partially overlap with disease symptoms 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 show weakness, especially if the disease is suspected to have spread.
- Role Functioning - refers to a person’s social, familial, and professional role and how this can change. Understanding how a patient feels and functions can impact how they feel and therefore, their disease state and recovery.
Several of the patient-report outcomes also present as symptoms prior to diagnosis and change as an individual undergoes surgery, treatment and clinical trials. A formal record of these parameters can help better define the 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.