Self-reported data on symptoms and overall quality of life during a drug trial can be instrumental in directing the proper resources and supportive care to a patient.
Patient-reported outcomes, or PROs, are becoming more commonly used as secondary endpoints in cancer clinical trials because the data derived from these questionnaires can be useful in understanding tolerability and toxicity of treatments and the long-term effects they have on patients.
Yet, only 20.7% of phase 3 adolescent and young adult (AYA) cancer trials included a PRO endpoint. Only one of those revealed the PRO data. That's according to new research from MD Anderson Cancer Center, Duke University School of Medicine and other groups that analyzed 87 phase 3 therapeutic trials in AYA cancer patients.
The percent of trials using a PRO endpoint barely increased from the first half of that period to the second half, inching upward from 18.6% to 22.7%, the researchers reported in Science Direct last week.
PROs include responses about symptoms experienced by patients, their perceived physical, emotional and social health, and functional outcomes as well as health-related quality of life. That last one entails the patient's overall physical, mental and social well-being, or the impact the disease and ensuing treatments have on a patient's daily functioning.
With PRO data in hand, supportive care and more direct resources can be given to patients with cancer, the researchers said. This is the first such study to look at PRO inclusions in AYA clinical trials, the researchers claimed.
The researchers, funded by the National Cancer Institute (NCI), looked at studies from 2007 to July 30, 2020, using the federal trial database, ClinicalTrials.gov. The study focused on cancer types common among AYA patients, including germ cell tumors, Hodgkin lymphoma, acute lymphoblastic leukemia, sarcomas and non-Hodgkin lymphoma. The researchers focused on trials that did not exclude patients aged 15 to 39 years.
This age group is difficult to assess because of the lack of AYA cancer patients who enroll in clinical trials. Some studies estimate a mere 2% to 14% of AYAs take part in NCI-supported trials, the researchers said, which is "significantly lower" than childhood and adult patient groups.
Because of a lack of AYA-targeted trials, paucity of physician awareness of available trials and barriers to access, enrollment is lower among this group. This makes it even more critical to get as many of the PRO data as possible, the researchers said.
AYA cancer patients have a greater than 80% improvement in five-year survival, which outpaces childhood and adult numbers. This means there's an increased length of life. Given the extended life, health-related quality of life data are even more important, the researchers said, to informing care intervention, better symptom control, patient satisfaction and care during survivorship.
For cancers with worse survival rates in AYAs, the PRO data would help with understanding symptom burden and treatment impact, the researchers concluded.