“PRO Domains of Interest”
Patients were asked to report at both data collection times any emotional, physical, and social symptoms they were experiencing, as well as their general quality of life. All of the questions were framed around symptoms that would be relevant to their forthcoming treatment, but patients were unaware of this when they filled out the survey for the first time. What researchers were looking for in both groups was any change in PROs between the first and second time they answered the questions.
Ultimately, when posttreatment answers from patients in the open-label trial were compared with those in the double-blind trial, the investigators determined that there were no significant differences in reporting between the arms. Patient knowledge of the treatment that was administered did not appear to bias PROs.1
This is good news for open-label cancer drug trials that include PROs as domains of interest, and Dr Basch said that he believes the FDA ultimately values these patient-reported details.
“Some have objected to [the FDA’s] rejection of quality of life as a construct in drug approvals, which is inconsistent with the European Medicines Agency,” he pointed out. “But overall, I feel the FDA has done much to advance the field of PROs in oncology regulatory science.”
Not everything is smooth sailing, though. In the second study presented at the same conference, the researchers (some of whom also participated in the first PROs study discussed in this overview), showed evidence that using PROs to monitor patient deterioration during trials is not always reliable.2
While it could be logical to assume that patients reporting deterioration in physical function would die during the trial, the researchers showed that this is not actually always the case: some patients are capable of later returning to a state of non-deterioration. They cautioned that a reversal or pause in deterioration is not necessarily an indicator that the drug is suddenly working again. Instead, the improvement in physical function could potentially be attributed to “treatment interruption, dose modification, or other clinical interventions” that are not adequately disclosed along with the PROs.
When questioned about what type of systemic change would be required to make PROs more useful in drug trials, Dr Basch said that “input from regulators would be optimal, to minimize missing data and to maximize meaningfulness.”
“If PROs were systematically collected during routine cancer care, that would both improve quality of care and provide a trove of data for analyses,” he added.
- Roydhouse J, Mishra-Kalyani P, Bhatnagar V, King-Kallimanis B, Kluetz P. Presented at: 26th Annual Conference of the International Society for Quality of Life Research; October 20-23, 2019: San Diego, CA. Does knowledge of treatment assignment affect patient-reported outcomes? An evaluation of open-label bias in multiple myeloma. Qual Life Res. 2019;28(Suppl 1):1-190. Abstract B204.5. doi: 10.1007/s11136-019-02257-y
- Roydhouse J, Lee H, Cheng J, Bloomquist E, Bhatnagar V, King-Kallimanis B, Kluetz P. Presented at: 26th Annual Conference of the International Society for Quality of Life Research; October 20-23, 2019: San Diego, CA. 26th Annual Conference of the International Society for Quality of Life Research. Evaluating time to physical function deterioration in multiple myeloma. Qual Life Res. 2019;28(Suppl 1):1-190. Abstract 303.4. doi: 10.1007/s11136-019-02257-y