A few days ago @DrYukselUrun shared on X the ORR ( objective response Rate) and CRR (Complete Response Rate and OS (overall survival) for the Phase 3 CLEAR trial ( combo of Lenvatinib and Pembrolizumab in advanced renal cell carcinoma).
In response, @StrijbosMichiel posted "Most impressive results from the CLEAR trial: the percentage of patients that have to endure grade 3-4 toxicities....82.3% in the 2021 NEJM publication. Yes you live longer....but at a very very hefty price. "
Deb Maskens (@DebMaskens)
a kidney cancer patient advocate posted: "Please do not post Efficacy numbers without posting what is equally important to patients: Quality of Life! Not just how long we live, but HOW we live. TY" (my emphasis)
What followed was an interesting discussion about QOL when testing new treatments and how they are measured and reported. What is tolerable for one patient may not be tolerable for another. The fatigue one patient experiences may mean they feel tired for another it may mean they are unable to move out of bed or stand to take a shower. Trials will report the AE's (Adverse Effects) as recorded by the clinicians and investigators or another person involved in the study. Occasionally patient reported outcomes (PROs) are included as part of the trial. At oncology meetings there is usually a slide reporting the % of patients enrolled in the trial who experienced AE's from grades 1-4, but the focus when presenting the data is usually on the ORR, PFS or OS.
It is important that patient reported outcomes become a standard part of the clinical trial process in oncology. The FDA issues a Draft Guidance on Core Patient-Reported Outcomes (PRO) in Cancer Clinical Trials in June 2021. The guidance includes various tools that can be used to measure patient reported outcomes in the areas of disease related symptoms, systemic adverse events, overall side effect measures , physical function and role functions. It also shows the frequency at which those measures shoe be recorded. The FDA considers PRO's( Clinical Outcome Assessments) as part of the COA's of a trial (https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-area-patient-reported-outcomes-and-other-clinical-outcome-assessments).
I've been thinking about the tools to measure PROs. There is a specific one for ovarian cancer called FACT-O (Functional Assessment of Cancer Therapy – Ovarian (FACT-O). The Assessment covers physical well-being, social/family well-being, emotional well-Being, functional well-being. Patients are asked to respond to statements that have been found to be important by others diagnosed with cancer with one of 5 responses from "not at all" to "very much". What about those issues not listed in the items others found important. There are vision issues with some anti-body drug conjugates now used to treat ovarian cancer but vision is not listed. Others including myself have had dental/ mouth issues during treatment which affect QOL and may fall under other categories but dental issues are not specifically mentioned. There is no space on the questionnaire to enter concerns that are not listed. I understand it is difficult to capture all of the patient experience in a single questionnaire.
At the recent ASCO 24 Annual Meeting there were multiple sessions and a keynote speech about artificial intelligence (AI). Can we use AI to read text from patients for example in a treatment diary or in an open ended question on a QOL form? Could those open ended responses be read by the study team in real time - not just for study analysis- so they can be responded to quickly? How difficult would it be to implement that analysis in all clinical trials in ovarian cancer and other cancers going forward? I don't have the an answer for these questions but I am sure their are other experts who could provide some information. I also would like to see the conclusion slide of each presentation at oncology meetings include those QOL measurements and not a simple statement that there are " no new safety signals" . What were the old signals?
We have work ahead but patient advocates, the FDA, Pharmaceutical companies and clinicians can work together to insure the patient experience and voice is recorded and used when considering approving treatments.
I'd love to hear from those in AI about the feasibility of implementing AI analyzed textual PROs or those with other ideas on how we can advocate for a greater use of PROs.
Dee
Every Day is a Blessing !
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