Patient-Reported Outcomes in Oncology: If No One Pays Attention to Data, Does it Really Exist?

In a recent piece published in the New England Journal of Medicine, oncologist Dr. Ethan Basch talks about his perspective that patient-reported outcomes (PROs) are not widely used in the development process for cancer drugs because they are not often measured in clinical trials.

Though this assessment is generally accepted, we questioned to what extent there is a lack of PRO data in oncology trials.

When analyzing both regulatory approval and global reimbursement decisions, Context Matters tracks PROs (along with many other outcomes and endpoints). So we were curious to take a look inside our database to see what the numbers said about the prevalence of PRO use in oncology.

Our Findings

Dr. Basch specifically points out that PROs are missing from U.S. labels and from published reports of clinical trials. When we looked at clinical trials cited in health technology assessments (HTAs) around the world, we got a different story. In seven solid tumor oncology diseases, 44% of the clinical trials cited in those reports included PROs.

In addition, Dr. Basch stated that preapproval trials for patients rarely measure symptoms or functional status (even when PRO data is used). We found that symptoms and functional status are the most commonly evaluated PROs. In fact, the PRO instrument cited most frequently in the HTA reports was the Functional Assessment of Cancer Therapy (FACT), an internationally recognized and validated questionnaire that has both general and cancer-specific subscales. FACT addresses physical well-being (energy, pain, nausea, feeling ill, and amount of time spent in bed), social/family well-being, emotional well-being, and functional well-being (working, sleeping, and quality of life).

Weighing All the Options?

Although we see the glass as being half full when we examine the frequency and nature of PROs in oncology, Dr. Bausch does make an interesting point that regulators and the pharmaceutical industry prioritize survival-based end points over patient-experience end points. This is consistent with our data, which shows that overall survival and progression-free survival are most often the primary outcomes of clinical trials. So while we may disagree with his assessment of low PRO use as evidence, we do think that Dr. Basch has raised several interesting questions for consideration.

PRO data is being measured and does exist, but it seems that PROs are not being weighed strongly in regulatory and reimbursement decision-making. In an environment that is increasingly focused on how best to incorporate patient-centered evidence, we find that this seeming disconnect may be an opportunity to explore a potential bias against PROs.

Some questions to consider:

• Does the hierarchy of evidence not address the role of PROs?

• How should PROs be weighted among the endpoints captured in clinical trials?

• What is the appropriate role of PROs in oncology?

• While we applaud Dr. Basch’s focus on the role of PROs in oncology, we believe that the real issue at hand is not about the lack of PROs or the lack of measurement of patient experience. The more interesting question and concern should be how (and how much) should PROs and patient-centered experiences be included in any overall assessment of oncology drug efficacy and effectiveness.