Two months ago, the Centers for Medicare and Medicaid Services convened an advisory panel of experts that voted in favor of supporting use of patient-reported outcomes, or PROs, in clinical trials of CAR-T cell therapies. The vote, by the Medicare Evidence Development & Coverage Advisory Committee, could result in PROs becoming a requirement for Medicare coverage of CAR-Ts if CMS follows the vote, a potential outcome that drew opposition from the drug industry and other groups.
MEDCAC voted that available evidence supported the ability of four of seven suggested PRO assessment systems could measure outcomes in future clinical trials. While not binding, the outcome of the vote illustrates how PROs and PRO measures are growing in importance as the healthcare system moves to a value-based model. That will be the subject of a panel discussion at the MedCity ENGAGE conference, which takes place Nov. 6-7 in San Diego.
Avalere Health Vice President Katherine Steinberg will moderate the panel, which will include Dr. Wei-An Lee, director for specialty care and clinical innovation at Los Angeles County+University of Southern California Medical Center; Epharmix founder Blake Marggraff; and Jean Slutsky, chief engagement and dissemination officer at the Patient-Centered Outcomes Research Institute.
PROs indeed have an important role to play in value-based care, Slutsky said. “Using PROs and measuring them in research studies can help us better communicate the value of interventions to patients and their families,” she wrote in an email. “PROs assess domains of importance to patients and the effect the treatment is having on the patient, from the perspective of the patient.”
From that angle, as value in healthcare is defined, it becomes important to do a better job representing a good outcome from the perspective of the patient, for which PROs offer an opportunity, Steinberg said. Quality has traditionally not been defined from the patient’s standpoint, despite quality and cost being the key inputs to defining value. “From my perspective, patients are the best source of information for insights about quality of life, functional status and symptom management, making PROs a critical part of understanding and evaluating value-based care,” she wrote in an email.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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Marggraff said maternal health is one example of where PROs are important. Postpartum depression can drive major costs at a time when a mother and baby are forming a relationship, he said. Just asking screening questions – such as queries about mood, followed if warranted by questions about thoughts of harm to self or child, can be powerful and straightforward, especially if clinically validated. “But if it’s not done, you’re sending two humans out into a void and hoping they don’t come back and cost you money and potentially be a major clinical risk,” he said in a phone interview.
Meanwhile, Steinberg said the most straightforward application is in patient-reported outcome measures, or PROMs, which can be used similarly to more traditional quality measures for evaluating and compensating providers. Depression provides an example. In that case, a PROM would be a standardized tool to evaluate symptoms, and using the results of the tool to evaluate performance on that measure in improving symptoms for patients would be the application of PROMs to value-based care. If the PROM is then used for performance management, she said, one might see reimbursement for care linked to improvement in those measures as a mechanism to drive increased value.
Still, PROs can have limitations as well.
“Collecting PROs is always a balance of rigor and comprehensiveness vs. burden to the respondents, the patients and family members,” Slutsky wrote. Any selected PRO must be “fit for purpose,” meaning that it must have demonstrated psychometric validity and reliability in the patient population, which might be difficult for a clinical center where that population is heterogeneous. There are also concerns about hurdles to operationalizing PRO metrics, and questions as to whether a metric was properly designed, including adverse selection or barriers to scalability could undermine a PROM’s reliability, Steinberg said.
One of the inherent attributes of PROs is that they can be subjective, but that does not diminish their value, Marggraff said. However, that does not make them any less valuable, like in behavioral health. The whole system for treating patients with depression and addiction, for example, relies on patients reporting their perceived predisposition to using an opioid when being treated for addiction or their mood when recovering from depression, and the trends that emerge become more valuable. “PROs are means to a few ends, but the main one is treatment and prevention, and the outcome of that effort is value creation,” he said.
Photo: asiseei, Getty Images
