Optimizing the Alignment of Value and Precision Medicine in Oncology
Chicago, IL—As healthcare resources become increasingly constrained, aligning precision medicine and value may offer a way forward, but traversing this path could be a challenge for oncologists. According to Deborah Schrag, MD, MPH, Chief, Division of Population Sciences, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, the vision of precision medicine is for accurate biomarkers to identify the appropriate candidates for targeted treatment, which is more effective and less toxic than traditional cytotoxic chemotherapy. Given the rapid pace of innovation, however, the reality is more complex.
“We have imperfect evidence right now to inform individual and policy decisions. We must work hard and focus on strategies that will help us optimize the value of precision oncology. This is going to come from technological advances in molecular profiling, delivery system innovations, patient and clinician education, informatics, and building culture and systems that promote knowledge sharing and collaboration,” said Dr Schrag.
As Dr Schrag reported at the 2017 ASCO annual meeting practice management sessions, genetic testing has undergone rapid evolution from single-gene targets, such as ALK, KRAS, and ERBB2, to multigene panels, but that is just the beginning. Multigene exome panels and paired somatic germline exome panels may soon become the standard of care, along with whole-genome, tumor, and germline-paired panels.
“As oncology undergoes this rapid evolution, the potential for discovery is immense, but there’s also an exponential increase in complexity, and oncologists are struggling to achieve balance between ordering too few genomic tests versus too many,” she said.
Although panel testing is expensive, when viewed in the context of many current cancer drugs, it almost seems like a bargain, said Dr Schrag, and major cancer centers are not the only ones that have adopted this technology. Integrated delivery systems, community practices, and an increasing number of commercial firms now offer testing.
Despite this rapid adoption, however, paying for testing is variable. In some contexts, this technology is still considered “investigational,” and is billed to research accounts, said Dr Schrag. In other contexts, insurance will cover the costs, but there are patients who support this testing out of pocket. There are also commercial firms that provide assistance and subsidization as they develop their assays and seek to gain more information.
“We all want to measure the value of genomic testing, but it’s exceedingly hard to quantify, because we lack standard metrics and transparency, and it clearly depends on one’s perspective,” Dr Schrag continued.
According to Dr Schrag, skeptics of precision medicine have argued that there is an insufficient number of randomized controlled trials of panel testing versus focus testing and that the tests themselves are heterogeneous. Although lung cancer is the poster child for precision medicine success, critics point to the rather modest yield of “actionable” information for other diseases.
“In many contexts, there’s an uncertain impact on clinical outcomes, and with constrained resources, next-generation sequencing and panel testing would have to be considered low value. Moreover, uncertain findings could cause harm, and they create a tremendous burden of information management,” said Dr Schrag.
Despite the complexity of the landscape, however, “putting our heads in the sand like the proverbial ostriches is not going to work. This is a huge innovation space, and emerging tools and systems are going to make this easier,” she added.
Critical Information Needed
Tumor profiling may be here to stay, but strategic implementation remains a challenge. Many oncologists receive integrated decision support in their workflows and electronic health records, and are starting to receive clinical trial matching and support with respect to patient and physician education. Nevertheless, said Dr Schrag, critical information is often unavailable to clinicians.
“Failure to integrate genomic, and what I describe as ‘phenomic,’ information limits our ability to realize the goals of precision medicine,” she observed.
To figure out whether precision medicine works, information concerning disease status and treatment response, specifically progression-free survival, overall survival, and response rate is required, but these data may be “trapped” in physician notes, imaging reports, or free text. Dr Schrag and colleagues are thus attempting to take the unstructured information that exists in every electronic health record, maybe even in paper documents, and code disease status at every visit with a simple “evident,” “not evident,” or “not evaluated.”
The researchers hope that the so-called PRISM system, a standard taxonomy for estimating treatment response, will augment the value of precision medicine and help identify effective and ineffective drug combinations from real-world data more expeditiously.
“I think we’re getting close to the tipping point where targeted versus systematic panel testing for cancer patients becomes the reality and becomes more valuable. Right now, we’re still going uphill, but I predict it will get easier,” Dr Schrag concluded.