Washington, DC—Value assessments for the management of patients with metastatic colorectal cancer (mCRC) focus more on cost-effectiveness and less on the line of therapy, according to a study presented by Ed Wang, PharmD, Deputy Director, Health Economics and Outcomes Research, Bayer HealthCare Pharmaceuticals, Whippany, NJ, and colleagues, at the 2016 International Society for Pharmacoeconomics and Outcomes Research annual meeting.
The definition of value in healthcare has evolved considerably in recent years, moving away from equating value with cost, drug costs especially, and toward greater consideration of the benefit or harm of a new therapy. This movement has been driven by the emergence of value frameworks, the investigators noted.
Evolution in the concepts and definition of value has also resulted in less emphasis on the payer perspective, and more emphasis on open dialogues between patients and physicians about value and the relative merits of cancer therapy as a key factor in informed decision-making.
“It is important to recognize that cost-effectiveness is only one component of a value assessment,” Dr Wang and colleagues said. “However, there is discordance between value frameworks that makes their real-world applicability challenging,” they added.
To examine the medical literature on value during this period of transition, Dr Wang and colleagues conducted a literature review of articles published from January 1, 2005, to December 31, 2015, and of abstracts presented at key conferences during 2015. They selected mCRC as a representative example of cancers that have been the focus of investigations on value, because of its prevalence as the third most frequently diagnosed cancer in the United States.
“As evidenced both in the published literature and in the value frameworks, line of therapy is often not a consideration in the assessment of value. Very few of the publications focused on advanced (third-line or later) mCRC,” Dr Wang and colleagues observed.
The search identified 88 publications that met inclusion criteria, including 37 from the United States, 35 from Europe, 8 from Canada and Australia, and 11 that were not specific to a geographic region.
The proportion of European-centered value publications increased steadily during the 10-year period, from 20% to 48%. Value-focused publications in the United States were uncommon (4%) between 2005 and 2008, but increased rapidly between 2013 and 2015, reaching 48%.
Economic modeling was the most common method for determining value in mCRC therapies, followed by retrospective database analyses. More than 80% of the economic models were cost-effectiveness or cost-utility analyses.
The majority (77.1%) of European studies were economic models, whereas the US studies were evenly divided between economic models (40.5%) and database analyses (45.9%). The majority (75%) of studies had a payer perspective. The patient perspective and physician perspective were represented in 3.4% and 8.0% of publications, respectively. Studies were funded by pharmaceutical manufacturers or by device companies (33%), academic institutions or health economists (38%), and physicians or physician organizations (25%).
Of the 73 publications reporting on line of therapy, 29 evaluated all lines of therapy, 29 evaluated only first-line therapy, 8 evaluated second-line therapy and subsequent therapies, 5 evaluated first-line therapy and subsequent therapies, and 2 evaluated third-line therapy and subsequent therapies.
Of the 37 US publications, 13 focused on cost-effectiveness, as assessed by incremental cost-effectiveness ratio, and 16 focused on healthcare resource utilization; the majority (19) evaluated the value of targeted therapies in mCRC.
The investigators compared the value frameworks developed by the American Society of Clinical Oncology, the European Society for Medical Oncology, the National Comprehensive Cancer Network, and Memorial Sloan Kettering Cancer Center. Formalized evaluations in the literature focused on cost-effectiveness, whereas the value frameworks consider cost side-by-side with the clinical benefit of a treatment rather than as a component of the assessment, excluded altogether, or included with subjective scales for “affordability.”
“Despite the substantial number of publications, the majority still focus on cost-effectiveness of mCRC therapies in the first-line setting,” the investigators concluded. “The observation that many publications were cost-effectiveness models in Europe is likely a reflection of the requirement by most European health technology assessments for an economic model submission for reimbursement consideration. In contrast, in the US [United States], the more common model is a budget impact analysis.”
