The ever-rising costs of pharmaceuticals continue to place an increasing burden on patients, physicians, and payers in the United States—none of whom are well-equipped to compare the value of different therapies. On July 23, 2015, the New York Times reported that “more than 100 prominent oncologists called for support…to stem the rapid increases of prices of cancer drugs, including by letting Medicare negotiate prices with pharmaceutical companies and letting patients import less expensive medicines from Canada. Similarly, the Wall Street Journal reported that the Laura and John Arnold Foundation is providing a $5.2 million grant to the Institute for Clinical and Economic Review (ICER), a Boston nonprofit that conducts cost-effectiveness research in the United States.
The ongoing debate around the high cost of drugs in the United States, especially in oncology, has produced interesting responses from special interest groups. It also demonstrates the insular nature of the United States’ approach to managing healthcare costs. The fact is, many countries outside of the United States depend on Health Technology Assessment (HTA) agencies to manage a systematic review process that evaluates drug efficacy and cost-effectiveness to assist with decision making in regard to pricing and reimbursement. Not only does the United States lack such a central agency—many citizens of the United States do not even know that such agencies exist. Even payers and policy makers in the United States often have little or no knowledge of the drug assessment methods used elsewhere in the world. The American Society for Clinical Oncology (ASCO) believes they may have found a solution for drug valuation in the United States.
In June 2015, ASCO published a paper detailing a “conceptual framework to assess the value of cancer treatment options,” a prototype of a tool allowing physicians and patients to better discuss and compare the values of different cancer treatment options. The proposed tool is intended for patient and physician decision making, not for payers and policy makers. However, ASCO “recognizes that this work has the potential to influence policymakers and payers as they consider preferred management options and evaluate the relative value of new treatments introduced into the cancer marketplace.”
Though their intended audiences differ, ASCO's tool and HTAs ultimately have the same aim: determining how a drug stacks up to other therapies and whether it is worth the money. ASCO's tool, to be useful to patients, has to be simple and clear. For example, ASCO emphasizes the value of data that can be “presented in a visually appealing, user-friendly way.” But what does this simplicity and clarity cost? ASCO's model only considers specific easily-obtainable and quantifiable survival outcomes, safety, and cost. Cost is not well-integrated in the model. As a result, a patient must consider cost and effectiveness separately and decide individually if the therapy is worth the price. In addition, ASCO's model does not consider the quality of evidence. As ASCO’s framework can only be applied to individual trials, choice of evidence source is absolutely imperative, but the ASCO framework provides no system for judging quality of evidence. Instead, it asks that patients and physicians be savvy enough to distinguish grades of evidence without any real guidance.
In comparison, HTA agencies can look at many parameters beyond the most easily accessible, can compare many different evidence sources and weigh their relative merits, and make use of teams of experienced professionals. While their reports are not tailored for the circumstances of individual patients, they can provide much more sophisticated and informed evaluations to support decision making.
HTA documents are not intended for direct use by patients or on individual cases, but as guidance government policymakers and payers can reference when making decisions that influence the entire healthcare system. In contrast, the ASCO framework was developed for a system where patients and physicians must regularly make decisions about the value of drugs on a case-by-case basis. ASCO provides physicians and patients with a rudimentary version of an HTA-style approach to drug evaluation for individual application that doesn’t help with the cost effectiveness discussion.
Any approach that sheds light on the relative value of therapies and facilitates informed decision making for patients, physicians, and payers has value. ASCO’s approach seeks to do so simply and with great clarity. However, one has to wonder why this responsibility is placed into the hands of patients. Cost-effectiveness and comparative effectiveness drug evaluations are produced by well-funded organizations in numerous countries. Their work considers a greater body of evidence, considers more contextual data, and is informed by teams of experts. How can patients and physicians examining individual trials based on narrow parameters hope to make comparably well-informed decisions? Is there an opportunity for United States to take the lead by establishing a valuation process that follows through from the population to the individual level?
ASCO’s goals are admirable—but rather than solving a problem they have revealed a need. Patients, physicians, and payers in the United States have few places to turn for comprehensive and detailed comparative efficacy and cost-effectiveness data on cancer therapies. American patients value their autonomy in making healthcare decisions. But autonomy only works well if the process and people involved are well-informed. Without benchmarks, nuanced process, or methodology, has ASCO simply provided a tool that confuses rather than clarifies?