Adapting Clinical Trials For The Upcoming Oncology Value Wave
By Jeremy Schafer, Precision for Value
In 2015, an estimated $107 billion was spent globally on oncology therapeutics, a year-over-year increase of 11.5 percent. Drug spend reports from leading U.S. pharmacy benefit managers and payers found similar trends in 2016. Express Scripts, MedImpact, and Prime Therapeutics all reported double-digit increases in oral oncology trend from 2015 to 2016. A Magellan report that focused on provider-administered therapies found 36 percent of all medication spend under the medical benefit for commercially insured patients was for oncology. The aging population in the United States means that the trends noted by major payers are likely to continue. Healthcare stakeholders, from payers to patients, are increasingly struggling to cope with the rising cost of oncology and are calling for new approaches that not only ensure access, but also provide value.
A Shift To Value
The increased cost of oncology treatment has led to a growing discussion around “value.” The idea is that rather than pay for oncology on a volume basis, as is currently done, payers would instead pay for oncology products and services based on the value provided. The definition of value varies but generally states that the better the outcomes in relation to cost, the higher the value. Under this definition, manufacturers can enhance value by providing products with greater efficacy, better safety, and lower cost, or a combination of these elements. However, the challenge remains: How is value measured?
Value frameworks have emerged that attempt to answer how much value an oncology drug provides. DrugAbacus, created by Memorial Sloan Kettering, was one of the first value tools; it uses a mix of data, including efficacy, safety, development cost, and disease rarity, to recommend a value price. Other frameworks soon followed, including the ASCO Value Framework, NCCN Evidence Blocks™, and more complex cost-effectiveness analyses by the Institute for Clinical and Economic Review (ICER). The target audience for each framework varies, but recent data indicate that payers have already begun using these frameworks in oncology management. The output of each framework varies, but all share a common goal: to help stakeholders determine how much value an oncology product provides in relation to its cost.
Payers, including the government, have begun to shift the oncology care payment model to include a value element. The CMS Oncology Care Model (OCM) aligns payers and oncology practices in a value approach that ties some of the compensation to improved outcomes and managing the total cost of care. UnitedHealthcare, the nation’s largest private insurer, has embarked on a value-based initiative with MD Anderson Cancer Center in head and neck cancers through value tracking and bundled payments. A similar model involving breast, colon, and lung cancer patients treated at five medical oncology groups saw a 34 percent reduction in the predicted total medical cost.
As stakeholders in oncology increasingly focus on value, an important question is raised for clinical researchers and manufacturers: Can oncology value be measured in a clinical trial program?
Conventional Oncology Trial Design
Currently, clinical trials in oncology capture limited value or cost-effectiveness data. Oncology drug trials are designed to measure a specific efficacy end point and to enroll as few patients and be completed as quickly as possible. The strategy is understandable given the considerable cost of studying oncology products and trying to meet the needs of patients with serious disease. Unfortunately, this strategy means that manufacturers are often scrambling to collect data for a value story post hoc, and the data may be lacking in both quality and scientific rigor. Prospective definition of value end points may enhance collection during clinical trials, particularly for data already generated incidentally in the current design.
Value Measurements In Oncology
For clinical researchers interested in incorporating value measurements into oncology trials, the first question is likely, “What should be measured?” Some potential value end points are described below. Researchers should note, however, that efficacy—particularly overall survival—and safety continue to be the most influential on value and should remain the focus.
Hospitalization: Hospitalization is a leading driver of healthcare cost. Measuring how often, and for how long, an oncology patient is hospitalized would be a valuable metric for stakeholders. Payers and health systems are exposed to more cost as patients spend more time in the hospital. In addition, hospital readmissions can negatively impact quality scores for health systems and payers. If an oncolytic can show reduced hospitalization in a clinical trial, the information would be an added benefit to the safety and efficacy profile of a new product.
ED utilization: Urgent emergency department (ED) visits are often indicative of poorly controlled or worsening disease and may also be the result of drug adverse effects. Measuring how often patients in a clinical trial end up at the ED may provide meaningful data on a drug’s real-world impact on safety and efficacy. Additionally, ED visits are costly and often lead to hospitalization; used as a trial end point, they can provide perspective on value.
Supportive care medication use: Medications used in oncology supportive care can vary from inexpensive and simple (pain relievers, oral antiemetics) to complex and costly (stimulating factors). The use of supportive care medications has a financial impact on both payers and health systems. Demonstrating a product’s differences in supportive care medication use can provide additional value data, as well as further define the safety profile.
Total cost of care: Capitated payment models provide a single lump payment to oncology providers to manage patients. Thus, a focus on total cost of care is important. Actually measuring the dollars spent on different patient interventions is complex due to regional differences and difficulty in capturing data. Researchers seeking to provide data on total cost of care may be able to achieve the objective by capturing other end points, such as those noted above, and assigning cost estimates.
Bringing Value Measurement Into Clinical Trials
The first step in measuring value in a clinical trial is to consider the end points prospectively in the trial’s design. End points such as hospitalization and supportive care medication use may already be captured to some degree by the clinical trial site. Identifying these end points before the trial begins would strengthen the protocol and increase the accurate gathering of this information. Upon trial completion, researchers would have supportive data showing the real-world implications of a studied drug’s safety and efficacy.
Researchers may also want to consider including an outcomes researcher or health economist in the data analysis portion of the clinical trial. Even if capturing relevant financial information is not possible, a specially trained researcher may be able to assign financial values to certain end points, strengthening the value story for the medication under study. This individual could also be useful in trial design and data methodology.
The primary obstacles to value measurement in clinical trials are data availability and added trial cost. Patients in an oncology drug trial may receive their oncology therapy at one site but go to another for conventional care or emergency services. Medication use may also come from disparate sources. Capturing these data is crucial to accurate value measurement. Clinical researchers should continue to advocate for the standardization of electronic medical records and the sharing of patient-level data between providers involved in a patient’s care. Researchers may also be challenged by the added costs required to measure value end points in a clinical trial. However, manufacturers tasked with gaining optimal access for an oncolytic may find the up-front investment worthwhile for the gains in the product’s value proposition at launch.
Value in oncology is increasingly a focus and unlikely to diminish anytime soon. While the emphasis on value will be most prominent after a product’s launch, there are steps that clinical researchers can take to initiate the value story in clinical trials. The additional investment of time and money in the clinical trial stage may reap significant rewards after product launch thanks to the enhanced value proposition the new product brings to the oncology market.
About The Author:
Jeremy Schafer, PharmD, MBA, is senior VP of specialty solutions at Precision for Value. In this role, he is responsible for enterprise leadership on specialty strategy across the payer team organization and integration with other enterprise groups, including health economics and outcomes research, provider marketing, access and analytics, and media outreach. He has more than 10 years of experience in specialty pharmacy programs and networks, pharmacy and therapeutics committee processes and review, fee schedule management, and pharmacy benefit migration.
Before joining Precision, Jeremy served as a corporate account manager for Grifols. Prior to his time there, he spent over seven years at Prime Therapeutics, one of the five largest pharmacy benefit managers, where he held a variety of leadership roles in formulary, utilization management, and specialty pharmacy.