The price of and access to oncology drugs remains both a national and international challenge to our patients, providers, and third-party payers. The debate on the methods to evaluate the value of both existing and newly developed anticancer therapies in the United States and around the world continues. The current ASCO position on addressing the affordability of cancer drugs calls for several steps in determining the price of drugs, while ensuring patient access to critical treatments and fostering innovation by drug manufacturers. ASCO’s work could inform a global effort to define value, reaching across borders. The position statement calls for value-based solutions that are patient-centered and evidence-driven. It calls for oncology professionals to define optimal care within a framework that assesses the comparative treatment options and focuses on a real and consistent relationship between the value of the drug and the cost to the patients. The trials sponsored by the pharmaceutical industry with the goal of drug approval are typically carried out in multiple countries. If we are going to call for a value-based pricing system around the world, there needs to be a consistent assessment of value that can be applied across different countries. This is a critical step to provide a dependable regulatory environment and the means for estimating a drug price and hence, potential profits from a new more effective treatment needed for ongoing drug development.
The ASCO/European Society for Medical Oncology (ESMO) Joint Education Session at the 2017 ASCO Annual Meeting, “Access to Clinical Care and Cancer Medicine,” pointed out the substantial differences between the systems being proposed and used by ESMO and ASCO. The 2015 and 2016 articles published in the Journal of Clinical Oncology and presented by Dr. Lowell E. Schnipper at that session outline ASCO's Value Framework for advanced and adjuvant disease. My following comments will focus on the advanced disease proposal. The value is determined by a clinical benefit assessment using the endpoints in the trial (increased survival, progression-free survival, and response rate in descending order), a score for decreased toxicity plus bonus points for an increase in the tail of the survival curve and a potential bonus for increased palliation compared to an existing treatment that give rise to a net health benefit score that has the potential to influence the cost of the treatment. Policymakers can then use the net health benefit score to help determine the value to the patient and hence guide the price of the drug.
The ESMO-Magnitude of Clinical Benefit Scale was published in the Annals of Oncology in 2015 and presented by Dr. Elisabeth De Vries at the 2017 ASCO Joint Education Session as well. The ESMO Scale differs from the ASCO Value Framework. Their systems for non-curative treatments roughly corresponds to the system for advanced disease in the ASCO proposal. The criteria for meeting clinical benefit for a new treatment regimen if the control survival is less than 12 months is a hazard ratio of 0.65 and a prolongation in survival of 3 months or an increase in 2-year survival of 10% over the existing therapy. Progression-free survival endpoints are also derived using a different method than the ASCO Value Framework. In the ESMO model the outcomes of the trials are given a score from 1 to 4 (1 is the best) and can be upgraded by another point to 5 if there is reduced toxicity or a substantial reduction in grade 3 or 4 toxicity. Five is the best score. Policymakers can then use these scores to help make decisions about approvals and potential increase in prices over existing therapies based on good scores. These examples are not given in detail, but are meant to show that the methods of deriving scores and the tools for decision-making currently vary in different countries.
The enormous costs needed for drug development and for carrying out registration trials leading to drug approval necessitate a stable and predictable environment for the pharmaceutical industry to guide their investments in new potentially more effective therapies. The variability in different countries for drug approval and different methods for setting drug prices contribute to uncertainty and may lead to underinvestment in drug development, a critical need for our field. The ASCO Value Framework and Magnitude of Benefit Scale are assessing similar endpoints so international efforts to harmonize our systems have the capability of leading to a more consistent and predictable means for drug approval and to guide our policy makers in setting drug prices. This calls for an international effort to standardize the value-based pricing of drugs around the world to provide a dependable regulatory environment and a consistent approach for identifying the value and pricing of new drugs.