The methodologies employed within value assessments, especially by the Institute for Clinical and Economic Review (ICER), primarily measure value through quality-adjusted life-years (QALYs). While also the common metric used in cost-effectiveness assessments by other countries, its application in the United States has generated significant controversy. While the focus on QALY pros and cons is valid and additional measures will be considered, another tremendous challenge with today’s value assessments is that they rarely evolve, even as new, relevant evidence becomes available. Given government pressures and threats to create the rules itself, the private sector has an opportunity to leverage greater use of structured methodologies and ongoing assessment to be part of the solution to addressing concerns about price by focusing on value.
Value assessments are rapidly evolving in the United States today. Routinely used to assess the value of treatments in countries around the world, methodologies for comparative and cost effectiveness only gained their first nationwide structure in the United States when the Academy of Managed Care Pharmacy released its Format for Formulary Submissions in 2000.1 Since then, the combination of value-based care initiatives across the health care spectrum, the emergence of value frameworks, and the heightened activity of ICER over the last 5 years has thrust value assessments of therapies increasingly into benefit coverage and even prescribing decisions.
Measuring Value, Improving Methods
The methodologies employed within value assessments, especially by ICER, primarily measure value through QALYs. While also a metric used in cost-effectiveness assessments by other countries, its application in the United States has generated significant controversy.2-4 While the focus on QALY pros and cons is valid and additional measures will be considered, another tremendous challenge with today’s value assessments is that they rarely evolve, even as new, relevant evidence becomes available. Most value assessments of therapies occur around the time of marketing approval (Figure 1). Yet, relevant evidence concerning the therapy, the condition it treats, and other treatments continues to evolve. It is often this compilation of evidence over time that provides the clearest view of a therapy’s value.
How often have entities making benefit coverage decisions assessed value based on a review of the handful of clinical trials at a product’s launch, and then made a subjective judgment of the comparative value with other treatments that were not used in those trials? From my past work on pharmacy and therapeutics committees and speaking with those directly involved today, the vast majority of decisions happen through this subjective judgment. Yet, there are methodologies specifically designed to provide comparative and cost-effectiveness assessments—the primary obstacle seems to be the premise that randomized controlled trials (RCTs) are the gold standard and all other evidence is inferior in strength. Even if that is the case (and there are sound arguments to be made for both viewpoints), in the vast majority of situations where value assessments are being made without RCTs including comparative therapies, would structured methodologies not provide a greater level of confidence than solely relying on subjective judgment? Moreover, as further evidence became available, would inputting that evidence into previously performed structured methodologies not create a clearer view on value?
As one of the primary roadblocks to utilizing structured methodologies that support comparative and cost-effective analyses, the reliance solely on RCTs is compounded as new evidence becomes available, which would suggest value assessments be updated periodically to improve the accuracy and precision of the findings. One example of the benefit of this type of approach is illustrated in evaluating the evidence generated for intravenous streptokinase in acute myocardial infarction (MI). Image 1 shows all the relevant clinical trials including the patient population and the outcomes. If we assessed the value solely on the first few studies, the conclusion would be that intravenous streptokinase was not effective in reducing the effects of an MI. When including new evidence over time, it appears the studies trend more toward an effect, yet many of the odds ratios still cross the line, ie, do not show a statistically significant effect. Compare that illustration of each piece of evidence evaluated independently to Image 2, where a structured methodology shows the cumulative effect by adding the effects of each study to the existing base of evidence. We see that, clearly over time, there is a positive treatment effect identified, both in amount of effect (distance to the left from the center line) as well as the precision of confidence that effect will occur (length of the line representing the confidence interval). While there have been a few recent examples of updating value assessments through these structured methodologies, they are exceptions.
In an environment where all payers are pushing toward a value-based system, we must elevate our diligence in assessing value, both by incorporating structured methodologies as well as updating when new evidence becomes available. While there are supporters and critics of the methods ICER has used to assess value, ranging from omitting key elements of value to ignoring patient choice to using QALY as the key measure, clearly, they and others proposing value frameworks have elevated the use of structured methodologies. Given government pressures and threats to create the rules itself, the private sector has an opportunity to leverage greater use of structured methodologies and ongoing assessment to be part of the solution to addressing concerns about price by focusing on value.
1. Gricar JA, Langley PC, Luce B, Lyles A, Sullivan SD; for Academy of Managed Care Pharmacy (AMCP). Format for Formulary Submissions. http://amcp.org/WorkArea/DownloadAsset.aspx?id=16275. Published October 2000. Accessed April 2, 2019.
2. Smith WS. Key questions for legislators about the Institute of Clinical and Economic Review (ICER) [white paper number 189]. Pioneer Institute. https://pioneerinstitute.org/featured/study-urges-caution-before-adopting-icer-reviews-to-determine-cost-effectiveness-of-treatments/. Published January 2019. Accessed April 2, 2019.
3. Cohen JT, Ollendorf DA, Neumann PJ. Will ICER’s response to attacks on the QALY quiet the critics? Center for the Evaluation of Value and Risk in Health (CEVR); Tufts. https://cevr.tuftsmedicalcenter.org/news/2018/will-icers-response-to-attacks-on-the-qaly-quiet-the-critics. Published December 18, 2018. Accessed April 2, 2019.
4. Partnership to Improve Patient Care (PIPC). Responding to stakeholder input: finding the patient voice in ICER’s value assessments. http://www.pipcpatients.org/resources/responding-to-stakeholder-input-finding-the-patient-voice-in-icers-value-assessments. Published October 1, 2018. Accessed April 2, 2019.
5. Lau J, Antman EM, Jimenez-Silva J, Kupelnick B, Mosteller F, Chalmers TC. Cumulative meta-analysis of therapeutic trials for myocardial infarction. N Engl J Med. 1992;327:248-254. doi:10.1056/NEJM199207233270406