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US Health Plans Addressing Rising Drug Costs With Step Therapy, Value Assessment Frameworks


As the cost of therapies increases, US health plans are utilizing tools like step therapy, to ensure patients try cheaper alternatives first, and value assessment frameworks, to assist with the decision-making process.

As the cost of therapies rise, approximately 40% of payers have step therapy protocols for specialty drugs that require patients to first fail an alternative cheaper treatment before accessing a more expensive drug, according to a poster presented at Virtual ISPOR 2021.1

Researchers utilized the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database, which includes information about how US payers cover specialty drugs. The researchers examined the frequency that plans applied step therapy by indication and identified the diseases step therapy was most often applied.

Of the 17 plans reviewed, step therapy protocols were applied to 38.9% of their coverage policies, ranging from as low as 20.6% and as high as 57.5%. On average, plans included 1.5 steps in their protocols with 66.6% of plans including a single step, 22.7% including 2 steps, 7.6% including 3 steps, and 3.1% including 4 or more steps.

The most common indications that plans applied step therapy were:

  1. Chronic migraine for 100% of decisions and a mean number of 2.2 steps
  2. Plaque psoriasis for 92% of decisions for a mean number of 2.0 steps
  3. Ankylosing spondylitis for 82% of decisions for a mean number of 2.0 steps
  4. Rheumatoid arthritis for 78% of decisions for a mean number of 2.0 steps
  5. Psoriatic arthritis for 74% of decisions for a mean number of 1.9 steps

Metastatic breast cancer and cervical dystonia were the least common indications that plans applied step therapy (6% of decisions for each).

According to the researchers, health plans apply step therapy protocols inconsistently. The reasons some diseases are affected more than others by step therapy is because some diseases have more indicated treatments and some specialty drugs have inexpensive alternatives available.

“Variations in health plans’ step therapy requirements can have important implications, as it means that patients enrolled in different health plans have different access to the same drugs,” they concluded.

Rising health care costs have also led to an increased interest in value assessment frameworks (VAFs). A poster at Virtual ISPOR 2021 updated insights on current trends, perceptions, and utilization of VAFs in decision-making processes among US payers.2

Xcenda fielded double-blinded, web-based surveys in November 2016 (N = 56), November 2018 (N = 47) and October 2020 (N = 47) to managed care professionals in Xcenda’s Managed Care Network. The surveys sought insights into familiarity with the Institute for Clinical and Economic Review (ICER) VAF, Memorial Sloan Kettering Cancer Center (MSKCC) DrugAbacus, and the National Comprehensive Cancer Network (NCCN) Evidence Blocks; use of these tools as supporting data to inform clinical, economic, or patient-related decisions; areas or disease states the ICER VAF was most used; and reasons these tools may have been used in a limited fashion to inform payer decisions.

Nearly all (≥ 90%) respondents said that inclusion of comparative clinical effectiveness information, appropriate metrics and outcomes, and methodologically rigorous and unbiased methods were extremely or very important. One-third (32%) said the inclusion of the patient perspective was not very or not at all important, while 47% said it was somewhat important and only 21% said it was extremely or very important.

Payers seemed to be more familiar with the ICER VAF and NCCN Evidence Blocks than the MSKCC DrugAbacus across all 3 years of the survey. “Across the 4-year period, the ICER VAF was used most frequently to inform economic decisions,” the authors noted. In 2016, 31% of respondents reported no use of the ICER VAF, which decreased to just 4% in 2020.

Among payers who used the ICER VAF in the 2020 survey, 41% said it was in oncology, 50% in rare disease/orphan drugs, 34% in primary care, 38% in a crowded therapeutic area, and 59% in a high-cost disease.

The MSKCC DrugAbacus had low utilization among respondents and use of the tool decreased from 2016 to 2020. In the 2020 survey, 60% of respondents reported no use of the tool. The most common reasons for not using MSKCC DrugAbacus were:

  • Insufficient validation of framework (30%)
  • Missing key outputs (30%)
  • Waiting for a better or revised version (28%)
  • Other, including being minimally aware of it, the tool not being easily available, and the tool not always being accurate (30%)

The NCCN Evidence Blocks were most frequently used to inform clinical decisions across all 3 surveys and in 2020, 45% reported using them to support major clinical decisions and 28% used them to inform minor clinical decisions. However, these were down from 2016 (55% and 45%, respectively).

“Given the influence of VAFs on payer coverage decisions, it is beneficial for stakeholders to remain current on VAF practices and proactively engage in assessments and methodology design when possible to inform the evolving value assessment landscape,” the authors concluded.


1. Lenahan KL, Gertler RM, Nichols DE, Chambers JD. Examining US commercial health plans’ use of step therapy protocols for specialty drugs: an empirical analysis. Presented at: Virtual ISPOR 2021; May 17-20, 2021. Poster PNS52.

2. Choate A, Yan A, Tan R, Tennant L. Current utilization and perceptions of value assessment frameworks among United States Payers. Presented at: Virtual ISPOR 2021; May 17-20, 2021. Poster PNS59.

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