Evaluating New Medications with Evidence-Based Value Analysis

Atlanta—Of the medications approved by the US Food and Drug Administration (FDA) in 2010 and 2011, many are specialty, biologic, and oncology treatments, making it especially important for healthcare plans to establish reproducible and transparent methods for assessing the value of these emerging therapies.

At a Managed Care Essentials session at the AMCP meeting, speakers addressed the issue of value assessment in a presentation titled “Evidence-Based Value Analysis of Mew Medications: 2011 Update.” Using a structured decision-making framework, Sean Karbowicz, PharmD, clinical pharmacy manager at RegenceRx, and Laurie Wesolowicz, PharmD, director of pharmacy services, clinical, at Blue Cross Blue Shield of Michigan, provided attendees with an assessment of the value of medications approved in the past 2 years.

They began the session by noting that when the FDA approaches the question of value assessment of a new medication, the agency attempts to answer questions about the safety and effectiveness of the therapy, and determine whether the benefit outweighs any associated risks. For managed care professionals, the questions focus on the comparative safety and effectiveness of a new therapy, with particular emphasis on how the therapy compares with other treatments.

Using an algorithm as a process for assessing the value of new medications, the speakers then presented several scenarios based on recently approved therapies. The process consists of 4 steps: (1) appraise the evidence of efficacy (the primary driver of formulary decisions), (2) evaluate other considerations, (3) synthesize the data, and (4) make a value assessment.

In scenario 1 (confidence in evidence for efficacy, other established options), the assessment of a new product begins with confidence in the evidence for efficacy of the therapy, leading to an evaluation of additional options. If there are other options, the next step in value assessment of the new therapy is an evaluation of the drug’s safety: a drug with proven safety advantages will involve less risk, followed by a drug with an established track record of minor adverse effects, to one with unknown or unconvincing safety data or concerns about safety that are “serious but manageable.” The level of safety concern indicating greatest risk is a drug with safety concerns that are significant enough to outweigh the benefits. The final step in this scenario is determining total overall cost. A medication with a high confidence in the estimation of efficacy would be of “high value,” whereas when a new therapy is of unknown safety or is riskier than other treatments and /or increases costs that are disproportionate to other options, the drug would be assessed as one of “uncertain value.”

In scenario 2 (confidence in evidence for efficacy, no established options), the assessment moves from high confidence in the evidence for efficacy, leading to a lack of other options, coupled with significant safety concerns and increases in cost that are disproportional to the benefit of the new therapy. The result of this value assessment is “uncertain value.”

In scenario 3 (lack of high confidence in evidence for efficacy, other established options), new drugs with no significant safety concerns, low cost when other options also lack evidence, or profound, proven safety advantages over other treatments would be of high value, whereas those with a lack of confidence in the efficacy data would be given a level of “uncertain value.”

In scenario 4 (lack of high confidence in evidence for efficacy, no other options), when a drug’s safety concerns are “manageable,” and benefit is “readily observable,” and “watch and wait” is not an option, the new therapy would be of high value. Absent those conditions, however, the lack of high confidence in the estimation of the efficacy of the drug would result in an assessment of “low value” for the new drug. In conclusion, the researchers said, “many innovative medication therapies with proven clinical benefit have recently become available, many with significant costs.

Quality evidence can allow confident estimations of efficacy for new medications in complex, severe diseases, or conditions with unmet needs. A standardized decision-making framework is essential in evaluating complex, new medications and describing their value.”