Robert J. Nordyke, MS, PhD

This article was published as part of a special joint issue and also appears in the 

 The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states.

We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study. We conducted in-depth qualitative interviews with the payers and asked them to rate the usefulness of CER study types across various disease states and market conditions. The results were analyzed for thematic content.

Our findings indicate that payers are interested in a broad range of CER study types, are unsatisfied with the current state of CER, and would like to partner with research groups to develop research and treatment guidelines to better leverage CER. Payers value CER less in oncology than in other disease states because of limitations in their ability to manage oncology therapies.

 To improve formulary design processes and support payers in providing more effective healthcare, policy makers should consider involving commercial payers in the development of CER as well as in the creation of research and treatment guidelines.

(Am J Manag Care. 2012;18(5 Spec No. 2):SP71-SP76)

Spending on pharmaceutical products from 2015 through 2020 is projected to increase an average of 7.2% per year, exceeding $500 billion by 2020.

 It has been suggested that comparative effectiveness research (CER) may improve the management and use of treatments, leading to more effective healthcare and decreased spending over time.

 Consequently, CER has received much attention recently. However, although two-thirds of those insured in the United States are covered by private health insurance,

 most research into CER has neglected the viewpoint of private payers.

Without the input and participation of commercial payers, comparative effectiveness data may not be optimal for making complex decisions on formulary design, access restrictions, and use controls for the bulk of US patients.

This study describes how US commercial payers use different types of CER to make reimbursement decisions in different disease states. Through qualitative interviews with commercial payers, we aim to provide useful insights into how these perspectives may improve CER.

We recruited 20 payers involved in pharmaceutical and therapeutic decision making. The sample was stratified by health plan scope (national vs regional) and respondent role (medical vs pharmacy director). Participants belonged to a diverse range of 15 different managed care organizations that collectively represented all regions within the United States. The payers participating in this study ranged from operating in 1 state to offering coverage spanning 50 states. The median number of lives covered was 2.5 million (range, <500,000 to >10 million), and the total number of lives covered totaled more than 95 million. Lines of business ranged from being 100% commercial to more than 50% Medicare or Medicaid.

We probed payers on their views of CER studies to compare the value of pharmaceutical treatments within 5 common scenarios (

). Payers were then asked to independently rate the usefulness of 6 different CER study types (

) on a scale of 1 to 7 and provide the rationale for their ratings. Ratings were averaged and stratified by respondent role and plan type; comments were transcribed and analyzed for thematic content.

We identified 4 themes in payer perceptions of CER value: 1) different CER study types are valued; 2) dissatisfaction with the current state of CER; 3) importance of payer involvement in CER policy efforts; and 4) diminished impact of CER in oncology.

We confirmed the commonly accepted belief that payers prefer randomized controlled clinical trials (RCTs) when comparing drug effectiveness for diseases in which coverage decisions can be implemented. However, payers were interested in CER study designs beyond RCTs. Payers found prospective nonexperimental studies, such as registries, to be valuable in assessing effectiveness and safety. Most payers valued retrospective analyses, such as claims analyses, more highly than RCTs when comparing real-world costs and adherence to drugs (

). Payers reflected the view that observational studies are valuable for real-world data with larger study populations to detect rare adverse events, more representative of their member populations, and able to assess resource use and overall treatment cost.

“Retrospective analyses are very useful; we’re comfortable with it and do it with our own data. Volume of data is important.” 

Generally, national payers valued retrospective analyses and modeling more highly than regional payers. Payers sometimes reviewed retrospective analyses from other research groups but preferred to analyze their own member population or review an analysis of a similar population. However, retrospective analyses require a significant amount of resources to extract and analyze large volumes of data from claims records. Regional payers often did not have sufficient resources and thus sometimes placed a lower value on retrospective analyses and were at a disadvantage in terms of application of CER.

“We’re looking at real world utilization. We would like to use a large claims database. We don’t have the resources to execute an analysis like this.”

National payers often had the resources to create, analyze, and review decision and economic models. Consequently, they were more likely to use models than were regional payers, who were sometimes less familiar with these tools.

“I’m biased against decision modeling. It is so complex with so many variables that it’s hard to translate into your situation.”

Dissatisfaction With the Current State of CER

Although payers valued and used CER, much of the available research was inadequate to inform decision making because of a lack of relevant head-to-head comparisons, perceived lack of credibility of manufacturer-funded studies, and paucity of economic data. The lack of useful CER often translated into difficulties in coverage decisions and sometimes led to inefficiencies in treatment.

“We’ve been really hungry for head-to-head data. What is tremendously important is to give clinicians and patients information that they can use to select the right treatment. It will be difficult for drugs to get any kind of first-line consideration unless they have that kind of data available.”

Throughout the interviews, payers expressed a deep suspicion of studies and models conducted by manufacturers. A few payers reported that they may discount manufacturer-funded studies entirely, because they expected intentionally biased studies to show products in a better light compared with competing drugs.

“Approximately 99% of the time [pharmaceutical-sponsored models] are skewed to make their product look better than competitors. We usually don’t even look at them.” 

“We don’t want registries funded by pharma. Biases are inherent as they select certain people that may respond better to treatment groups; government-sponsored registries are more believable.”

Given the importance of industry funding to CER, it is essential that this evidence is seen as having the highest integrity in development, analysis, and reporting so that it is used by payers. The internal and external validity of CER should be underpinned by detailing assumptions, adjusting for biases, and reporting methodology in a transparent manner to dispel suspicion of biases. CER may be more broadly used when current observational research standards are followed and research is collaborative and published in top-tier journals.

“If data was authenticated by third parties in well-established peer-reviewed journals we could look at that. We will incorporate concepts as we look at our own data, as long as methodology makes sense.” 

One of the most common concerns was the lack of cost data in CER. Payers preferred to have economic end points, such as resource use, when comparing drugs in competitive, mature markets. Although payers preferred head-to-head RCTs with economic end points, there are clear difficulties in designing and funding these studies both pre- and postlaunch. The reality is that much of the necessary comparative economic evidence will be generated in observational studies.

“[CER] has to include cost; if you don’t have cost in there I don’t know how you’re going to use that information. We need to take into consideration the clinical and financial aspects when we make a formulary decision.”

Importance of Payer Involvement in CER Policy Efforts

The Patient-Centered Outcomes Research Institute (PCORI) aims to assist patients, providers, policy makers, and purchasers in making informed health decisions by prioritizing, funding, reviewing, and disseminating CER.

The launch of PCORI, coupled with CER conducted by independent and manufacturer-based research groups, provides excellent opportunities for collaboration with payers. Partnerships with payers will facilitate communication about what CER data payers require to make more informed decisions.

“If [researchers] are going to do something, ask us what our needs are, what conclusions we want to draw. The most important thing is ask us what our needs are; don’t assume.”

Articles

To improve formulary design processes and support payers in providing more effective healthcare, policy makers should consider involving commercial payers in the development of comparative effectiveness research and creation of research and treatment guidelines.

Robert J. Nordyke, MS, PhD

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US Payer Perspectives on Evidence for Formulary Decision Making