It has been suggested that comparative effectiveness research (CER) could improve the management and use of treatments, leading to more effective health care and decreased spending. But most research involving CER has neglected the viewpoint of commercial payers. A new study in the American Journal of Managed Care seeks to change that.

Anthony Wang, MPH, and others at PriceSpective, a consultancy, determined how commercial payers use different types of CER study designs to make coverage decisions in different disease states. They recruited 20 payers involved in pharmaceutical and therapeutic decision making, stratified by health plan scope (national vs. regional) and respondent’s role (medical director vs. pharmacy director). The researchers probed respondents about their views of CER studies of pharmaceutical treatments in five common scenarios.

Overall, insurers preferred randomized controlled clinical trials (RCTs) when comparing drug effectiveness for diseases in which coverage decisions can be implemented. However, the value of CER was reduced when insurers were not able to manage certain diseases because of limited treatment options (e.g., orphan diseases) or political sensitivity (e.g., cancer), according to the study. In fact, the value to health plans of study designs in cancer were rated at nearly half the value of study designs in chronic degenerative diseases.

Plan executives reported being “wary of reimbursing oncology drugs that have marginal effectiveness” but “also wanted to avoid being perceived as interested only in profit at the expense of patients’ health,” says the study. Insurers were not able to use CER and were compelled to cover all oncology drugs, trying to manage oncology products through tiering, prior authorization, specialty pharmacies, and coinsurance.

The researchers point out that payers were looking to “professional and governmental organizations to develop more specific treatment guidelines to define which drugs are most effective and of reasonable cost.” Insurers would also like to see economic end points and other relevant data comparing new drugs with the standard of care.

“Our research highlights the fact that payers are constrained from utilizing CER in oncology by various factors, and it highlights the importance of using non-RCT study designs to capture certain endpoints of interest to payers,” says Wang. “If future health care costs are to be controlled, these issues will be need to be addressed.”

Value of study designs for comparing the effectiveness of drugs, by disease

1 = least valuable, 7 = most valuable

Chronic degenerative Orphan Advanced cancer
Randomized controlled trials6.475.663.58
Pragmatic clinical trials5.743.912.58
Prospective observational studies4.163.782.58
Retrospective observational studies3.662.472.13
Decision modeling2.471.751.37
Systematic reviews3.052.601.82

Source: Wang A, Halbert RF, Baerwaldt T, Nordyke RJ. U.S. payer perspectives on evidence for formulary decision making. Am J Manag Care. 2012;18(5 Spec No.2):SP71–SP76

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