WellPoint has released guidelines for the submission and evaluation of comparative effectiveness research that it will use in reviewing drugs for its formulary.

Issued May 20, the ¹ CER guidelines are the first of their kind to be released publicly by a health insurer. They are meant to complement WellPoint's previously published health technology assessment guidelines. The company said in April that it was working on CER guidelines to be released by the end of the year (² Also see "WellPoint Plans CER Guidance, Building On Progress With Outcomes Data" - Pink Sheet, 19 April, 2010.).

"While a few organizations have made some strides, there is little in the way of publicly available, comprehensive evaluation criteria" for CER studies, WellPoint Chief Pharmacy Officer Brian Sweet said in a news release. "Substantial literature does exist on observational study techniques and analysis methods. Our job is to judge the proficiency and methodology of the study design and its relevance to our population against these techniques and methods."

WellPoint sees the potential for CER and observational studies to provide valuable information that goes beyond the typical drug clinical trials used for gaining FDA approval.

Investment in CER is set to grow substantially with funding available through the American Recovery and Reinvestment Act and the establishment of a permanent U.S. CER oversight organization via the Patient Protection and Affordable Care Act.

WellPoint said it may use CER to assist in selecting a position on its formulary for drug products. The new guidelines will aid both the insurer and drug manufacturers in determining just how well each CER study suits the plan's needs.

Observational Studies Of "Real-World" Questions

The guidelines state that, "while randomized, controlled clinical trials remain the gold standard for producing reliable efficacy and safety data, WellPoint recognizes that there are circumstances in which RCTs alone may not be sufficient for decision-making. Accordingly, a well-conducted CER or observational study may complement RCT-based information by providing effectiveness data, or data on outcomes achieved in a 'real-world' setting."

The guidelines explain that "observational studies provide information regarding product safety, cost-effectiveness, budget impact, and the validation of key assumptions used in cost-effectiveness and budget impact models. Furthermore, WellPoint may want to gain more insight regarding drug comparisons not found in the literature, such as off-label use and use in sub-populations."

Should a drug sponsor desire to submit results of a CER study, the guidelines list 20 specific criteria relating to the study for which information should be included. These are to include not only details of the study design, patient population and results, but a discussion of potential bias, the limitations and validity of the conclusions, and the relevance of the findings to WellPoint's patient population.

WellPoint's role will be to evaluate the scientific credibility of the study and the study's relevance and/or applicability to the WellPoint population, the guidelines state. After evaluating each study, WellPoint will rate it as useful, possibly useful or not useful for its reviews.

The company tested the CER guidelines retroactively with two studies. In one case, WellPoint used the new CER criteria to determine that research comparing only two osteoporosis drugs was not useful. It then went on to conduct its own analysis comparing three bisphosphonates for osteoporosis, which led to a new formulary requirement that a patient has to try Merck's Fosamax or Warner Chilcott's Actonel before being prescribed Roche's Boniva .

- Scott Steinke ( ³ s.steinke@elsevier.com )

WellPoint's CER Guide Describes How It Will Determine Usefulness Of Studies

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