Sorting out the varying standards for pharmacoeconomic guidelines

As we provide and interpret pharmacoeconomic (PE) evaluation results for decision makers in different settings around the world, we should consider the standards being followed. And there are several of these: the ISPOR website presents a summary of international standards , grouping them into two broad categories: “PE guidelines” and “submission guidelines”.

Even the labels for the two types of guidelines suggest that there is likely to be significant variation. PE guidelines largely reflect the best technical approach for conducting an economic evaluation without a specific objective. In contrast, submission guidelines deal with a very specific objective of pharmacoeconomic evaluation—submission for formulary consideration or other coverage-related decisions.

Those reading manuscripts in peer reviewed journals or conducting studies that are intended for publication need to familiarise themselves with how the recommendations vary and what this implies for how peer reviewed work translates into actual decision making. For those who hope for a single set of standards for pharmacoeconomic evaluation, it may be disheartening to know that PE guidelines vary significantly among different countries.

Even so, PE guidelines are more likely to recommend the societal perspective (as primary), have longer time horizons, and recommend QALYs as the preferred outcome measure. Thus, without some analysis from a perspective that is less general than the societal perspective and employing less time than the ‘lifetime’ or similar horizon allowing for all effects of an intervention, the information presented in peer reviewed manuscripts will not be easily used for formulary decisions. This ultimately means analysts must perform more work in order to use the information for decision making. Additionally, the variation in standards makes reviewing international manuscripts all the more challenging.

All this variation may leave us longing for a single standard. While the community of researchers interested in and users of pharmacoeconomic evaluation might consider the pros and cons of a single standard, this would not unambiguously improve outcomes for the populations that are affected by formulary decisions as some of the variation reflects different national interests, and may be warranted.