Adopting a Framework for CER Assessment

Although the way in which comparative effectiveness evidence will be applied remains uncertain, companies will benefit from a systematic mechanism for forcing the dialogue into both early and later stages of pipeline review. While a strategy to pursue a broad market indication with limited comparative data at launch may be appropriate in some cases, the question must be asked and it must be asked early. For example, the targeted cancer therapeutic Erbitux found sustained marketplace success only after the dissemination of data demonstrating superior efficacy in an identifiable subgroup of patients with the wild-type KRAS mutation in their tumors. A framework for considering these issues early—both at the category level and the individual product level—will prove increasingly useful.

The likelihood of evidence review and other forms of comparison that may affect a product's commercial success rely on two factors: 1) the potential budget impact of the therapy in question and 2) the availability of comparable alternative therapies. The former creates the incentive for payers to measure, even if cost is kept nominally out of the equation, and the latter creates the opportunity for action. Thus we see that chronic and highly prevalent diseases such as hypertension, with many available therapies showing some degree of efficacy, have already been the subject of comprehensive evidence review from bodies such as AHRQ. However, because the generic mechanism has created a vehicle for cost control in mature and relatively undifferentiated categories, the practical focus of much evidence review will be associated with the diseases experiencing the most innovation, where newer therapies are expanding treatment at higher cost.

Figure 1. CER Assessment Framework Applied to Selected Competitor Groupings

In general, the further "north" or "east" on the graph a disease may fall, the more important the innovator's comparative effectiveness strategy will be. The need to demonstrate superiority to standard of care, a focus on health outcomes rather than intermediate measures, and selection of patient subgroups in which evidence is stronger are all hallmarks of development requirements in these diseases. Areas such as diabetes and rheumatoid arthritis (RA) have become intensely demanding for innovators. Recent launches such as Onglyza in diabetes and Simponi in rheumatoid arthritis have resulted in slower-than-anticipated uptake. These products have good data but lack the comparative superiority that is becoming essential to gain share over incumbents in these categories. Not surprisingly, biologics for inflammatory diseases such as RA also topped IOM's list of priority drug classes for comparative effectiveness review last year. At the other end of the spectrum, certain orphan diseases such as Gaucher's and Fabry disease present a combination of limited potential budget impact and an absence of alternatives. Both factors offer greater insulation from the effects of CER initiatives.

Figure 2: CER Assessment Framework Implications for Developers