Comparative Effectiveness Research: Will You Get a Seat on the Bus?

This industry keeps evolving, and this evolution defines the relationships between the pharmaceutical industry and its customers; in this case, the managed markets or payers. When the American Recovery and Reinvestment Act of 2009 (ARRA) was signed into law, $1.1 billion was earmarked for development of Comparative Effectiveness Research (CER), the study of new and available evidence to compare one therapeutic intervention against another (note: not a placebo or control group). Among other things, ARRA implemented the Patient-Centered Outcomes Research Institute (PCORI), a nonprofit, nongovernmental body that complements the CER already being performed by the Agency for Healthcare Research and Quality. The big difference here is that absent the repeal of the legislation, PCORI’s funding is guaranteed to be nearly half a billion dollars in just a few years (and it has been in existence for only one year). This means that the expectation of more head-to-head studies should be fulfilled in the coming years, and it may be possible that managed care organizations will come to expect this information before covering a new product.

An important aspect of this new environment is that pharmaceutical and biotechnology companies may need to start planning for CER and incorporating the types of outcomes studies that MCOs will demand, much earlier in the clinical trial process. Only a few years ago, standard operating procedure for the industry was to await the outcome of phase III trials before addressing the key managed care questions, like burden of illness, cost of care, value proposition, and placement on the formulary copayment structure. These variables will have to be included in phase III trial deliverables (in other words, planning will need to start during phase II and IIb trials). This could substantially change funding and study types needed for phase III research.

Not all CER will involve costs, although the implication is that by using treatments found to be most effective, we’ll get more value for our $2.7 trillion in health expenditures. Let’s assume, for example, that of two diabetes treatments, one works better or one causes less hypoglycemia than the other with the same treatment efficacy. This can help guide practice and reduce the widespread practice variation seen around the country. Importantly, P&T Committees can use this information to help them interpret the evidence and whether utilization of the more effective product should be encouraged. Or, if there is a finding that one intervention that was previously thought to work based on clinical trials by the manufacturer does not have the intended effect in real-world practice, this type of “observational” trial result can have tremendous implications for how care is provided.

At the Academy of Managed Pharmacy meeting last week in Atlanta, a couple of things were clearly stated with regard to CER: (1) Efforts to expand CER activity and reach are moving forward, and (2) the most popular way to move in this direction is through collaborative efforts. Some pharmaceutical manufacturers have already gotten on the bus. Pfizer has entered into a venture with Humana to do CER around some of Pfizer’s areas of interest, including Alzheimer’s disease and cardiovascular disorders. And the National Pharmaceutical Council has begun collaborating with other organizations--professional and governmental--to ensure it has a voice.

Perhaps the biggest concern from the pharmaceutical side regarding CER is that there will be winners and losers, and the easy mentality is to prevent “losing.” However, resistance to the concept doesn’t seem to be a viable option anymore: There is too much money being funneled toward these CER efforts, and payers are extremely hungry for CER results. It may be time to get on the bus, before all of the seats are taken.