Has the Time Come for Comparative Effectiveness Research?

There has been a great deal of talk about comparative effectiveness research during the healthcare reform debate: about $1.1 billion worth. In general, the conversation has gone something like this: we can do a better job of focusing treatments, improving care and health outcomes, and saving healthcare dollars when we know what care works best.

Comparative effectiveness is not a new concept. In fact, healthcare leaders such as Gail Wilensky, PhD (Project Hope), and Sean Tunis, MD, MSc (Center for Medical Technology Policy), have been touting the virtues of comparative effectiveness research for some time. Only recently has it seemed to catch on (or at least get funding). A number of questions remain. Who will oversee such work? What outcomes do we expect from this work? How will the results be communicated? How will the results get utilized? Who will benefit from the results? In short, what will this hefty price tag get us? Many of these questions will get answered over time. What I want to focus on today is the conversation on how the results will affect each of the constituencies in the healthcare equation. Who will benefit?

The public conversation around comparative effectiveness has focused on the clinical ramifications and has tended to stay clear of the financial implications. That being said, behind closed doors the financial conversations are occurring.

There is no question that the patient will be one of the major beneficiaries of comparative effectiveness research. The benefits bestowed may be both clinical and financial. If a patient is given a medication that has been found to be more effective, the likelihood of a positive clinical outcome is improved. This outcome also may decrease long-term complications related to the condition being treated. Choosing a successful drug the first time also decreases the likelihood of a medication switch. Both the decreased need to change medications and potentially fewer long-term health complications can decrease the patient’s out-of-pocket costs. The fact remains, however, that as a society we like choice. So what happens if patients in conjunction with their physician choose a medication that was found to be less effective than some of its therapeutic peers? Will the patients be able to get that medication? Will there be financial ramifications in the form of higher out-of-pocket costs?

For patients to receive the benefits of comparative effectiveness research, physicians must have the most up-to-date information. However, in studies of physicians’ prescribing habits, it has been noted that most utilize a small number of medications. These medications are those that they are comfortable using and tend to prescribe most frequently. Many of these medications are ones that physicians learned about during their residency training and early in their professional career. Their knowledge about new medications comes from journals, their peers, and occasionally the drug representatives who visit their offices.

An effective strategy to communicate with physicians is necessary to change their prescribing habits. Information from comparative effectiveness studies must be presented in a timely manner and in an easily received and easily utilized format. One method being considered is communicating through the use of the e-prescribing or electronic medical record tools.

The payers and purchasers of healthcare (health plans, governmental entities, and self-insured employers) see comparative effectiveness data as a way both to improve a patient’s health and very importantly, to decrease the costs associated with less effective care. Today, a number of medications for the same condition are perceived as “me too drugs,” meaning that they differ little in terms of safety and efficacy. Healthcare payers rely on pharmacy benefit managers and physicians to advise them on the best, most cost-effective decision regarding medication coverage and tiering. Most often, however, these decisions are made by using a combination of clinical information and contractual agreements between the pharmacy benefit manager and the manufacturer, with few or no comparative data on the medications. In the past, accusations have been made that medication coverage decisions are made based on a bidding process by the manufacturers. Although my experience is that this is not the case, it is the hope that comparative effectiveness research will bring better information to the decision-making process. There has been a great deal of conversation regarding the effect of comparative effectiveness data on the pharmaceutical manufacturer. As we have seen during those rare times when head-to-head trials of competing medications occurred, superiority of one drug over another can have a chilling effect on the use and financial future of the medication found to be less effective. That being said, drugs found to be more effective may command a significant price premium. I have had conversations with a number of my peers who work for pharmaceutical manufacturers on issues such as when in the development process comparative effectiveness studies should occur and whether comparative effectiveness research would stifle new drugs that might be found to be highly effective for a subpopulation.

Although many questions have yet to be answered regarding both the process of comparative effectiveness research and the resultant data, I believe that the more information we have available in the healthcare decision-making process, the higher the potential quality of care will be. As Editor in Chief, I assure you—our readers—that we will stay close to these issues and keep you informed through both peer-reviewed articles and commentary as we progress down this new path in healthcare.