PCORI to Study More Head-to-Head Comparisons in 2014

At a conference last week, Patient-Centered Outcomes Research Institute (PCORI) Executive Director Dr. Joe Selby announced plans to fund more comparative effectiveness research (CER) examining head-to-head treatment alternatives. While such research could help patients understand what treatments work best for specific conditions, the National Pharmaceutical Council (NPC) cautioned that the research topics and processes should be meaningful, prioritized and transparent.

NPC has previously noted that PCORI’s research should “focus on conditions with a high burden of illness and cost such as chronic diseases and provide evidence that will facilitate good decision-making by health care professionals and patients.” In addition, the research “should encompass all health care services, including devices, diagnostics, health care delivery methods, pharmaceuticals and medical and surgical procedures, and establish priorities for research in an explicit and transparent manner.” It is unclear what research projects PCORI plans to prioritize and fund in 2014, but Dr. Selby has acknowledged that fewer of those projects will be investigator-initiated.

NPC pointed out that much is already known about how drugs work, but less is known about treatment alternatives, such as medication vs. surgery, imaging vs. CT scans or treatment provided by a specialist vs. a primary care doctor.

“CER is much more than a ‘red pill vs. blue pill’ equation,” said NPC President Dan Leonard. “Meaningful CER will consider a broad range of treatment options as well as individual patient outcomes. And while PCORI has acknowledged the need to conduct research that takes these individual treatment effects into account, we hope that will be the case with any head-to-head comparisons they decide to fund.”  

With respect to head-to-head comparisons, Leonard added that these types of assessments should be rooted in the real world and take into account biological variations, socio-economic factors and patient preferences when navigating decisions around treatment options. Whether the choice is among medications, surgery, medical devices, lifestyle changes or other treatment approaches, it is possible for CER to reflect the variability inherent in treating individuals rather than populations.

As NPC has pointed out in previous research, if CER is used to simply determine what works best on average, we may lose sight of what works best for individuals. This could lead to coverage and/or reimbursement decisions being made based on this information, which could result in poorer health outcomes and increased health care costs.

The reason for this is also simple—those patients who do not respond to the average treatment could end up costing more to treat if access is limited to other treatments that work better for them. Even if these patients are granted access to a second treatment, they likely will be forced to pay a higher copayment. For some patients, this could delay access to treatments that are deemed most effective for their condition.

“Patient differences matter a great deal. PCORI has an important role to play in funding meaningful research that answers questions patients care about,” said Leonard. “That’s why we hope that PCORI will continue to be thoughtful in its approach to funding research and engaging the entire research community in its efforts.”