“The nation has made a commitment to CER,” Sally Morton, PhD, professor and chair of biostatistics at the University of Pittsburgh. Dr. Morton noted that law established the Patient-Centered Outcomes Research Institute (PCORI) as a source of CER funding. “The national research agenda places the interests of patients and the community at the forefront,” she said.
CER involves many steps, each with its own challenges, said Dr. Morton. The first, systematic review is “the foundation for CER,” she said. Systematic reviews identify, select, assess, and synthesize the findings of similar but separate studies and can help clarify what is known and not known about the potential benefits and harms of drugs. It can be difficult to choose among several existing different standards for conducting systematic reviews, she said.
Systematic reviews must assess both benefits and harms of interventions, said Dr. Morton, adding that evaluation of harms can include consideration of both randomized clinical trials and observational studies. Institute of Medicine standards state that observational studies should complement, rather than serve as a substitute for, randomized studies, but finding high-quality observational studies and synthesizing them can be difficult, she added.
Other challenges include a lack of direct evidence for comparing benefits and harms of treatments and difficulties inherent in evaluating the strength of the body of evidence for treatments. Such evaluations are imperfect processes, Dr. Morton said, recalling a study in which she asked pairs of reviewers to grade evidence from randomized controlled trials and observational studies on their own, and reach a consensus. “Even experienced reviewers didn’t reach the same conclusions on the strength of evidence,” she noted. “For CER, this highlights the importance of communication and transparency that meets the needs of all stakeholders.”
HTA’s Impact Grows
HTA—which involves risk–benefit analysis in determining if government-funded health services are safe, efficacious, and cost effective—is becoming more important in drug development, said Richard Willke, PhD, acting head of the Global Market Access Primary Care Business Unit at Pfizer, Inc.
Noting that the U.S. lags behind Europe, Canada, and other countries in use of HTA, private U.S. insurers and, to a certain extent, Medicare, now use it, too. For example, Wellpoint includes CER and observational data in its outcomes-based formulary process, and looks at “how drugs perform in the real world” by conducting internal analyses of claim data, Dr. Willke said.
Medicare uses HTA in National Coverage Determinations about controversial or expensive interventions, primarily for outpatient drug treatment covered by Part B. Dr. Willke cited a 2012 study of 195 such determinations made between 1999 and 2007. It found positive Medicare national coverage decisions were associated with fair to good quality supporting evidence, lack of an alternative intervention, and evidence of cost effectiveness.2
Looking at Particular Patients
In Europe, such decisions are made with more focus on different sorts of patients. In Britain, the National Institute for Clinical Excellence (NICE) is well known for its consideration of patient subgroup data. NICE coverage decisions about particular treatments are based on cost effectiveness in specific patient subgroups and are not always the same for all subgroups, Dr. Willke said.
