Real-World Data Better than RCTs for Formulary Decisions

November/December 2008, Vol 1, No 9 - Meeting Highlights, Conference Highlights AMCP
Alice Goodman
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Real-world data on a new therapy and a userfriendly assessment tool to evaluate real-world studies facilitate formulary and reimbursement decisions, said Diana Brixner, PhD, RPh, Professor and Chair, Department of Pharmacotherapy, and Executive Director, Pharmacy Outcomes Research Center, University of Utah, at the 2008 educational conference of the Academy of Managed Care Pharmacy.

Payers need information beyond randomized controlled trials (RCTs) to make the best decisions about how to deploy limited budgets for large patient populations. RCTs are conducted in a select patient population, according to strict protocols, and the results are not generalizable to large patient populations. Supplemental observational open-label trials and postmarketing surveillance provide data on the effect of new treatments that are more relevant for managed care organizations (MCOs), such as quality-adjusted life-years, productivity, and absenteeism.

Dr Brixner summarized the proceedings of 2 roundtables that addressed issues surrounding use of real-world data in managed care. A roundtable in July 2007 included 4 MCO directors and 4 economists. The panel tackled the quality of real-world evidence, the complexity of real-world research, the lack of consistency in quality assurance and assessment in decision-making, and the time delay between accrual of real-world data and product launch. Recommendations for further study include:

  • Analysis of tools for quality assessment of observational studies
  • Development of a consolidated instrument to measures the quality assurance of these data
  • Establishment of a process to support application of a comprehensive quality assessment tool
  • Development of training for producers and users of observational studies on the consolidated tool.

"Our healthcare system is fragmented. A consolidated tool would reduce the impact of fragmentation," said Dr Brixner.

Developing a new tool for quality assurance to evaluate real-world studies is a daunting task, and is still ongoing, Dr Brixner explained. In analyzing existing checklists, participants found that none of them fulfilled the criteria for a simple-to-use, comprehensive checklist to evaluate published real-world studies. Existing checklists were either too long or too academic to support decision-making.

A second roundtable, held in March 2008, discussed how to act on the recommendations of the first roundtable. Participants agreed on the need:

  • For quality and convenience in assessment of realworld data
  • To recognize that the process of implementation of a new tool is as important as the new tool itself
  • For peer validation of a new tool
  • To educate all constituents about the tool and its implementation.

A small survey of 4MCOs found that although none of them currently uses a checklist to evaluate realworld studies, they would use a tool if it was short and concise, validated, and recommended by a national organization, Dr Brixner said.

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