The appropriate timing for outcomes research is after a product is approved for commercial marketing, FDA Commissioner McClellan told a recent conference on outcomes research sponsored by Merck and AARP.

McClellan appeared at the conference along with other stakeholders including payors, providers, government agencies and consumer groups.

The purpose of the conference was to initiate a discussion of the current science and future directions for collecting and using comparative effectiveness information (¹ 'The Pink Sheet' Jan. 12, 2004, In Brief).

McClellan appeared at the conference to talk about the appropriate use of outcomes research, not to assert any FDA mission regarding comparative effectiveness research.

The agency continues to believe that placebo controlled trials are the best standard for determining safety and efficacy, and that marketing approval should not involve any superiority standard.

FDA's contribution to outcomes research may best come from innovative approaches to information technology for post-marketing surveillance, the agency believes.

McClellan's position that the post-marketing setting is the appropriate place for comparative studies is in accordance with the position taken by the Pharmaceutical Research & Manufacturers of America in a statement on government-funded outcomes research (² Also see "PhRMA Adopts Outcomes Research Policy; Set For Cost-Effectiveness Debate" - Pink Sheet, 19 January, 2004., p. 16).

Industry is concerned that other countries use comparative effectiveness trials to delay the uptake of new medicines (³ Also see "Outcomes Research May Be “Next Frontier” In Drug Development Science" - Pink Sheet, 2 February, 2004., p. 27).

McClellan addressed the conundrum of rising biomedical innovation costs and pressure to contain health care costs at a "Value of Investment in Health Care" briefing in Washington, D.C. Feb. 11.

"I don't think this is a very sustainable situation: on the one hand, rising costs of discovery and developing new treatments, and on the other hand combined with increasing pressure - appropriately increasing pressure - to contain rising health care costs," McClellan said.

"It's not a good combination for medical innovation," he declared.

McClellan may soon be asked to take on a more direct role in assessing the value of new technologies. The Administration appears to be considering asking him to take over as head of the Centers for Medicare & Medicaid Services (see ⁴ [A#00660070023]).

The Merck/AARP roundtable, held at AARP's Washington, D.C. headquarters, was intended to be a brainstorming session to initiate an outcomes research discussion.

The roundtable included a large cross-section of stakeholders, including the Agency for Healthcare Research and Quality.

Participants did not come to any conclusions about future directions for effectiveness research, but the conversations provided input for AHRQ in taking the first step - drafting a research agenda.

AHRQ is responsible for establishing "an initial list of priorities for research" within six months of enactment of the Medicare legislation.

The legislation authorizes $50 mil. in FY 2004 for AHRQ to conduct research on drug effectiveness and outcomes, and "such sums as may be necessary" in following years (⁵ Also see "Cost-Effectiveness Makes Medicare Bill; Research Agenda Due In 6 Months" - Pink Sheet, 24 November, 2003., p. 7).

However, the funding was not included in AHRQ's FY 2004 appropriations nor was it requested in the White House's FY 2005 budget.

The Administration explained that the comparative studies provision was added to the Medicare legislation too late in the budget drafting process to make it into the February document. The Administration said it supports the effort to fund the studies.

Senate Majority Leader Bill Frist has also said that he will push for funding for the cost-effectiveness and comparative effectiveness research provision.

Outcomes Research Should Come After Approval, FDA’s McClellan Says

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