Costs appear to be an almost inevitable part of the expanded federal comparativeness research program, and the UK's 10-year old cost-effectiveness watchdog, the National Institute of Clinical Excellence, offers strategic clues for how the U.S. biopharma industry should respond, experts from the law firm Sidley Austin law firm advise.
They spoke at an April 21 FDC/Windhover webinar on "U.S. Comparative Effectiveness Policy: What It Is And What NICE Can Teach Us."
Bill Sarraille, a senior member of Sidley Austin's Healthcare Practice group based in Washington D.C., noted Congress' deletion of language in an early House report on comparative effectiveness research suggesting that cost effectiveness would be part of the evaluations.
But he went on to point out some recent concrete examples of how cost concerns are creeping back into the program, which allots $1.1 billion through the economic stimulus package to HHS, the Agency for Healthcare Research and Quality and the National Institutes of Health.
As one instance, he cited the composition of the Federal Coordinating Council. The group of 15 federal officials, named in March, is charged with overseeing how the $1.1 billion is actually spent (1"The Pink Sheet," March 23, 2009, p. 26).
The stimulus law requires that "at least half" of the council members are required to have clinical expertise, Sarraille said, pointing out that that means the other half need not and those others are more likely to focus on financial issues.
He also noted that despite meetings convened to gather public input on CER issues and study topics, NIH has already circulated its research plan, which includes a number of studies incorporating cost effectiveness.
One of the NIH study abstracts notes that cost effectiveness will "guide future policies that support allocation of health resources" (2"The Pink Sheet" DAILY, March, 30, 2009). Senate Republicans pressed HHS Secretary Kathleen Sebelius on the issue during her confirmation hearings, noting that one study on NIH's plan would address "Integrating Cost-Effectiveness Analysis Into Clinical Research" (3"The Pink Sheet," April 20, 2009, p. 26).
During the webinar, Sarraille said similar topics can also be found on AHRQ's research plan.
And, in the bigger picture, Sarraille pointed to the context in which the CE program has come about.
"'Value' is a central rhetorical element of the call for [health] reform in the Obama administration," Sarraille observed. The goal of expanding health care coverage to the 43 million Americans now without it will be prohibitively expensive without some form of cost control, he suggested, saying that an "aggressive" coverage expansion and the current financial crisis mean there is a "premium placed on cost containment."
Congress Hopes CER Is "Magic Bullet"
And Congress is trying to isolate itself from being seen as saying "no" to patients and hopes CER is the "magic bullet" to limit spending, Sarraille said.
He and Brett Rowland, an associate in Sidley Austin's London offices, described lessons gleaned from NICE. The parallels aren't perfect since the UK's National Health Service is government-run and covers all citizens, but NICE nevertheless "offers one of the most important points of comparison," according to Rowland.
"NICE does not commission primary research such as comparative clinical trials," explained Rowland. Much of its work is instead based on meta-analyses and economic modeling.
He and Sarraille noted the U.S. CER provisions do not limit studies to clinical trials either, thus opening the door to meta-analyses of existing data.
So for those in industry hoping that the CE program represented a long-term threat rather than an immediate one (because $1.1 billion is a lot of money, but would not be sufficient to fund many head-to-head trials, and these would in any case take years to deliver a result), it's time to be disillusioned. "This program can do a lot more with $1.1 billion than it could if head-to-head trials were involved. This leveraging effect is important to keep in mind," pointed out Sarraille. "U.S. policymakers have seen the use of meta-analyses in NICE ... and specifically wrote that in" to the U.S. program.
Sarraille urged stakeholders to keep an eye on the quality of data used in meta-analyses, noting that this approach frequently results in "garbage in, garbage out."
His comments also suggested that stakeholders should continue to push for as much transparency as possible. U.S. legislators may have observed that NICE has come under fire for a lack of transparency, both in topic selection and in precisely how it measures value. But the U.S. record so far on transparency is mixed, the Sidley Austin experts said.
While there have been public meetings and reports on priorities, some of the fundamentals, such as how "effectiveness" will be assessed, and what precisely it means, remain opaque. The lack of public representation on the federal council also fuels concerns by would-be stakeholder participants.
Sarraille also said that the fact that NIH and AHRQ had research plans before the program has truly gotten off the ground raises further reservations.
Procedural Challenges May Be Fruitful Avenue
Sarraille emphasized that "right now is a critical time in understanding" how accountable the CER program will be long term, and, in the near term, what the procedures are for selecting and conducting reviews.
There have been a small number of "relatively successful" challenges to some NICE reviews, but those have focused on procedural failings and not the substance of reviews. Substantive challenges often pose a higher bar than procedural ones. For example, if the review process calls for 180 days of notice and comment, that is a cut-and-dried issue for challenge, he said, and thus stakeholders should work on a clear understanding of such procedural definitions.
In the UK, about 75 percent of reviews since 2006 have involved a prescription drug, Sarraille reported. The initial CER research agendas unveiled so far suggest the pharmaceutical focus may not be as disproportionate but will still be high, he added.
Rowland noted as well that new procedures and services tend to be overrepresented in NICE reviews, adding to the commercial hurdles for these items. And it's often not clear how the review topic was selected until the end of the review process. Given the uncertain outcome and sometimes lengthy review periods, it can often seem to manufacturers that "getting selected for review is itself a huge negative."
Melanie Senior (firstname.lastname@example.org)
This article also appeared in "The Pink Sheet" – May 4, 2009
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