WASHINGTON — Post-marketing surveillance of products by the FDA is clearly coming to the forefront as more and more drugs and devices are being either withdrawn or found to have serious risks, and it is becoming obvious that the agency does not have sufficient resources for thorough post-market review. And what happens in the drug sector soon seems to appear in the device arena.

A Senate hearing last month focused on a report from the Institute of Medicine (Washington) that charged that the FDA is failing in the application of science to drug approvals and post-market assessment. And this discussion was combined with an evaluation of S. 3807, which is titled "Building a 21st Century FDA: Proposals to Improve Drug Safety and Innovation," also indicating the need for upgrading the agency's efforts in post-market surveillance.

Speaking on behalf of IOM, Sheila Burke, who co-chaired the institute's panel that produced the report on the FDA, said that the recommendations constitute "a lifecycle approach" to drug evaluation — from initial regulatory filings to an understanding of effectiveness in the market place. She said that this is not a new concept, but a concept only "implemented in a limited and fragmented manner."

Burke acknowledged that absolute product safety is unattainable, remarking that "delaying approval until absolute safety has been established" and pulling a product at the first hint of problems "are simply not options." Still, she argued that the culture at the agency's Center for Drug Evaluation and Research "is at times dysfunctional," blaming "turnover and instability at the commissioner's office." She said that the agency might be better served if the post was assigned for "a six-year fixed term."

Sen. Mike Enzi (R-Wyoming) suggested that the FDA has its share of organizational unhappiness but noted that this may be "because we don't have an FDA commissioner." He said that the acting commissioner, Andrew von Eschenbach, "left a job he loves ... for a thankless job" at FDA and called on the Senate to get past partisan bickering and put von Eschenbach in place.

Sen. Ted Kennedy (D-Massachusetts) noted that while FDA sees to the health and safety of 300 million Americans, he pointed to recent events indicating that "the FDA itself needs treatment." The former and future chair of the committee pointed out that the agency's budget amounts to less than $6 per year for each American citizen.

User fees found no more welcoming an audience at IOM than at industry gatherings. "A substantial and sustained financial effort is needed" to enable the FDA to roll out the recommendations in the IOM report or in the Enzi/Kennedy bill [S. 3807], but "we prefer that the additional funding … come from appropriated funds," Burke said.

As for the notion of creating a separate body to review drug safety, Burke said "we do not believe … setting up a freestanding" office for drug safety is a sensible way to go about the task of beefing up post-market tracking "because it is inconsistent with the lifecycle approach" advocated by IOM. Burke also noted that the IOM believes the lifecycle approach involves tracking a drug until it is no longer being used.

Kennedy asked Burke how important it is to pass S. 3807. She replied that it is "enormously important" and that the agency "is starved for resources." She pointed to the adverse events reports system as an example of a function that collects quantities of data that the FDA is not staffed to fully analyze. And she made the case for inserting "safety staff earlier in the approval process."

Burke also said that "the agency needs far greater clarity in its authority," suggesting that one of the agency's beefed-up powers should include the ability to "call unilaterally for changes in labeling." The IOM's aversion to increased reliance on user fees is not absolute.

Study: Wheelchair cuts not cost-effective

WASHINGTON — Attempts by Congress and the Centers for Medicare & Medicaid Services to rein in Medicare costs always face significant pushback, often relying on conflicting statistics. Entering the fray with its set of data, the American Association for Homecare (Alexandria, Virginia) last month released a study of the impact of the impending reductions of per-unit reimbursement for power mobility, with a focus on the K0011 category of powered wheelchairs.

Economic consulting firm RRC (Bryan, Texas) conducted an economic analysis of the 30% reduction in reimbursement, and the numbers argue for suspending the cuts and building a case that the powered mobility industry could sustain severe damage should the cuts hold. Clifford Fry, executive vice president of RRC, author of the report, said that the CMS numbers are "below market price" and that, as a result, there may be a "massive short-run shutdown of supplier firms as they escape the costs of providing services for which they will not be reimbursed."

According to his numbers, "there may be declines on the order of 30% to 50% in access to quality power mobility" due to the loss of "at least 1,500 power mobility suppliers from major city markets" and an unspecified number from rural markets. And Fry said that the regulations could result in "a net cost on Medicare in the range of $2.7 billion to $9.5 billion" between 2007 and 2015 "due to higher expenditures for hospitalization, physician services and home healthcare services."

Hence, Fry urged CMS to rely on "market forces, not price controls" because the latter "will impair the functioning of market forces and decrease access to power mobility." Fry stated that Medicare expenditures for the K0011 reimbursement category for powered wheelchairs rose from roughly $156 million in 1998, for just over 46,000 units, to more than $663 million in 2005 for about 175,000 units. These statistics yield a per-unit cost in 1998 of about $3,383 and in 2005, slightly more than $3,786.

Other sources provide different numbers for the K0011 category. In congressional testimony in 2004, Dara Corrigan, then acting principal deputy inspector general at the Department of Health and Human Services, said that Medicare coughed up $1.2 billion in 2003 for K0011 purchases, a significant difference from the almost $898 million cited by Fry. Both Fry and Corrigan cited fraud as important components of the ballooning K0011 layouts. Corrigan said that a 2001 audit disclosed that 31% of the audited claims "did not meet Medicare's coverage criteria for any type of wheelchair" and that another 45% met criteria not for K0011, but "for another, less expensive mobility device."

Fry told Biomedical Business &Technology that routine tracking of applications by provider and by region might have caught the fraud seen in 2002 and 2003. And he projected the CMS price control proposal as "very disruptive" to the marketplace. He said that he has no data on the profit margins enjoyed by the various companies, most of which are privately held and hence not subject to SEC disclosures. He also couldn't say whether price leadership has any influence on non-Medicare prices for wheelchairs.

Fry commented that the reduction in CMS reimbursement for powered wheelchairs could spur a market for used equipment "over time," but he was unsure as to how long the market would take to move in this direction. "These devices are durable, but there is [currently] no efficient mechanism" for bringing used equipment to market.

Benchtop-to-beside centers formed

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health said it has provided grants to develop four new Centers of Research Translation (CORTs) intended to bridge the bench-to-beside gap. It said that CORTs will bring together basic and clinical research in a way that helps translate basic discoveries into new drugs, treatments and diagnostics.

The four new centers are:

• Center for Translating Molecular Signal Pathways to Orthopaedic Trauma Care, headed by Randy Rosier, MD, PhD, chair of orthopedics at the University of Rochester (Rochester, New York), to study the biology of fracture healing and the efficacy of a potential new treatment, teriparatide, an injectable form of human parathyroid hormone that stimulates new bone formation.
• Center for Lupus Research, headed by M. Virginia Pascual, MD, of Baylor Research Institute (Dallas), to study the role of different cell types in the origin of lupus, and develop markers of disease activity and severity and look for new treatment targets.
• Center for X-Linked Hypophosphatemic Rickets Research, led by Thomas Carpenter, MD, of Yale University (New Haven, Connecticut), to study molecular contributors to this genetic form of rickets and develop treatments.
• Center for Research Translation in Scleroderma, headed by Frank Arnett, MD, professor of internal medicine in the Division of Rheumatology at the University of Texas Medical School (Houston), to study the molecular basis of scleroderma to understand its underlying causes using functional genomics and gene networks.

CORT grants are a new funding mechanism for NIAMS, and require centers to encompass at least three projects, including one clinical and one basic research study.

Consumers catch on to healthcare dilemmas

WASHINGTON — A November briefing on public opinion regarding modern healthcare revealed some apparently contradictory ideas about healthcare.

Daniel Gaylin, the executive vice president of the National Organization for Research at the University of Chicago, said that his organization's survey showed that "80% of respondents think that the system is in trouble." "A substantial majority want a system that expands coverage and increases choice, but does not increase cost," Gaylin noted. But he noted that while roughly half of respondents say that mandatory enrollment is a good idea, only one in four of the uninsured are of the same opinion.

Another feature of the data that policy makers might find interesting is that roughly nine in 10 feel that the cost of insurance should not vary by healthcare status, but "the majority of respondents say smokers should pay more," an attitude that was echoed when it comes to those who are overweight, Gaylin noted.

Despite the suggestions of political turmoil seemingly embodied in the just-completed congressional elections, "[t]here was surprising commonality between Republicans and Democrats" Gaylin remarked. The support for mandatory enrollment was at 48% for registered Republicans, and Democrats supported the idea by 63%. Independents came in last, but by a still-substantial portion at 43% (the data table indicated that only a yes or no answer was available to respondents, opening the question of how the numbers might have tumbled out if "don't know" was an option).

Marjorie Ginsburg, the executive director of Sacramento Healthcare Decisions (California), said that her research into consumer preferences, which employed an Internet chat board, suggested that consumers are aware that simply expanding the current base of coverage to all residents of the U.S. "is going to be extremely difficult." She said that in the project, designed to elicit some ideas about what sort of basic coverage consumers might opt for in a limited insurance scenario, "a number of values came to the fore."

The participants, after several rounds of virtual negotiations on the chat board, came to the conclusion that "some healthcare needs could not be covered," including some quality of life solutions. Among these were treatments for impotence and infertility, the latter of which "got the most discussion," Ginsburg said.

Many participants concluded that "the $100,000 cancer treatment" for a patient who seems unlikely to survive for a substantial period of time even with the treatment was not a compelling expense. However, "the most controversial of the 14 measures" discussed was a restriction of providers, and the controversy regarding this aspect of insurance "points to how we decide as individuals how we feel about healthcare" in contrast to how one might see an issue as a policymaker, Ginsburg said.

Mark Smith, president/CEO of the California HealthCare Foundation (Oakland), said that insurance now covers four elements of concern for the typical consumer. One is "what if I get hit by a bus?" The other three concerns address cost, availability of drugs and access to doctors. He argued that "much of the tinkering ... seeks to enroll people in a healthcare system that they cannot afford."

Smith said that little change for the uninsured is on tap unless policy makers address the question of "how can we make the underlying asset more affordable," recommending that insurers "unpack" basic healthcare services from other services in order to create a system that is workable for low-income Americans. He said that he sees "great promise in the retail clinic model" that provides after-hours care without incurring emergency-room charges, as well as increased use of telemedicine and non-physician providers.

Public meeting for e-submissions

The FDA will hold a hearing Dec. 8 on converting all its submissions to an electronic format. At the hearing, the agency will seek the public's views on issues related to creation of an electronic information exchange platform, presumably a reference to interoperability issues.

Scott Gottlieb, the agency's deputy director for scientific and medical affairs, pointed out that the agency "sits on the world's single largest repository of drug safety and effectiveness information, but so long as a lot of it remains locked away in paper archives," the data is tough to make use of without risking disclosure of proprietary information. The use of "a consistent electronic format would help FDA make more effective use of important drug information, especially in safety situations," Gottlieb said.

The Center for Devices and Radiological Health (CDRH) currently uses an electronic filing format originally dubbed the Turbo 510(k) and now is named CeSub, for CDRH electronic Submissions.

However, this limited filing process is available only for in vitro diagnostics and some radiological products and, as the name states, cannot be used to file a PMA. In keeping with the naming conventions of the electronic world, CeSub has gone through several iterations and the current version is known as CeSub v. 1.14.01.

The FDA might not be more interested maintaining such a system than it is in writing the code for the proposed electronic submission gateway. The announcement said that among the topics for discussion are the prospects for a "public/private partnership" to write the code and to manage the platform after its creation.