FDA officials followed through last month with promises supporting an earlier pledge from Commissioner Mark McClellan to speed device review times. The details of the goal, which appeared three weeks after McClellan's pledge on the agency's web site, will "reduce by 30 days the FDA time to 50% approval for both expedited and regular PMAs [premarket approval applications," according to the statement from the agency.

The 50% approval acceleration is one of several metrics the agency uses to evaluate the timeliness of premarket reviews. The metric was successful in establishing goals for drugs and biologics, noted the FDA, so the goals should complement performance measures included in the Medical Device User Fee and Modernization Act (MDUFMA) of 2002, it said.

The catch is that the goals won't be immediately noticeable to device manufacturers. In fact, the FDA isn't bound to the reduced time until Oct. 1, 2004. The goal then applies to the fastest 50% of applications approved during fiscal years 2005 to 2007. The agency is basing the goal on the three-year average of review times for the fastest 50% of approved PMAs from fiscal years 1999 to 2001, according to the FDA.

Additionally, the performance goal, as with other goals and timelines established in MDUFMA, depends on support from Congress. "The performance goal is based on the assumption that we will receive full appropriations," said Thinh Nguyen, director of the premarket approval section of the program operations staff in the Office of Device Evaluation in the Center for Devices and Radiological Health. "If the appropriations do not meet the specified levels in MDUFMA, then the commissioner will have to revisit the goal," Nguyen said.

McClellan's pledge was hailed by the Advanced Medical Technology Association (AdvaMed; Washington) and its president, Pamela Bailey. McClellan's goals for CDRH will complement the aggressive performance goals mandated in the medical technology user fee agreement, she said (see following item). Increased collaboration, more efficient review procedures and early communication of premarket requirements will shorten and increase the predictability of product development and review, Bailey added.

Bailey hopes, however, that the FDA also will focus its attention on applications that fall outside the "fastest 50%" margin. "For FDA to implement Dr. McClellan's action plan effectively, it will need the additional appropriations mandated by MDUFMA," she said. "Congress must realize the important role the agency plays in getting safe and effective technologies to patients. I urge our nation's lawmakers to allocate the needed resources to ensure the FDA has the tools and resources it needs to meet these ambitious performance goals."

Shortfall means 'worst fears realized'

Device manufacturers nearing a finish line to ready products for submission to the FDA should sprint to get the application in before Oct. 1 or face a stiff increase in user fees. The new fees, amounting to a 34.9% increase over the FY03 rates, are a structured tier of payments as part of the Medical Device User Fee and Modernization Act of 2002 (MDUFMA). The increase is due to a shortfall in the number of applications submitted during the previous year, according to an FDA statement. The budgeted amount for user fees that was not met in FY03 amounted to more than $5 million, the agency said.

The increase amounts to "our worst fears being realized," said Mark Leahey, executive director of the Medical Device Manufacturers Association (MDMA; Washington). "Last year when this issue was being negotiated, we raised our concerns and foreshadowed this very scenario, and now it's unfortunately coming true and has a negative effect on everyone in the industry," Leahey said.

Additional factors contributing to the increase include an adjustment for inflation, amounting to slightly more than $1 million, and an increase of 8.5% in revenue from user fees, which was outlined in the MDUFMA legislation, according to the FDA. The statutory revenue for the fiscal year was $25,125,000, but as of the end of June, the FDA had only collected $14,360,000, it reported. Estimates for the remaining quarter of the fiscal year are set at $5,287,000.

"The MDUFMA statute recognized the importance of a stable FDA funding from year to year to enable the agency to develop greater long-term expertise," the agency said in its statement. "Under provisions of the statute's compensating adjustment, this revenue shortfall is to be added to the revenue target for the next year. This is the largest component of the fee increases for FY 2004. For the limited time period that MDUFMA has been operational so far, the number of FY 2003 full PMA fees and the number of PMA supplements paying higher fees are running about 30% lower than expected, thus creating one reason for this year's shortfall. The numbers of 510(k) fees are coming in at the anticipated rate."

Another change in FY04 is the tiered rates for 510(k) submissions. "In FY 2004, the two-tier fee rate for 510(k)'s goes into effect for the first time," the agency stated. "FDA anticipates that about 80% of the 510(k) submitters will qualify for the small business rate in FY 2004 which, for those submitters, will amount to less than a $600 increase, for a total fee of $2,784. A majority of the medical device industry is composed of small companies that have revenues equal to or less than the $30 million small business user fee definition, thus they will qualify for the reduced 501(k) fee."

Leahey said that the compensation adjustment "is something we were adamantly opposed to from the beginning, but other players in the industry made some detrimental concessions that are starting to show today." He named the Advanced Medical Technology Association as the "other players" referred to.

The rate increase is "more than expected," according to AdvaMed, which pointed out, however, that the compensating adjustment feature was agreed to when negotiating the language in MDUFMA with lawmakers. "It is important to note that the compensating adjustment was agreed to in lieu of fees for product listing and establishment registration. Unlike user fees, these other fees would not have been tied to performance and provided no benefit to companies," AdvaMed noted in a statement.

Leahey said that the Center for Devices and Radiological Health "pointed out today that one of the reasons for the shortfall was the bundling of submissions. They attributed $154,000 in a shortfall due to the expanded definition of bundled submissions, which we were worried about." None of the shortfall, he said, "was due to the two-tiered fee structure. This situation is 100% the responsibility of provisions put into MDUFMA. We don't blame the FDA for this at all."

He said MDMA will work with Congress to gain full appropriations for the bill, adding that the shortfalls in user fees weren't attributable to the congressional appropriations. "There was no relation to the appropriations levels in this fiscal year," Leahey said. "If the appropriations aren't there from Congress for the next fiscal year, however, the [user fee] program is clearly headed in the wrong direction."

NCI in new study of partial-breast radiation

Partial-breast radiation, already an option for women in America to prevent breast tumors from recurring, should be expanded to include more women throughout North America, researchers are proposing. The treatment's endorsement could pave the way for wider use of MammoSite radiation therapy system, a treatment offered following lumpectomy in early stage breast cancer. MammoSite is manufactured by Proxima Therapeutics (Alpharetta, Georgia).

A large study, scheduled to begin this fall, is being developed to determine if the faster treatment keeps women cancer-free as long as the traditional treatment, as well as to identify which patients should receive it. The study is being developed by the National Cancer Institute (NCI), a unit of the National Institutes of Health (NIH; Bethesda, Maryland). The NCI is rushing to implement the study due to a recent article appearing in its Aug. 20 issue of its Journal of the National Cancer Institute, which showed that partial-breast radiation produced comparable results to traditional radiation over a five-year period. That study, which followed 398 women over a five-year period, compared 199 patients whose early stage breast cancer was treated with partial-breast radiation vs. 199 patients who received whole-breast radiation.

The study was led by Frank Vicini, radiation oncologist and corporate chief of oncology services at William Beaumont Hospital (Royal Oak, Michigan). "The importance of the study is that it represents, to the best of our knowledge, the largest group of women treated with partial-breast radiation and it has the longest follow-up," Vicini said in a statement.

An accompanying editorial from NCI researchers suggested the treatment option should be expanded to women who would otherwise not be able to conserve their breast. "For many medical conditions, decision-making about treatment options includes factors other than outcome statistics; now partial-breast radiation is one of those treatment options,"the researchers wrote.

NCI's goal is to launch research that would allow more women throughout North America to receive the therapy. To achieve the goal, the institute is enlisting Vicini. The NCI study hopes to answer questions concerning whether the treatment is effective for more women. Researchers plan to enroll at least 6,000 women, comparing the two radiation treatments.

More hearings set on GPO dealings

Federal agencies are taking allegations of unfair contracting practices by group purchasing organizations (GPOs) seriously by announcing a continuing investigation into those practices this fall. The Federal Trade Commission (FTC; Washington), in conjunction with the Department of Justice (DOJ; Washington), will begin a series of hearings this month to investigate hospital group purchasing organizations (GPOs) and healthcare competition law. The hearings are scheduled for Sept. 24-26 and Sept. 30-Oct. 1.

Specifically, officials will be looking at product bundling practices, lengthy manufacturer and GPO sole-source contracts, and high hospital/GPO commitment contracts.

This month's hearings aren't the first for the FTC. Last year, it conducted a workshop on GPO decision-making and how it influences manufacturer rebates, weighing cost vs. quality, and whether or not the GPO code of conduct addresses the concerns raised about GPO business practices.