A Medical Device Daily

The steady march of guidances from the Center for Devices and Radiological Health at FDA continued last week with the publication of a guidance dealing with PMAs for treatments of urinary incontinence. This is at least the third guidance this year published by the agency for a device, preceded by guidances for drug-eluting stents (Medical Device Daily, March 28, 2008) and total artificial spinal discs (MDD, April 21, 2008).

FDA urged all interested parties to submit comments within 90 days of the posting of the guidance Sept. 19 posting in the Federal Register, which FDA states at its web site will be Dec. 18.

The guidance document noted that the condition, depending on the precise definition, affects between 3% and 55% of the population, but that the "prevalence ... also increases with age," implying that the impact of this condition is almost certain to increase.

The agency warns would-be sponsors that urinary incontinence is a somewhat subjective phenomenon and because of this fact, studies are subject to "a significant placebo effect." Between this and the fact that a heterogeneous population makes it difficult to balance covariates between the study and the control arm of a controlled study, "we believe sound clinical study design is essential." FDA also noted its well-known preference for "a randomized, controlled trial," which the agency described as "the least burdensome means of collecting data to demonstrate safety and effectiveness."

However, the guidance states that CDRH reviewers "will consider alternatives" assuming support from "an adequate scientific rationale."

The guidance covers devices that employ electricity as well as mechanical and hydraulic forces to reduce or eliminate incontinence, and FDA recommends that a pilot study randomize patients into a the study article and a control treatment regime. Regarding pooling pilot study data with pivotal study data, the agency's position is that differences in the protocols make this impractical unless the sponsor can demonstrate "that is it statistically and clinically valid" to do so.

Some of the characteristics FDA sees as essential to the control arm are the use of the current standard of care to treat the patients and a risk/benefit ratio that is similar to that of the study device. The ability to impose a double blind is also an important consideration.

The guidance notes that sham surgical procedures, while an option, nonetheless "involve more risk than the placebo" in a drug study and hence "should only be considered when methodologically necessary." Given the difficulty of blinding the surgeon in a sham device study, a sponsor may have to engage the services of a third-party evaluator, the document states.

Among the efficacy endpoints FDA sees as appropriate are a one-hour pad weight test to determine leakage, which has the advantages of being standardized and objective, but which suffers from variability and "poor-to-moderate sensitivity." A 24-hour weight test retains the objectivity of the one-hour variant, but is not as standardized. Other outcome measures include quality of life.

FDA recommends that patients in a pivotal trial be followed for a year, and sponsors who obtain a PMA may have to check with their patients annually for an undetermined period, although sponsors may be able to transition patients from the pivotal study into the post-approval study.

The agency had little to say about the size of enrollment in the pivotal study other than to point out that enrollment will have to be adjusted to account for drop-outs and to control for "incorrect assumptions regarding the treatment and placebo effects and their variance[s]."

Part B Medicare premium flat for '09

The Centers for Medicare & Medicaid Services disclosed the fee schedule for beneficiary premiums for Part B last week, and beneficiaries got the welcome news that their payments will not go up next year. The agency said in a Sept. 19 announcement that the standard monthly premium will hold at $96.40, which will make 2009 the first year since 2000 that Part B premiums held a flat line.

CMS indicated that while it expects the cost of Part B services to rise next year, the expected increase is "offset by a substantial reduction in the premium 'margin' needed to maintain an adequate contingency reserve in the Part B trust fund account."

The agency explained that because of "legislative changes that increased Part B spending for a year after the financing had been determined for that year," the Part B portion of the Supplementary Medical Insurance trust fund was below par for the four years between 2003 and 2006. Because of this state of affairs, Part B premiums and general revenue financing were reset to compensate, and have slightly overcompensated.

CMS said "because of the more-than-adequate asset level expected at the end of 2008, no increase is needed in the Part B premium to maintain an adequate asset level for 2009, and the margin included in prior years for this purpose can be eliminated."

Caregivers get web site at CMS

Those who care for the sick at home are under enormous stress, and CMS has launched a web site to put a few tools into the hands of these anonymous caregivers in the hopes of making their tasks and their lives a bit more manageable.

The Sept. 18 announcement states that the Ask Medicare web site and an associated newsletter will give these 44 million caregivers "links to key partner organizations that assist caregivers and beneficiaries, and present personal stories from caregivers in the community." The site will also provide "support information and tools to help caregivers address common problems." The newsletter will be delivered electronically into the subscriber's e-mail box.

According to acting CMS administrator Kerry Weems, the site and the newsletter "will help provide more information to caregivers, helping to streamline caring for Medicare beneficiaries and ultimately improve their quality of life and that of their loved ones." The CMS statement also noted that the dollar value of these tens of millions of caregivers has been estimated at more than $350 billion a year. The URL for the site is www.medicare.gov/caregivers.

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