WorldHeart Will Learn Fate Of Novacor As Destination Therapy Within 90 Days

WorldHeart may ask FDA's Medical Device Dispute Resolution Panel to re-evaluate the firm's PMA supplement for the Novacor left-ventricular assist device as a destination therapy, according to CEO Rod Bryden.

On Sept. 27, FDA issued a "non-filing" letter in response to the Ottawa company's Aug. 14 PMA supplement submission. The filing was based on the 270-patient INTrEPID trial, supporting the Novacor as an alternative for end-stage heart failure patients ineligible for transplant (¹ (Also see "INTrEPID Data Supports Expanded LVAD Medicare Coverage Request" - Medtech Insight, 23 Sep, 2002.), p. 9).

"In our view, the issue is really one in which the scientific experts advising us say that the data that we have submitted is clearly appropriate and in their view it also merits approval," Bryden explained during a Nov. 11 earnings call.

"That's a scientific, not really a factual, question," he asserted, "and we will probably seek to use that [dispute] panel review process as a part of the next step if we go that next step."

The PMA supplement relies on a Bayesian statistical analysis of the INTrEPID data to show that the survival rate of end-stage heart failure patients implanted with the Novacor is significantly better than historical rates for patients treated with optimal medical management.

In the non-filing letter, FDA indicates that the company needs to clarify the issue of "exchangeability" of historical controls used in the INTrEPID study, in lieu of a randomized control cohort.

WorldHeart was scheduled to meet with the director of the Office of Device Evaluation, Daniel Schultz, the week of Nov. 11 to discuss the non-filing decision.

"In the event that the director decides not to make that change, we have a right to submit that matter for further review by the overall director of the device and radiology [center]," the CEO added.

As a last resort, "if we do not get a favorable response from the director...there is also the opportunity to have the matter reviewed by the panel that was established by the FDA for the resolution of scientific disputes," Bryden concluded.

Bryden explained that WorldHeart expects ODE to tell the company by Nov. 22 whether or not the PMA supplement will be accepted. If ODE accepts the application as filed, the company would expect action by the end of 2002, or 135 days after the original submission in August.

If the ODE director decides not to overturn the non-filing letter, then WorldHeart has 30 days to appeal to the CDRH director, who then has 60 days to respond to the appeal.

"So it's somewhere in the 90-day range that it may take before we can finally determine if it's going to get filed," Bryden noted.

Before the CDRH director reviews the company's appeal, WorldHeart will notify FDA that it may bring the issue before the dispute panel, which is presided over by the CDRH director. In that case, the deputy director, Linda Kahan, will review the company's appeal so that CDRH Director David Feigal will not be placed in the position of reviewing his own decision should the issue appear before the dispute resolution panel.

The MDDRP, mandated by the FDA Modernization Act of 1997, reviews scientific disputes between industry and FDA's device center. The eight-member panel has issued one decision in its brief history, ruling in favor of Lifecore Biomedical (4-0) in September 2001. The panel found that the firm's Gynecare Intergel adhesion prevention solution demonstrated a statistically significant treatment effect (² (Also see "FDA Acts Quickly On Lifecore’s Intergel PMA Following Dispute Resolution" - Medtech Insight, 26 Nov, 2001.), p. 3).

On Nov. 6, FDA approved Thoratec's PMA supplement for the HeartMate SNAP VE as an "alternative-to-transplant" therapy for end-stage heart failure (³ (Also see "Thoratec HeartMate Receives Long-Awaited Destination Therapy Indication" - Medtech Insight, 11 Nov, 2002.), p. 15).

By year-end, CMS is expected announce the date of an upcoming Medicare Coverage Advisory Committee (MCAC) meeting to consider the new indication. A final decision could be rendered within 60 days of a formal MCAC coverage recommendation. Both WorldHeart and Thoratec have submitted data to CMS supporting coverage of destination LVAD therapy (⁴ (Also see "Destination LVAD National Coverage Determination Referred To MCAC" - Medtech Insight, 4 Nov, 2002.), p. 27).

The Novacor has been available in the U.S., through a distribution agreement with Edwards Lifesciences, as a bridge to transplant since 1998 and in Europe under a general indication since 1994. Approximately 1,400 patients have received the Novacor under the current indications.