Four device companies seeking Medicare hospital inpatient new-technology add-on payments for fiscal 2014 made pitches to CMS Feb. 5 on how their technologies represent a substantial clinical improvement.

CMS held its annual new-technology Town Hall at its Baltimore headquarters to hear presentations from Cook Medical Inc. for its Zilver PTX drug-eluting peripheral artery stent; NeuroPace Inc. for its RNS responsive neurostimulation system for epilepsy seizures; Abbott Vascularfor its MitraClip transcatheter mitral valve repair system; and Second Sight Medical Products Inc. for its ArgusII retinal prosthesis system.

Pharmaceutical company CSL Behring had also submitted a new-tech add-on application for its Four-Factor Prothrombin Complex Concentrate (Human) (4F-PCC), a replacement therapy for patients with an acquired coagulation factor deficiency due to warfarin and who are experiencing severe bleeding. But the firm did not make a presentation at the Town Hall.

New-tech add-on payments are intended as a temporary measure to ensure adequate payment for promising but costly new medical technologies as they enter the market. Technologies that qualify can be reimbursed at a significantly higher level compared with the standard payment rate for the procedure category they fall into. The special payments may continue for up to three years, until CMS has collected enough claims data to understand the true costs.

The threshold for getting an add-on payment is not trivial, and companies routinely run into hurdles. To qualify, a technology must meet CMS’ definition of “new;” it must have a cost that exceeds the standard Medicare payment for the applicable procedure; and it must be deemed to offer substantial clinical improvement over existing treatments or technologies.

Further, the product needs to be approved or cleared by FDA as of July 1 of the preceding year (2013). Meeting that standard remains an open question for three of the four device applicants; each of the three is still awaiting a decision from FDA.

Cook’s Zilver PTX Drug Eluting Peripheral Stent

Cook does not have that particular worry this time around for its Zilver PTX paclitaxel-eluting stent. CMS denied a new-tech add-on for the stent last year because it was not approved in time. (See Also see "Cook’s Zenith Fenestrated AAA Graft Nets Positive Payment Rulings In Medicare Reg" - Medtech Insight, 6 August, 2012..) But on Nov. 15, FDA approved Cook’s PMA for Zilver PTX, making it the first drug-eluting stent in the U.S. labeled for treating peripheral artery disease, specifically in the superficial femoral artery. (See Also see "New Products In Brief" - Medtech Insight, 19 November, 2012..)

Peripheral arterial disease affects approximately nine million Americans, according to Mark Burket, director of vascular medicine at the University of Toledo Medical Center in Toledo, Ohio, who presented at the CMS meeting on behalf of Cook.

Clinical trial safety results for Cook’s Zilver PTX showed “really dramatic and clinically important improvement for patients with this disabling disease,” said Mark Burket, University of Toledo Medical Center.

Burket reported three-year data from the Zilver PTX pivotal trial, which showed that the primary patency rate in patients treated with Zilver PTX as a first-line therapy was 68.7 percent compared to 46 percent for patients who were treated successfully with angioplasty alone, demonstrating a 42 percent reduction in three-year restenosis with Zilver PTX.

In the study, half of the 474 patients who were enrolled received the conventional standard of care of angioplasty alone, and half received angioplasty with the Zilver PTX stent. In randomization of the subgroup of patients for whom the angioplasty alone failed and provisional stenting was required, half were treated with Zilver PTX and half received a Zilver bare-metal stent.

In the main safety results, 471 patients treated with Zilver PTX from the start experienced a 83.7 percent freedom from target lesion revascularization – the predominant major adverse event seen in the study – at three years compared to 70.2 percent in patients who were either treated successfully with angioplasty or who required a provisional bare-metal stent, amounting to a 45 percent reduction by three years in target lesion revascularization for Zilver PTX patients.

This result showed “really dramatic and clinically important improvement for patients with this disabling disease,” Burket said.

A single-arm study conducted in addition to the randomized controlled trial showed results consistent with those seen in the pivotal study. At 12 months, 89.5 percent of patients treated with Zilver PTX achieved freedom from target lesion revascularization and 82.8 percent achieved primary patency. In conclusion, Burket asserted that Zilver PTX provides a substantial clinical improvement relative to previous technologies, including angioplasty, bare-metal stents and bypass surgery.

Although unsuccessful last year with Zilver PTX, Cook was awarded a new-tech add-on payment for another of its products in fiscal 2013: The firm's Zenith fenestrated abdominal aortic aneurysm graft was the only device awarded a new-tech add-on payment by CMS last year.

Argus II Retinal Prosthesis System

The Argus II, meanwhile, is expected to receive FDA humanitarian device exemption approval “any day,” as the first artificial retina available to restore vision to the blind, according to Brian Mech, VP of business development for Second Sight. Last September, FDA’s Ophthalmic Devices Panel voted 19-0 that the benefits of the Argus II implant outweigh the risks for restoring vision to the blind. (See Also see "FDA Panel: Argus II Artificial Retina’s Benefits Outweigh Risks" - Medtech Insight, 8 October, 2012..) Mech said the company expects to launch the device in the third quarter of 2013.

The device, which also received a CE mark in 2011, is specifically designed for patients with severe to profound retinitis pigmentosa, a collection of genetic eye diseases that cause light-sensitive rods and cones of the retina to degenerate and die. (See [A#01110321007].) There are 250 cases a year in the United States a year, Mech said.

The system includes a 55-electrode array that is implanted in the back of the eye, a specialized pair of eye glasses with a tiny video camera and a visual processing unit worn on a patient’s belt. Images are collected by the camera and transferred to the processing unit, which sends signals back to the glasses, where they are transmitted to the eye. The signals trigger the implanted electrode to stimulate a patient’s surviving retina cells, allowing the brain to perceive light and visual patterns.

Except for the Argus II, there are no treatments or therapies currently available for the small group of retinitis pigmentosa patients with profound visual loss, said another Second Sight presenter, Eugene de Juan, a professor of ophthalmology at the University of California, San Francisco.

Data from a prospective, single-arm, study of 30 patients at 10 centers in the U.S. and Europe to evaluate the safety and benefit of the Argus II found that the system provided substantial clinical improvement to subjects in all three domains assessed to evaluate benefit: visual function, functional vision and quality of life, de Juan reported.

About a quarter of subjects regained measurable visual acuity, among other improvements.

Furthermore, the study demonstrated device safety and improvement sustained beyond two years.

“We meet all criteria for the new-technology add-on payment, and granting this payment will be critical to ensure access to beneficiaries who have no other alternatives,” Mech said.

NeuroPace’s RNS

NeuroPace is still looking ahead to an FDA panel for its RNS System, which the company says would be the first implantable closed-loop, responsive system to treat partial onset seizures. The agency’s Neurological Devices Panel will meet Feb. 22 to consider the NeuroPace’s PMA for the implantable device.

It is specifically developed as an adjunctive therapy to reduce the frequency of seizures in patients aged 18 and older whose partial onset seizures have not been adequately controlled with anti-epileptic medications.

“We have performed a rigorous class I clinical trial … that has demonstrated substantial clinical improvements in patients who were not responsive or were not eligible for other treatments,” said Martha Morell, NeuroPace chief medical officer.

Through a cranially implanted programmable neurostimulator that senses and records brain activity through one or two electrode-containing leads that are placed at the patient’s seizure focus or foci, the device is able to detect electrographic patterns previously identified by the physician as abnormal, the company explained. The device then provides brief pulses of electrical stimulation through the leads to interrupt those patterns. The typical patient is treated with a total of five minutes of stimulation a day, according to NeuroPace.

A prospective, randomized controlled trial, in which 191 patients were implanted with the device at 32 comprehensive epilepsy centers in the U.S., met both its primary safety and effectiveness endpoints. For the latter, the trial achieved a 37.9 percent reduction in seizures with the device versus a 17.9 percent reduction in seizures without the device.

The serious adverse event rate was found to be not worse than historical comparator procedures, nor was a difference found in the adverse event rate between treatment and sham groups during blinded periods.

“We have performed a rigorous class I clinical trial … that has demonstrated substantial clinical improvements in patients who were not responsive or were not eligible for other treatments, and have shown that not only is there a reduction in seizure but there’s an improvement in quality of life, including a number of domains involving cognition and mood that are not seen with anti-epileptic medications,” Morrell said.

Abbott’s MitraClip

Abbott Vascular's MitraClip system is also PMA-pending. If approved, it will be the first mitral valve repair product available that can be delivered by catheter rather than with open surgery. However, the FDA approval process appears to be behind schedule. Abbott had previously signaled the potential for an FDA panel meeting and approval decision in 2011, but a panel has yet to be scheduled. (See Also see "Abbott Expects To Resume Shipments Of Recalled MitraClip In Several Months" - Medtech Insight, 23 May, 2011..)

Data from the firm's EVEREST II pivotal trial has shown MitraClip to have an improved safety profile compared to surgery, but to be not quite as effective as surgery in reducing mitral regurgitation. (See Also see "ACC Meeting In Brief" - Medtech Insight, 11 April, 2011..)

The MitraClip system addresses a “large and prominent cohort” of patients who do not have access to other disease-modifying therapies, said William Gray, director of endovascular services, Columbia University Medical Center, New York-Presbyterian Hospital, who spoke on behalf of Abbott Vascular at the Town Hall meeting.

Gray discussed trials done in addition to EVEREST II, including a high-risk, single-arm study called EVEREST II High-Risk Surgical Cohort, in which 78 patients were enrolled. When patients in the high-risk cohort were examined at the one-year baseline for the degree of mitral regurgitation, it was found that 82 percent of those patients had mild-to-moderate mitral regurgitation, and those findings were sustained at two years, Gray said. He added that reductions in overall left ventricular volumes, sizes and dimensions and sometimes improvement in function served as evidence that mitral regurgitation was effectively reduced.

MitraClip, which has been available in Europe since 2008 and is now available in more than 40 countries, “is [a] new medical intervention which provides substantial clinical improvement over existing standard of care for patients who are too high risk for surgery,” Gray said.

During the meeting, AdvaMed once again called on CMS to modify existing new-tech add-on policies. (See Also see "Cook Medical Makes Its Case For Medicare New-Tech Add-On Payments" - Medtech Insight, 20 February, 2012..)

“We believe that the restrictive framework for approving applications has had a chilling effect on the interest of the medical technology industry – one of the country’s most innovative industries – in submitting applications for consideration of approval,” said Richard Price, AdvaMed senior VP of payment and health care policy.

AdvaMed suggested that CMS increase the add-on payment levels from 50 percent-80 percent of the difference between the standard MS-DRG payment and the cost of the procedure with a new technology, and that the agency make the assignment of a new ICD code or FDA approval – whichever date is later – “the controlling date, for starting the eligibility window for a new technology payment.” Under current policy, the two- to three-year period of a technology’s newness is based on the date of FDA approval.

CMS will accept written comments on the new-tech add-on proposals through Feb. 26. Decisions will be included in the agency’s final fiscal 2014 hospital inpatient rule, which will be released by August.

Four Firms Vie For Medicare 2014 New-Tech Add-On Payments

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