Physicians Push for Proof of Diaphragm Pacer Benefit

by Amy Madsen on Tue, 2012-01-03 10:59
Article Highlights:
  • MDA ALS physicians suggest more evidence is needed to show that the diaphragm pacing system (DPS) provides benefit.
  • Additionally, those physicians polled said people with ALS should carefully consider the risks associated with the surgery required to implant the device; and the DPS does not prevent the need for BiPAP or other supplemental ventilation.
  • People with ALS who are interested in the DPS should review all available information about the device and then discuss it with their physician.
  • Read A Closer Look: Diaphragm Pacing System to learn more about one man's experience with the DPS.

The U.S. Food and Drug Administration approved the NeuRx Diaphragm Pacing System as a humanitarian use device (HUD), a designation given to medical devices intended for use in rare diseases. For HUD approval, the FDA requires sufficient evidence the device does not pose “an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use.”

FDA approval followed findings from a multicenter clinical trial that enrolled 106 patients with ALS and treated 86 for chronic hypoventilation at seven U.S. sites and one French site. Manufacturer Synapse Biomedical reported that study findings showed “the NeuRx DPS helps people with ALS live longer and sleep better than the current standard of care alone.”

Those who are interested in the device are strongly urged to discuss it with their ALS physician and find out as much as possible about the procedure before considering having it done. Although some physicians don’t think anyone with ALS qualifies for the DPS, because the surgery is too risky and benefit is unknown, others estimate that up to 25 percent will meet the FDA’s criteria to qualify for the device.

MDA/ALS center physicians polled about the DPS expressed some common themes.

More evidence is needed. Although the DPS has been shown to be safe in people with ALS, there remains a need for solid evidence that it actually provides benefit. Long-term follow-up must be done on those who participated in the Synapse trial, and additional well-controlled, large-scale clinical trials of the device are needed.

There are risks. Individuals must be aware that (as with any surgery, and especially when it involves someone with ALS) there are risks associated with the surgery to implant the pacer electrodes.

It is not a cure-all. The DPS does not prevent the need for BiPAP or other supplemental ventilation, nor does it stop or slow the progression of the disease.

“This is still a controversial procedure,” one ALS physician said. “It does provide some hope for ALS patients, but we must temper that enthusiasm with caution.

“While it is always optimal to have choices, it should be made very clear to patients that there is still no clear-cut choice when [considering] assisted respiration [options] in ALS.

“Patients must be provided with as much education and information as is available so they can make informed decisions on how they will approach their treatment.”

Be sure to read A Closer Look: Diaphragm Pacing System to learn more about one man's experience with the DPS.

Amy Madsen
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