Nearly one year after Columbia researchers announced that a heart pump extends the lives of terminally ill heart patients, the Food and Drug Administration approved the permanent use of the pump in some patients with end stage heart failure, the first pump to gain such approval in the United States. The FDA estimates 20,000 to 30,000 additional patients may benefit from the device.
For Dr. Eric Rose, the leader of the pump's clinical trial, the FDA approval marks the end of the beginning for the field. "The decades of work spent on the development of the pump have been more than worth it," says Dr. Rose, chairman of surgery at Columbia Presbyterian Medical Center and Morris and Rose Milstein/Johnson & Johnson Professor of Surgery, who began working with external pumps in dogs in 1977. "The pump will now be available to the sickest of the sick across the country."
The device, the HeartMate VE, takes over the pumping of a weakened left ventricle and sends the blood to the aorta, which distributes it to the rest of the body. The heavy pump, about the size of a personal CD player, is implanted in the abdomen and connected to the left ventricle and aorta. A narrow tube also runs from the pump through the skin to the pump's controller and battery outside the body.
Previously, FDA allowed implantation of such left ventricular assist devices only as a temporary measure for patients waiting for a heart transplant. The new approval allows surgeons to implant the pump in patients who are terminally ill but not eligible for a heart transplant because of age or other serious medical problems.
Results from the three-year, multi-center clinical trial, published last November in the New England Journal of Medicine, were used in winning FDA approval, according to executives at Thoratec Corp. of Pleasanton, Calif., the company that manufactures the device. Columbia researchers showed the one-year survival rate of end-stage heart failure patients with the pump was 52 percent, twice as high as the 25 percent survival rate of patients on drug therapy. Just as important, the research also showed that patients with the device felt better than the patients on medication and some were able to return home after lengthy stays in the ICU.
The FDA approved the heart pump only for patients who are not eligible for heart transplants because the device may cause serious complications. Infections acquired from the skin-piercing tube and mechanical failure of the pump caused most of the deaths in the trial. Nevertheless, Dr. Rose says future improvements in the pumps and medical care will make the pumps acceptable for patients who have less severe forms of heart failure.
"The experience we gain taking care of a larger population of pump recipients will lead to better devices and better management of patients so that patients will live longer," Dr. Rose says. "Eventually we will use the pumps in patients who are not so ill, but now the devices are not good enough."
Dr. Yoshifumi Naka, Herbert Irving Assistant Professor of Surgery and director of the Mechanical Circulatory Support Program at NewYork-Presbyterian Hospital, says he will soon test a smaller pump containing fewer moving parts, which should make the device more durable.
But currently, most end-stage heart failure patients now eligible for a permanent pump will not be able to afford the $206,000 procedure until the Centers for Medicare and Medicaid Services approve reimbursement.
In a few months, Dr. Rose, with Dr. Annetine Gelijns, associate professor of surgery and public health, and Dr. Alan Moskowitz, associate professor of clinical medicine, clinical public health, and surgery, both of Columbia's International Center for Health Outcomes and Innovation Research, which analyzed all the data from the trial, will present the trial data and cost analysis to the payer community. Dr. Gelijns and Dr. Moskowitz say the pump's cost is similar to the cost of a heart transplant, which society has already accepted as reasonable.
"We're very optimistic about getting coverage," Dr. Rose says.
In the future, heart failure therapy may look a lot like kidney failure therapy today. "Kidney dialysis was first envisioned as a bridge to kidney transplant," Dr. Rose says. "Now there are 300,000 people on dialysis and only a few thousand receive transplants. The pumps will probably never be better than a heart transplant, but it will be an important therapy."