Kidney Patients Skirt FDA Delay by Flying Drug in From Europe
The drug comes by mail order, a clear liquid packed in slender ampules, then carefully insulated in dry ice.
The package begins a 6,500-mile journey from a pharmacy in Switzerland, travels by Swissair to Boston’s Logan Airport and, after being loaded onto a Federal Express jet, is flown to a medical clinic in Pasadena. There, Warren Bacon, 72, who suffers from kidney disease, gets injections of a new biotechnology drug called erythropoietin (EPO) that has helped transform his life.
His wife, Nita, said that before the three-times-a-week treatments began in February, her husband “never had any energy. He’d just sit with his head in his hands most of the day,” and his life was like the “living dead.”
Now, Nita Bacon said, “The change in this man is incredible. He has much more energy. He swims, he’s started riding his bicycle, he cooks, he works in the garden. Now he may have one bad day in 6 or 10.”
Valerie Buhler, 65, another Pasadena resident who suffers from kidney disease, got her first mail-order package of EPO from West Germany in March. She is on a twice-a-week schedule, with the drug administered by her doctor. Before, “running a few errands just tired me out,” she said. “The difference has been remarkable. I don’t sleep all afternoon. I have energy and zest for life. I’m planning to take a cruise and go to Hawaii.”
Buhler pays $650 a month for her EPO supply, Bacon about $1,200. Not a penny is covered by their insurance. The drug is manufactured in the United Kingdom under license by a fledgling biotechnology company called Amgen, which has its own new EPO factory in Thousand Oaks, a mere 45 miles from where Buhler and Bacon live. But that factory might as well be in Antarctica.
The problem is that EPO isn’t legally for sale in this country--at least not yet. The drug has won rave reviews for its clinical test results, including in the New England Journal of Medicine, and in February the U.S. Food and Drug Administration was all but set to approve EPO for sale. Then a messy legal squabble erupted between Amgen and its marketing partner, postponing FDA approval indefinitely.
So Buhler, Bacon and a growing number of kidney disease patients who have had their fill of waiting are importing the drug themselves. Remarkably, it’s all perfectly legal thanks to an obscure 30-year-old FDA policy that allows individuals to import drugs that are sold overseas so long as they are for personal use and in amounts no larger than a three-month supply.
“Since my husband developed kidney problems, we’d heard about this drug. They kept telling us momentarily this drug would be released,” Nita Bacon said. The FDA’s delay “is ludicrous,” she said.
Wishes Others Could Get Drug
Said Buhler: “I can’t say I feel guilty I’m getting the drug. I just wish all these other people could get it.”
Dr. Michael S. Linsey, a Pasadena nephrologist who administers EPO to Bacon and Buhler, has been impressed by how well they feel. “It’s a very good drug,” he said. Linsey has heard about the many clinical tests of EPO. “Everyone has reported exceptional results with few side effects,” and he feels comfortable suggesting that other patients consider buying the drug by mail.
Buhler and Bacon aren’t alone. Susan Burkhart, a supervisor at a Pasadena kidney dialysis clinic, said that at a meeting last month of the California Dialysis Council about 20 clinics said they were treating patients who bought EPO from overseas.
Amgen spokesman Mark Brand said: “The Zurich airport pharmacy has quite a little EPO business going” to travelers headed to the United States.
All this activity is easy to explain. In the dialysis community, the advance word on EPO is that it offers a better life. Patients and physicians are excited because it is a drug for which there is no alternative. About 80,000 patients in this country suffer from kidney failure and need dialysis to cleanse their blood of impurities; more than 20,000 of them also need frequent blood transfusions and suffer chronic anemia.
EPO can help those 20,000 patients. A protein produced in minuscule amounts in the kidneys, EPO triggers production of red blood cells that transport oxygen throughout the body. The problem until recently has been how to obtain enough of it. In biotechnology, scientists play medical detectives who, by resplicing genes that contain the blueprints of life, can trigger laboratory production of rare hormones and proteins.
Much Energy Returns
In EPO clinical tests, 97% of the patients no longer need blood transfusions, and much of their old energy returns. Higher blood pressure is an occasional side effect of the drug, and patients still need dialysis treatments so their condition must be closely monitored. Last week, Warren Bacon had a one-day setback when fluid built up from his dialysis treatment. But for most patients, getting EPO is like pumping high test into your tank.
EPO also shows promise in combating anemia from chemotherapy and liver disease, and it’s being tested in concert with the AIDS drug AZT in hopes of minimizing anemia. Denise Gilbert, an analyst with Montgomery Securities, pegs the domestic EPO market at $450 million a year by the early 1990s; other analysts say the annual EPO market will hit $2 billion. With a dollar to be made from the drug, the action has shifted from the laboratory to the courtroom.
Amgen is a modest company that struck a deal in 1985 with a mighty one, Johnson & Johnson, to help pay for EPO research. Amgen would keep domestic rights to the EPO market for kidney dialysis patients, a J&J; subsidiary could sell EPO for any other use in this country, plus J&J; would get all rights to the European market.
Along the way, Amgen agreed to include J&J;’s medical data when it applied for FDA approval. The two partners kept squabbling about, among other things, how much EPO that J&J; would buy from Amgen and at what price. Finally, Amgen refused to file J&J;’s documents. By last winter, when the FDA was about to give Amgen the green light to sell EPO, J&J; sued. Amgen believes that J&J; was worried that doctors would start prescribing EPO for all manner of treatments and that would cost J&J; sales.
Amendment Ordered
In March, a federal judge in Delaware ordered Amgen to amend its FDA application and include J&J;’s data, and that put a brake on the FDA’s approval. Analyst Gilbert guesses that the FDA will approve EPO for kidney dialysis patients by June, but no one really knows how long it will take.
So some crafty patients have made it their business to master the FDA’s obscure import policy, which came into focus last summer when FDA Commissioner Frank Young, under pressure because of the acquired immune deficiency syndrome epidemic, reaffirmed the policy.
He was moved in part by a rush of imports of dextran sulphate, a 20-year-old Japanese drug that is used to treat high cholesterol. When some lab tests showed that the drug can slow the AIDS virus, anxious patients in the United States began ordering the drug, and soon a flood of dextran sulphate was intercepted by confused postal and customs officials.
“We allow individuals to bring in personal-use quantities of drugs available overseas, provided the drug is not on our list of things that are clearly fraudulent or dangerous,” said FDA spokesman Brad Stone.
The FDA’s contraband list contains about 40 items, including Laetrile, the controversial cancer drug made from apricot pits. But EPO isn’t on the list, and that was the window of opportunity that Warren Bacon needed.
Lost 30 Pounds
Four years ago, when Bacon was stricken with kidney disease, he lost 30 pounds in a month. “It’s been downhill all the way,” said his wife. When the EPO delays began, the Bacons turned to a friend who was traveling abroad. The friend contacted a sympathetic Swiss doctor who asked for Bacon’s medical history. Last winter, Nita Bacon said, “I received a wire from this pharmacy in Switzerland” that offered to ship the drug directly to a medical clinic. She checked with customs officials, then she sent $600 for her husband’s first batch of EPO.
Much like Bacon, Valerie Buhler has become a student of her disease. Her kidney problems were first diagnosed in 1978 and gradually her energy level dwindled. “I’d always been a very active person so it was very devastating,” she said. Her doctors mentioned EPO. “All the doctors told me it was supposed to be released by the end of last year. But I knew this thing would go on forever.”
Since last summer, EPO has been on sale in France, Germany and Switzerland, among other European countries, so Buhler got in touch with a friend at the University of Munich and eventually a German pharmacy agreed to sell her the drug. When the first package arrived two months ago, she was a bit nervous about whether the drug would really help. In a week, she felt the difference.
But Buhler hesitates before telling other patients about her good fortune. She manages to pay for her EPO supply with the help of her late husband’s pension, but for many other kidney patients, she said, buying EPO out of their own pocket would be too much. “Most people on dialysis are on Medi-Cal and it’s still a terribly expensive thing,” she said. “They live such a limited life. I don’t want to tell them how great I feel.”
Warren Bacon, a retired furniture designer, has to dig even deeper to pay for the drug. An EPO dosage varies, in part, according to the patient’s size. At 6-foot-2, Bacon needs a lot more of the drug than Buhler, who is 5-foot-2. At this rate Bacon faces a $14,000-a-year bill and he is eager not only for the FDA to hurry up and approve EPO, but hopes Medicare will pick up most of the costs for the drug.
For now, Nita Bacon enjoys receiving an occasional Swiss postcard from her friendly, distant pharmacist. “Of course he’s making a profit,” she noted. But if you love your husband and his life is fuller than in years, there is no choice. “I’ll keep buying it until it’s released here,” she said.
