Lottery Will Determine Who Gets New MS Drug
Multiple sclerosis patients and their families cheered earlier this summer when the U.S. Food and Drug Administration approved Betaseron, the first drug ever shown to be effective in treating the debilitating neurological disease.
But those cheers turned to groans when the drug’s distributor, Berlex Laboratories of Wayne, N.J., recently announced that it only has enough of the drug to initially treat 20,000 patients, perhaps a fifth of those who could benefit from it.
Now, to ensure fairness, Berlex has organized a unique computerized lottery to dispense the new drug. It will select patients to receive the drug regardless of their age, geographic location and, perhaps most important, economic status.
The company has also established a sliding price scale in which some patients will receive the expensive drug free and others will be charged according to their ability to pay. As production is expanded, the number receiving the drug will expand until everyone has access, probably by 1995.
“This is an interesting, innovative and commendable plan,” said medical ethicist Arthur Caplan of the University of Minnesota. “It’s a very thoughtful maneuver and I think it is a very good thing.”
Stephen Reingold, vice president of the National Multiple Sclerosis Society, said it was unfortunate that the drug will not be more readily available. But he acknowledged difficulties faced by the manufacturer in making enough of the drug for mass distribution.
“It would have been wonderful if (the manufacturer) had started stockpiling Betaseron even before its tests were completed,” Reingold said. “Yet, is it reasonable for a company to make such an investment before a drug is proven useful?”
Multiple sclerosis is a chronic, often disabling disease of the brain and spinal cord that is thought to occur when the body’s immune system mistakenly attacks the sheaths that insulate nerves, causing “short circuits” that impair muscle control. It affects as many as 350,000 people in the United States, two-thirds of them women.
Until the advent of Betaseron, no therapy could impede the course of the disease.
Betaseron is the trade name for a naturally occurring immune system protein called beta-interferon that is produced by genetic engineering techniques. Scientists once had high hopes that beta-interferon, discovered in the 1960s, could prove effective against a variety of ailments ranging from viral infections to cancer. Despite those hopes, beta-interferon failed in more than 70 clinical trials.
The exception was a trial against multiple sclerosis. Researchers tested the drug against a mild form of the disease, called relapsing-remitting MS, that affects one-third to one-half of MS victims. Patients with this form suffer one to five periods of neurological dysfunction each year with spasms, tingling and paralysis that often require hospitalization, interspersed with periods of relative well-being.
In a trial on 372 patients with relapsing-remitting MS, those who received an injection of Betaseron every other day had significantly fewer relapses, the duration of relapses was shortened, and the number of hospitalizations was reduced.
Betaseron is manufactured for Berlex by Chiron Corp. of Emeryville, in the Bay Area, and neither company was fully prepared for the deluge of requests for the drug when FDA approval was given, said Dr. Jeffrey Latts, Berlex’s vice president for clinical research and development.
Because of the complexity of the beta-interferon molecule, Latts said, the production process is tedious. Large quantities will be required because, unlike most other products of the biotechnology industry, the new drug will have to be taken every other day for the rest of the patients’ lives.
And finally, the clinical trials indicated that the effectiveness of the drug is severely reduced if patients do not take it continuously, as would happen if patients had to stop taking it for short periods because they could not find it in drug stores. “No one benefits when everyone gets sub-optimal therapy,” Latts said.
Under the lottery, neurologists must register eligible patients with Berlex by Sept. 15. Over the following two weeks, a computer program will take the list of potential patients and electronically reshuffle it to randomly determine who will get the drug. Distribution of the drug will begin about Oct. 1.
Latts said the company will charge $65.96 per vial, each of which contains one dose that a patient can inject. At 182 doses per year, that amounts to $12,005 per year. Even if a patient has to pay the full price, Latts said, he or she would save money because that is still less than the typical cost of one hospitalization.
But most patients will not pay full cost. After a patient buys 10 months’ worth, Latts said, the next two months will be free. Patients with no insurance and an annual income of less than $20,000 will receive all of their doses free. Patients with incomes between $20,000 and $50,000 will pay an increasing percentage of the list price, while those over $50,000 will pay full price.
For patients whose insurance requires them to pay for prescriptions and wait to be reimbursed, Berlex will, in effect, grant a short-term loan, allowing them to charge the drug and pay for it after reimbursement.
