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What’s Causing a Shortage of Pediatric Cancer Drugs?

While more than 250 drugs were declared in short supply in the U.S. this past year, the latest worries centered on one called Methotrexate, considered essential for children battling leukemia. Ray Suarez discusses the problem and latest developments with Dr. Peter Adamson of The Children's Hospital of Philadelphia.

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    And finally tonight, a drug shortage problem and its impact on a critical treatment for childhood leukemia.

    Ray Suarez has our update.


    There have been more than 250 drugs declared in short supply in the past year. The latest worries centered around a pivotal one called Methotrexate. Used to treat several kinds of cancers, Methotrexate is considered essential in battling acute lymphoblastic leukemia among adults and especially among children.

    ALL is the most common form of cancer in children, affecting about 3,500 kids in the U.S. each year. Late last year, one of four U.S. manufacturers of the drug was forced to shut down a plant, triggering alarms that hospitals could run out of the drug within days.

    This afternoon, FDA officials told the NewsHour and other news organizations they have a plan that will avert the shortage. But there are still questions being asked about the problem.

    Dr. Peter Adamson of the Children's Hospital of Philadelphia has been one of the leading voices in all this. He chairs a nationwide children's oncology group.

    Dr. Adamson, why did it even get to this point? Help us understand why, if one manufacturer is forced to close a plant, the remaining ones simply don't make more medicine?

    DR. PETER ADAMSON, Children's Hospital of Philadelphia: Well, I think there are a number of complex factors.

    But, quite simply, we've known for the last several years that a number of drugs have been on FDA's shortage list. And the reasons are that these are all generic, these are all injectable drugs. And the capacity of the generic industry to make these drugs is very limited. And so the supply is so tenuous, if we lose one plant, the house of cards collapses.


    The FDA for its part said this afternoon: "This issue has been of the highest priority for the FDA. As of today, Bedford" — that's the maker — "advised FDA it will release emergency supplies. This additional quantity of medicine was produced before the company voluntarily shut down, and the company has worked to ensure that drug was not impacted by the issues that led to the plant shutdown."

    What is a sufficient amount of supply? If they release this stockpiled drug onto the marketplace, what's the safety zone? Three weeks? Four week-supply?


    Well, probably at least a month's supply.

    Fortunately, childhood cancer isn't common. But we have over 200 centers that treat children with cancer in all parts of the country. And it's hard to predict when a child will be diagnosed, when they'll come in. But the drug has to be available early in diagnosis and throughout those first three months of treatment.

    So we can easily manage several days without the drug. Several weeks becomes increasingly problematic. If it were ever to extend to several months, that would definitely impact our ability to cure a number of these children.


    As you mentioned, this is now produced as a generic. The drug is more than 50 years old. How come supply problems have not been addressed in the past?


    Well, this has been a topic in Congress, amongst many sub-specialties.

    And I think solutions are going to begin to emerge. Part of it has to do with the level of reimbursement for these drugs. In general, the reimbursements are low. The generic industry hasn't uniformly been able to invest in its manufacturing plants.

    So some of the plants were superb in the 1970s and 1980s, but technology has advanced, and what we demand of our drugs today is greater than what we demanded many years ago. And when these plants simply haven't had the investment they needed — they need, then issues around safety, around purity of the drugs come into question, and plants close down.


    There's a bill now circulating on Capitol Hill that would call upon the drug manufacturers to have an early warning system, so people would be well aware if problems develop in the supply chain. Do you think this is part of an answer?


    I would say that's the first step.

    Clearly, an advance notice gives the FDA time to look for foreign suppliers, to try to help suppliers correct problems that may exist. Right now, they only hear from it in large measure when pediatric oncologists and childhood cancer centers call up the agency and say, we can't get drugs.

    It's the first step, but that's clearly not going to be the long-term solution by itself.


    As we mentioned earlier, there have been shortages over the past year of more than 200 drugs. How come that wasn't a sufficient alarm for the FDA, for the medical community and the manufacturers to get together on some durable long-term solutions?


    I think that's a really important question.

    It has been discussed. It has been discussed with members of Congress, as well as the FDA. I think in the pediatric oncology community, we've known that we have been skating on thin ice for a long time. And what we have also known, it wasn't a matter of if this was going to happen; it was really a matter of when it was going to happen.

    And it is so unfortunate that we have to put our children at risk for this kind of event in order to get the attention that this really deserves. With all that said, I think many people are working very hard to come up with solutions to a complex problem.


    Will the developments over the last 24 hours be enough? Are you reassured? Are you satisfied that no child, for instance, suffering from leukemia will have to go without Methotrexate?


    Well, Ray, I'm encouraged.

    But the Children's Oncology Group is a research network that cares for children across the country. And until we know that every center that cares for children with leukemia has drug available that they can give that day, we're not — going to maintain our concern, maintain our pressure.

    Yes, we're reassured and encouraged, but, no, we don't think we're out of the woods by any means yet.


    And, very quickly, before we go, Doctor, is missing a dose or two doses or a few days' worth a serious setback in the treatment of this disease?


    I think, over the course of a day or one to two weeks is not going to be a serious setback. It's when it extends longer than that, that we're putting our children really at an unacceptable risk.


    Dr. Peter Adamson joined us from the Children's Hospital of Philadelphia.

    Good to talk to you, sir.


    Thanks for covering this, Ray.

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