Does it get much worse than this?
A story in the New York Times last week highlights the dwindling supply of the drug methotrexate, which is vital in the treatment of a form of childhood acute leukemia. The closure in November of a key manufacturing plant-without other readily available sources of the drug-means that children with a very treatable form of leukemia may go without a drug that can make a difference in their lives. And there are no immediately obvious solutions to the problem.
This is-unfortunately-only the latest chapter in a saga that has been unfolding over the past year. For a variety of reasons, the supplies of vital drugs necessary to treat cancer and other diseases have been in various periods of short supply, and we as a nation have been unable to find answers that make sense.
Maybe, just maybe, it's time to take the actions necessary to deal with the problem. Too many lives are at stake to expect otherwise.
Over the past several years, there have been increasing reports of drug shortages in the United States. Contrary to what you might expect, there are no routine mechanisms in place requiring manufacturers to notify anyone formally that a drug may be entering into a shortage phase, why the drug is entering the shortage phase, and how long it will be in short supply. So we are left with alternative mechanisms which rely in no small part on the goodwill of many sources to try to get our arms around the problem and get some answers.
In the absence of formal governmental reporting mechanisms, the private and non-profit sectors have responded by creating online information resources such as the websites of the American Society of Health-System Pharmacists and the University of Utah to give us the best information available on the supply problem. The Food and Drug Administration has a website listing drug shortages, but as they note the information provided may not be complete or timely, since there is no legislative/regulatory requirement that such information be reported. And the companies themselves may provide the information, but there can be no sharing of competitive information, since there are in fact anti-trust concerns when companies start talking to each other about business practices or issues. Legislation pending in Congress is intended to address this problem.
How did we find ourselves in this situation?
Most of the drugs in question are generics, that is drugs that have come off patent and can be manufactured by companies that apply to the FDA and demonstrate the equivalence of their product to the available "branded" alternative. In cancer treatment, many of the drugs we have used for decades fall into this category.
Over time, however, the number of generic manufacturers has dwindled, which means the supplies of some of these drugs may be provided only one, two or perhaps three suppliers. And when one company has a problem it doesn't necessarily provide a timely warning so that others can ramp up their production. And then there are the production issues involved, since several drugs can be manufactured on a single production line, such that everything has to be scheduled "in order" and in anticipation of need-which can be many months into the future.
There are issues with the raw materials that come from various parts of the globe, and there can be quality problems in the manufacturing plants themselves, as outlined in a recent United States Government Accountability Office report. Some have pointed out that the reasons for the problem are the economics of paying for these drugs under our current Medicare reimbursement policies for intravenous cancer chemotherapy given in doctors' offices. But others deny that the problem is economics.
As one who has testified at a hearing on this matter, I can tell you that at the end of that day I came away with a very distinct sense that quality must be job one. We also need better information exchange, which is currently the focus of an effort in Congress with proposed legislation to do just that. We need to address the "gray market" abuses, where unscrupulous sources prey on the hopes of cancer patients and their families by offering drugs at exorbitant costs-while unable to verify a chain of custody that confirms whether or not the drug is in fact the real deal.
The President has issued an executive order strengthening the staffing of the FDA office which deals with these drug shortages. When I attended a hearing the FDA held on this issue there was general agreement that the four staff members of that office where doing yeoman work with the resources at their disposal. Yet the problems continue, and the impact on cancer patients is enormous.
I wish I could say that there are simple solutions, but there are not. The problem is multi-factorial, and what causes a shortage for one drug may not be the cause of the shortage for another. But that doesn't make life any easier for the parents of children with cancer who may not be able to get methotrexate, or for doctors who can't treat other children or adults with other forms of leukemia because they can't get cytarabine. And ovarian cancer patients can't get Doxil, a drug frequently used in the treatment of women who have advanced or recurrent disease. The list goes on and on.
There are no secret stashes of drugs. It isn't a matter of knowing someone in a special place who can answer the call for an individual's treatment needs. It is a problem faced by many, and it is a problem that many cancer centers are finding requires a full time effort to track down supplies of a given medication on a given day to keep things moving. And with all that effort, there are still patients who can't get their treatments on the days they are scheduled, and now the specter of some patients not getting their drugs at all.
We should be better than this. We need efficient and effective exchange of information. We need a plan to accelerate production of drugs in imminent and existing shortage. We need the quality problems addressed as rapidly as possible. We need to know the root causes of the problem and we need to address those causes with all of the tools we have at our disposal.
Our children deserve the best care we can offer, as do all of our patients facing cancer treatment. The disease itself is tough enough to deal with. Whether or not the drug you need to save your life is available should not add to that burden.