Imagine that you're a cancer patient. There's a highly effective drug for your condition, your doctor prescribes it, your health care covers it. Now the bad news: Even though it's approved by the U.S. FDA, it's out of stock at every pharmacy you call.
Apparently, you'll have to wait -- but try telling that to your cancer.
National data indicate that's the news more and more patients are facing, with seemingly no meaningful halt to the trend. A study released by the non-partisan Government Accountability Office (GAO) in December found that the number of drug shortages have tripled since 2006, with 2011 the highest year on record, beating the previous record holder, 2010.
Within these data is another troubling trend. Between 68 percent and 77 percent of the shortages involved injectable drugs, including biologics -- a relatively new type of medicine made from living organisms (as opposed to most prescription drugs, which are chemically-based). A good example of an injectable drug is methotrexate, which is used to treat childhood leukemia -- and for some young patients is the only effective therapy.
So why is this happening -- and what's the fix?
According to the FDA, product quality issues -- namely, microbial contamination and impurities -- are partly to blame: Mostly being injectables, biologics must be highly pure to be safe, so factories have to recall or halt their sale if impurities are found. One study indicated that up to 42 percent of the shortage is due to quality issues. The GAO study similarly placed the blame on "factory flaws and shutdowns," such as manufacturing problems.
This highlights a classic problem for the FDA: balancing drug access and patient safety. As life-saving biologics have come off-patent and drug companies seek to make imitative versions (called biosimilars), a key question has been how to ensure that the copycat versions of the drug are equally safe and effective. With biologic or biosimilar drugs, the manufacturing process is arguably the biggest indicator of whether the drug will be effective, with even minuscule changes in the process potentially having a dramatic effect on the product's safety. Thus, even if there's a shortage, the FDA must insist that manufacturers of biologics and biosimilars not cut corners. After all, it doesn't do much good to have adequate supply of a drug if the drug is unsafe or doesn't work in the first place.
There are some things that patients can do to be proactive, such as directly asking their prescribing physician about the issue, and asking their physician to be aware of any long-term supply issues, for example. But the GAO's analysis suggests that a systemic, long-term solution that ensures adequate drug supply without sacrificing safety will require Congress giving additional authority to the FDA.
The problem, according to the GAO report, is that the FDA can't fix a problem it doesn't know exists. In addition to lacking a database to track drug supply levels, the FDA has limited ability to require manufacturers to alert the FDA and the public as to impending or possible drug shortages, so the FDA is often in the dark until it's too late. When it does know about an impending shortage, it can -- for example -- temporarily open the market up to, and provide safety oversight for, imported drugs that aren't approved in the U.S. but are in other countries. It can also require that manufacturing facilities with some safety problems identify the lots that were safe and distribute those, rather than throwing out the good with the bad.
But these solutions also involve some risk: How do we accurately target the "safe" batches, and ensure that imported drugs are safe? Alongside these import efforts to ensure adequate drug supply, particularly of biologics, the FDA should ensure that adequate resources are put into drug authenticity and safety monitoring programs such as "track and trace" (to monitor the integrity of drug supply chains).
Drug shortages are a complex problem with multiple causes, not least of all manufacturing issues that can affect the safety of injectable medicines like biologics. As Congress considers how to ensure that patients have access to the life-saving medications they need, it must make sure that the FDA has not only the tools to foresee impending drug shortages, but also the tools to ensure that any biologics or biosimilars are actually safe and effective to help protect patients.
Follow John Horton on Twitter: www.twitter.com/legitscript