This article is more than 5 years old. Information may no longer be current.
Not again … but I thought the government and Pharma said the problem was fixed?
Or losing your bladder because somebody didn’t do his job.
Click Here to Manage Email Alerts
Bladder cancer is one of the commonest malignancies in Western society.
There are more than 75,000 new cases each year in the United States, and about 80% of incident cases are noninvasive (formerly called “superficial”) disease.
For decades, the standard approach to this problem has been to ensure adequate and prompt diagnosis, including careful endoscopic assessment of the bladder and urinary tracts, with meticulous biopsy and pathologic staging and grading.
Derek Raghavan
For tumors that are high grade (including carcinoma-in-situ), multi-focal or recurrent, randomized clinical trials have shown unequivocally a clinically relevant and statistically significant improvement in relapse rates, disease-free interval and freedom from cystectomy for those patients treated with the instillation into the bladder of bacille Calmette-Guérin (BCG) via urinary catheter after resection of the bladder tumor(s).
The idea is simple: BCG acts to alter suppressor/helper T-cell ratios within the bladder immune system, and essentially contributes to “rejection” of local tumor cells. In the past, other immune manipulation strategies have been tried, but BCG has always been shown to be superior in randomized clinical trials.
Other options are feasible, including the intravesical delivery of cytotoxic agents such as mitomycin C, doxorubicin, thiotepa and gemcitabine.
Level 1 evidence has confirmed that mitomycin C and doxorubicin have anticancer activity, but the level of effectiveness appears to be inferior to BCG.
Thiotepa is rarely used because it is a small molecule, and there have been reported cases of systemic absorption from a post-biopsy bladder and even subsequent occasional cases of apparently iatrogenic leukemia. Despite that, thiotepa also has spent much of the past 2 years on the drug-shortages list in the United States, so it is hard to obtain if one chooses to use it for this unlikely indication.
Gemcitabine has strong activity against invasive bladder cancer when used systemically, but the level of evidence to support its use against noninvasive disease is less strong. The bottom line is that BCG is the standard of care, proven by level 1 evidence, and has been an effective means of avoiding or delaying relapse and/or cystectomy.
The problem is that, once again — despite numerous pharmaceutical industry and government assurances — we are faced with a shortage of product.
Who cares what the reason is? Whatever the proffered explanation/excuse, the drug is unavailable for clinical use, there was no significant advanced warning, and the companies that manufacture and supply the product have publically shrugged their shoulders and said that it is a “manufacturing problem.”
What it really shows us — again — is that government filled the space with platitudes the last time we were faced with significant shortages of anticancer drugs and announced that they had taken care of the problem, but in reality the issue is still here.
The FDA tells us that federal legislation has not empowered it to act in this space. When I last checked, there were a bunch of bills stuck in the overly partisan impasse inside the beltway.
Why should we care? Apart from the fact that it is disappointing when ineffective, overly partisan government lets down the voters and taxpayers, this potentially has a huge impact for patients.
There is some evidence that systemic chemotherapy doesn’t work very well (if at all) for noninvasive bladder cancer. Even if it did have efficacy, who would want the side effects of systemic chemotherapy as an alternative to moderate local complications restricted to the bladder that characterize intravesical BCG?
Although it is clear that there is some anticancer efficacy from doxorubicin and mitomycin C, both have unpleasant side effects, and they present a different spectrum of biohazards to staff and patients. With the unavailability of the standard of care, the sad fact is that patients with noninvasive bladder cancer must now face an increased prospect of a radical cystectomy to achieve definitive control of their disease.
This is a big deal. Although we have improved the morbidity of bladder removal, it is still a tough procedure, with the risk for death, loss of potency, problems with self-image and many other issues.
Why should so many patients have to face this added concern when effective government and a responsible pharmaceutical industry could have avoided it by developing a strategy to ensure the production of potentially life-saving medications?
Another important issue is cost. At a time when we simply cannot afford health care as currently configured, this situation will add another quantum to the cost blow-out because of the need to do additional complex operations, the complications of those surgeries in some patients, and the use of more expensive — and potentially less effective — alternative options of medical management.
This is just another example of a passive, otherwise-focused voting public not holding their politicians accountable.
It’s an election year, and it is time to place a value on our votes. I imagine that the leading patient advocacy organization for this disease, the Bladder Cancer Advocacy Network, will be taking a position on this issue, and I hope the American Cancer Society and other advocacy groups also involve themselves. This is not a tumor-specific issue. It’s a national disgrace!
For more information:
Derek Raghavan, MD, PhD, FACP, FRACP, FASCO, is HemOnc Today’s Chief Medical Editor, Oncology. He can be reached at derek.raghavan@carolinashealthcare.org.
Disclosure: Raghavan has served as a member of the Expert Oncology Panel of Sanofi-Aventis.