Today I would like to share with you some thoughts on the topic of the costs of cancer treatments. It is the result of a moment on Saturday morning while, in the midst of listening to hurricane coverage on television, I was scanning the pages of my morning paper. There in the headlines was the comment that the Food and Drug Administration on Friday-presumably a bit later in the day, since the article was posted online at 8PM-approved a new drug called crizotinib (Xalkori®) for the treatment of lung cancer.
The news didn't get much attention, likely because it was overwhelmed by the hurricane. But at any other time, I suspect it would have been all over the media since this drug in fact represents a breakthrough treatment for some patients with lung cancer (more on that later).
But as I read the rest of the story, I almost choked on my coffee when I saw the cost of the new treatment: $9600 a month for a medicine that consists of two pills a day. Now, to me in my world, that was news. Just off the top of my head, that seemed a pretty steep price for success.
I have blogged about crizotinib previously. It is a drug that takes advantage of a mutation called ALK found in a small minority of patients with advanced lung cancer. According to the FDA, that number ranges from 1 to 7 percent. Most of those patients who are positive for the mutation-and thus likely to benefit from crizotinib-are non-smokers with a type of lung cancer called adenocarcinoma. When the drug works, it works quickly. And patients who are symptomatic from advanced lung cancer can have remarkable responses.
According to the FDA, in studies of 255 patients crizotinib produced responses in 50 percent in one study, where the median duration of response (that is, half the patients responded for less than that time, and half responded longer) was 42 weeks. In the second study, the response rate was 61%, and the median duration was 48 weeks. And, it is important to note that in these trials, crizotinib was given to patients who had already received and failed prior chemotherapy.
Those are remarkable numbers, thus my comment that this is truly an exciting drug and represents a significant step forward in the treatment of some patients with lung cancer. No one yet knows how effective this drug might be if given earlier in the course of lung cancer treatment, perhaps as first line therapy for recurrent or advanced lung cancer or possibly even as adjuvant therapy (although I would note that other targeted therapies which treat advanced disease with some success have failed to improve survival when used as an adjuvant therapy following primary surgical treatment).
But the cost of the treatment was what surprised me. For one year, the cost of crizotinib alone would be $115,200, not including other related costs such as lab tests, scans, physician visits, and so on.
We have known about the success of crizotinib in clinical trials through presentations at scientific meetings and publications in medical journals for some time, so the approval of the drug by the FDA was anticipated. But we didn't know what the cost of the drug would be, and now we have another in a growing list of treatments that are phenomenally expensive, and certainly beyond the reach of anyone who doesn't have the means to pay, and even for many who think they do.
Although I don't have a complete list of drug costs, I suspect this one ranks right up there near the top for treatments that might be expected to have fairly widespread clinical use throughout the world. (For comparison purposes, the immune therapy Provenge®, which is used to treat advance prostate cancer that no longer responds to hormonal manipulation and extends life by a little over 4 months, costs $93,000 for a cycle of three treatments.)
There is, as always, more to the story.
Drug costs have always been a difficult subject. The traditional arguments have been that the cost of drug development has skyrocketed, and is now well over $1 billion for each successful drug brought to market. There are the costs of basic drug research and development, the costs of large clinical trials, and the fact that many drugs fail to get approval after all the testing is done because they are either too toxic or don't meet their treatment expectations.
Then there are the so-called "targeted therapies" in cancer treatment, drugs which are designed to take advantage of the weaknesses in a cancer cell and exploit those weaknesses to improve the life of the patient unfortunately afflicted with that particular cancer.
There have been several successes with targeted therapies, including long-standing ones such as Gleevec® for chronic myelogenous leukemia (and other cancers), and Herceptin® for breast cancer. Those drugs are also expensive, but not so much as crizotinib (although it should be noted that when some of these drugs are combined with standard chemotherapy drugs the total annual costs of treatment can be greater than the cost of crizotinib.)
One of the questions that has always been in the background regarding targeted cancer chemotherapy drug development is how much we would have to pay for the drugs. It's a topic that was recently addressed in a blog by Matt Herper in his blog post on crizotinib.
A little over five years ago, when thinking about this, I wrote a blog highlighting the issue: If we were successful in developing effective targeted therapies, and if we had tests which would tell us which patients would benefit, then it would be very possible that the market for those drugs may be so small that there wouldn't be much motivation for pharmaceutical companies to make the needed investments to develop and market those drugs.
On the other hand, if we had true targeted drug development, then the costs associated with developing those drugs-from the basic science aspects to the clinical trials-may be substantially less than has traditionally been the case.
Well, folks, here we are.
In the old days we would have a drug that might work for a small number of patients but we couldn't tell in advance who would benefit. So many patients would get a drug-thus spreading the costs over more people-but few would improve. Now, in the good new days, we can target who will benefit from the drug, but only a few will get it so the costs are more concentrated over a smaller population.
It is difficult for me to estimate how many people might be expected to be candidates for crizotinib in the United States each year. An editorial in the New England Journal of Medicine last October estimated that 10,000 patients may well be eligible for treatment with this drug annually. That number is not too far out of line with my own crude calculations. If every one of those patients received crizotinib-which is highly unlikely-for the median of 10 months, that would total just short of $1 billion a year.
The company that has developed and will be marketing crizotinib was quoted in the newspaper article as saying that "eligible, private-insured patients" will pay no more than $100 out of pocket a month.
I guess that's a relief, but what about people without insurance, or on Medicaid or Medicare? Remember, this is an oral drug, not one given intravenously in a doctor's office. So the coverage rules are different than traditional intravenous chemotherapy given in a doctor's office, where much of the cost is covered by public plans.
Recently, I had someone I know face this dilemma.
On Medicare, they had enjoyed excellent health throughout their lives. Never a pill, never a problem. So, although they had Medicare, they didn't have drug coverage. When they were diagnosed with advanced lung cancer, and prescribed another oral targeted therapy, they scrambled hard because they suddenly had to come up with $4,000 a month for the pills in the bottle. That was a shocker for a number of people. They had no idea about the costs of cancer treatment.
So we move forward one more step along the path that has been predictable for years: the costs of new drugs in cancer treatment are huge and going higher. Maybe that's justifiable, maybe it's not. I certainly have no way of knowing.
What I do know is the end result: how are we going to continue to offer the results of the best science we have at a cost that many people simply cannot afford?
I know that if someone I loved needed a medicine that would save their lives, I would do everything in my power to do whatever was needed to find a way to pay for that drug. But I also know that there are limits to my resources, and when the resources don't match the need, what would I do? And I also know that there are many who are less fortunate than me who may be forced with the more difficult choice of just saying goodbye.
I can't help but ask if that is a choice that anyone would want to make. Somehow, I don't think so. Yet people are doing it every day.
Our miracles aren't getting any less expensive.