Nov 7, 2015

Clinical Trials, Big Pharma, the FDA, and Expen$ive Drugs

...turns out this is not always a great combination for patients
Many of us, including this author, are taking one or more of the new and very expensive cancer drugs in our attempt to live longer and have a better quality of life. New and expensive does not necessarily mean we will live longer and better, however. We keep doing this (at least I do) because there is not much in the way of alternatives. We seem to be at the mercy of the most powerful companies in the U.S.

In a new book, Ending Medical Reversal: Improving Outcomes and Saving Lives, Dr. Vinay Prasad looks at ever increasing prices, secretive clinical trial results, questionable procedures, and low drug effectiveness in dozens of the new cancer drugs on the market. Dr. Prasad is an Associate Professor of Medicine at OHSU.

Here are some excerpts from Dr. Prasad’s book in his own words:
"Cancer drugs are outrageously expensive; but they are worth every penny, right?
When you pay a lot for something, you want it to be great. This is true of a new car, a new computer, a new pair of shoes—but it doesn’t seem to be true for cancer drugs.
Recently, we heard that profiteers are raising the price of old, established medicines. For many Americans, Turing pharmaceutical and Martin Shkreli have become four letter words.
But the problem of inappropriate prices is not just a few exceptional cases; it is the norm in cancer medicine.
Before we talk price tag, let’s consider what we get for our money. In medicine, there is always nuance, and it exists here. For example, there are some excellent cancer drugs. Two that come to mind are imatinib—a revolutionary pill that transformed a highly fatal leukemia into a manageable condition—and rituximab—a monoclonal antibody that has improved survival in several cancers. These drugs are expensive, sure, but no one can say they aren’t terrific. But we can say that Novartis, the maker of Gleevec, has raised the price of the drug from an already high $30,000 in 2001 to $76,000 today despite the fact that it costs less than $200 dollars to manufacture a year's supply.
But at least Gleevec is a great drug. Most cancer drugs are not close to being great—they have only a tiny positive impact on longevity and often cause many side effects.
The median improvement in survival for 71 drugs approved by the US Food and Drug Administration for treating solid cancers over the last decade is only 2.1 months. And there are two good reasons to assume that this remarkably short reported extension is in fact an overestimate. First, the average age of cancer patients in the real world is much older than those included in the studies submitted to the FDA —60% of cancer patients are over 65, but they account for just 36% of patients in key trials. Older patients—who are frailer and have other medical problems—experience more side effects from cancer drugs and less benefit.
Second, the majority of cancer drugs approved by the FDA are not included in the 2.1 month figure, because their effects on overall survival are unknown. Amazingly, most new drugs (2/3s) are approved on the basis of improvement of a surrogate endpoint—e.g. a new drug shrinks a tumor on a CT scan. We don't know that this shrinkage actually translates into a longer or a better life.
So, the efficacy of most cancer drugs is minimal and may be even less in the real world. How do we then explain why they are already so ridiculously expensive and increasing in cost at a rate of 10% a year. Cancer drugs now routinely cost more than 100,000 dollars for one year of treatment.
One way doctors measure the value of drugs is to ask how much is the total drug cost to get us 1 year of quality life. Since most drugs just add a few weeks or months, you have to treat many people to get just one year of extra life. By this measure, most cancer drugs are outrageously expensive. A new breast cancer drug costs ~$700,000 for a quality adjusted life year, and a new colon cancer drug costs $900,000. And these figures apply only if you give the drug the benefit of the doubt, and assume it works as well in the real world as it does in FDA studies.
Not surprisingly, Pharma is our most profitable industry—double digit returns ranging from 10% to 42% are routine, despite the overall disappointing benefits of their products.
Reasonable people would say it’s fine to make a tidy profit when you make a great drug—a drug like Gleevec. But we might also say it doesn’t seem fair to keep raising its price over time, as the cost to make it is trivial, and the research and development are already done. Steep price increases feel like profiteering.
Reasonable people might also say that it doesn’t seem right to pay so much for drugs that add so little—and only add that little bit under just right conditions. Especially when the costs to make drugs are so low and profits are so high.
What prevents us from being reasonable people? That is not just a million dollar question, but a tens of billion dollar one. It involves rules that prevent Medicare from negotiating the price of drugs and the intricacies of the patent system. Solutions won’t be simple or easy, but the price of cancer drugs is way too high, and we get far too little for it. The time for reform is now."
As someone who is about to embark on yet another expensive cancer drug experience I applaud Dr. Prasad. I wish him well and hope he can convince enough of the right people (maybe even Congress) to take another look and maybe even take action to decrease drug costs. My life, and many of yours, may depend on it.

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To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker