In 2004, the FDA approved Avastin for patients with advanced colon cancer, based on studies that suggested that Avastin extended life for four months. Subsequent larger studies, however, suggested that although the drug slowed tumor growth, it did not extend life in a statistically significant way.
In contrast to the FDA, the National Institute for Health and Clinical Excellence (NICE) in the UK concluded that Avastin "would not be a cost-effective use of NHS resources."
The New York Times quoted many experts who suggested that the benefit Avastin provides is small, and - in their view - NICE is correct in its judgment. For example, Roy Vagelos, a former CEO at Merck, said that "there is a shocking disparity between value and price, and it's not sustainable."
Here are the two comments that jumped out for me with regard to our inability to get a grip on costs. I have put the key comments into boldface:
1. Dr. Winer says that when he is not sitting in front of a patient, he thinks about whether drugs like Avastin are worth it to society. But when facing a seriously ill patient, who, based on clinical trial results, might benefit - even if only a little - from Avastin along with chemotherapy, he has to think about his patient's needs. "I can't say, 'Let's not use Avastin; it's a very expensive drug and I am worried about the cost to society...'"
2. What does it mean to say an expensive drug works? Is slowing the growth of tumors enough if life is not significantly prolonged or improved? How much evidence must their be before billions of dollars are spent on a drug? Who decides? When, if ever, should cost come into the equation?
Dr. Winer's use of the word "needs" for his assessment of Avastin's role for his patient is absolutely typical medical parlance. But it's wrong. We don't have evidence that allows us to say "If we want the patient to live longer he must have Avastin." A more accurate unpacking of "needs" would be - "I desperately want my patient to live longer. I hope that Avastin might help that happen. And, in the U.S. system, I am not expected to consider the cost to society."
The second quote raises questions about cost effectiveness and then asks the key question - "who decides?" In the UK, NICE decides, and is given that responsibility by the public, via the political process. In the US, no one is authorized to decide. The result is that cost-effectiveness is debated in the classroom and the media, but not in decision-making venues.
Only in the US is it possible to ask "when, if ever, should cost come into the equation?" with regard to a drug that produces ambiguous benefits at best for which billions of dollars are spent.
The correct answer to the question is that cost should always come into the equation. Every expenditure has an opportunity cost. Rather than anguishing as to whether it is ethically acceptable to consider costs, we must learn that good ethics requires us to consider opportunity costs in spending shared resources.
The practical question is - how do we, as a society, accomplish this learning? Moralizing won't do the job. If any readers see a way of advancing our public understanding without creating global budgets for health care, so that trade offs can't be avoided, please let me know.
Of course we could shift more and more of the cost to individuals, so that they could bring cost into the equation in light of their personal budgets. Sadly, for the time being, that's the American way!