The Conundrum of Clinically Meaningful Benefit
The Conundrum of Clinically Meaningful Benefit
Dr. Miller: The problem that you are likely to have, however, is that what one person describes as benefit doesn't match what someone else says is benefit, and we need to get regulatory approval for drugs. Even if they are less expensive, they will still have some cost and someone will have to pay, and the forces above or next to the patient will be making those value-based decisions. If we make you the chief regulator and you have those patient survey data, do you shoot for making something available that matches the value list from the spectrum of Maurie's patients? Do you shoot for the high end?
Dr. Winer: This is a real challenge. In clinical practice, this is much less of a problem when you have a thoughtful doctor who has a good relationship with the patient. In those situations, doctors and patients together make these tradeoffs about disease control and toxicity, both implicitly and explicitly. The challenge is trying to sort out what kind of endpoint in a trial is sufficient to approve the drug. A drug that doesn't make you live longer and better is a drug that I don't want available.
On the other hand, it is sometimes hard to demonstrate those aspects of a drug in a trial. Sometimes we do have to compromise and accept the fact that we may be approving a drug that later on turns out to be less than ideal, which is why the drug approval process has to be flexible. We should have a lower threshold for approving drugs but a much higher threshold for keeping them approved.
Wisdom of Approving Drugs for the Wrong Reasons
Dr. Miller: The problem that you are likely to have, however, is that what one person describes as benefit doesn't match what someone else says is benefit, and we need to get regulatory approval for drugs. Even if they are less expensive, they will still have some cost and someone will have to pay, and the forces above or next to the patient will be making those value-based decisions. If we make you the chief regulator and you have those patient survey data, do you shoot for making something available that matches the value list from the spectrum of Maurie's patients? Do you shoot for the high end?
Dr. Winer: This is a real challenge. In clinical practice, this is much less of a problem when you have a thoughtful doctor who has a good relationship with the patient. In those situations, doctors and patients together make these tradeoffs about disease control and toxicity, both implicitly and explicitly. The challenge is trying to sort out what kind of endpoint in a trial is sufficient to approve the drug. A drug that doesn't make you live longer and better is a drug that I don't want available.
On the other hand, it is sometimes hard to demonstrate those aspects of a drug in a trial. Sometimes we do have to compromise and accept the fact that we may be approving a drug that later on turns out to be less than ideal, which is why the drug approval process has to be flexible. We should have a lower threshold for approving drugs but a much higher threshold for keeping them approved.