Framework for balancing risks and benefits

Whenever we make a decision about whether to use a particular medical treatment for a particular patient we decide whether the expected benefit for the patient outweighs the potential risks to the patient from the treatment. Sometimes the benefit is very significant and the risk is small so the decision is easy. Many times the benefit is less certain and the risks are higher and the decision can be very difficult. When the decision is more difficult the values and priorities of the person making the decision are what determine whether the benefit or the risks are more significant. One of the important aspects of respecting the autonomy of patients is to allow the values that impact that balancing of benefit and risk to be the values of the person being treated.

It becomes more complicated when the decision being made is a policy decision that impacts a large number of people. Whose values and priorities do we use to make decisions that affect a whole group of people? That is a concern faced by the FDA as it makes decisions about approving safe and effective medicines for use in the US. The decision to approve a medicine involves weighing the benefits of that medicine against the risk of harm for the medicine. They use the best scientific studies available to assess the benefits and risks of the medicine, but whose values should decide whether the risks are worth the benefits expected? That value decision is ideally made by individual patients, but how do you make it for all the people who might potentially use a particular medicine? Should the physicians, scientists and other experts that the FDA calls upon to make those decisions use their own values?

A recent article in Nature News reports on how the FDA is trying to deal with this difficult issue. They are developing a framework for how to evaluate the balance of risks and benefits for the drugs they are called on to approve. One part of that framework is a planned series of meetings with patients who are representative of those who have 20 different common diseases. They will be looking at how those patients put value on the risks and benefits of potential treatments. That will allow them to be guided by the values that patients have expressed when they evaluate drugs for those diseases.

The FDA is commonly criticized for approving drugs that turn out to have more problems than expected or being too slow at approving needed medicines or sometimes both at the same time. They should be commended for recognizing the important place that patients’ values play in the medical decision making process and trying to incorporate that into their own process for approving new drugs.

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Jon Holmlund, M.D.
Jon Holmlund, M.D.
8 years ago

I agree. This is some of what was discussed at the DIA meeting. See also my post for June 26. The techniques are getting quite sophisticated, and can yield some really interesting and useful information. Of course, you still have the payers–governments or insurers, or, should I say, you and I speaking through/being spoken for by agents–to decide what different outcomes are “worth.”