Compassionate use: the use of a new, unapproved drug to treat a seriously ill patient when no other treatments are available.
The primary way for a patient to gain access to a medication not yet approved by the FDA is by being a part of a clinical trial. Clinical trials are set up to learn the benefits/burdens of a study drug in a controlled setting to determine its overall utility, critical for receiving FDA approval.
Seriously ill patients are often willing to take great risks to have access to potentially beneficial medications/treatments. Designating a medication as being given for compassionate use allows a patient access to the drug without participating in a study. This can be particularly attractive to patients searching for a ‘miracle’.
sounds like a good thing, but is it really? and is it necessarily compassionate?
An op ed in the NY Times called attention to some issues around this topic: The Problem with Miracle Cancer Cures by Dr Robert Wachter.
A point of contention (with me) is that the author conflates palliative care with hospice/comfort care in this piece. But aside from that, his main thesis is an important one. Thanks to compassionate use, patients who have failed standard chemotherapy may now be eligible to start one of the new immune therapies or targeted therapies as a last ditch means of treatment. These are the new classes of drugs which have shown dramatic, almost miracle-like responses in some types of cancer (think of former President Jimmy Carter who just turned 95 and is still alive and well several years after a diagnosis of brain mets from melanoma). Critical research is ongoing to identify the best uses for these drugs and there is still much is to be learned about their benefits/ burdens and overall utility. Because of the sometime seemingly ‘miracle’ response to these drugs, there is a clamoring for more widespread availability.
So now when a patient with whose cancer continues to progress despite standard treatments, and asks their oncologist ‘isn’t there anything else?’ their oncologist may be able to offer compassionate use of one of these new therapies.
the problem
In reality, the chance of this treatment being effective in these circumstances is very small. But due to the miracle responses making their way into the lay and scientific press, many patients will try it if offered. And oncologists feel it is their responsibility to offer this potential path.
Which has consequences.
Financial costs: the drug companies may or may not be covering the cost of the medication, are likely not covering the administration costs, nor the cost of treating complications from the drugs themselves.
Health costs: contrary to how these drugs are portrayed in the press, the true extent of the side effects associated with these medications is still being worked out. They are not as benign as often portrayed. That’s why the ongoing clinical trials are so important. Although the drug approval process can seem maddeningly slow- there are good reasons for much of this process as there are significant risks with new medications. And they could even hasten death.
In my mind, what is most concerning is the activation of HOPE– for a cure that likely will not happen. So instead of shifting towards acceptance of their new reality, patients/families continue in ‘keep fighting’ mode. This typically pushes off the difficult conversations about how someone wants to spend the time they have left which can lead to the loss of opportunities to spend quality time with family/friends, to put off completing their ‘bucket list’, or whatever. And in the process, more suffering at end of life may result.
how to make this process truly compassionate
- The drug companies should have to pay for the drug and its administration and treatment of any ill-effects.
- All patients receiving a drug under compassionate use terms should be seen by a provider that is not involved with the treatment. Be it their primary care provider or a palliative medicine provider (i.e., a provider with no skin in the game who can be true honest arbiters to help patients/families weigh the benefits/burdens of potential treatments), a true conversation to help the patient think about their overall goals is critical at this time. In this way, it is ensured that the patient is not giving up more than they are gaining by going down this path.
- And because the costs of the compassionate drug and its effects are now covered by the drug company, patients who otherwise would have shifted to comfort focused care should still be able to enroll in hospice. Because the reality is that they are very likely nearing end of life, and this is an important level of extra support. If the miracle response occurs, they can be discharged from hospice- a truly win-win result.
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