Risk Evaluation and Mitigation Strategies

We think we understand risk when we talk about chances flipping a coin, but are all outcomes equally good or bad?
We think we understand risk when we talk about chances flipping a coin, but are all outcomes equally good or bad?

Earlier this month, I was approach to come and present my view as a patient to the Food and Drug Administration’s conference on Risk Evaluation and Mitigation Strategies (REMS). Prior to 2007, the FDA was tasked solely with determining whether a drug was “safe.” It was under this directive, that they pulled Tysabri, the drug I take to slow the progression of multiple sclerosis. They pulled the drug from the market because some people who took the drug died, and in fairness, death is a pretty good indication something may be unsafe. However, many patients wanted to make the same decision my wife and I eventually did, to take the drug because the benefits outweighed the risks. Not many had died on the drug, and many had benefited. Tysabri and MS were not the only drugs caught in the predicament of patients wanting a drug they felt outweighed the risks, and eventually the Food and Drug Administration Amendments Act of 2007 allowed the FDA to declare the benefits of a drug worth the risks of taking it. No longer were potential downsides of taking a medication the only determining factor for approval.

Years later, they are looking specifically at the impact of the various REMS on the healthcare delivery system and patient access. I was thrilled to present my impressions at the two day conference. In the first morning of the conference, we received the history of the REMS which the FDA is authorized to require. The strategies range from special certification of doctors, nurses and treatment delivery locations to specialized pharmaceutical dispensers to patient registries and monitoring.

When they got to the part on Patient Access and asked for questions, I asked if they thought Patients generally acted in their own best interests. I then gave the example of Tysabri and all the questions patients are routinely asked before each infusion. If we answer yes, we get no infusion until the doctors can clear us. If we say, “no, nothing new to report…” we are god to proceed. Why would we answer yes? To make matters worse, Tysabri is not approved for progressive forms of MS. However, there have been trials of it for progressive MS, and as of yet, there is no treatment approved for progressive MS. So if you transitioned to progressive MS, would you volunteer that information? The choice is between “may help” and “no help.” It is not in our interest to be honest, so how predictive do you think our answers will be? What’s more, we are setting up a situation where it is not in a patient’s best interest to tell their medical provider all of the information.

At the end of the first morning, I got to present. I went through my spiel about how poorly we all understand large numbers. Then I went into ways we deal with that inability to grasp large numbers and how I used the most common remedy for this failure, compare one large number with another. For me it was the risk of using Tysabri versus the risk of treating the most treatable kind of cancer with the treatment giving the best odds for long life (chemo for breast cancer caught early). The mortality rate for chemo is roughly 1 in 200. When I started Tysabri, my odds were estimated at 1 in 750, or 3.5 times better than if I took the most effective treatment for the most treatable kind of cancer.

Then I asked if we are sure what we are measuring when we look to approve drugs is correct in the first place. With my MS, the FDA looks at numbers of new lesions in a relatively short period of time. However, these are just spots on an MRI until a patient experiences symptoms, and the correlation between lesions and symptoms is poor. I have roughly 18 lesions on my brain and a half dozen on my spine. Yet, I work full time and run 5 miles multiple times a week. Still, one lesion near my brain stem had me in the hospital due to swallowing problems. It is not the number of lesions that matter to the patient. It is the impact on their life.

Finally, I closed recognizing the FDA has the power to regulate the availability of a treatment, and doctors have the ability to proscribe or refuse a treatment. Ultimately, it is the patient who has to make the final call on whether to pursue a treatment. Should not we do this with the best data available so that we can best assess whether the benefits are worth the potential risks? As a project manager, I have to write risk assessments all the time. However, my list of risks includes risks things may go right. That is what is taught in modern project management decision making, and yet all of the papers given to the patients with every drug speak only of the potential negative outcomes. If we are to make informed choices, how about giving us all of the known information about possible outcomes and known likelihoods?

The amazing thing to me as I finished was to see and hear some people applauding. That made for a great start to my last week as a 30 something. I sometimes find it a great boon to my sometimes tired spirits to think people take my words as meaningful. I’ll grant the final document from the two day conference mentioned only that “patients may be willing to take more risks,” but at least I think many in the crowd understood. That’s enough to make me feel I represented our position as best I could.

For more information about the conference, including a link to the presentations, go to Risk Evaluation and Mitigation Strategies (REMS)

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25 thoughts on “Risk Evaluation and Mitigation Strategies”

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