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Measuring Response in Goal-Based Therapy


A discussion on how to accurately measure response to goal-based therapy and ensure patients are receiving appropriately effective treatment.


Neil Minkoff, MD: You mentioned how you measure responding. You brought up the 50% reduction. That’s something that gets bandied about a fair amount, in terms of looking for a reduction in the frequency and duration of the attacks. But Dr Nahas, could you build on that 50%? Could you add some nuance to that? When we evaluate the drugs as a former payer, we look for things like a 50% reduction, based on the studies. What are you looking at in your practice when you’re talking about goal-based therapy?

Stephanie J. Nahas, MD, MSEd, FAHS, FAAN: Yes, 50% would be great. That’s typically 1 of the secondary end points for these clinical trials, but it’s something that is easy to grab onto. Intellectually we can conceptualize getting 50% better. We see that as a round number indicating that we’re halfway there. It’s a goal worth striving for. Honestly, even 25% or 30% is pretty good, especially for someone who has 20 or 25 days on which they’re severely affected in a month. If you take away 5 or 10 of those, they’re going to be very happy.

But we keep striving for 50%, and these other exploratory end points of how many people got 75% better, or maybe even had a migraine-free month during the trial, which is a 100% response. You’ll see that the numbers pan out. Depending on the trial and the population, the difference may be pretty small. But these are achievable goals. We can certainly strive for them, but not right off the bat. Fifty percent better is something I’ve been telling my patients is our goal for a long time, even before any of these monoclonal antibodies came out. I’ll say, “Let’s focus on what that means for you. Is it 50% fewer days with migraine symptoms? Is it 50% lowering of the intensity of pain?”

Neil Minkoff, MD: Right.

Stephanie J. Nahas, MD, MSEd, FAHS, FAAN: “What does it mean for you? Are there 50% fewer days that you have to take medicine for the attack?” These are all other kinds of exploratory measures in these clinical trials. It’s about what is the most meaningful for that patient, at that moment, and that can change over time.

Neil Minkoff, MD: Dr Dodick, you wanted to add to that?

David W. Dodick, MD: Yes. I’d like to say that the reason why the American Headache Society’s position paper took a position on this was because there are patients whose quality of life is dramatically improved by a treatment that didn’t reduce 1 headache day per month.

Neil Minkoff, MD: Yes.

David W. Dodick, MD: If you have a patient for 20 years who’s had chronic, continuous pain, you can forget about trying to reduce the frequency of headache days by 50%. Maybe it happens in some patients, but in many of those patients, it doesn’t. We said, “Yes, you can have a 50% reduction. Alternatively, you can have a clinically meaningful reduction in impact using the Headache Impact Test or the MIDAS [Migraine Disability Assessment Test]. It’s about the disability. There are validated reductions as to what’s clinically meaningful.

In this new instrument that measures performance in daily activities and physical impairment, you don’t need to benchmark this with 50%. You need to show that the patient is getting better. Sometimes you can’t achieve that by showing a 50% reduction in the number of headache days.

One more comment I’ll make is that recently we published evidence with some of these newer therapies. We looked at patients with a 20% reduction and a 30% reduction and correlated that with some of these other patient-reported-outcome measures, including disability and headache impact. That showed a substantially better effect with the drug therapy, compared with placebo, on these other measures, even if they didn’t achieve a 50% reduction.

Neil Minkoff, MD: Dr Stephens, let me bring that back to you. We know that payers, by and large, are trying to do some level of utilization management around these medications. How is that being managed in your neck of the woods? Is there any difference between injectable medications vs oral or IV [intravenous] medications in this space?

Kevin Stephens Sr., MD: That’s a very good question. We have an interesting predicament. As in the commercial market, our customer, on the 1 hand, is the employer. On the other hand, our customer is also the employee. The employer looks at missing things and that kind of stuff. If you have some type of utilization management that restricts access to current therapeutic modalities, then patients miss more days of work. Then employers get upset because they want healthy employees.

On the other hand, it’s the same for the employee. Their quality of life, however you want to measure it, is affected. Being able to work is 1 issue, but in terms of functionality, they want to be able to engage in all the other things they love to do. That’s very important.

We try to bridge the gap, and we try to stay out. Then we dive in between the patient and physician. We think that’s something that is unique to the neurologist, headache specialist, PCP [primary care physician], or whoever is managing the patient. We really try to facilitate and help them if there is information that we can pass on to the patient. If there’s something else we can give the patient, that’s what we try to do.

But this is so complicated and complex. As I mentioned earlier, 1 size does not fit all. We have to let the physicians manage this disease. Then we try to be as facilitative as we can. We want to be not obstructive but within the guidelines.

That’s the other thing. We have national guidelines. We typically follow them to the letter. If it says we have 2 months of trial therapy and 1 month of this and that, then that’s what we do. Treatment works because that’s what the national experts say. As we mentioned earlier, they have come up with a statement on this, and we follow it.

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