There Are Elephants In The Room

A little over a week ago we got the wonderful news that a drug had been approved for ALS in the US.  Since then we have had multiple news reports and webinars and communication from ALS organizations and MT Pharma America about the drug, Radicava (edaravone).

Don't get me wrong.  I ecstatic about having another tool in the toolkit to treat ALS.  There are just these two elephants bothering me.

Elephant 1 - The Awkward Age Of Precision Medicine

This is a drug that was developed in Japan in the late 1980s and was approved for use in strokes there in 2001.  Based on later ALS studies in Japan, it has been used there for ALS since 2015.

I sensed the first elephant right away.  We've been so conditioned of late to the heterogeneity of ALS and the genetic differences that I wondered how people with ALS in Japan differ from those in the United States.  What are the most common ALS genes in Japan compared to the the US? Is there anything in the Japanese data that will help us predict which people in the United States are most likely to respond?

The elephant sat quietly until I listened to Friday's webinar when Dr. Palumbo from MT Pharma America showed a slide modeling comparable blood levels between people of Japanese ethnicity and Caucasians.  I think that showed us that the drug gets to where it needs to be in both groups, but it really doesn't tell us anything about how the groups responded to the drug, does it?

The concepts of precision medicine today constantly drive us to know more about individual cases of ALS and what interventions are likely to work in specific individuals.  New concepts are to know everything possible that distinguish an individual case of ALS.  Can old clinical trial data shed that kind of light? The elephant emerges. How can we figure out which people in the United States are likely to respond (and as important, not respond) to Radicava?

Elephant 2 - The Price

I'm the person in the room who normally defends drug pricing.  New drugs are expensive to discover and develop into commercial products.  As expensive as pharmaceutical products are, they usually provide results that are both cost effective and beneficial to our lives.  I'm pretty immune to drug price sticker shock.

Right after the Radicava approval announcement when some pricing information was revealed, an ALS caregiver asked me what I thought about the price.  I said that I thought if they had a slam-dunk product and could say that people with specific flavors of ALS would receive significant therapeutic benefit, that the extremely high price was probably in the right ballpark.  Unfortunately, the data that we've seen don't make it likely that this is a slam-dunk product for anyone.  It will be something to consider and try, and it may help, and it may not help, and it may be hard to perceive the difference individually.  Elephant 2, meet Elephant 1.

People with ALS will try it and I'm glad they can.

It's a fact that people who are not going to be responders to this product are going to pay the astronomical price, and we don't have data to minimize the size of that group. It's not like Costco where you get your money back later if it doesn't work.

The webinars we've heard have been great about explaining the process of getting payers to pay for new indications.  If it's likely that Medicare will pay for Radicava and its administration for people with ALS, that will be good.

That doesn't mean that we shouldn't talk about the price elephant.  We absolutely should.  It's not harmless for the government to overpay for something, especially when we may be on the verge of more product approvals for ALS in the US.

Over the past couple of years I have gained huge respect for the way DMD parents advocate for approval of therapies for their kids and then for access to those therapies.  We could learn from them about not ignoring elephants.