50 Million Reasons

October 22, 2014

by Mark Dunning

Fifty million dollars is a big number, an intimidating number, a number beyond the imagination of most of us. Personally I think in hundreds and thousands. Anything larger than my mortgage is almost beyond comprehension to me. Oh, I dream of winning the lottery and I can imagine owning an island in the South Pacific. But that is fantasy. It’s not something that I can really see happening. No, fifty million dollars is forever going to be beyond my reach.

A couple of years ago, Susie Trotochaud represented the Usher Syndrome Coalition and testified before the House appropriations sub-committee. She said she believed that Usher syndrome should be receiving fifty million dollars a year in federal research funding from the National Institutes of Health (NIH). It was a massive number, an intimidating number, but within the scope of the federal budget, it is pocket change. That is not to say we chose fifty million dollars because we figured they could find it among the seat cushions of the couch. No, we chose fifty million dollars for some very specific reasons.

Other Federal Spending

We looked at three similarly sized diseases and the funding amounts NIH provided to them. I’ve written about this before, but here it is again. ALS (30,000 in the US), Huntington’s disease (30,000 in the US), and Duchenne Muscular Dystrophy (48,000 in the US) affect about the same number of people as Usher syndrome. The average of the annual NIH funding for those three diseases is fifty million dollars per disease. 

And that’s not the only reason for choosing that fifty million dollar figure. In fact, it pops up all over the place. To explain you’ll need some background information.

One in Twelve Clinical Trials Succeed

Good science is about trial and error. A treatment may be tried and not succeed, but we learn from it. This is an expected part of the process. The hope is that with each iteration we get further and eventually we succeed. But this approach means that there are a lot of failures. According to Avik Roy, a Senior Fellow at the Manhattan Institute for Policy Research, “only one in 12 drugs that enter human clinical trials end up gaining approval from the FDA.” These are clinical trials we are talking about, not just basic science. They are big productions. And you almost need to plan on having twelve of them to produce a single treatment.

Clinical Trials are Expensive

The drug Solaris is a good model for calculating the expense of a clinical trial for an Usher syndrome treatment. Solaris was developed for an orphan disease of the blood (paroxysmal nocturnal hemoglobinuria) that affects about 4,000 Americans. That’s roughly the same population as Usher 1d. According to the paper Stifling New Cures: The True Cost of Lengthy Clinical Drug Trials, Solaris cost $22.8 million to develop ($1.4 million for the phase I trial, $4.7 million for phase II, and $16.7 million for phase III).

The Real Cost to Develop a Treatment

That $22.8 million figure for Solaris was for a success. To get the real cost to develop a treatment we need to account for the cost of the failures as well. Remember, eleven out of twelve treatments that reach clinical trial never reach the clinic. Most don’t make it past phase I, but some will get further before failing. It is safe to assume that most won’t get to phase III without a high degree of confidence, so there will be fewer failures there. For our calculations we’ll assume that 3/4 fail to get out of phase I and that there is only one phase III trial before we have success. Using those assumptions, the costs would breakdown like this:

A picture of stacks of money.

12 phase I clinical trials at $1.6 million each

3 phase II clinical trials at $4.7 million each

1 phase III clinical trial at $16.7 million

Grand total to successfully develop one Usher syndrome treatment: Fifty million dollars.

Hey! Where have I heard that number before? But wait, there’s more.

Total Cost to Treat Everyone

Most of the treatments in development are for a specific genetic type of Usher syndrome. There are 12 known genetic types of Usher syndrome. Hold on for more math…

We just figured that it will cost fifty million dollars to develop a treatment. We need twelve of them, one for each genetic type. That means we need $600 million dollars for clinical trials. But even that figure is low.

We need to do basic science, the stuff that develops a treatment that is worthy of a clinical trial. That’s expensive, too. If we assume we have to spend at least half of what we spend on clinical trials just to develop treatments in the lab, we would need another $300 million dollars. All totaled, we probably need close to $900 million to find treatments for every person with Usher syndrome. 

Annual Cost to Develop Treatments

We don’t need $900 million right now. We wouldn’t be able to spend it if we had it. Science takes time, building infrastructure takes time, developing Usher syndrome experts takes time. So let’s agree on a goal. A child born with Usher syndrome today will have treatments before he or she becomes an adult, regardless of the genetic cause. That means we have 18 years to find treatments for everyone with every type of Usher syndrome of every age. It is aggressive but possible. And what annual figure do you get when you divide $900 million over 18 years? You got it. Fifty million dollars a year.

The Goal

So that’s our goal: fifty million dollars annually for the next eighteen years. Now you probably know that we are in the midst of the Usher Syndrome Coalition annual appeal. We are NOT looking to raise fifty million dollars with this appeal. But in the next few posts I’ll explain how your investment in the Usher Syndrome Coalition is critical to ultimately getting to that fifty million dollar annual figure. Who’s up for some more math?

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