DC Dream

March 1, 2016

by Mark Dunning

I am traveling to Washington, DC this week with a dream. It looks something like this, but remember, at the moment, this is just a dream:

In this dream we, the Usher Syndrome Coalition, work with researchers and the National Institutes of Health, to identify the most valuable research, the steps that will move us most quickly to delivering viable treatments to the Usher syndrome community.

This becomes the roadmap for NIH Usher syndrome funding. With the confidence that the best researchers in the world have agreed on this critical path, NIH offers up challenge grants to encourage researchers to do this important research.

These challenge grants attract submissions from new researchers with new approaches to solving these critical problems.

The roadmap, and the progress on it, is made public for the Usher syndrome community to see. People with Usher syndrome draw hope from the visibility in to the progress being made. Some invest their own money, or get private institutions, to invest in the roadmap, adding additional resources to the public financing.

This increased pool of researchers then works again with the Usher Syndrome Coalition and NIH to update that roadmap and we do the whole thing over and over again, year after year.

The result is 1) an ever increasing pool of talented researchers focused on the most important research, 2) targeted federal spending that NIH can be confident will give the best return on investment, 3) a well-informed community that includes families and researchers and 4) real hope for those with Usher syndrome.

We have been traveling to Washington, DC regularly for several years. We have made a lot of progress but there is much work to be done. The dream described above remains, mostly, a dream. But it need not stay that way. On Wednesday we are meeting with the National Institute on Deafness and Other Communication Disorders and with the National Eye Institute. On Thursday we have meetings lined up with offices of a number of influential members of the House and Senate. Hopefully we can work together to make the above dream a reality.

Here is a quick summary of the world in which we currently operate and some of the concerns we hope to address with our visit. According to the NIH categorical spending reporter, NIH has invested almost $16M in Usher syndrome related research this year. That’s the good news. The bad news is they invested almost $19M last year. Prior to the Usher Syndrome Coalition’s advocacy, Usher syndrome was not listed on the categorical spending reporter so we’re not sure how much was invested. We have a lot more visibility in to the spending on Usher syndrome. Now we need to work together to make sure the investment remains consistent.

There are a lot of legitimate reasons why the spending could go down but this is one of the reasons we make yearly visits. We want to make sure that Usher syndrome remains a priority at NIH while understanding the challenges they face in funding Usher research.

Our search of the categorical spending reporter turned up 27 total grants issued. Of those, seven were what are known as intramural, meaning it was money granted to NIH scientists. It never left the campus. Those seven grants made up more than half ($8.5M) of the total money spent on Usher syndrome research by NIH. Sixteen other organizations received Usher syndrome related grants. Most were for around $300,000. Several institutions received more than that amount.

University of Iowa $1,000,000
Oregon Health and Science University $1,000,000
Stanford University $577,000
University of Oregon $510,000

It’s great that NIH is investing internally on Usher syndrome and the external organizations that have received federal funding have done great work. But we need more organizations working on Usher syndrome research and we need more funding from NIH going to those organizations. Right now there are not enough researchers, not enough organizations, and not enough money per organization. We want to draw in new talent with new ideas. We need to cast a wider net. That’s part of the dream.

However, it’s not solely about the amount of money spent. We also want it to be spent wisely.

Most of these grants are for broader research that could impact Usher syndrome but is not focused on Usher syndrome. In fact, only five of the grants totaling $1.5M contain Usher syndrome in the title. To be clear, these other grants are very important. But we want to ensure that if they are called Usher syndrome research that they represent the best investment to advance Usher syndrome research.

To know that, we need a public plan, a roadmap, for Usher syndrome research. The goal is to get treatments, viable treatments, to people with Usher syndrome as quickly as possible. What is the critical path to make that happen? That’s where our investment dollars should be going.

During our visit to Washington, DC we hope to discuss that critical path with NIH. We want to know how we, the Usher Syndrome Coalition, the research community, and NIH can work together to develop a roadmap, a viable, valuable roadmap, for Usher syndrome research that will yield Usher syndrome treatments while ensuring that our precious research dollars are invested as wisely as possible.

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