Perspectives, Questions and Ideas for What We Can Do

FDA-blueThe question posed in the headline above is really multiple questions, and which one to ask depends on your perspective.

The first questions, from the parent’s perspective: Will there be another medication that works as well or better? If so, can I enter a trial of that medication, and when is it likely to be approved and made available by the FDA?

The next question is from the policy perspective: Was there anything inherent in the structure of the FDA clinical trial and approval process that contributed to the failure of the Seaside trials and ultimately to the failure of Seaside as a company?

As the director of public policy for the NFXF, it is this latter question that I will attempt to answer here.

The official result of the Seaside trials was that STX209 did not do what its makers thought it would. The present structure of FDA trials requires that the proponents of a new drug predict what it will do and then conduct trials to determine whether the drug delivers the predicted results. If it does not, that particular trial is deemed a failure and cannot be relied upon to make the case for formal approval of the drug.

Many parents who participated in the trials of STX209 have no doubt that they saw dramatic improvements in their children in areas such as behavior, anxiety, sensory overload and language. Many in the scientific community believe that the structure of the trials and the difficulty in predicting (only) one aspect of what a new drug will do is very difficult when looking at conditions as complex as Fragile X syndrome or autism. Combining these two observations could lead one to the conclusion that STX209 may have worked, but that the predicted result was not the observed result, and so the trial had to be deemed a failure. If that’s what Seaside believed, normally it would be their next course to propose and carry out another trial. This is not uncommon with new drugs. But in this case Seaside simply ran out of money.

From a policy perspective, we at the NFXF have been carefully considering what if anything we as a community of passionate and dedicated advocates can do to increase the likelihood that this doesn’t happen again, with another drug company. While the next players we see coming down the road are certainly better capitalized, other start-up ventures are also entering the Fragile X market. So whatever we can do to smooth out the road they all must travel will be in all of our self-interests. This brings us back around to the last question I posed at the beginning of this article. Here’s what we’ve learned.

FDA Approval Issues

Those on a quest to develop drug treatments for FXS are not the only ones facing the difficulty of predicting what a new drug might do (an endpoint) and needing more flexibility in demonstrating to the FDA what a new drug can do. There are many other, mostly rare, also complex and unheard of conditions that are struggling with the same hurdles. Fortunately, we’ve identified a group, the Rare Disease Legislative Advocates (RDLA), that we believe is right on top of this issue. Together with our friends at Autism Speaks, we’ve already held a series of meetings with them to explore how we can all join forces and advance a policy agenda that will aim toward greater flexibility at the FDA in establishing endpoints and demonstrating drug success.

One bill that the RDLA was instrumental in moving forward is the Food and Drug Administration Safety and Innovation Act (FDASIA). Although there are some regulatory shortcomings with the current version of the act, we believe that the kind of flexibility envisioned by FDASIA in designing trials and establishing more flexible endpoints are exactly the kind of changes that can decrease the likelihood that a drug developer gets “seasided” again. So the NFXF has joined forces with the RDLA to bring about changes to the newly enacted regulations for FDASIA. Watch for more details and action alerts coming soon. We also anticipate that our advocates will have something to say about this on NFXF Advocacy Day 2014.

Funding Challenges

Another issue we’ve identified has to do with the fact that much of the funds available to support the work of the FDA come from “user fees” paid by drug companies. These user fees are not tax dollars but fees paid directly by the drug companies to subsidize the work of the FDA.

Now, I’m certain you’ve all heard the term “sequestration,” and while I have no intent of taking a political position here, suffice to say that the across-the-board cuts that have been imposed as a result of sequestration have impacted the operation of the FDA (even before the more recent government shutdown). But what’s a bit odd is that sequestration has been used not only to reduce the amount of federal tax dollars made available to run the FDA, but also to reduce the amount of user fees that the FDA can access to subsidize their operations. Many in the clinical trials arena believe this is wrong, and since these fees are paid by private drug companies and not tax dollars, sequestration should not keep the FDA from accessing this money. We at the NFXF agree, and that’s why we are also supporting a bill recently introduced in the House of Representatives to fix this.

The bill is known as the FDA SOS (Safety over Sequestration) Act, H.R. 2725, and the NFXF has joined forces with a broad coalition that’s come together to secure its passage. Watch for more details and action alerts coming soon on this as well. We also anticipate that this bill is another matter our advocates will have something to say about on Advocacy Day if it has not become law.

So, although we share in the deep disappointment over the flameout of STX209, we’ve been hard at work looking at what the NFXF can do that will soon translate into what you as our most passionate advocates can achieve on behalf of the Fragile X community. That’s what we’ll be working toward in coming months.

Hope to see you in Washington next March!

Jeffrey CohenJeffrey Cohen
is the director of government affairs and advocacy and manages the federal affairs agenda of the NFXF and its annual Washington, DC Fragile X Advocacy Day. He has 2 children living with Fragile X syndrome.

Questions and comments can be directed to the author at