I just turned in my answers to the FDA questions. A member of the SCW Facebook page said this, “What is the big deal with the FDA meeting, and why should we even try to go? Especially since most of us that have sickle cell are on a limited income, it makes no sense to trudge into a cold city in the middle of February just for our opinion.”
I can see his points. However, if we sit back in complacency, then we don’t have a right to complain. Even if you can’t physically attend, we need to take this opportunity to speak up. The FDA is the one that has the ability to fast track new medications for sickle cell disease. There are over 15 treatments for sickle cell in clinical research now, but most of them get caught up in the FDA circuit and do not get approved for one reason or another. There are so many of us living and dying with sickle cell disease, and it’s mind boggling to me that we only have 1 approved drug, when other fringe conditions that are more complicated have dozens to hundreds of effective therapies. So the first issue I would want to talk about is improved delivery channels for research to actually make it to market if successful.
The second thing I would want to improve is the educational focus on tissue ischemia. Tissue ischemia happens every time you have a pain episode, and your tissue dies from lack of circulation. But because pain is the loudest symptom, it is the one that always gets treated first. Meanwhile our tissues are dying and leading us more at risk for complications and organ failure.
Another improvement that I see is that all the transcripts and records, and comments made by all registered on the public docket will be available for every one to see. This includes those that finance sickle cell centers, and maybe it will prevent our funding from being cut, since they understand all the quality of life issues that we face. The NIH and Minority Health often have to make drastic cuts every year, and sickle cell has lost legislative traction. This means that we are often not prioritized when it comes to fiscal dollars, and this means that you will not have a sickle cell center in your town able to treat you. It is a trickle down effect and we feel it all across the nation.
Another reason why I think this is so important, is that this is the first open meeting for sickle cell disease in over 5 years. We are being called to the table with the lawmakers, lobbyists, and health care industry. If we don’t show up to speak for ourselves, then we can’t complain about anything. I understand that the money can be a factor, but those in the area that can attend, should attend. And if you can’t fly there, just register for the webcast and watch it online. You can also submit your comments and your experience to the public docket and this will be included in the final docket. These are just my thoughts at 1am. I know that there is a huge benefit, and we should not let this opportunity slip us by.
For those of you that are interested in learning more, visit the FDA page. And if you want to submit your answers, do so.HERE