“Government’s first duty is to protect the people, not run their lives.”Ronald Reagan
On to ALS enemy #2 – U.S. Food and Drug Administration. So much could be said about this bureaucratic quagmire. It’s a behemoth of an organization that is so confusing and has an unbelievable amount of red tape. It seems, from the outside, they are protecting us to death. But pALS are already dying so that doesn’t make any sense.
In 2019 we had an in-person meeting with some of the officials that were instrumental in the Nurown trial. Honestly, I had a hard time staying awake. I feel like it started off well. Through some contacts we had in Congress, the acting head of the FDA joined the meeting, but left about 5 minutes in. From there I felt it went downhill. One member of the FDA in particular was incredibly condescending. Another treated the group as if we were his children, not in a condescending way, more in a placating way. Once I was insulted for the group I tuned out. Through these 2 individuals I feel like we can completely sum up the basis of the problem with the FDA in how it approaches ALS and our community.
For starters, they absolutely believe they know better than us when it comes to this disease. I do not feel like they honor or respect our opinions or input. And isn’t that crazy?! We are the ones living this hell. We are the ones with firsthand knowledge of what is going on. And with my husband there – freakish smart guy – there are people in the community who know and understand the science behind the disease as well. In fact, I would venture to say that the ALS community as a whole is very versed and informed on the science behind the disease. So to treat us like we are somehow less than them is rude, to put it nicely. Then we come to the paternal nature. Like we are petulant children throwing a hissy fit over a drug. They try to placate us with words but no action. I don’t know how long they promised the Guidance Document for ALS Trials before they finally published it. It took Matt saying he was going to starve himself for them to publish 12 pages of guidance. I think it was over 5 years it took them to review and publish the document. That’s the equivalent of a parent saying “2 minutes” when we know it is really going to be 10.
The community has asked for more modern trial design. Did you know that the trial requirements for an ALS treatment are the same for an acne treatment? In what world does that make any sense? A terminal illness is treated the same way as a cosmetic inconvenience. If that doesn’t blow your mind you are special. The placebo arm of a trial is somewhat cruel when it comes to a terminal illness. In regards to NurOwn®, the procedure involves taking a bone marrow aspiration then injecting manufactured stem cells into the spinal fluid. It’s not a comfortable procedure. Half the trial participants essentially got sugar water – that’s cruel.
Another problem with the FDA is conflict of interest. Panelists have to disclose any conflicts when they are selected to review a drug, but there are loopholes, like in everything. While they might not get incentives up front, a couple years down the road they might get some kickbacks. In the way of speaker fees, donations to their research, etc. The larger pharma companies can afford to incentivize panelists. Unfortunately, small companies are the ones researching ALS. These companies cannot provide the same sort of future bonuses that big pharma can nor do I think they should for ethical reasons. Big pharma can also make it worthwhile for panelists to not let the small guys get a leg up. I would hate to say that corporate pharma is keeping a treatment from market, but I will imply it. Big corporations do not like the little guys succeeding, in any market. A long read but check out this article giving real numbers to this issue: Hidden Conflicts?
Obviously the FDA is broken in how it approaches ALS. It is also not helping many other orphan diseases. The market for a treatment is not as lucrative for these diseases. There are not enough people dying and it is not a communicable disease. The FDA needs to stop protecting us to death. They need to allow innovation so we can save our pALS and find an end to this disease.