One Drug: Many Uses
Supply Issues Continue
As supply issues are still impacting some patients, I’ve seen an alarming push for restricting usage of immunoglobulin replacement therapy to on-label uses.
Why do I call that an alarming trend? Because it ignores the full range of patients, who need IgG therapy for a multitude of diseases. Currently, the FDA only lists a handful of uses as on-label for particular products. Those on label uses include specific dosing instructions.
Should we eliminate elective use of immunoglobulin therapy in times of shortage? Yes. But is all off-label use unnecessary? No.
Consider for a moment, the patients for whom the “usual” dosing or schedule don’t work. A great example is my IC colleague Colin, who uses a non-traditional approach to dosing to remain healthy, or the many patients who may need more product to keep them healthy because they metabolize product outside what is considered “normal.” Everyone is different, so it can be hard to classify specific dosing, especially if the prescribing guidelines don’t work for that individual patient. Please know I am not advocating to pull the plug on Colin, but more so saying that there are a wide spectrum of uses of IgG within the primary immunodeficiency community that are both necessary and appropriate. Being honest about this diversity is the only way we can truly build sustainable long-term solutions.
In addition to the wide ranging use in the PI community, consider other rare diseases for which there is:
No other treatment
No economic incentive for manufacturers to bring those uses on-label
Reasonable evidence of the efficacy of immunoglobulin therapy to limit damage
Insurance companies, forever the bane of patient existence, don’t even restrict paying for these plasma-based drugs to on-label use. Sure, they consider some applications to be experimental, but there is plenty of research behind using plasma for patients who aren’t immunodeficient. There are patients who have an urgent need for off-label IgG therapy and have no other option. In this case, those who are crying out for restriction to on-label use could be considered more draconian than your average insurance company.
We are ultimately part of a broader community that relies on plasma, and we should be aware of that. There are those in our community who are off-label in Ig use. I would propose that we would, instead, become more cautious as patients about pushing others to go on therapy without adequate evidence it’s necessary. Remember, just because it worked for you, does not mean that it will work for everyone else or that they are entitled to the medication. There are several instances of folks I can think of who were taken off therapy for testing because they did not receive appropriate testing when first diagnosed and have sky-high immunoglobulin levels. Some have gone on to show no clinical evidence of the need for IgG therapy. Those same people are found crying foul around social media because they don’t have access. Even though they should not have access to treatment by even the most liberal clinical measures.
Maybe that is a harsh thing to say, but we wouldn’t tell someone who did not have diabetes that they needed insulin shots, when there was no clinically established reason or need for it. I’ve seen cases like that in our community. Patients who are presenting their case, test results, etc, and are not even borderline, and may even suggest against treatment due to unusual or extreme reactions.
My immunologist and I have talked at length about the problem of over- and under-diagnosis for immunodeficiencies. He believes there is a lack of recognition for some patients, and other patients are put on replacement when they have nothing more than chronic sinusitis as evidence of disease. It takes far more proof than that to establish a true PI diagnosis worthy of immunoglobulin replacement, but these patients will be tagged with CVID and put on therapy.
It concerns me that we do not like to evaluate these cases in our own community but are quick to judge the needs of others. We must strike a balance in how we use immunoglobulin therapy, but that must include patients who are using IgG in off-label ways.
How do we do that? We must increase the visibility of people who are on plasma products and tell their stories. We should also encourage plasma donation to ensure we have a steady, adequate supply for every use, including those to come. Additionally, we must hold companies accountable and ensure they are good stewards in how they supply the demand through their contracting.
One thing is for sure; we need more plasma to ensure a steady, affordable supply, so please share your story and encourage more people to donate plasma. You can help your friends and family find more resources at the Donating Plasma website. Ultimately, we are all powered by plasma, and together, we can solve this current issue.