Thanks to last week’s post, a lot of people have taken action, but a lot of people are also confused and wanting to know more about what’s really happening.
Here’s the Cliff Notes version of last week’s PANDAS/PANS controversy in the community…
A private company wants to fund FDA trials to get their particular IVIG product FDA-approved. This sounds wonderful, except they’re trying to do it with the Orphan Drug program, which requires PANS and PANDAS be classified as a rare disease with the HHS.
Long story short: this company can only pursue Orphan Drug Status if PANS Advocacy organizations change their websites to imply that PANS is a rare disease. This is what was asked of them last week.
Why an HHS rare disease classification would be harmful for PANS and PANDAS:
1) Anyone who doesn’t meet the very strict definition of PANS and PANDAS from the trial could have a more difficult time accessing treatment.
We’re likely talking about teens and adults, as well as kids who don’t have an abrupt-onset, and even kids who have already been sick for a year or more. One common myth about PANS in the past was that it couldn’t be treated successfully after a few months. Indeed, in the first trial this company ran, only kids with a new onset were considered eligible for treatment.
It could be catastrophic if doctors and insurance companies began to believe there was no point treating PANS if someone has been sick for more than a few months. As most of you know, most people still take several months, if not years, just to get a diagnosis.
2) Large numbers of people will likely not get an appropriate diagnosis at all.
If your doctor believes PANS affects 1 in 11,000 people, do you really think they’ll ever consider a PANS diagnosis? Will they even bother to educate themselves on a condition they think they’ll never encounter?
3) The prevalence studies cited as “evidence” to support PANS/PANDAS as rare are deeply flawed.
You can’t estimate the prevalence of an under-recognized disease by looking at how commonly it was diagnosed. All you’re looking at is how likely doctors are to diagnose it, not how often PANS actually occurs.
4) Pretending there’s hard evidence that PANS/PANDAS is rare will damage the credibility of the entire community.
Let’s face it: we already have a credibility problem. Too many unscrupulous providers peddle quackery at desperate families, promoting themselves as offering treatment for PANS. This has caused physicians to see PANS as a fake disease with fake treatments.
If major PANS advocacy organizations suddenly update their websites to call PANS “rare” without well-designed studies to back this up, we are only feeding into the idea that PANS isn’t an evidence-based diagnosis.
5) Calling PANS/PANDAS rare undermines broader neuroimmune research.
There are many other pathways for companies to study PANS and seek FDA-approval without seeking Orphan Drug status. Though companies benefit from the seven years of market exclusivity that an Orphan Drug receives, they also benefit from having a larger pool of patients eligible for their drugs. Saying that PANS is NOT rare is actually beneficial for both patients and industry partners in the long-term.
Will drug companies really want to donate their drugs if they think PANS is rare, and they’ll never see a return on their investment? There is a lot of valuable research to be had from studying existing drugs rather than just new drugs and new formulations of existing drugs.
How can we stop this PANS/PANDAS rare disease effort?
PANS/PANDAS can’t be classified as rare unless advocacy groups imply it could be rare on their websites.
Read that again: the fate of future PANS diagnosis and treatment hinges on what advocacy groups say on their websites.
Many advocacy organizations have already taken a stand for what’s right and refused to change their websites.
Others are enabling this Pharma company without realizing it. The latter hear “this company wants to fund research,” and they’re ready to do whatever they ask, even if it means compromising what they’ve stated they believe for many years: that PANDAS/PANS is NOT rare, but rarely diagnosed.
Most disturbingly, organizations need only say, “the incidence is unknown” for PANDAS/PANS to be reclassified as “rare” with the HHS.
This, along with the flawed studies I mentioned last time, is taken as sufficient evidence by HHS to classify PANS/PANDAS as rare.
How this can possibly be true defies logic, but this is indeed the case for this particular situation. It’s not the case for every disease seeking Orphan status, but for PANS and PANDAS it is at this time. I wish I were making this up (or exaggerating, as some have accused me of), I really do.
I certainly understand the desperation for more research—we definitely need more of it. But any new research studies need to be based on solid evidence—not the faulty premise that PANDAS/PANS is rare and exclusively pediatric.
A lot of the pressure about changing websites comes after a certain very powerful researcher asked organizations to do this, citing the specific IVIG trial as the reason. This is what started the whole conversation.
The pharmaceutical company who wants to fund the new trial has paid this “powerful researcher” in previous years. This is publicly available data on the Open Payments system, by the way. One has to wonder who really stands to benefit if the company achieved Orphan Drug Status. Nevertheless, I also think this all probably goes even deeper than this one researcher, and this one company, hence I don’t name either of them.
Ultimately, patient advocacy groups should answer to patients and families, not researchers, not drug companies, not foundations, and not even clinicians. Patient advocacy groups are there to serve the best interests of the patients, and I don’t think calling PANS rare would be doing that. I hope every organization understands this and will take a strong stand against it.
What’s needed is for these organizations to return their websites to the language they were using a couple of weeks ago.
They need to state: “PANS/PANDAS is estimated to affect 1 in 200, but more studies are needed.”
This is all that’s necessary to foil one of the greatest setbacks to PANS advocacy in the past two decades. That’s it. So simple, yet so critical right now. Please keep writing to organizations, sharing this post, and commenting.
Tell organizations including the influential PANDAS Network to explicitly say PANS/PANDAS is NOT RARE.
On a personal note, I want to take a moment to explain more about why I’ve written this post and the last one, as some have questioned my motives. I’m not here to point fingers and stir up drama just because. My goal was simple journalism: to inform the community and hold decision-makers accountable. I found myself in a unique position to speak up for transparency as an unaffiliated, anonymous blogger.
If the PANS community shows up against Orphan Drug status, the advocacy organizations have more leverage to resist any powerful people or companies that could be pressuring them.
To be honest, I’ve been incredibly stressed over the whole situation and how people would respond. Doing the right thing is all too often not the easy thing…
About the author: The Dreaming Panda is the pseudonym of a 30-something PANS patient who has lived with the illness for two decades. Since 2014, they have blogged about their journey from diagnosis at age 19, through years of treatments, through remission and relapses, to their present truce with ongoing neuroimmune complications. TDP wants everyone to know they’re very tired of people still calling PANS a rare pediatric disease after twelve years of blogging and advocacy.
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