Dear Dr. Curay,
 

We write as a concerned group of doctors, patients and caregivers who for at least two decades have attempted to muddle through an increasingly complex and difficult landscape tied to the need for treatments and the potential for cures for the nearly 5 million Americans afflicted with the conditions of Digestive Tract Paralysis.

We have been in  communication with the Agency since 2007; the conditions of Gut Paralysis including Intestinal Pseudo-Obstruction, Gastroparesis and Colonic Inertia remain the most serious unaddressed conditions of the digestive tract known to humankind.  Patients who previously exhibited good health can quickly and without warning develop one or more of the aforementioned conditions and face the peristaltic failure of the digestive tract; with insufficient available treatments we face pain and malnutrition, the potential for digestive tract failure, the need for tube feeding and the end stage possibility of full intestinal transplant.

Since the 2000’s, in much the same way that the NIH’s NIDDK has been a partner to our community through the formation of the Gastroparesis Clinical Consortium, we would have hoped that the FDA would be a partner to the Gut Paralysis community; we want the same thing.   The development of safe and effective treatments that restart and restore the function of the digestive tract should not be some unattainable goal that we cannot see our lifetimes; in point of fact, that clarity is more greatly seen in early every other industrialized democracy on this planet.

Puzzingly, since the withdrawal of Cisapride in the early 2000’s, this Agency has consistently sought to block medications, primarily tied to the possible and limited incidence of a cardio-vascular side effect known as Long QT Syndrome.  What is troubling, is that when the FDA has withdrawn drugs like Cisapride and Zelnorm or blocked the approval of compounds such as Domperidone, our patients are forced to make even riskier choices.  We are forced into surgeries that permanently remove portions of our digestive tracts, we face tube feedings that prevent starvation but present the incumbent risks of liver damage and inflammation, and we face desperation in terms of intractable pain where we seek uncontrolled opioids, CBD and Kratom.  These concerns have only intensified upon the onset of the COVID 19 pandemic where it is clear that gut paralysis is a sequalae in states of LONG COVID.

Our community has sought discussion and dialogue for nearly two decades tied to the Patient Centered Drug Development Initiative to develop more effective clinical guidelines for the passage and approval of drugs for Gastroparesis; hundreds of pages of testimony attest to this fact; your office of Patient Affairs is well aware of our concerns.

But if the last year is a test of the Agency’s commitment to this dialogue and a new and better path to care; it is profoundly clear that we have all failed. Namely,

a.      The failure of essential IND’s for the drugs Domperidone, Zelnorm and Cisapride.

b.     Ongoing tensions between this Agency and the manufacturer Vanda, as well as a lack of clarity for the future pathways for others who would seek to develop solutions.

c.      Ongoing and significant shortages of infusion based medications and nutritional supplies, and even the most basic over the counter medications that stabilize our conditions.

This state of affairs is profoundly unacceptable; we want the Federal government to be a partner not a continued barrier to our care; we recognize the severity of Crohn’s Disease and Ulcerative Colitis, but the pathways to care for those conditions are decades ahead of where we are right now; it should not be that a patient with neurologic failure of the digestive tract should need to feel that they would have been better off to get ulcerative bleeding of the gut.

Over the course of 2025, this community will be coming back together to bring to public light these concerns; it has been suggested by the Agency, if we could just identify a small subclass of rare and ultra rare disease for our conditions, we could develop better standards.

We will abandon no one; we follow the model of remarkable groups like Emily’s Entourage for Cystic Fibrosis; we have all felt the loneliness of having the wrong condition where peristalsis of our digestive tracts may never start again; we refuse to believe that we should feel as we would be better off living in Europe or Asia to get access to the care that we need.

We have lost too many remarkable and young members of this community particularly as a result of gut failure provoked by COVID 19 that we can any longer sit quiet; a positive and productive dialogue that leads to a future that we can all be proud of needs to start immediately, we will sit silent for no longer.