Breakthrough T1D Submits Formal Response to HHS Request for Information Urging the Reclassification of Deceased Donor Islets as Organs
The landscape of type 1 diabetes care in the United States could face a profound regulatory transformation following a decisive policy push by patient advocacy organizations and leading medical professionals. Breakthrough T1D, a prominent global organization dedicated to type 1 diabetes research and advocacy, announced today that it has officially submitted its comprehensive response to a federal Request for Information regarding the regulatory status of deceased donor islets. This submission represents a critical milestone in a concerted nationwide campaign to redefine how insulin-producing cell clusters sourced from deceased organ donors are classified, regulated, and delivered to patients suffering from severe forms of the condition.
At the core of the ongoing federal debate is a regulatory mismatch that has persisted for years. Currently, deceased donor islets are regulated by the U.S. Food and Drug Administration under the legal framework governing biological drugs and cellular therapies rather than as human organs under the National Organ Transplant Act. Advocates, clinicians, and researchers argue that this classification creates administrative bottlenecks, manufacturing-style regulatory burdens, and systemic hurdles that render a clinically validated, life-changing therapy largely inaccessible to the American public. By urging the Department of Health and Human Services to reclassify unmodified deceased donor islets as organs, the type 1 diabetes community aims to transition oversight to the Health Resources and Services Administration and the Organ Procurement and Transplantation Network, aligning regulatory reality with established biological science and international clinical practices.
Chronology of a Grassroots and Policy Campaign
The momentum culminating in today’s formal RFI submission began earlier this year when members of the type 1 diabetes community approached Breakthrough T1D with an urgent mandate: lobby federal authorities to remove deceased donor islets from the stringent drug regulation pathway and integrate them into the nation’s established organ transplantation infrastructure. Recognizing the immense potential to expand patient access, Breakthrough T1D mobilized immediately, engaging government officials, policy leaders, and health agencies across the Department of Health and Human Services.
Within two months of receiving the community’s mandate, the organization delivered a formal, comprehensive reclassification proposal to federal leadership, including a series of legal and regulatory white papers designed to map out a clear administrative path forward. This proactive strategy was bolstered by active partnerships with key institutional champions, including the Diabetes Research Institute Foundation, the Cure Alliance, and the Islets for US Collaborative, alongside vital support from prominent congressional leaders who advanced formal letters of congressional backing.
Medical experts who have dedicated decades to advancing islet transplantation technology—such as Dr. Piotr Witkowski, Dr. Camillo Ricordi, and Dr. Norma Kenyon—lent their profound clinical expertise to the effort. Following the publication of the federal government’s Request for Information, Breakthrough T1D’s internal teams of medical, scientific, and policy specialists spent weeks dissecting the complex scientific, technical, and regulatory questions posed by federal regulators. The resulting formal submission, delivered to HHS this morning, integrates decades of clinical trial data, regulatory analysis, and real-world international outcomes to present an airtight case for administrative reform.
Clinical Validation and the Scientific Framework
To understand the urgency behind the push for reclassification, one must examine the clinical nature of deceased donor islet transplantation. The procedure involves isolating insulin-producing islet cells from the pancreases of deceased organ donors and infusing them directly into the liver of a recipient living with type 1 diabetes. Once established in the liver, these donor cells begin producing insulin autonomously in response to fluctuating blood glucose levels. While the procedure offers a profound restorative benefit, recipients must maintain a regular regimen of immunosuppressant medications to prevent immune rejection of the transplant.
Presently, deceased donor islet cell transplants are explicitly intended for, and FDA-approved solely for, adults with type 1 diabetes who experience severe, debilitating hypoglycemia—characterized by dangerously low blood sugar episodes—or hypoglycemia unawareness, a hazardous condition in which patients lose their physiological warning signs before a catastrophic blood sugar crash occurs. Despite its proven efficacy, the procedure remains remarkably scarce in the United States due to the heavy compliance frameworks imposed by drug manufacturing regulations, which treat human-derived cell clusters akin to synthetic pharmaceutical compounds.
The formal regulatory arguments submitted by Breakthrough T1D draw extensively from landmark, NIH-sponsored clinical research, specifically the Clinical Islet Transplantation Consortium phase 3 trials. Data from the CIT-07 trial—which evaluated isolated islet transplantation alone—demonstrated that 87.5 percent of participants achieved a glycated hemoglobin level below 7.0 percent while entirely eliminating severe hypoglycemic events at the one-year mark. Furthermore, long-term follow-up data from the CIT-08 study revealed that freedom from severe hypoglycemia remained above 90 percent for up to eight years post-transplant, with 74 percent of recipients achieving insulin independence at some point during the evaluation window.
Comparative international frameworks provide further validation for the proposed shift. Programs such as the long-standing clinical islet transplantation protocol in Edmonton, Canada, alongside frameworks utilized in various other developed nations, have successfully delivered deceased donor islet transplantation as a standard element of clinical care under traditional organ or tissue regulatory structures for years. Proponents note that the operational infrastructure required—including medical standards, personnel qualifications, and organ allocation protocols—already exists within the Organ Procurement and Transplantation Network and can be rapidly adapted to accommodate islet cells without compromising patient safety.
Core Recommendations Outlined in the Federal Submission
The executive summary of Breakthrough T1D’s formal submission outlines a precise, multifaceted recommendation initially presented to federal health leadership earlier in the spring. The primary proposal urges the Department of Health and Human Services to define unmodified deceased donor islets explicitly as organs under the statutory authority of the National Organ Transplant Act.
Under this recommended framework, oversight of unmodified islets would shift to the Health Resources and Services Administration and the Organ Procurement and Transplantation Network. This transition would mirror the governance structures applied to kidneys, livers, hearts, and lungs, integrating islets into standard organ procurement and distribution channels. Simultaneously, the Food Information and Drug Administration would retain its traditional jurisdiction over manufactured cell therapies and heavily modified deceased donor islets—such as those that undergo genetic engineering, encapsulation, protein-coating, cellular expansion, or cellular differentiation.
The submission carefully defines "unmodified" deceased donor islets as cell clusters isolated and purified directly from a deceased donor pancreas—matching the exact methodology utilized in the phase 3 Clinical Islet Transplantation Consortium trials—and transplanted into a recipient without subsequent biological or chemical alteration. To bridge the gap between regulatory reclassification and practical clinical implementation, the organization’s recommendations also urge federal health authorities to apply enforcement discretion during the transition period while simultaneously calling upon the Centers for Medicare & Medicaid Services to establish favorable insurance coverage pathways.
Broader Implications and the Path to a Cure
The potential implications of reclassifying deceased donor islets extend far beyond administrative realignment; they represent a fundamental modernization of American healthcare policy that could fundamentally alter the trajectory of type 1 diabetes management. If adopted, the reclassification would remove significant financial and institutional barriers that currently discourage academic medical centers and specialized surgical hospitals from establishing and maintaining islet transplant programs. By streamlining oversight through existing organ networks, hospitals could integrate islet procurement seamlessly into established organ donation workflows, exponentially increasing the availability of the procedure for eligible patients.
Moreover, proponents argue that aligning regulatory oversight with biological reality would foster a more stable environment for academic research and clinical innovation. While manufactured and genetically modified cell therapies continue to represent the cutting edge of futuristic diabetes cures—requiring rigorous, drug-style oversight by the FDA—unmodified donor cells represent a proven, mature therapeutic modality that functions biologically as an organ and should be regulated accordingly. Strengthening the regulatory pathway for deceased donor islets not only directly benefits adults suffering from severe hypoglycemia today but also fortifies American competitiveness in regenerative medicine and translational research on the broader path toward universal cures for type 1 diabetes.
Next Steps in the Federal Regulatory Process
While the submission of the formal response to the Request for Information marks a monumental victory for collaborative advocacy, healthcare experts emphasize that the administrative battle is far from over. An RFI represents a critical information-gathering phase in the federal rulemaking process, but it does not guarantee immediate government action or formal policy transformation.
Following the closure of the public comment period, federal agencies will face the monumental task of reviewing more than 10,000 public comments submitted by patients, clinicians, researchers, and institutional stakeholders. This rigorous review process will inherently require time, during which policy adjustments may occur quietly behind the scenes without immediate public announcements.
In light of this timeline, leadership at Breakthrough T1D has pledged to maintain continuous, behind-the-scenes engagement with the Food and Drug Administration, the Health Resources and Services Administration, and executive leadership within the Department of Health and Human Services to keep regulatory progress moving forward. The organization continues to encourage individuals living with type 1 diabetes, their families, and medical professionals to stay informed through official updates, follow the unfolding administrative developments, and consider joining national volunteer advocacy networks to ensure that the patient voice remains central as federal regulators weigh the future of islet transplantation in the United States.
