The landscape of Type 1 Diabetes (T1D) treatment in the United States stands at a critical regulatory crossroads. This week, Breakthrough T1D (formerly JDRF) submitted a formal response to the Department of Health and Human Services (HHS) regarding a Request for Information (RFI) that could fundamentally alter how Deceased Donor Islets (DDIs) are governed, funded, and distributed. At the heart of the matter is a push to reclassify these insulin-producing cells from "biological drugs" to "organs," a move advocates say will bridge the gap between life-saving science and patient accessibility.
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Main Facts: The Regulatory Bottleneck and the Quest for Accessibility
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For decades, the T1D community has grappled with a paradoxical reality: a clinically proven cure-like therapy exists, yet it remains functionally out of reach for the vast majority of those who need it most. Deceased Donor Islet (DDI) transplantation involves isolating insulin-producing clusters from a donor pancreas and infusing them into the portal vein of a recipient’s liver. For patients with "brittle" diabetes—characterized by severe, life-threatening hypoglycemia unawareness—this procedure is transformative.
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However, under current U.S. law, the Food and Drug Administration (FDA) classifies these isolated cells as "biological drugs." This classification subjects the procedure to the same rigorous and expensive Biologics License Application (BLA) processes required for mass-produced pharmaceuticals. Because DDIs are naturally occurring tissues harvested from human donors rather than synthetic compounds, the "drug" framework has created a prohibitive environment for academic medical centers and hospitals.
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Breakthrough T1D’s formal recommendation to HHS Secretary Kennedy is clear: unmodified DDIs should be defined as "organs" under the National Organ Transplant Act (NOTA). This would shift oversight to the Health Resources and Services Administration (HRSA) and the Organ Procurement and Transplantation Network (OPTN), aligning the U.S. with international standards in Canada, Europe, and Australia. Under this proposal, the FDA would retain jurisdiction over manufactured or genetically modified cell therapies, while naturally derived, unmodified islets would be treated as transplantable tissue.
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Chronology: The Road to the 2026 RFI
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The journey toward this regulatory shift has been a multi-year effort that accelerated significantly in early 2026. The timeline reflects a coordinated campaign between scientific experts, advocacy groups, and federal legislators.
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- Early 2026: The T1D community and various advocacy groups, including the Diabetes Research Institute Foundation and the Cure Alliance, formally approached Breakthrough T1D to spearhead a national push for reclassification.
- March 6, 2026: Breakthrough T1D leadership met with HHS Secretary Kennedy to present a formal proposal. This meeting served as the catalyst for the current administrative review, outlining the legal and scientific justifications for moving DDIs from FDA to HRSA oversight.
- Spring 2026: In the months following the meeting with the Secretary, Breakthrough T1D developed and circulated comprehensive legal and regulatory white papers. These documents were shared with the FDA and HRSA to address technical concerns regarding safety and quality control.
- Summer 2026: Congressional champions joined the effort, sending three separate letters of support to HHS. These letters emphasized the bipartisan nature of the issue and the urgent need for patient access.
- Late Summer 2026: HHS officially published the Request for Information (RFI), seeking public and expert input on the classification of DDIs.
- Present Day: Breakthrough T1D submitted its formal response to the RFI, backed by a coalition of medical experts and thousands of patient testimonials. The public comment period is set to close on November 9th, having already garnered over 10,000 submissions.
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Supporting Data: Clinical Validation of DDI Transplantation
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The argument for reclassification is not merely administrative; it is rooted in decades of robust clinical data. Breakthrough T1D’s submission leans heavily on the results of the National Institutes of Health (NIH)-sponsored Clinical Islet Transplantation (CIT) Consortium trials.
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The CIT Trials (CIT-07, CIT-06, and CIT-08)
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The CIT-07 Phase 3 trial is often cited as the gold standard for DDI efficacy. The trial focused on patients with T1D who suffered from impaired awareness of hypoglycemia. The results were definitive:
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- Primary Outcome: 87.5% of participants achieved an HbA1c (average blood sugar) of below 7.0% at one year post-transplant, with a complete absence of severe hypoglycemic events.
- Long-term Success (CIT-08): Follow-up studies demonstrated that freedom from severe hypoglycemia remained above 90% for up to eight years.
- Insulin Independence: Approximately 74% of recipients achieved insulin independence—the ability to stop daily injections entirely—at some point during the study period.
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Global Precedents
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The U.S. is currently an outlier in its regulatory approach. In Canada, the "Edmonton Protocol" has been the standard of care for over two decades, where islets are treated as organs. Similarly, in many European nations, DDI transplantation is integrated into the national organ transplant infrastructure. These models have shown that islet transplantation can be delivered safely and sustainably without the pharmaceutical "drug" label, utilizing existing organ procurement networks to ensure quality and equitable distribution.
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Official Responses and Expert Testimonials
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The push for reclassification has unified some of the most prominent voices in diabetes research. In its formal response, Breakthrough T1D highlighted the contributions of pioneers such as Dr. Piotr Witkowski, Dr. Camillo Ricordi, and Dr. Norma Kenyon. These experts argue that the current FDA classification ignores the biological reality that islets are a functional component of the pancreas, not a synthetic product.
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The Breakthrough T1D Executive Summary
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In its formal submission, Breakthrough T1D emphasizes a nuanced "two-track" regulatory approach:
- Unmodified DDIs: Islets isolated and purified from a deceased donor pancreas should be overseen by HRSA/OPTN. This aligns with how whole-organ pancreases are currently managed.
- Modified/Manufactured Cells: The FDA should maintain its authority over cells that have been genetically modified, encapsulated in protective coatings, or derived from stem cells (differentiated). This ensures that "next-generation" therapies, which involve significant laboratory engineering, still undergo the rigorous drug-approval process.
The Role of Advocacy
The Diabetes Research Institute Foundation and the Islets for US Collaborative have been instrumental in mobilizing the patient voice. "The T1D community is not just asking for a change in paperwork; they are asking for the right to access a validated therapy that can save their lives," noted a representative from the collaborative. The 10,000+ comments submitted to the RFI represent a groundswell of support from families who live with the constant fear of nocturnal hypoglycemia—a condition where blood sugar drops dangerously low during sleep, sometimes resulting in death.
Implications: What Reclassification Means for the Future
If HHS acts on the recommendations provided by Breakthrough T1D and its partners, the implications for the American healthcare system and T1D patients would be profound.
1. Increased Patient Access and Insurance Coverage
Currently, because DDIs are classified as "investigational drugs" in many contexts, insurance providers—including Medicare and Medicaid (CMS)—are often reluctant to cover the procedure. Reclassifying them as organs would trigger CMS action on coverage, making the procedure financially viable for hospitals and accessible for patients regardless of their socioeconomic status.
2. Sustainability of Academic Research
The current BLA requirements are tailored for large pharmaceutical companies with the capital to build massive "clean room" facilities. Most academic hospitals cannot afford the overhead required to maintain "drug manufacturing" status for a procedure that is, in essence, a tissue transplant. Reclassification would allow these centers to utilize their existing transplant infrastructure, fostering innovation and clinical excellence.
3. Strengthening American Competitiveness
By aligning with international standards, the U.S. would regain its position as a leader in T1D clinical care. Many American patients currently travel abroad to receive islet transplants; reclassification would ensure that world-class care is available domestically.
4. The Path to a Cure
While DDI transplantation currently requires immunosuppressants, it serves as a critical "proof of concept" for future cures. Perfecting the delivery and management of donor islets is a necessary stepping stone toward stem-cell-derived therapies that may one day eliminate the need for immunosuppression entirely.
What Comes Next?
The closing of the RFI comment period on November 9th marks the end of the public input phase, but the beginning of a complex internal review within HHS. Government officials will now be tasked with synthesizing the thousands of comments and expert recommendations.
Breakthrough T1D has stated it will continue to work "behind the scenes," engaging with leadership at the FDA and HRSA to ensure that the momentum generated this year does not stall. While an RFI does not guarantee a change in policy, the sheer volume of scientific evidence and public support has made the status quo increasingly difficult for the government to maintain. For the millions of Americans living with T1D, the hope is that 2026 will be remembered as the year the "biological reality" of islet transplantation finally caught up with its "regulatory reality."
