WASHINGTON, D.C. — In a move that could fundamentally reshape the landscape of Type 1 Diabetes (T1D) treatment in the United States, Breakthrough T1D (formerly JDRF) has formally submitted a comprehensive response to the Department of Health and Human Services (HHS) regarding the classification of deceased donor islets (DDIs). The organization, alongside a coalition of scientific leaders and patient advocates, is urging the federal government to reclassify unmodified DDIs as organs rather than biological drugs.
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This regulatory pivot, long sought by the medical community, aims to dismantle the bureaucratic and financial barriers that have rendered islet transplantation nearly inaccessible to thousands of Americans suffering from life-threatening hypoglycemia. As the public comment period for the government’s Request for Information (RFI) concludes, the T1D community stands at a historic crossroads that could align U.S. medical policy with global standards and biological reality.
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Main Facts: The Argument for Reclassification
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At the heart of the debate is a jurisdictional distinction that carries profound consequences for patient access. Currently, the U.S. Food and Drug Administration (FDA) classifies deceased donor islets—clusters of insulin-producing cells harvested from a donor’s pancreas—as "biologics" or drugs. This classification subjects islet transplantation to the same rigorous and prohibitively expensive clinical trial and manufacturing standards as synthetic pharmaceuticals.
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Breakthrough T1D’s central recommendation, first presented to HHS Secretary Xavier Becerra and department leadership, is for the government to define "unmodified" DDIs as organs under the National Organ Transplant Act (NOTA). Under this proposed framework:
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- Jurisdictional Shift: Unmodified DDIs would fall under the oversight of the Health Resources and Services Administration (HRSA) and the Organ Procurement and Transplantation Network (OPTN), similar to kidneys, hearts, and whole pancreases.
- FDA Role Preservation: The FDA would retain jurisdiction over "modified" islet products—those that have been genetically engineered, encapsulated in protective coatings, or expanded in a lab setting.
- Definition of "Unmodified": This refers specifically to islets isolated and purified from a deceased donor pancreas and transplanted without further modification, a process validated by years of NIH-sponsored research.
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By moving DDIs into the organ framework, advocates argue that the procedure can be integrated into the existing national transplant infrastructure, making it a standard-of-care surgery rather than an experimental drug therapy.
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Chronology: The Road to the RFI
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The journey toward this regulatory milestone has been driven by a grassroots surge within the T1D community and a concerted effort by advocacy groups.
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- Early 2024: The T1D community formally petitioned Breakthrough T1D to prioritize the reclassification of DDIs. The impetus was a growing frustration over the "Catch-22" of islet therapy: while the science had proven the procedure’s efficacy, the "drug" designation meant that insurance companies rarely covered it, and few hospitals could afford the regulatory burden of maintaining a biologic manufacturing license for a transplant procedure.
- March 6, 2024: Breakthrough T1D leadership met with HHS officials to present a formal proposal for reclassification. This meeting included the submission of legal and regulatory white papers outlining how the change would function under existing law.
- Spring/Summer 2024: Advocacy intensified. Breakthrough T1D worked with "Congressional Champions" to draft three separate letters of support to HHS. During this period, the organization collaborated closely with the Diabetes Research Institute Foundation, The Cure Alliance, and the Islets for US Collaborative to build a unified scientific front.
- August 2024: In response to the mounting pressure and the clear scientific consensus, the federal government issued a Request for Information (RFI), seeking public and expert input on potential changes to DDI classification.
- November 2024: Breakthrough T1D submitted its final, formal response to the RFI, backed by over 10,000 public comments from patients, physicians, and researchers.
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Supporting Data: Clinical Validation of Islet Transplantation
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The push for reclassification is not based on theory alone but on decades of rigorous clinical data. Breakthrough T1D’s submission highlights the results of the NIH-sponsored Clinical Islet Transplantation (CIT) Consortium trials, which provide the gold standard for DDI efficacy.
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The CIT-07 and CIT-08 Findings
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The CIT-07 phase 3 trial focused on islet transplantation alone for patients with type 1 diabetes and severe hypoglycemia. The results were transformative:
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- Success Rate: 87.5% of participants achieved an HbA1c level below 7.0% at one year post-transplant.
- Hypoglycemia Elimination: Participants experienced a near-total elimination of severe hypoglycemic events.
- Long-term Efficacy: The follow-up study, CIT-08, demonstrated that freedom from severe hypoglycemia remained above 90% for up to eight years. Furthermore, 74% of recipients achieved insulin independence at some point during their follow-up.
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Global Context and "The Edmonton Protocol"
The United States currently lags behind several other developed nations where islet transplantation is already regulated as an organ or tissue transplant. In Canada (specifically the University of Alberta’s Edmonton Protocol), the United Kingdom, and Australia, DDI transplantation is a recognized standard of care. These countries have successfully integrated islet isolation into their national health systems, providing a blueprint for how the U.S. could adapt its OPTN policies to include DDI.
Official Responses and Expert Advocacy
The formal response to the RFI was a collaborative effort involving some of the most prominent names in diabetes research. Breakthrough T1D specifically acknowledged the contributions of Dr. Piotr Witkowski, Dr. Camillo Ricordi, and Dr. Norma Kenyon, pioneers who have spent decades refining islet isolation techniques and advocating for patient access.
"DDI transplantation is a scientifically and clinically validated therapy that remains largely inaccessible in the United States because of a classification that does not match the biology of DDI or global practice," the organization stated in its conclusion.
The submission also emphasizes that the infrastructure for this change already exists. The OPTN already has standards for organ procurement, personnel requirements, and allocation rules. These could be adapted "quickly and efficiently" to include islets, provided the HHS issues the necessary directive to reclassify them.
The response from the T1D community has been overwhelming. More than 10,000 comments have been filed in the RFI docket, many containing harrowing personal stories of "hypoglycemia unawareness"—a condition where patients cannot feel their blood sugar dropping to dangerous levels, leading to seizures, loss of consciousness, or death. For these individuals, DDI transplantation is not just a convenience; it is a life-saving intervention.
Implications: What Reclassification Would Change
If HHS moves forward with the recommendation to reclassify unmodified DDIs as organs, the implications for the American healthcare system and T1D patients would be seismic.
1. Sustainable Academic Research and Innovation
Currently, academic medical centers wishing to perform islet transplants must navigate the FDA’s Investigational New Drug (IND) or Biologics License Application (BLA) processes. These are designed for pharmaceutical companies with massive capital, not for research hospitals. Reclassification would allow academic surgeons to innovate and perform transplants under the same peer-reviewed medical standards used for other organ transplants, fostering a new era of clinical advancement.
2. Expanded Patient Access and Insurance Coverage
The "drug" designation has long been a hurdle for insurance reimbursement. Most private insurers and the Centers for Medicare & Medicaid Services (CMS) view experimental "drugs" differently than "organ transplants." Reclassification as an organ would pave the way for CMS to provide standardized coverage, making the procedure available to patients based on medical need rather than their ability to pay out-of-pocket or participate in a limited clinical trial.
3. American Competitiveness
By aligning with international standards, the U.S. could regain its position as a leader in T1D cure research. Currently, many U.S. patients travel abroad to receive islet transplants, and American researchers often find fewer regulatory hurdles in Europe or Canada. Reclassification would keep American talent and patients within the domestic healthcare system.
4. Preservation of Regulatory Integrity
Crucially, the proposal does not seek to strip the FDA of its power. By maintaining FDA jurisdiction over "modified" islets, the framework ensures that any product involving genetic engineering or synthetic materials still undergoes the rigorous testing required for manufactured medical products. This "dual-track" system respects the biological nature of human tissue while protecting patients from the risks associated with novel biotechnologies.
Looking Ahead: The Path to November 9th and Beyond
The public comment period for the RFI is set to close on November 9th. Following the closure, HHS, the FDA, and HRSA will begin the arduous task of reviewing the thousands of submissions.
Breakthrough T1D has cautioned that while the RFI is a monumental step, it is not a final guarantee of action. The organization has committed to "working behind the scenes" to ensure that the momentum generated by the community translates into policy.
"We urge HHS to define unmodified DDI as organs under HRSA and the OPTN, to consider enforcement discretion in the interim, and to pair reclassification with CMS action on coverage," the organization’s summary stated.
For the millions of Americans living with Type 1 Diabetes, and particularly the subset for whom modern insulin pumps and continuous glucose monitors are not enough to prevent life-threatening lows, the reclassification of deceased donor islets represents more than just a regulatory change—it represents a path toward a safer, more stable future. As the government deliberates, the T1D community remains vigilant, ready to move from advocacy to implementation in what could be the most significant regulatory update in the history of diabetes care.
