Islets for US
Collaborative
Why passing the ISLET Act now
is so important?
Islet transplantation is approved for clinical use worldwide...
... But why not in the US?
"...this is because, human islets have been regulated as drugs,
instead of as organs for transplantation like in all other developed countries worldwide..."
...scientific evidence strongly supports the need for an urgent update in the US, regarding the regulation of islets as organs...
Professor of Surgery, Medicine, Biomedical Bioengineering, Immunology at University of Miami
Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center
expert, inventor and "father" of the field of islet transplantation
THE ISLET ACT
GREAT NEWS
June 22, 2023
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In response to the direct request from the Presidents of the American Society of Transplant Surgeons, the American Society of Transplantation, and experts from the Islet for US Collaborative representing concerns of the entire transplant community, Senator Mike Lee and Congressman Matt Rosendale introduced the ISLET Act to the Senate and to House of Representatives, respectively..
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The ISLET Act is long-overdue and includes human cadaveric islets into the regulatory framework developed for organ transplantation under HRSA, UNOS and OPTN.
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THE ISLET ACT mandates islet transplantation to be provided safely and effectively in accredited organ transplantation programs, which have appropriate infrastructure, resources, and experience to provide medical care to transplant patients requiring life-long immunosuppression.
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The ISLET Act allows open access to islet transplantation for the most disadvantage patients with type 1 diabetes in the US and coverage by a medical insurance as a standard of care, adopting the same approach proven to be safe and effective in other countries (Canada, EU, Australia, Japan).
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It will promote healthy competition between transplant centers, cost-effectiveness, innovation, and progress towards the reduction of immunosuppression medication, and ultimately, the cure for diabetes.
BAD NEWS
Clouds over the ISLET Act
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In contrast to many other countries, the FDA regulates human cadaveric islets as drugs hampering the progress and transition of the islet transplantation from the experimental to the standard of care procedure in the US over the last 10 years.
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As experts in the field and representing the entire transplant community, we have been voicing our concerns and calling for the necessary regulatory update for last several years. However, the FDA has not been in favor of any changes (Am J Transplant 2020) (Am J Transplant 2021) (Transplant International 2021) (Front Endocrinol 2022).
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In response to our call, Senator Mike Lee and other Congressmen introduced the ISLET Act, which would allow proper regulation of islets as organs for transplantation in the US.
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Unfortunately, a few days later, on June 28, 2023, to oppose the ISLET Act, the FDA granted a license and exclusive rights to a for-profit company (CellTrans) for cadaveric islet distribution as a drug in the US. It is so disappointing as it happened despite multiple warnings from the transplant community and experts in the field about the serious negative effects of such a decision on our patients and the entire field. (J Clinical Medicine 2021)
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The decision was a big surprise, as the FDA had previously reported that the quality of the CellTrans islet product after processing could NOT be objectively confirmed, which is required for any drug safety and efficacy assurance and new drug license approval. In addition, there is a lack of quality consistency between the product batches, which is required for drugs. Even sterility of the islet product was unknown (Transplantation 2021). It is hard to interpret the FDA's decision differently than purely political.
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By granting exclusive rights to a private company, the FDA not only compromised patient safety but substantially complicated the path for passing the ISLET Act and the implementation of the critical regulatory update.
Here is a list of serious negative downstream effects of the FDA’s decision of granting the exclusive rights to human islets distribution to a private company (Front Endocrin 2022):
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For-profit company received seven years of marketing exclusivity under the Orphan Drug Act. This is inconsistent with the federal prohibition on commercialization of human organs under the National Organ Transplantation Act, which includes islets as subparts of an organ into the definition of human organs (Am J Transplant 2021) .
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Patient safety is in jeopardy as a private, for-profit company will exclusively distribute human islets, which quality cannot be verified before the transplantation (even the sterility cannot be). (Transplantation 2021).
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Patient safety will be additionally compromised since:
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Islets regulated as drugs can be transplanted outside the UNOS/OPTN accredited transplant programs. After islet transplantation patients require complex supporting medical therapy, which includes immunosuppression. Patient care by a team from the UNOS/OPTN accredited programs is critical for patient safety and the effectiveness of the therapy.
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Thorough patient oversight and public reporting of clinical outcomes are not required for drugs as is mandated for organ for transplantation..
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A single private company received unprecedented control over the entire field of islet transplantation in the US.
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As the only islet supplier in the country, a single company can make its own profit-driven decision about who, where, how and if it will be transplanting the islets.
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Transplant centers will have no alternative source of islets for clinical use.
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Transplant centers will have no control over the quality of islets for their patients
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The company has a significant leverage in terms of the contract with any transplant center to provide islets for transplantation, including the price of islets.
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CellTrans will remain an exclusive islet distributor even if properly declares waiving exclusive rights to islet distribution. None of the commercial entities will risk investing tens of millions of dollars and many years of work and investment knowing that waiver of exclusive rights can be revoke by CellTrans at any time (see Orphan Drug Designation rules).
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Lack of healthy competition between islet processing centers will NOT promote cost-effectiveness, innovation and progress in the field.
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This will limit access to islet transplantation due to increased costs and decreased availability of islets compared to a situation where islets are regulated as organs and are exempt from BLA.
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Profit-driven decisions may lead to shutting down islet distribution at any time and subsequent lack of access to the procedure for patients in the US. In one of the scenarios: CellTrans could potentially be acquired by a pharmaceutical competitor leading to the closure of the islet distribution for their potential advantage, displacing islets with a competing product.
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For a better illustration of the complete negative impact, imagine consequences if FDA has granted rights to the exclusive distribution of human hearts or livers for clinical transplantation to only one for-profit entity in the US.
The current situation is very concerning and disappointing, which happened despite warnings from the transplant community.
In contrast, the ISLET Act shifts cadaveric islets from drugs to the regulatory framework for organ transplantation which is constantly optimized to provide safe and effective procedures.
ISLET Act will allow existing UNOS/OPTN accredited transplant centers to develop their own islet transplantation programs. Patients will get open access to the procedure reimbursed by insurance like other organ transplantations. It will stimulate healthy competition between centers, cost-effectiveness, innovation and progress in the field.
Please support the ISLET Act now, before it is too late.
Please send an email with the confirmation of your support to isletact@gmail.com
Please include your first and last name and the name of the state you live in.
See list of current supporters
Watch stories of our islet transpant patients supporting the Islet Act
Ashley's story
Mohammed's story
Stacy's story
