Islet cell transplant's "perfect storm" results in new legislation, CMS reg, $75 million in research funding.
In the last 7 months, pancreatic islet cell transplantation has been swept to the forefront of public consciousness by the confluence of several powerful political forces: the passage of its own legislative act; the publishing of a proposed rule for federally-sponsored clinical trials; and the awarding of $75 million to a "network" of clinical research centers to conduct research on patients with type 1 diabetes.
The events leading to the convergence began last February with the introduction of the Pancreatic Islet Cell Transplantation Act of 2004 by Representative George Nethercutt, Jr., (R-WA). The purpose of the bill was to "increase the supply of pancreatic islet cells for research, and to provide for better coordination of Federal efforts and information on islet cell transplantation." The bill-H.R. 3858-was passed by the House and the Senate on October 5th.
On August 3, the Department of Health and Human Services (HHS) Centers for Medicare and Medicaid (CMS) published a "Final Rule on Pancreatic Islet Cell Transplantation in Clinical Trials." Although the transplant community has expressed "significant concern" over some provisions, on October 1 Medicare payments began for Medicare beneficiaries participating in a pancreatic islet cell transplant clinical trial.
Finally, on October 4, the National Institutes of Health announced plans to award about $75 million over the next 5 years to 5 clinical research centers and 1 data coordinating center to conduct studies on islet cell transplantation in patients with type 1 diabetes.
Pancreatic Islet Cell Transplantation Act of 2004
Representatives Nethercutt, and the bill's co-sponsor Diana DeGette (D-CO) are credited with directing the islet cell legislation through Congress and on to the White House where it is expected to be signed into law by President Bush.
"My daughter was diagnosed with juvenile diabetes as a little girl. Since that day nearly 20 years ago, I have worked for a cure for this disease," Nethercutt said in a press release announcing passage of the bill. "Right now more research is needed, and roadblocks need to be removed to enable more eligible patients to receive transplants. [The legislation] will help enable more testing of this procedure, and help more patients live without being dependent on insulin injections."
DeGette emphasized that the legislation will help provide access to islet cell transplantation that up-to-now has been slowed because of non-scientific roadblocks. The bill addresses these concerns "by increasing the pancreas supply, disseminating information on this treatment and coordinating federal involvement in this procedure," she said.
Islet cell transplant surgeon Dixon Kaufman, MD, said the ultimate impact of the legislation will be determined by the amount of funds made available to take advantage of the scientific breakthroughs that occur.
"If the policies give incentives to organ procurement organizations to procure more pancreata for transplantation, and more federal funding for research is available the bill will be very effective," Kaufman told Transplant News. "My interpretation of the bill is congress has recognized there is a tremendous shortage of islet cell tissue and we need to maximize what is currently available."
Kaufman pointed out the legislation specifically cites research on xenotransplantation as a potential source for islet cells. "I think it great that the bill mentions xenotransplantation," he said. "In my opinion the first breakthrough in xenotransplantation will be in islet cell transplantation before any other kind of cellular procedure."
Kaufman is professor and vice chair of research in the department of surgery division of transplantation at the Feinberg School of Medicine at Northwestern University in Chicago, IL.
Here are the provisions of the bill:
Sec. 2 Organ Procurement Organization Certification
( c ) Pancreata procured by an organ procurement organization and used for islet cell transplantation or research shall be counted for purposes of certification or recertification.
Sec. 3 Annual Assessment of Pancreatic Islet Cell Transplantation
"In each annual report prepared by the Diabetes Mellitus Interagency Coordinating Committee . . .the Committee shall include an assessment of the Federal activities and programs related to pancreatic islet cell transplantation. Such assessment shall, at a minimum, address the following:
(1) The adequacy of Federal funding for taking advantage of scientific opportunities related to pancreatic islet cell transplantation;
(2) Current policies and regulations affecting the supply of pancreata for islet cell transplantation;
(3) The effect of xenotransplantation on advancing pancreatic islet cell transplantation;
(4) The effect of United Network for Organ Sharing policies regarding pancreas retrieval and transplantation;
(5) The existing mechanisms to collect and coordinate outcomes data from existing isle cell transplantation trials;
(6) Implementation of interagency clinical investigations; and
(7) Recommendations for such legislation and administrative actions as the Committee considers appropriate to increase supply of pancreata available for islet cell transplantation.
CMS Final Rule for Pancreatic Islet Cell Transplantation at NIH
The Final Rule, published in the August 3 Federal Register, directs the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to "conduct a clinical investigation of pancreatic islet cell transplantation that includes Medicare beneficiaries. [The law] "provides for Medicare payments, beginning no earlier than October 1, 2004, for the routine costs as well as the costs of the transplantation and appropriate related items and services for Medicare beneficiaries who are participating in a clinical trial as if such transplantation were covered under Medicare Part A or Part B. Routine costs are defined as reasonable and necessary routine patient care costs including immunosuppressive drugs and other follow-up care.
An informal collaborative of 10 major transplant organizations played a major role in crafting the provisions contained in the CMS Final Rule. The organizations included: American Society of Transplant Surgeons, American Society of Transplantation, Association of Organ Procurement Organizations, Juvenile Diabetes Research Foundation, United Network for Organ Sharing, Cell Transplant Society, IPITA, The Transplantation Society, North American Transplant Coordinators Organization, and ASMHTP.
In a letter to ASTS members in early September, Richard Howard, MD, PhD, the society's president, outlined the collaborative's efforts over the past 5 months, noted 2 major objectives achieved, and outlined several "significant areas of concern" that will continue to be addressed with CMS.
Howard explained the group had major concerns with the original draft of the rule implementing the payment methodology for pancreas recovery, islet cell isolation, and islet transplantation.
"We disagreed with CMS' proposal to pay for islet cell acquisition costs on the basis of a flat 'add-on' rate in lieu of using the standard organ acquisition cost methodology that is used to determine payment for pancreas and other solid organ acquisitions," Howard wrote. "We also asked CMS to reconsider diagnosis-related group (DRG) assignments to more clinically appropriate DRGs or create a new DRG for islet transplantation alone."
NIH funding centers to study islet cell transplantation
The final element in the perfect storm -- $75 million to fund 5 clinical centers to do type 1 diabetes research over the next 5 years -- was the result of a little known provision in the Balance Budget Act of 1997 which appropriated a total of $1.14 billion for type 1 diabetes research for FY 1998 through FY 2008. The Special Statutory Funding Program augments regularly appropriated funds that the Department of Health and Human Services (HHS) receives annually for diabetes research through the Labor-HHS-Education Appropriations Committee.
The NIH announced October 4 that the $75 million has been awarded to 4 centers located in the US-the University of Iowa (Data Recording Center), Iowa City-Dr. William Clark, principal investigator; University of Miami, FL-Camillo Ricordi, MD, principal investigator; University of Minnesota, Minneapolis-Dr. Bernhard Hering, principal investigator; University of Pennsylvania, Philadelphia-Ali Naji, MD, principal investigator; and 2 international centers-University of Alberta, Edmonton, Alberta, Canada-James Shipiro, MD, principal investigator; and Uppsala University, Uppsala, Sweden-Olle Korsgren, MD, principal investigator.
Of the centers, 3 have developed collaborations with other centers: The University of Miami-University of Virginia and Baylor University in Houston, TX; University of Alberta, Edmonton - Emory University in Atlanta, GA; The University of Minnesota - Northwestern University, Chicago, IL, and the University of California, San Francisco.
Two NIH institutes sponsor the consortium - the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), and the National Institute of Allergy and Infectious Diseases (NIAID). Dr. Thomas Eggerman oversees the consortium for NIDDK.
"This is a huge development," Kaufman told Transplant News. "What the NIDDK has done [with the funding] is set up some incredible programs for diabetes research and finding new treatments. This is just one of the multiple programs set up as a result of the special funding program. All these things are linked-the new islet cell transplant act, the CMS regulations and this huge new clinical trial. It's no accident that all of these developments are related. All are part of a choreographed effort that works."
NIH said the studies will focus on improving the safety and long term success of methods for transplanting insulin-producing islets in people whose own islets have been destroyed by the autoimmune process that characterizes type 1 diabetes. Some studies will focus on improving combined islet and kidney transplants in patients with type1 diabetes and kidney failure, a common complication of diabetes.
NIH said the consortium researchers will design studies to:
*improve isolation and viability of islets;
*reduce complications of the transplant procedure, e.g., bleeding and clotting;
*reduce the side effects of immunosuppression;
*trace the fate of islets after transplantation and determine why donor islets sometimes fail; and
*evaluate new ways to safety prevent immune rejection of donor tissues.
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|Title Annotation:||Pancreatic Islet Cell Transplantation Act of 2004; Centers for Medicare and Medicaid Services|
|Comment:||Islet cell transplant's "perfect storm" results in new legislation, CMS reg, $75 million in research funding.(Pancreatic Islet Cell Transplantation Act of 2004)(Centers for Medicare and Medicaid Services)|
|Date:||Oct 15, 2004|
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