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IMPLEMENTATION OF THE PANCREATIC ISLET CELL TRANSPLANTATION ACT OF 2004
Through Public Law No. 108-362, the Pancreatic Islet Cell Transplantation Act of 2004, the function of the DMICC has been extended to include in its annual reports an assessment of the Federal activities and programs related to pancreatic islet cell transplantation. To this end, the following examples are provided.
The Department of Health and Human Services (HHS) vigorously continues to pursue scientific opportunities in the field of islet transplantation through resources provided through the annual appropriations process, as well as through support of the Special Statutory Funding Program for Type 1 Diabetes Research, which is a special appropriation that the NIDDK administers on behalf of the Secretary, HHS, in collaboration with multiple NIH Institutes and Centers and the CDC. These resources are supporting multifaceted research efforts that aim to overcome current barriers that limit the widespread clinical use of islet transplantation—such as the shortage of available islets. As improvements in islet transplantation are realized, the shortage of islets threatens to pose an even greater challenge in the future. Research is ongoing through numerous efforts, such as by the NIDDK-led Beta Cell Biology Consortium, to understand development and function of insulin-producing beta cells, which are found in pancreatic islets. Increased knowledge could help researchers learn how to grow an unlimited supply of beta cells in the laboratory for use in transplantation. Research is also being pursued on alternative sources of islets. For example, the NIH remains committed to supporting research on xenotransplantation. The NIAID, in collaboration with the NIDDK, has sponsored a recent initiative on “Immunobiology of Xenotransplantation.” The purpose of this initiative is to support cooperative, interdisciplinary research programs to pursue studies on pre-clinical, porcine to non-human primate models of xenotransplantation. Two of the grants recently awarded through this initiative involve islet transplantation. The NIDDK also supports small business innovation research (SBIR) grants involving xenotransplantation, including one involving an encapsulation technology for transplanting pig islets into non-human primates. If successful, this approach has the potential to eliminate the need for immunosuppression. The report of the HHS Secretary’s Advisory Committee on Xenotransplantation is expected to be published soon.
To overcome additional barriers in islet transplantation, the NIDDK and NIAID created a major new Clinical Islet Transplantation Consortium (CIT). Investigators in the CIT are in the process of developing seven clinical trials that have the potential to greatly improve the safety and long-term success of methods for islet transplantation. One of these trials is mandated by Title VII, Section 733 of the Medicare Prescription Drug, Improvement, and Modernization Act (Public Law 108-178). That law requires the Secretary, HHS, acting through the NIDDK, to conduct a clinical investigation of pancreatic islet cell transplantation which includes Medicare beneficiaries. Implementation of this law requires coordination between the NIH and the CMS because CMS will pay for the routine patient costs, such as immunosuppression and patient followup care, as well as for pancreas acquisition and islet preparation. In addition to NIDDK, NIAID, and CMS, other Federal agencies have also been involved in a series of meetings regarding this clinical investigation, including FDA, HRSA, and the Office of the Assistant Secretary for Planning and Evaluation/HHS. Broader input from other relevant Federal agencies was solicited at a DMICC meeting on “islet transplantation” on November 23, 2004. This meeting included discussion about the Medicare trial. Numerous steps have been taken to implement a well-designed and well-controlled investigation that will advance the field of islet transplantation. In February 2005, the NIH convened an ad hoc expert panel of scientists who were asked to make recommendations to the CIT investigators involved in designing the Medicare trial with respect to parameters such as clinical trial design, including appropriate numbers of patients to enroll and appropriate trial endpoints. Based on recommendations garnered at this meeting, the investigators have developed a protocol, which was reviewed by the NIDDK Data and Safety Monitoring Board (DSMB) in September 2005. Additionally, three meetings have been held with the FDA in September and November 2005. The coverage group at CMS has also been reviewing the study protocol. The protocol is being revised to reflect comments received from the FDA and the DSMB. It is anticipated that the congressionally mandated clinical investigation will begin in calendar year 2006.
Important components of clinical trials, such as those being conducted through the CIT, are careful monitoring and reporting of findings. To facilitate the collection and coordination of data from islet transplantation trials, the NIDDK created the Collaborative Islet Transplant Registry (CITR). The Registry collects, analyzes, and communicates comprehensive and current data on all islet cell transplants performed in North America and five European centers (www.citregistry.org). The Registry released its second annual report in September 2005, to disseminate the knowledge gleaned from researchers in the United States and Canada. CITR works closely with the United Network for Organ Sharing (UNOS), which is overseen by HRSA, and the Islet Cell Resource Centers (ICRs), which are supported by the NCRR and the NIDDK. In addition, the CIT has developed agreements to share data with the CITR and the ICRs. Federal agencies involved in islet transplantation will continue these types of coordination efforts to share data across multiple entities that are utilizing data from islet transplantation trials.
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