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Public Statements

Statements on Introduced Bills and Joint Resolutions

Floor Speech

Location: Washington, DC



Mr. SPECTER. Mr. President, I have sought recognition today to introduce Nino's Act, to provide for the continuance of successful treatment for children who are required to leave National Institutes of Health, NIH, research studies. The NIH provides the greatest medical research in the world on innumerable diseases, including cancer, Alzheimer's, Parkinson's. The NIH also conducts excellent research on diseases that affect children. To conduct that research many brave children must partake in research studies including observational, or natural history, studies and clinical trials to test experimental therapies. This participation is critical to understanding diseases and ultimately finding cures at the NIH.

To participate in the trials and studies, children and their families often make considerable sacrifices. Families will travel great distances to receive treatment that may provide relief from the child's illness. In many cases, parents and doctors will have tried many treatments for the child's disease about which little may be known or understood. The NIH studies represent an opportunity for both the medical community to learn more about the disease and the child to be studied and potentially treated by the best researchers in the world.

When the experimental treatments are successful, it is cause for great celebration for the child. The joy, however, can end quickly as the studies come to end but the children who have been part of them continue to be stricken by these terrible illnesses.

Nino's Act seeks to transition children out of the NIH studies as they end so they don't experience a gap in their important treatment. This legislation continues the successful treatment initiated in NIH studies by providing access to the same prescription drugs for children who are required to leave NIH clinical studies due to the studies ending, researcher leaving, or other reason. Often drugs that are used successfully in these studies have not yet been approved by the Food and Drug Administration or have not been approved for treatment of the child's specific disease. As such, it is nearly impossible for children to get access or insurance coverage for these drugs. This bill makes that access possible by requiring Medicaid to cover the cost of treatment in the event that the children's health insurance does not.

On occasion, insurers will cover the cost of the treatment for these children if they have adequate insurance and the FDA has approved the drug for off-label uses. More often then not, however, children do not have health insurance, or have insufficient insurance to obtain these drugs. As a result, children suffer their diseases without relief from the treatment as established in the clinical NIH studies. To ensure that these children have access to successful care post-study, Nino's Act requires Medicaid to cover the cost of treatment for these children. While Medicaid access is traditionally based on income, due to the importance of these drugs to the child's well-being the income component will be waived. To ensure Medicaid is not unnecessarily covering medication, Nino's Act requires the physicians participating in the research to certify the treatment as successful and essential.

This important issue was introduced to me by Lori Todaro of Newville, PA. Lori's son Nino suffers from Undifferentiated Auto-Inflammatory Periodic Fever Syndrome. This disease takes a devastating toll on those who suffer from it. The auto-inflammatory disease can cause joint inflammation arthritis, Crohns, colitis, irritable bowel syndrome, and cyclical high fevers. Treatment for Periodic Fever Syndrome is experimental at best; Lori and Nino have visited a number of doctors and tried many medications in an effort to control the disease.

In 2003, Nino was fortunate to be selected to take part in an observational study at NIH in Bethesda, Maryland for Undifferentiated Auto-inflammatory Periodic Fever Syndrome. During the course of the study, Nino was given a new medication and his condition greatly improved. Before he participated in the study he was being fitted for wheelchairs and was home schooled because his symptoms were so disruptive and unpredictable. The NIH treatment allowed him to resume a normal life and enabled him to attend school and play soccer. While Nino's treatment was successful he could not remain part of the study indefinitely and was encouraged to seek coverage for his treatments through his private insurer. Initially, the Todaro's insurer would not agree to cover the cost of the experimental drug and only after an intense lobbying effort by Lori, did the insurer agree to cover Nino's prescriptions.

Nino's story is a successful one, but also serves to highlight the issue that children and their families are facing as they transition out of NIH studies. For many, NIH trials are a source of hope for relief from the worst diseases known to man. The excellent doctors and research teams at NIH make invaluable contributions to our understanding of complex and debilitating diseases. This legislation seeks to amplify the NIH's contributions by allowing America's sickest children to continue their successful treatment under Medicaid coverage. I encourage my colleagues to work with Senator Casey and me to move this legislation forward promptly.

Mr. President, I ask unanimous consent that the text of the bill be printed in the Record.


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