[Congressional Record Volume 155, Number 27 (Tuesday, February 10, 2009)]
[Senate]
[Pages S2082-S2083]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mr. SPECTER (for himself and Mr. Casey):
  S. 406. A bill to amend title XIX of the Social Security Act to 
provide Medicaid coverage of drugs prescribed for certain research 
study child participants; to the Committee on Finance.
  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.

[[Page S2083]]

  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 than 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.
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