[Congressional Record Volume 154, Number 24 (Wednesday, February 13, 2008)]
[Senate]
[Pages S975-S977]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mr. SPECTER (for himself and Mr. Casey):
  S. 2629. 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.
  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

[[Page S976]]

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.
  There being no objection, the text of the bill was ordered to be 
printed in the Record, as follows:

                                S. 2629

       Be it enacted by the Senate and House of Representatives of 
     the United States of America in Congress assembled,

     SECTION 1. SHORT TITLE.

       This Act may be cited as the ``Nino's Act''.

     SEC. 2. MEDICAID COVERAGE OF DRUGS PRESCRIBED FOR RESEARCH 
                   STUDY CHILD PARTICIPANTS.

       (a) Mandatory Coverage if State Provides Drug Coverage.--
       (1) State plan requirement.--Section 1902(a) of the Social 
     Security Act (42 U.S.C. 1396a(a)) is amended--
       (A) in paragraph (69), by striking ``and'' at the end;
       (B) in paragraph (70), by striking the period at the end 
     and inserting ``; and''; and
       (C) by inserting after paragraph (70) the following new 
     paragraph:
       ``(71) in the case of a State plan that provides medical 
     assistance for prescribed drugs under section 1905(a)(12), 
     provide for such medical assistance to include coverage for 
     any drug, biological product, or insulin prescribed for a 
     child (including any such drug, product, or insulin that is 
     self-administered) who--
       ``(A) is eligible for medical assistance under the State 
     plan (including a child who is eligible only on the basis of 
     paragraph (10)(A)(i)(VIII));
       ``(B) is a current or former participant in a research 
     study conducted or funded (in whole or in part) by the 
     National Institutes of Health; and
       ``(C) satisfies the requirements of subparagraphs (B), (C), 
     and (D) of subsection (dd)(1).''.
       (2) Mandatory coverage of drugs of research study child 
     participants who are not otherwise eligible for medicaid if 
     the state offers drug coverage.--
       (A) In general.--Section 1902(a)(10)(A)(i) of the Social 
     Security Act (42 U.S.C. 1396b(a)(10)(A)(i)) is amended--
       (i) in subclause (VI), by striking ``or'' at the end;
       (ii) in subclause (VII), by adding ``or'' at the end; and
       (iii) by adding at the end the following new subclause:

       ``(VIII) who are research study child participants 
     described in subsection (dd)(1), but only if the medical 
     assistance made available by the State includes prescribed 
     drugs under section 1905(a)(12),''.

       (B) Group described.--Section 1902 of the Social Security 
     Act (42 U.S.C. 1396a) is amended by adding at the end the 
     following new subsection:
       ``(dd)(1) Research study child participants described in 
     this subsection are individuals who--
       ``(A) are not otherwise eligible for medical assistance 
     under the State plan;
       ``(B) have not attained age 19;
       ``(C) have been certified by a physician participating in a 
     research study conducted or funded (in whole or in part) by 
     the National Institutes of Health to be current or former 
     participants in such trial or study who have a specific 
     disease or condition that--
       ``(i) is or has been successfully treated under such trial 
     or study with a prescribed use of a drug, biological product, 
     or insulin that is not approved under the Federal Food, Drug, 
     and Cosmetic Act; and
       ``(ii) is likely to continue to be successfully treated 
     with such drug, product, or insulin; and
       ``(D) do not have other health coverage for such drug, 
     product, or insulin.
       ``(2) A State shall redetermine not less than every 2 years 
     the eligibility of an individual for medical assistance who 
     is eligible solely on the basis of subsection 
     (a)(10)(A)(i)(VIII).
       ``(3) For purposes of this subsection and paragraphs 
     (10)(A)(i)(VIII) and (71) of subsection (a), the term 
     `research study' means a clinical study, including an 
     observational (or natural history) study, or a clinical 
     trial, to test an experimental therapy.''.
       (C) Medical assistance limited to coverage of the research 
     or observational trial drugs, biological product, or 
     insulin.--Section 1902(a)(10) of the Social Security Act (42 
     U.S.C. 1396a(a)(10)) is amended in the matter following 
     subparagraph (G)--
       (i) by striking ``and (XIV)'' and inserting ``(XIV)''; and
       (ii) by inserting ``, and (XV) the medical assistance made 
     available to a research study child participant described in 
     subsection (dd)(1) who is eligible for medical assistance 
     solely on the basis of subparagraph (A)(10)(i)(VIII) shall be 
     limited to medical assistance for a drug, biological product, 
     or insulin that is prescribed for the participant as a result 
     of participation in such trial or study (including any such 
     drug, product, or insulin that is self-administered)'' before 
     the semicolon.
       (D) Conforming amendment.--Section 1903(f)(4) of such Act 
     (42 U.S.C. 1396b)(f)(4)) is amended in the matter preceding 
     subparagraph (A) by inserting ``1902(a)(10)(A)(i)(VIII),'' 
     after ``1902(a)(10)(A)(i)(VII),''.
       (b) Presumptive Eligibility.--Section 1920B of the Social 
     Security Act (42 U.S.C. 1396r-1b) is amended--
       (1) in the section heading, by inserting ``or research 
     study child participants'' after ``patients'';
       (2) in subsection (a), by inserting ``or a child who is 
     eligible for medical assistance under the State plan 
     (including a child who is eligible only on the basis of 
     section 1902(a)(10)(A)(i)(VIII) but subject to the limitation 
     on medical assistance for such a child under clause (XV) of 
     the matter following section 1902(a)(10)(G)), is a current or 
     former participant in a research study conducted or funded 
     (in whole or in part) by the National Institutes of Health, 
     and satisfies the requirements of subparagraphs (B), (C), and 
     (D) of section 1902(dd)(1)'' after ``patients)'';
       (3) in subsection (b)(1)(A), by inserting ``or subsection 
     (a)'' after ``1902(aa)''; and
       (4) in subsection (d), in the flush language following 
     paragraph (2), by striking ``for purposes of clause (4) of 
     the first sentence of section 1905(b)'' and inserting ``for 
     purposes of the first sentence of section 1905(b) (and, in 
     the case of medical assistance furnished to an individual 
     described in section 1902(aa), for purposes of clause (4) of 
     such sentence)''.
       (c) Notice of Medicaid Coverage for Research Study Child 
     Participants.--
       (1) In general.--Not later than 90 days after the date of 
     enactment of this Act, the Secretary of Health and Human 
     Services, in consultation with the Director of the Institutes 
     of Health and State Medicaid Directors, shall--
       (A) develop a written notice for child participants in 
     research studies (as defined in section 1902(dd)(3) of the 
     Social Security Act, as added by subsection (a)(2)(B)) 
     conducted or funded (in whole or in part) by the National 
     Institutes of Health who are likely to eligible for medical 
     assistance for a drug, biological product, or insulin 
     prescribed for such participants as a result of participation 
     in such a study (including any such drug, product, or insulin 
     that is self-administered) in accordance with paragraph 
     (10)(A)(i)(VIII) or (71) of section 1902(a) of the Social 
     Security Act (42 U.S.C. 1396a(a)) (as added by subsection 
     (a)), of the availability of such assistance; and
       (B) establish procedures for making such notice available 
     to the child participants through physicians participating in 
     such research studies or such other means as the Secretary 
     determines appropriate.
       (2) Authorization of appropriations.--There is authorized 
     to be appropriated for fiscal year 2008 and each fiscal year 
     thereafter such sums as may be necessary to carry out this 
     subsection.
       (d) Effective Date.--The amendments made by this section 
     apply to medical assistance for items and services furnished 
     on or after the date of enactment of this Act, without regard 
     to whether final regulations to carry out such amendments 
     have been promulgated.

  Mr. CASEY. Mr. President, I rise today to speak about a critical 
health issue affecting thousands of our children every day but about 
which few people have ever even heard. All across this country, 
thousands of children suffer from rare genetic diseases called ``orphan 
diseases,'' thus named because

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of the relatively small number of people these diseases strike.
  An orphan disease is defined as affecting fewer than 200,000 people. 
The National Institutes of Health, NIH, estimate that there are 
approximately 6,000 of these orphan diseases, affecting about 25 
million Americans on the whole. Most of these rare diseases are genetic 
and many affect children.
  Last spring, I met with a group of mothers who shared their struggles 
and frustrations in getting ongoing and consistent treatment for their 
children, each of whom suffers from an orphan disease. Many of these 
parents had been able to enroll their children in clinical trials at 
the NIH and had found experimental treatments for their children that 
had proven extremely successful. The doctors at NIH do miraculous work 
in finding treatments for children with rare genetic diseases. But 
oftentimes, when the trial ends, these children and parents are left on 
their own, with no access to the previously free and effective 
treatment that their children were getting.
  Imagine if you can, for one moment, the predicament of these children 
and their parents? After months and sometimes years of first not 
knowing what was ailing their sick children, desperately seeking help, 
then finally getting a diagnosis, only to find out that there was no 
FDA approved treatment. Then after searching for some kind of treatment 
and then finally, finally finding--and being admitted to--a clinical 
trial on medication that miraculously gave their children the ability 
to function like other kids--to be able to play soccer and go to school 
and have friends over and just have the energy to be a child. For all 
of us who are parents, you can imagine the joy of seeing your child 
finally alleviated from the suffering he or she has been going through, 
finally able to enjoy him- or herself and do all the things that 
children are supposed to do.

  Then imagine, if you can, what it would be like to suddenly have that 
taken away. The clinical trial ends, or funding for the trial ends. 
Suddenly, you no longer have access to this drug that your child needs 
to be able to function, to do their homework, eat well and have fun. If 
it is a drug that has not been approved by the Food and Drug 
Administration or specifically approved for a child's particular 
disease, then insurance companies typically will not cover it because 
the treatment is considered ``experimental.'' In some cases, a drug has 
been approved for other uses than the orphan disease, known as ``off-
label'' use. If a family has enough insurance, and there is off-label 
FDA approval, sometimes families can get coverage of the drugs. If not, 
the resulting cost to families is astronomical--ranging anywhere from 
$10,000 to $30,000 per month.
  This is what happened to Nino Todaro, a young boy from Newville, 
Pennsylvania, and that is why Senator Specter and I are today 
introducing Nino's Act. Nino suffers from Periodic Fever Syndrome, an 
unpredictable genetic condition that can cause uncontrolled 
inflammation throughout the body. When this disease acts up, Nino has 
days where he cannot do much more than lie on the couch. Left 
untreated, this condition could leave Nino unable to walk and even be 
life-threatening. Fortunately Nino found help through an NIH clinical 
trial, but funding ran out last year. The drug that returned Nino to a 
joyous soccer-playing kid was approved for arthritis and Crohn's 
disease, but not Periodic Fever Syndrome. Facing costs of $12,000 a 
month, and initial rejections from their insurance company, Nino's 
parents turned to Congress.
  Nino's Act will allow children to transition out of successful 
treatment in NIH studies without a gap in treatment. There are 
thousands of children like Nino across this country who desperately 
need the continuity of ongoing successful treatment for their rare 
disorders. These are children who have been very ill, sometimes 
incapacitated, and have been able to resume normal childhoods through 
successful drug treatment. Parents advocating for their children 
understandably refuse to accept that their children have no choice but 
to regress because their insurance company will not cover humongous 
medical bills that no middle class family could even begin to absorb.

  No parent should ever have to face a situation in which the care they 
need for their seriously ill child is too expensive or held up by 
regulatory red tape. It is unthinkable to me that any ill child in this 
country, the richest nation on earth, with all our medical 
advancements, should ever be denied medical treatment that is available 
and proven successful. Our bill will give these children and their 
parents peace of mind that when a study ends, their children's 
successful ongoing treatment will not be threatened. To address this, 
Nino's Act will require Medicaid to cover the cost of treatment of in 
the event that a child's health insurance does not.
  This is the least we can do for these children and families. No child 
for whom treatment is available should have to forego that treatment to 
the serious detriment of their health. That is just plain wrong. 
Senator Specter and I share the belief that ensuring ongoing treatment 
for children with rare disorders is something this Congress should get 
behind. I urge my colleagues to support Nino's Act and I will work hard 
for its passage. My hope is it will go a long way toward ensuring that 
children with orphan diseases can get the successful treatment they 
deserve, freeing them and their families to focus on what is truly 
important--keeping them well, and living out happy and productive 
lives.
                                 ______