[Congressional Record Volume 165, Number 206 (Thursday, December 19, 2019)]
[Senate]
[Page S7211]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
By Ms. COLLINS (for herself, Mr. Jones, Ms. McSally, and Mr.
Menendez):
S. 3116. A bill to enable States to better provide access to whole
genome sequencing clinical services for certain undiagnosed children
under the Medicaid program, and for other purposes; to the Committee on
Finance.
Ms. COLLINS. Mr. President, I rise today to introduce the Ending the
Diagnostic Odyssey Act. This legislation gives States the option of
providing whole genome sequencing WSG clinical services through
Medicaid for children with a disease that is suspected to have a
genetic cause, at an enhanced Federal matching rate for 3 years. I am
pleased to be joined by Senators Jones, McSally, and Menendez.
Children with rare diseases will spend on average 5 to 7 years on
diagnostic odyssey, and 30 percent of those children will not survive
beyond the age of 5 years old. The average patient sees seven different
physicians in that time. The wait to find a cause--nevermind a cure--
can be excruciating. Parents try to project a calm and reassuring
presence for their child while facing a whirlwind of doctor
appointments, hospital visits, and unanswered questions.
Undeniably, we are making progress in both accelerating research
funding for rare diseases as well as in the development of diagnostics.
In 2014, the National Institutes of Health, NIH launched a program
called the Undiagnosed Disease Network UDN. In its first 20 months, the
UDN accepted 601 participants undiagnosed by traditional medical
practices. Of those who completed their UDN evaluation during this
time, 35 percent were given a diagnosis. Many of these diagnoses were
rare genetic diseases, including 31 previously unknown syndromes.
In May, the Director of the National Institutes of Health, Dr.
Francis Collins, wrote a blog post on how whole genome sequencing--
combined with artificial intelligence, AI--can now be used to diagnose
genetic diseases in seriously ill babies in fewer than 24 hours. Dr.
Collins writes: ``I would submit that there is no other technology in
the history of planet Earth that has experienced this degree of
progress in speed and affordability.''
For parents of children with an undiagnosed illness, answers cannot
come soon enough. There are approximately 7,000 rare diseases known
today. Approximately 80 percent of rare diseases are genetic, and about
one-half of all rare diseases affect children. For example, Alstrom
syndrome is an extremely rare and complex genetic disorder.
Approximately 1,200 affected individuals have been identified
worldwide, which makes a obtaining a correct diagnosis challenging.
Characteristics of Alstrom syndrome include vision disturbances,
sensorineural hearing impairment, cardiomyopathy, obesity, kidney
dysfunction, and diabetes.
Robin Marshall, executive director of the Alstrom Syndrome
International, located in Mount Desert Islanas, ME, has said that
``whole Genome Sequencing has changed the lives of those we represent
by enabling earlier and more accurate diagnosis, fostering more timely
and appropriate medical care, and unlocking a host of social services
to combat the educational and psycho-social complications that our
children confront.''
By giving States an incentive to provide whole genome sequencing for
eligible children through Medicaid my legislation will ensure that more
children and their families can obtain the right diagnosis and
treatment the start. The Ending the Diagnostic Odyssey Act has the
support of more than 100 patient advocacy organizations, including
Alstrom Syndrome International, the Genetic Alliance, the Personalized
Medicine Coalition, and many others. I urge my colleagues to support
this legislation.
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