There was a time in history,
not very long ago,
the discovery of PKU was made,
and to many, much thanks we do owe.
For lives were saved
from the devastating consequences of this disease,
a rare, metabolic disorder
which results in neurological toxicities.
There was a woman,
who shaped PKU history some years long ago,
before the formation of any alliances
or The Orphan Drug Act,
which in later years we have come to know.
Over 40 years ago,
she gave birth to her first child
diagnosed with classical PKU,
A phone call came in the middle of the night,
please return to the hospital
regarding your daughter’s life.
What is this PKU?
What will the child look like?
Will she ever be normal?
Is it too late to be spared institutional life?
A dietary treatment exists,
the doctors brought this news.
In 1958, Lofenalac had been FDA approved.
However, there was a landmark decision back in 1972,
Lofenalac, the prescription drug
became what is known today, as a “medical food.”
The dietary treatment worked,
this was later proven, you know.
In later years, research had shown
phenylalanine (phe) levels must always be kept low.
But in the early years of this young child’s life,
the diet presented many obstacles,
issues with compliance, cost, and insurance strife.
What is this special diet, the norm in this patient’s life?
Eat fruits and vegetables.
Dairy, eggs, meat, grains, fish, poultry, bread constitute a “red light.”
The instructions were: you need to eat “phe free.”
Therefore, it is essential to consume Lofenalac,
for protein, for growth, and for calories.
In the early days, as part of a Multi-State Collaborative Study,
this child received comprehensive healthcare services,
intensive home visits, dietary monitoring, and
therapeutic blood phe drawing.
But one day the woman received a letter in the mail,
an Explanation of Benefits,
that really made her wail.
Your future claims are denied,
we erroneously covered your child.
Lofenalac the drug is used for weight reduction,
it is not necessary for her survival.
Yes, Lofenalac was a drug.
Not a nutritional supplement, not just a vitamin.
It was used for dietary management, for there was no other PKU treatment
that could prevent mental retardation.
What is this woman to do?
For her PKU children now include not one - but two.
And, a 3rd sibling is on the way,
how does one get reimbursement for this costly treatment these days?
At this time in history, “diet for life” was not known.
There was no other option….
no orphan drug, no Kuvan
but this woman was not alone.
She appealed the denial
to Members of Congress, - the Senate and the House,
to the FDA, Mead Johnson, the government, doctors, research experts,
and anyone else who would listen to her shout.
She wrote and she wrote,
but not until 1980, was success obtained
after years of tireless, persistent, advocacy efforts,
these calls to action were not in vain.
For the first time in this history,
in the Federal Employee Benefits Plans, what did appear?
Lofenalac became included,
as a covered benefit effective that plan year.
The story does not end here.
Sadly, the access and reimbursement legacy remains.
For after 40 years, her daughter stands before you,
my name is Jennifer Payne.
The Federal Employee Health Benefits Plan has become
one of social injustice and discrimination.
The Plan continues to deny
essential health benefits that I need for life.
Could the Federal Government again set the precedent?
Failure to step up, not covering PKU treatment would be a societal and economic shame.
They need to convince other insurers to follow suit,
and for all ages, for life, every state, and employer - cover treatment and to all, the same.
If we want to shape the future,
we must learn from the past.
The baton will be handed to future generations
unless we can work together at last.