THE INTRODUCTION OF A NATIONAL NEONATAL
SCREENING PROGRAM TO DETECT AND TREAT PKU IS ONE OF THE SUCCESSES OF
MODERN MEDICINE THE LANCET,312 (1256), 1991
TREATMENT OF NBS DISEASES
DRUGS: SCD, CH, CAH, biotinidase deficiency, tyrosinemia
DIET: PKU, galactosemia, MSUDCOMBINATION: biopterin defects, CF, homocystinuria, fatty acid oxidation disorders (7), organic acidemias (15), urea cycle disorders (3)
MEDICAL FOOD: A food which is formulated to be consumed or administered under the supervision of a physician and is needed for treatment of a specific disease….
Publ L 100-290
DIETARY THERAPY FOR INBORN ERRORS
ØSpecial formulas with disease specific amino acid restriction and amino acid supplementation. These provide 80-85% of protein, vitamin and mineral needsØ Low protein medical foods supply calories
and varietyØ Low protein natural foods supply variety
some calories
COSTS OF DIETARY THERAPY/YEAR
FORMULA LO-PROINFANT $2,000 $ 90PRESCHOOL $3,200 $ 750CHILD $7,600 $1,600TEEN/ADULT $9,400 $1,800
SOURCE: OHSU Metabolic Clinic
COSTS OF DIETARY THERAPY/YEAR
8 YR OLD PKU CHILD NEEDS 28 GRAMS PROTEIN, 1900 CALORIES, TOLERATES 5 GRAMS NATURAL PROTEIN (PROVIDES 200 CALORIES)
FORMULA ONLY: $13,200
COSTS OF DIETARY THERAPY/YEAR
8 YR OLD PKU CHILD NEEDS 28 GRAMS PROTEIN, 1900 CALORIES, TOLERATES 5 GRAMS NATURAL PROTEIN (PROVIDES 200 CALORIES)
FORMULA ONLY: $13,200FORMULA + LPP: $ 7,600
COSTS OF DIETARY THERAPY/YEAR
8 YR OLD PKU CHILD NEEDS 28 GRAMS PROTEIN, 1900 CALORIES, TOLERATES 5 GRAMS NATURAL PROTEIN (PROVIDES 200 CALORIES)
FORMULA ONLY: $13,200FORMULA + LPP: $ 7,600
DIFFERENCE $ 5,600
EFFECT OF “DIET FOR LIFE” ON PATIENT LOAD
0
20
40
60
80
100
120
140
160
year 1 year 6 year 15 year 20 year 30
EFFECT OF “DIET FOR LIFE” ON COSTS
$0
$200,000
$400,000
$600,000
$800,000
$1,000,000
$1,200,000
year 1 year 6 year 15 year 20 year 30
SOURCES OF PAYMENT FOR DIETARY THERAPY
PUBLIC Ø TITLE XIX (WELFARE)Ø WOMEN, INFANTS & CHILDREN (WIC)Ø CHILDREN WITH SPECIAL HEALTH CARE NEEDS
(CSHCN)Ø TITLE XXI STATE CHILDREN’S HEALTH INSURANCE
PROGRAM (SCHIP)Ø STATE GENERAL FUNDØ MCH BLOCK GRANTPRIVATEØ NBS FEESØ INSURANCEØ PARENTSØ FOUNDATIONS/PARENT GROUPS
NBS PROGRAM RESPONSIBILITY FOR TREATMENT
IT IS NOT THE STATE’S RESPONSIBILITY TO PAY FOR TREATMENT, BUT TO ENSURE TREATMENT COSTS ARE COVERED FOR EVERY AFFECTED INFANT.
IT IS POSSIBLE FOR EVERY STATE TO DEVELOP A SELF SUSTAINING FUND FOR ENSURING TREATMENT OF METABOLIC DISORDERS WITHOUT THE USE STATE GENERAL OR MCH FUNDS
INSURANCE RESPONSE TO MEDICAL FOOD COVERAGE
• NOT A COVERED BENEFIT• MEDICAL FOODS AND FORMULAS ARE SUPPLEMENTS
• TREATMENT NOT EFFECTIVE• HAS TO BE GIVEN BY TUBE
LEGISLATION MANDATING INSURANCE COVERAGE OF NUTRITIONAL THERAPY
LEGISLATION……………………………39 STATESFORMULA ONLY……………………14FOODS AND FORMULA…….25OTHER DISORDERS…………..30PKU ONLY………………………………..9CAPS/LIMITATIONS………….17
SOURCE: NATIONAL PKU NEWS, PERSONAL COMMUNICATIONS
FUNDING SOURCES IN THE U.S.
#STATES
INSURANCE MANDATE…………39PUBLIC FUNDS………………………..40NBS FEES…………………………….…….19PARENT FEES…………………………….47
SOURCES: NATIONAL PKU NEWS, PERSONAL COMMUNICATION
WE’VE COME ALONG WAY, BUT WE CAN DO BETTER!
ØTHREE STATES HAVE NOTHING IN PLACEØFOUR STATES STILL USE ONLY MCH & BLOCK GRANT MONEYØMANDATES ARE NOT PERFECTØNEED FEDERAL LEGISLATION
INGREDIENTS FOR STABLE FUNDING OF MEDICAL FOODS
ü USE AS MANY SOURCES OF FUNDING AS YOU CAN
• WIC, WELFARE, CSHCN, MCH, GENERAL FUND, CHIP
• INSURANCE• PARENTS• NBS FEES
INGREDIENTS FOR STABLE FUNDING OF MEDICAL FOODS
ü USE AS MANY SOURCES OF FUNDING AS YOU CANü PROBABLY WILL NEED STATE LEGISLATION
AND MAY NEED FEDERAL LEGISLATION• SHOULD INLUDE ALL DISORDERS, ALL
AGES, BOTH SEXES• SHOULD INCLUDE FORMULAS AND LOW
PRO MEDICAL FOODS• KEEP CAPS AND LIMITATIONS TO A
MINIMUM
INGREDIENTS FOR STABLE FUNDING OF MEDICAL FOODS
ü USE AS MANY SOURCES OF FUNDING AS YOU CANü PROBABLY WILL NEED STATE LEGISLATION AND MAY
NEED FEDERAL LEGISLATIONü DEVELOP A ‘METABOLIC FOOD STORE’
• CONTROLS ACCESS TO KNOWN PATIENTS• INCREASES THE VARIETY OF PRODUCTS• CENTRALIZED BILLING AND DISTRIBUTION• CHEAPER THAN DISTRIBUTION FROM PHARMACIES• GENERATES A FUND FOR INDIGENT PATIENTS
INGREDIENTS FOR STABLE FUNDING OF MEDICAL FOODS
ü USE AS MANY SOURCES OF FUNDING AS YOU CANü PROBABLY WILL NEED STATE LEGISLATION AND MAY NEED
FEDERAL LEGISLATIONü DEVELOP A ‘METABOLIC FOOD STORE’ü EDUCATION OF ALL PAYERS
• EFFICACY OF TREATMENTS• INCREASED USE OF LOW PRO MEDICAL FOODS
INCREASES BIOCHEMICAL CONTROL AND DECREASES COSTS BY OVER 40%
• COST EFFECTIVENESS OVER NO TREATMENT• IT IS IMPORTANT TO ENSURE TREATMENT TO
EVERY AFFECTED INFANT REGARDLESS OF ABILITY TO PAY BOTH MORALLY AND TO REAP THE GREATEST BENEFIT OF SCREENING
INGREDIENTS FOR STABLE FUNDING OF MEDICAL FOODS
ü USE AS MANY SOURCES OF FUNDING AS YOU CAN
ü PROBABLY WILL NEED STATE LEGISLATION AND MAY NEED FEDERAL LEGISLATION
ü DEVELOP A ‘METABOLIC FOOD STORE’ü EDUCATION OF ALL PAYORSü USE EXISTING PROGRAM MODELS FROM
OTHER STATES