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