1. Newborn Screening and Health Care Reform Presented at the NCC/RC PI Annual Meeting, November 16, 2009

2. Newborn Screening and Health Care Reform: ACHDNC paper OVERVIEW As with health care system overall, there is unequal access to newborn screening (NBS) services across the country As with health care system overall, there is unequal access to newborn screening (NBS) services across the country The reasons behind disparities in NBS mirror problems with the broader health system: The reasons behind disparities in NBS mirror problems with the broader health system: Public financing Public financing Payment systems Payment systems Administrative inefficiencies Administrative inefficiencies Insurance coverage issues Insurance coverage issues

3. Unequal Access to Services Recommended Reforms: 1 Convene an expert panel to establish a minimum recommended standard of service and care for each component of the newborn screening system—education, screening, diagnosis, follow- up/tracking and evaluation services.

4. Public Financing Features of Current NBS System Combination on funding streams from fees, Maternal and Child Health Title V Block Grant funds, state appropriations and general revenues Existing support only provides for some education efforts, screening, diagnosis, and initial confirmation of treatment in half of the states Fees do not correlate with number of mandated tests

5. Public Financing Recommended Reforms: 2 Develop national guidance on creating public health budgets for newborn screening systems in order to minimize geographical disparities and highlight budget alternatives that may better serve the needs of a particular state program. The guidance should incorporate the flexibility in funding design that states may require and identify areas that the federal government may target for additional support to help states deliver the minimum standard of service and care set forth in recommendation 1.

6. Payment systems Features of Current NBS System Billing and payment practices vary from state to state Lack of financial incentive to coordinate care

7. Payment systems Recommended Reforms: 3 Convene an expert panel to examine coding changes to streamline the billing process for newborn screening services and to put forth recommendations that enhance the standardization of health care transactions.

8. Payment systems Recommended Reforms: 4 Work with the Centers for Medicare and Medicaid to pilot a payment method for providers treating the same child with a disorder diagnosed as a result of screening that incentivizes care coordination.

9. Administrative Inefficiencies Features of Current NBS System Lack of funding to support e-health activities for state public health departments Efforts to promote electronic exchange of NBS information (ONC, NICHD)

10. Administrative Inefficiencies Recommended Reforms Further define and adopt the meaningful use case for newborn screening for health information exchange endeavors by the Department

11. Insurance Coverage Issues Features of Current NBS System State policies that require insurance coverage for medical foods vary and are not comprehensive Gaps in coverage of necessary medical foods and foods modified to be low in protein result in financial burden for some families

12. Insurance Coverage Issues Recommended Reforms Close gaps in insurance coverage for medical foods and foods modified to be low in protein as recommended by the ACHDNC in April 2009.

13. Insurance Coverage Issues ACHDNC Recommendations April 2009 1. Federal legislation be enacted to establish a uniform requirement that health plans offer coverage of medical foods and foods modified to be low protein for those conditions recommended by the Committee. Health plans would include Federal insurance programs coverage plans (Children’s Health Insurance Program, Tricare, and Medicaid) and those plans governed by the Employment Retirement Income Security Act (ERISA) and would not be subject to state exclusions.

14. Insurance Coverage Issues ACHDNC Recommendations 2. Medicaid’s enabling legislation (Title XIX of the Social Security Act) be amended to ensure more uniform coverage by state Medicaid programs of medical foods and foods modified to be low protein for those conditions recommended by the Committee. (Medical foods are not mentioned in the federal Medicaid statute allowing significant variation across states with respect to the coverage of medical foods. Amending §1905(a) of the federal statute would encourage best practices and ensure greater uniformity.)

15. Insurance Coverage Issues ACHDNC Recommendations April 2009 3. The following specific requirements be included in the legislation: a. Medical foods (as defined by the FDA and for those conditions recommended by the Committee) delivered either orally or by tube (both are enteral) and foods modified to be low protein used under the direction of a physician for the treatment of an inborn error of metabolism should be included as medical benefits and not restricted to pharmacy benefits. b. Pharmacological doses of vitamins and amino acids used specifically for the treatment of inborn errors of metabolism for those conditions recommended by the Committee under the direction of a physician will be covered.

16. Insurance Coverage Issues ACHDNC Recommendations April 2009 c. A minimum yearly coverage should be set for all health insurance plans, including those covered by the Children’s Health Insurance Program, Tricare, and Medicaid and those governed under the ERISA. The Secretary will have authority to set age-specific minimum levels of coverage and periodically update these levels based on a standard cost of living index.

17. Federal Medical Foods Legislation S 2766 FDA definition of medical foods “… a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation.”(section 5(b) of the Orphan Drug Act (21 U.S.C. 360ee (b) (3))) In addition to nutritionally modified counterparts of traditional foods, medical foods are recognized for purposes of this Act to include, but not be limited to, other forms of foods such as formulas, pills, capsules and bars, so long as consumed or administered enterally. The term “enterally” refers to consumption or administration through gastrointestinal tract, whether orally or by tube. (Source: National PKU Alliance) “… a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation.”(section 5(b) of the Orphan Drug Act (21 U.S.C. 360ee (b) (3))) In addition to nutritionally modified counterparts of traditional foods, medical foods are recognized for purposes of this Act to include, but not be limited to, other forms of foods such as formulas, pills, capsules and bars, so long as consumed or administered enterally. The term “enterally” refers to consumption or administration through gastrointestinal tract, whether orally or by tube. (Source: National PKU Alliance)

18. Federal Medical Foods Legislation Coverage requirements apply to federal health insurance plans, plans governed by the Employee Retirement Income Security Act (ERISA), individual and group health insurance plans Coverage requirements apply to federal health insurance plans, plans governed by the Employee Retirement Income Security Act (ERISA), individual and group health insurance plans Coverage requirements address medical necessary foods, foods modified to be low in protein, pharmacological doses of vitamins and amino acids Coverage requirements address medical necessary foods, foods modified to be low in protein, pharmacological doses of vitamins and amino acids Secretary, HHS to determine minimum yearly coverage Secretary, HHS to determine minimum yearly coverage (Source: National PKU Alliance)

19. Federal Medical Foods Legislation HR 3262 Internal Revenue Code of 1986 (relating to medical, dental, etc., expenses) amendment:`(C) for foods for special dietary use, dietary supplements (as defined in section 201 of the Federal Food, Drug, and Cosmetic Act), and medical foods,'. Internal Revenue Code of 1986 (relating to medical, dental, etc., expenses) amendment:`(C) for foods for special dietary use, dietary supplements (as defined in section 201 of the Federal Food, Drug, and Cosmetic Act), and medical foods,'. SPECIAL RULE FOR INSURANCE COVERING FOODS FOR SPECIAL DIETARY USE, DIETARY SUPPLEMENTS, AND MEDICAL FOODS- Amounts paid for insurance covering foods and supplements referred to in paragraph (1)(C) shall be treated as described in paragraph (1)(E) only if such foods and supplements comply with applicable good manufacturing practices prescribed by the Food and Drug Administration or with other comparable standards.'. SPECIAL RULE FOR INSURANCE COVERING FOODS FOR SPECIAL DIETARY USE, DIETARY SUPPLEMENTS, AND MEDICAL FOODS- Amounts paid for insurance covering foods and supplements referred to in paragraph (1)(C) shall be treated as described in paragraph (1)(E) only if such foods and supplements comply with applicable good manufacturing practices prescribed by the Food and Drug Administration or with other comparable standards.'.

20. Contact Information Alissa Johnson Johnson Policy Consulting Ph: (703) 272-7847 ajohnson@policyconsult.com www.policyconsult.com