MEDICAL FOODS AND NUTRICEUTICALS: A NATIONAL CARE DILEMMA

1. MEDICAL FOODS AND NUTRICEUTICALS: A NATIONAL CARE DILEMMA Medical Foods Expert Workgroup of the Committee’s Subcommittee on Follow-up and Treatment Susan A. Berry, MD

2. Why did we do this?

3. FDA Definition: Medical Food "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." http://www.cfsan.fda.gov/~dms/medfguid.html

4. What are the treatments? • Medical foods • Specially compounded formulas that supply a substantial portion of nutrition for the treatment of inborn errors of metabolism • “Nutriceuticals” • Pharmacologic doses of cofactors or vitamins • Amino acids provided to give substrate or prevent specific amino acid deficiency • Other vitamin-like drugs that may provide benefit, e.g. carnitine • MCT oil • Both classes of agents require physician supervision • Both are required therapies for treatment of inborn errors of metabolism Also at issue: Specially manufactured low protein foods

5. What is the problem? • Medical foods (etc) are NOT drugs, they are materials of nutritional value • Medical foods are substantially more expensive than traditional foods • Everyone needs food, yet traditional foods are harmful to persons with inborn errors of metabolism • They are not optional, they ARE the treatment • Because they are foods, they are EXCLUDED from coverage by many insurers • Costs may be prohibitive; coverage is at best variable Affected persons cannot survive without them but cannot afford to buy them

6. What are (some of) the barriers? • Each insurer has its own practices • Private insurers • Public practices vary state to state • Each policy, even with the same company, may have differing coverage • Each state has different rules / laws covering provision of medical foods • (seehttp://www.ncsl.org/programs/health/lawsfoodsformula.htmfor list of laws) • Even when laws exist they don’t cover all insurance carriers • Even when laws/guidelines exist they are subject to interpretation by insurers and the states

7. How I got started on this: Minnesota (pretty average) 2007 Minnesota Statutes 62A.26 • COVERAGE FOR PHENYLKETONURIA TREATMENT.     • Subd. 2. Required coverage. Every policy, plan, certificate, or contract referred to in subdivision 1 issued or renewed after August 1, 1985, must provide coverage for special dietary treatment for phenylketonuria when recommended by a physician. History: 1985 c 49 s 41; 1Sp1985 c 9 art 2 s 1; 1992 c 564 art 1 s 54 • For MN: note law is specific to PKU

8. Nutraceutical prescriptions: denied… • Reviewed denials of health insurance claims for reimbursement of metabolic medications in my MN clinic (01/01/04-08/05/05) • 78 pharmacotherapeutic agents initially denied • All denials appealed by metabolic center • 19 additional re-appeals submitted by metabolic center Total appeals/re-appeals: 97 Final outcomes: 50 requests for coverage had appeals ultimately approved 11 requests for coverage had appeals ultimately denied 17 requests for coverage had unknown final outcome

9. Medical food prescriptions:also denied! Medical Food Denials (01/01/04-08/05/05) • Most requests handled by dietician, reviewed only if denied on initial submission • 17 appeals by metabolic center after initial denial • 4 required re-appeals by metabolic center Final outcome: • 9 ultimately approved • 2 denied • 6 unknown status

10. Lessons Learned? • Many nutraceuticals and medical foods ultimately covered by insurance BUT many paid only after repeated appeals • Responses not uniform, each takes its own strategy with varying success • In the meantime… • Some went without treatments • Some changed private insurer or obtained Medicaid • Some found charity groups who graciously paid for a month of formula or medicine while we continued to appeal • Each year the process starts over (coverage granted for 12 month time periods) Is there another way?

11. Medical Foods Expert Workgroup • Workgroup of the Follow-up and Treatment Subcommittee • Ensure that families of children with inborn errors of metabolism have coverage for medically necessary treatments INCLUDING medical foods

12. Medical Foods Expert Workgroup • Susan A. Berry, Workgroup Chair • Coleen Boyle, Committee Member • James J. Figge, Consultant • Carol Greene • Jennifer N. Kraszewski • Jill Levy-Fisch • Mary Kay Kenny • Michele Lloyd-Puryear • Rani H. Singh • Jill F. Shuger • Regional Collaborative investigators for survey implementation • Region 2 NYMAC: Katharine B. Harris • Region 3 Southeast: Rani H. Singh • Region 4: Cynthia A. Cameron

13. Defining the scope of the problem • Find out what families are experiencing • Examine mandates and regulations governing public and private coverage

14. Defining the scope of the problem • Find out what families are experiencing • A family survey regarding coverage for medical foods, nutriceuticals, and feeding supplies • Examine mandates and regulations governing public and private coverage

15. Medical Foods SurveyInformation sought • Demographics: age, disorder, state • Type of coverage • Financial burdens incurred • Success (or not) in coverage for • Medical foods (formulas) • Low protein foods • Nutriceuticals • Feeding supplies (G-tubes, equipment, etc.) • Room for free text comments

16. Survey Work Plan • Phase 1 – Initial survey development • Workgroup met by phone/email • Cognitive testing with two parent focus groups • Phase 2 – Pilot testing • Qualitative testing to determine response patterns • Done in 3 Regions • small sample • children and young adults • Tests also feasibility (IRB, procedures) • Phase 3 – Full survey implementation

17. Phase 2 - Accomplishments • Region 2 NYMAC – 8 surveys submitted • Region 3 Southeast – 15 surveys submitted • Region 4 – 21 surveys submitted Surveys analyzed for utility of format and questions

18. Phase 2 – pilot testing • Issues and Solutions: • “Type of insurance” – distinguish between State subsidized private insurance vs. unsubsidized private insurance • Re-format – condense, collapse and reorganize to simplify recording of responses and distinction between Medical Foods and Dietary Supplements • Provide for genetic center staff review of completed surveys to verify patient information • Re-pilot in a small sample from two geographic areas

19. What next? • Assess issues encountered in survey use, revise for full implementation • Present Roundtable at APHL • Phase 3: Full implementation of survey • Goal: Spring 2009 • 200 surveys per Region Use the information derived to • Develop manuscript • Identify potential actions

20. Region 2 NYMAC Collaborative (expressed interest) AI duPont Children's Hospital Albany Medical Center Children's Hospital of Pittsburgh Mount Sinai Medical Center SUNY at Stony Brook University of Maryland University of Virginia University of Rochester Westchester Medical Center Region 3 Southeast Collaborative (centers confirmed) Emory University Tulane University University of Florida University of South Carolina University of Tennessee Region 4 Great Lakes Collaborative (centers confirmed) University of Cincinnati University of Minnesota Phase 3 – Survey Implementation

21. Defining the scope of the problem • Find out what families are experiencing • A family survey regarding coverage for medical foods, nutriceuticals, and feeding supplies • Examine mandates and regulations governing public and private coverage

22. Meeting on Reimbursement Issues for Medical FoodsJune 2, 2008 • Purpose: • examine barriers to reimbursement • develop recommendations for Advisory Committee actions • Invitees: • Medical Foods Workgroup • Representatives: • Private insurance • Industry • U.S. Dept of Labor/IRS (employer-based health plans) • CMS – State and Federal

23. Possible Actions to Impact Financing of Medical Foods: Medicaid • Federal: broaden Federal statute to cover medical foods • State: Develop a model state policy for medical food

24. Possible Actions to Impact Financing of Medical Foods Private Insurers • Develop a model state insurance law to minimize variation from state to state • Work with AMA Editorial Board to develop reimbursement codes (CPT) that facilitate billing • Work with insurers to recognize and reimburse for appropriate CPT codes • Work with insurers to improve knowledge-base of staff re: medical foods issues

25. Possible Actions to Impact Financing of Medical FoodsOther • Explore options to influence employer-based health plans including federal mandate under ERISA (exempt from state statutes – 60% of those with private insurance) • Seek advice from FDA about updating the definition of medical foods

26. Defining the scope of the problem • Find out what families are experiencing • A family survey regarding coverage for medical foods, nutriceuticals, and feeding supplies • Examine mandates and regulations governing public and private coverage • Workgroup meeting June 2008 • Document mandates and regulations in place in each state