2015 STATE POLICY PROGRESS REPORT: A ROAD MAP FOR STATE IMPROVEMENT

Transcription

1 2015 STATE POLICY PROGRESS REPORT: A ROAD MAP FOR STATE IMPROVEMENT October 2015

2 Copyright 2015 NORD - National Organization for Rare Disorders, Inc. All rights reserved. Headquarters 55 Kenosia Avenue Danbury, CT 068 Phone: Fax: Washington, DC Office 1779 Massachusetts Avenue Suite 500 Washington, DC Phone: Fax: Massachusetts Office 1900 Crown Colony Drive, 4th floor Quincy, MA Phone: Fax: Media Inquiry Jennifer Huron Phone: (203) jhuron@rarediseases.org Policy Inquiry Tim Boyd Phone: (202) tboyd@rarediseases.org

3 Contents Introduction and Methodology... 3 National Overview... 5 Policy Scores: Alabama-Maryland... 8 Policy Scores: Massachusetts-South Dakota... 9 Policy Scores: Tennessee-Wyoming... Medical Foods Table 2: Medical Foods Scoring Rubric Prescription Drug Cost Sharing Table 3: Prescription Drug Cost Sharing Scoring Rubric Newborn Screening Table 4: Newborn Screening Scoring Rubric Newborn Screening: Newborn Screening: Newborn Screening: Medicaid/CHIP Eligibility Table 5: Medicaid/CHIP Eligibility Scoring Rubric Resources for Advocates Special Thanks Appendix I: Medical Foods.. 34 Appendix II: Rx Cost Sharing. 44 Appendix III-A: Newborn Screening Core Conditions. 49 Appendix III-B: Newborn Screening Secondary Conditions. 55 Appendix III-C: Newborn Screening Non-Recommended Conditions. 59 Appendix IV: Medicaid/CHIP Eligibility 63

4 Introduction and Methodology Since its inception, NORD has been a key influencer of federal policy governing rare disease research and care. Most notably, the founders of NORD were instrumental in the creation and passage of the Orphan Drug Act in 1983, which has transformed the health care landscape for people living with a rare disease by facilitating the development of breakthrough therapies. Prior to its passage in 1983, there were only 38 orphan-designated medications approved. Today, there are more than 360 rare diseases that have an FDA-approved treatment resulting from an orphan designation. During this same period, NORD has increased its involvement in state-based policy matters in order to ensure that patients are able to access the treatments the rare community has fought so hard to get developed and approved. NORD recognizes that now more than ever, the cost and availability of health care services for patients with a rare disease is dependent upon state-based policy decisions This is welcomed news, as it means national goals are closer to benefiting patients at the local level. However, it also means that the rare community is increasingly dependent on the earnestness with which their state implements better policies. This is where the first annual NORD State Policy Progress Report comes in. This Progress Report serves as an initial evaluation of state progress in implementing policies pertinent to the rare disease community. With it, NORD hopes to provide a road map for the rare disease community of where each state can improve and, ultimately, push forward the discussion on rare disease public policy. The 2015 Progress Report focuses on four key policy sections: 1. Coverage of medical foods in private and publicly-funded health plans 2. Capitation of prescription drug cost sharing requirements in commercial health plans 3. State coverage of newborn screening 4. State Medicaid eligibility levels, including the Children s Health Insurance Program (CHIP) This report is also a tool kit. In addition to in-depth appendices concerning each policy section, we have provided a list of resources that stakeholders can use to advocate on these issues in their state. Chief among these resources is NORD s Rare Action Network. Rare Action is NORD s grassroots advocacy network that connects advocates and gives them the tools to support the rare disease community in their state. At the end of this report, you will find information on how to sign up for Rare Action and a list of local Rare Action contacts in your region. Finally, NORD recognizes that while the issues in this report are critical, there may be other important topics that are not covered. In particular, we plan to incorporate policies concerning genetic testing and medical child abuse into future iterations of the Progress Report. These issues were not included in this release partly because NORD s policy position on these matters is not yet fully developed (we want to 3

5 make sure we get it right before recommending to states how to act). If you have feedback on these and other issues, we welcome it. Feel free to contact us at How NORD Evaluated State Progress States were evaluated and scored on four policy sections (medical foods, Rx drug cost sharing, newborn screening, and Medicaid eligibility). Each of these sections was evaluated independently, i.e., there is no overall score for a state only a score for each policy. This was done in order to ensure that insufficient state progress in one area would not unfairly skew perception of other policy areas where a state is excelling (and vice versa). The overall score for each policy section is expressed as a percentage on 0% to 0% scale as follows: Ø 0% to 59%: Insufficient Ø 60% to 79%: Satisfactory Ø 80% to 89%: Good Ø 90% and above: Excellent To determine an overall score for each policy section, NORD broke up each policy into subcategories that were scored using a tiered system. For example, for medical foods, publicly-funded coverage and privately-funded coverage were scored as separate subcategories within the medical foods section. These scores were then combined to calculate the overall score for a state s medical foods policy. The specific criteria for each policy subcategory varied depending on the issue being analyzed (each section of the report goes into more detail on the methodology for that particular issue). However, the basic structure of the tiered scoring was the same for all sections: Ø Tier Score of 0: State has no policy provision for the relevant issue Ø Tier Score of 1: State has some policy in place, but it does not meet the standards of higher tiers Ø Tier Score of 6: State policy on the given issue meets minimum standards Ø Tier Score of 8: State policy meets most, but not all desired standards Ø Tier Score of : State policy meets all desired standards. A score of is considered model policy that other states should seek to enact. To reiterate, each section of this report goes into further detail on the methodology and includes a scoring rubric for each policy area. 4

6 National Overview This section provides a brief overview of some of the areas where states have excelled and where they need to improve. NORD also identified key themes that cut-across multiple policy issues: Ø States with insufficient benefit requirements for commercial health plans tended not to supplement coverage by offering robust publicly-financed programs. Ø State coverage requirements for certain disorders tended to either specify a list of actual conditions or to broadly define medical criteria (e.g., all inborn errors of metabolism). A handful of states linked coverage to the medical discretion of a patient s physician. Ø As expected, there is not a one-size-fits-all approach to addressing many of these issues. Many states chose to tackle coverage gaps through enhancing their Medicaid coverage, others developed supplemental programs specific to the medical issues at hand. Likewise, improved policy did not always emanate from new legislation. In many instances, states chose to make quicker policy changes through stronger regulation (such as to limit prescription drug cost sharing requirements in certain health plans). Medical Foods Coverage State policies concerning mandated coverage for medical foods are highly inconsistent. Many states only mandate coverage for infant formula as opposed to low-protein solid foods. Such a policy limits care options available to adults living with a metabolic disorder. Further, more than a handful of states do not mandate coverage of medial foods at all, leaving patients to rely on whatever assistance they can obtain from the government or fend for themselves (for more, see the chapter on Medical Foods on p. 11). State Policy: Medical Foods Coverage 5 Excellent Good Satisfactory Insufficient

7 Prescription Drug Cost Sharing Overall, only 8 states have enacted comprehensive policies capping drug cost sharing in commercial plans (for more, see the chapter on Cost Sharing on p.13). State Policy: Prescription Drug Cost Sharing Excellent Good Satisfactory Insufficient Medicaid Eligibility There are still 20 states that have not expanded eligibility under the Affordable Care Act (ACA). This report also looked at other aspects of eligibility and found that some states that have expanded their adult eligibility lag behind when it comes to coverage for children up to age 18 (see the Medicaid chapter on p. 22). 6

8 State Policy: Medicaid/CHIP Eligibility Excellent Good Satisfactory Insufficient Newborn Screening One area where most states earned solid scores is newborn screening (see p. 16). Nearly all states cover the full panel of recommended Core conditions. However, far too few states cover all of the Secondary recommended conditions. State Policy: Newborn Screening Coverage 7 Excellent Good Satisfactory Insufficient

9 Policy Scores: Alabama-Maryland Medical Foods (%) Prescription Drug Cost Sharing (%) Newborn Screening (%) Medicaid/CHIP Eligibility (%) Alabama 53% 0% 80% 25% Alaska 65% 0% 89% 90% Arizona 90% 0% 69% 85% Arkansas 75% 0% 55% 90% California 0% 0% 90% 90% Colorado 95% 0% 83% 95% Connecticut 0% 0% 95% 0% Delaware 85% 80% 90% 95% District of Columbia 40% 0% 97% 90% Florida 90% % 80% 43% Georgia 40% % 83% 48% Hawaii 95% 0% 88% 90% Idaho 35% 0% 87% 20% Illinois 80% 0% 93% 90% Indiana 80% 0% 95% 90% Iowa 35% 0% 95% 95% Kansas 55% 0% 56% 38% Kentucky 85% 0% 82% 90% Louisiana 80% 80% 57% 25% Maine 85% 0% 83% 60% Maryland 0% 0% 97% 95% 8

10 Policy Scores: Massachusetts-South Dakota Medical Foods (%) Prescription Drug Cost Sharing (%) Newborn Screening (%) Medicaid/CHIP Eligibility (%) Massachusetts 93% 0% 87% 90% Michigan 47% 0% 96% 90% Minnesota 93% 0% 0% 0% Mississippi 40% 0% 97% 43% Missouri 80% % 95% 43% Montana 93% 80% 80% 85% Nebraska 80% 0% 88% 43% Nevada 87% 60% 87% 85% New Hampshire 73% 0% 77% 95% New Jersey 93% 0% 0% 90% New Mexico 87% 0% 84% 0% New York 80% 0% 92% 95% North Carolina 53% 0% 77% 43% North Dakota 73% 0% 94% 68% Ohio 40% 0% 81% 90% Oklahoma 40% 0% 92% 25% Oregon 0% 0% 87% 90% Pennsylvania 67% 0% 82% 95% Rhode Island 87% 0% 63% 95% South Carolina 40% 0% 96% 43% South Dakota 73% 0% 93% 25% 9

11 Policy Scores: Tennessee-Wyoming Medical Foods (%) Prescription Drug Cost Sharing (%) Newborn Screening (%) Medicaid/CHIP Eligibility (%) Tennessee 73% 0% 94% 60% Texas 87% 0% 87% 43% Utah 87% % 91% 25% Vermont 93% 0% 76% 95% Virginia 47% 0% 80% 38% Washington 67% 0% 73% 95% West Virginia 40% 0% 88% 85% Wisconsin 60% % 91% 90% Wyoming 93% 0% 83% 25%

12 Medical Foods Map: Medical Foods Excellent Good Satisfactory Insufficient Background There are multiple rare disorders which require special nutrition in order to prevent serious disability and allow for normal growth in children and adults. For patients living with these conditions, effective medical foods are the only viable treatment option available. The manufacture of these medical foods is highly specialized, making them more expensive for patients. For example, the average annual cost of formula for the metabolic disorder PKU (phenylketonuria) can cost as much as $12, Third-party payment for foods for special dietary use is inconsistent, and state statutes regarding reimbursement vary widely. Some states require coverage only for inherited metabolic diseases, such as PKU, and others include a range of metabolic conditions. While much can be done at the federal level to increase access to medical foods all for inborn errors of metabolism, states also play an integral role in ensuring access to these critical therapies. 1 Buist, N., Huntington, K., Winter, S. Healthcare Coverage for Medical Food Treatment of Inborn Errors of Metabolism. National Organization for Rare Disorders. June

13 Because insurance is regulated primarily at the state level, many states have mandated the inclusion of medical foods within private plans sold within their state. However, in the states which do not have medical food mandates, individuals in need of these particular treatments are faced with a huge burden of access and require assistance in paying for medical food expenses. Inclusion of medical foods within each state s Medicaid program is also essential, yet only some states mandate coverage. For states that do not provide coverage through Medicaid, a few states have chosen to provide access to medical foods through other publicly-funded health programs. While mandating coverage of medical foods in states is a big step forward, too many states place arbitrary cost, age, or gender limits on these coverage requirements. NORD encourages each state to adopt coverage mandates for medical foods without arbitrary limitations. Methodology Table 2 provides the scoring rubric for the medical foods. States were scored on a tiered system and earned a score of 0, 6, 8, or for each of four separate subcategories. Scores were assigned to the following four subcategories, for a total possible score of 40 (maximum score of for each category): 12 Covered Diseases for Private Insurance This subcategory covers which diseases are eligible for medical foods coverage for private insurance. Mandated Private Insurance Coverage: This subcategory covers whether or not a state mandates private insurance coverage of medical foods. State-Funded Coverage: This subcategory covers the level and type of coverage provided by state programs. Covered Disorders for State Programs: This subcategory covers which diseases are eligible for medical foods coverage for state programs (such as Medicaid). Note on Exclusion of Eosinophilic Disorders NORD did not incorporate state coverage of medical foods for eosinophilic disorders in this report. This was done, in part, because a clear national standard on coverage for these disorders is still evolving. However, given the importance of medical foods for this population, particularly individuals with rare allergic disorders, this category will likely be incorporated into future iterations of the report. Sources 1. National PKU Alliance (NORD Member Organization), State Coverage Database. Accessed online at: 2. HRSA, State Statutes and Regulations on Dietary Treatment of Disorders Identified Through Newborn Screening Accessed online at: dations/reports/statelaws.pdf 3. Nutrica Metabolics, State Coverage Database. Accessed online at

14 Table 2: Medical Foods Scoring Rubric Tier Score Description Subcategory Covered Disorders for Private Insurance and State services State does not mandate coverage of any disorders. Covered disorders include two or fewer metabolic conditions (such as PKUonly). Covered disorders include three or more metabolic conditions, but not all inborn errors. All inborn errors of metabolism. Private Insurance Coverage State does not mandate private insurance coverage of medical foods. States mandates private insurance coverage, but sets a limit on eligibility (such as age) or coverage (such as a dollar cap or covering formula only). States mandates private insurance coverage for both formula and low-protein foods. State has a coverage limit, but it is not less than $2,500 perperson per-year. State mandates private insurance coverage for both formula and low-protein foods with no limits on eligibility or coverage. State-Funded Coverage State does not mandate coverage for Medicaid. The state does not offer supplemental programs to provide coverage. State mandates Medicaid coverage for formula, but does not cover low-protein foods); Or State does not mandate Medicaid coverage but provides coverage on a case by case basis. State mandates Medicaid coverage for formula and lowprotein foods, but coverage includes age restrictions; Or State provides formula and low-protein foods through a supplemental program that may have age restrictions. State mandates Medicaid coverage for medical foods with no age restrictions. Or State provides formula and low-protein foods through a supplemental program with no age restrictions 13

15 Results (For a complete breakdown of results for every state, see Appendix I) Overall, 25 states have policies scored Good or Excellent, with four states earning perfect scores (CA, CT, MD, and OR). Most states that earned Satisfactory scores could easily improve by mandating coverage of low-protein foods instead of just formula-based nutrition. States that scored Insufficient typically do not mandate coverage of any kind for either private insurance or Medicaid. One area of concern regarding state policy is the process by which certain diseases are covered. In many states, each covered condition is specified in the law, making it difficult to extend coverage for new disorders. NORD believes a better policy is for states to specify coverage based on whether a given disease is part of the newborn screening panel. One obvious drawback of this approach is that it is dependent on state coverage of newborn screening. Currently there are only 6 states that take this approach. 16 States Do not mandate coverage of medical foods for private insurance plans 8 States Mandate private insurance coverage for formula only 11 States Do not mandate Medicaid coverage of medical foods 14

16 Prescription Drug Cost Sharing Map: Rx Cost Sharing Excellent Good Satisfactory Insufficient Background Under the Affordable Care Act (ACA), many people with rare or chronic diseases who previously have been denied insurance coverage on the basis of a pre-existing condition are now able to purchase commercial plans. Insurance coverage means these individuals now have reliable access to medications that can help treat and manage their condition. Unfortunately, some insurance providers have implemented policies that place orphan therapies on the so-called "specialty-tier" of a drug formulary. For drugs placed on this tier, plans often require that enrollees pay co-payments each time they fill their prescription that can be as much as 50% of the actual cost of the medication. 2 For many people with a rare disorder, as well as those with other severe chronic diseases, these costs are untenable. As a consequence, patients in need of life saving treatment are forced to go without their medication or use options that are less effective and less safe. 2 "Appleby, J. "Got Insurance? You Still May Pay A Steep Price For Prescriptions." Kaiser Health News. Published Oct 14, Accessed online at 15

17 The utilization of specialty tiers in these plans is staggering. For example, it is estimated that up to onefifth of Exchange plans require cost sharing of 30% or higher for the entire class of drugs for common chronic diseases. 3 To assist patients who find themselves in this difficult situation, several states have passed legislation mandating a cap on out-of-pocket costs for specialty medications. These caps range from $0 to $500 per-month per-medication, depending on the type of plan (Bronze, Silver, Gold, or Platinum). NORD strongly supports the enactment of these types of policies as they greatly benefit rare patients at a minimal impact to the overall insured population. In fact, recent analysis has demonstrated that these types of limits on co-pays can be instituted with little to no impact on overall plan premiums for all beneficiaries. 4 Methodology When it comes to addressing the issue of high drug cost sharing, there are several different policies states can implement that are effective. For example, some states have chosen to cap co-pays on a perdrug, per-month basis. Others have mandated total caps for all drug cost sharing. One state is considering a policy by which prescription drug costs cannot exceed 20% of a plan s out of pocket limit for all medical costs. States were scored on a tiered rubric with possible scores of 0, 1, 6, 8, or (see Table 3). Bills that have not passed both legislative chambers were not factored as part of a state s score (as of the release of this report no bill introduced this session has passed).these proposed bills are included in Appendix II. Sources: In completing this section of the report, NORD solicited feedback from the Leukemia & Lymphoma Society and members of the State Access to Innovative Medicines Coalition. Other sources: 1. NORD identified state legislation and regulation through a state by state legislative analysis. As part of the State Access to Innovative Medicines (SAIM) coalition, NORD also had access to legislative tracking information shared by SAIM members. 2. State Patients Equal Access Coalition (SPEAC). National Landscape of Caps on Patient Out-of- Pocket & State Oral Chemotherapy Parity Laws. speac.myeloma.org/uploads/2014//national- Landscape-of-State-Oral-Chemotherapy-Parity-Laws_.1.14.pdf 3 "Analysis of Prescription Drug Tier Placement and Cost Sharing in Health Insurance Exchange Plans." Avalere PlanScape. Published Feb 11, Accessed online at /-/-/-/ _Avalere%20Planscape%202015_Class%20Tiering%20Analysis.pdf 4 Pharmacy Cost Sharing Limits for Individual Exchange Benefit Plans: Actuarial Considerations. Milliman, Inc. March 5, Sharing-Limits-for-Exchange-Plans.pdf 16

18 Table 3: Prescription Drug Cost Sharing Scoring Rubric Tier Score Description ate does not have a cap on cost sharing. State has enacted cost sharing limits for a limited number of treatments (such as oral chemotherapy only). State has instituted a per-drug cap or total cap on cost sharing that does not apply predeductible. State has instituted a per-drug cap on cost sharing that applies predeductible. Cap only applies to specialty-tier drugs. OR State has a total cap on Rx cost sharing that applies pre-deductible. Cap only applies to specialty-tier drugs. State has instituted a total c on Rx cost sharing that appl pre-deductible and for all prescription drugs. 17

19 Results (For a complete breakdown of every state, see Appendix II) Only a handful of states have enacted comprehensive legislation to limit cost sharing requirements for prescription drugs. In total, only 8 states have enacted some form of comprehensive cap on prescription drug cost sharing (either a per-drug cap and/or a total cap). Unfortunately, most states that scored Insufficient are not even considering legislation to limit cost sharing for prescription drugs. Some of these states do have limits of cost sharing for oral chemotherapy, but these restrictions do not apply to any other medication or condition. States that have enacted an Rx cap that did not earn a perfect score typically did not cap total costs, or the per-drug cap was high compared to other states. Overall, the lack of enacted state limits on drug cost sharing reflects the recent development of this issue. With the ongoing implementation on the ACA, NORD expects more states to recognize the need for limits on drug cost sharing. Note on the dollar limit of individual state caps Given that only a handful of states have implemented caps on prescription drug cost sharing, this iteration of the Progress Report did not look at specific dollar value of a cap in determining the category score. However, as more states implement cost sharing policies, future iterations of the report will take the total value of the cap into account. 14 States Considered cost-sharing legislation in States Passed a new cost sharing law or regulation as of October

20 Newborn Screening Map: Newborn Screening Excellent Good Satisfactory Insufficient Background Newborn screening is one of the most successful public health programs ever enacted, saving tens of thousands of lives over the past 50 years. Newborn screening allows physicians to catch a heritable disease early and start treatment almost immediately following birth. In this way, many of the worst effects of a disease can be mitigated. Newborn screening programs are regulated and operated almost entirely at the state level, allowing customization of their program to the state s specific needs. For example, states have great leeway in terms of what conditions to screen for or how samples are used following a blood spot test. NORD supports robust, well-funded newborn screening programs in every state, and encourages state lawmakers to prioritize the early detection of these debilitating diseases. NORD encourages every state to adopt the Uniform Newborn Screening Panel developed by the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children 5, and will continue to advocate for this adoption in each state that currently does not screen for the disorders included within the panel

21 Methodology NORD scored states based on their implementation of screening of the core and secondary conditions on the Uniform Newborn Screening Panel (a full list and definitions of each condition can be found on p. 19). States were not scored on non-recommended conditions identified by the panel, or on conditions only recommended this year. States were scored separately for the subcategories of Core Conditions and Secondary Conditions. These subcategory totals were then combined to calculate the overall newborn screening score. For each individual condition, states were scored on a tiered rubric with possible scores of 0, 6, 8, or (see Table 4). A score of zero was utilized because many states do not cover certain recommended conditions at all. Appendix III-C lists coverage status for non-recommended disorders; however, states were not scored on these conditions. As NORD s policy on non-recommended conditions develops, future iterations of the Progress Report will be expanded to evaluate at least some of these conditions. Sources 1. HRSA, Advisory Committee on Heritable Disorders in Newborns and Children. Recommended Uniform Screening Panel Baby's First Test, Conditions Covered by State National Newborn Screening and Genetics Resource Center (NNSGRC), National Newborn Screening Status Report. November 14, Association of Public Health Laboratories, NewSTEPS Screened Conditions Report. Available online at 20

22 Table 4: Newborn Screening Scoring Rubric Tier Score Description Condition is universally covered; Or State is implementing universal coverage Screening is universally offered but not required 8 Or The Condition is detected as part of Multiple Reaction Monitoring 6 6 Coverage is only mandated for select populations 0 Not covered 6 HRSA, Newborn Screening: Toward a Uniform Screening Panel and System. P

23 Results (For a complete breakdown of every state, see Appendix III) Nearly all states earned a score of Good or Excellent for their implementation of newborn screening recommendations. However, while all states cover nearly the entire panel of core recommended conditions, only 12 states cover all core recommended conditions. There was much greater variance of state coverage of secondary conditions. For example, multiple states only mandate screening for a few secondary conditions or only for select populations. Three states (Arkansas, Kansas, and Louisiana) scored Insufficient because they only cover a single secondary condition. Overall, the rate of state coverage of newborn screening is encouraging. However, more states need to put policies in place to identify and report on secondary conditions, consistent with RUSP recommendations. Pompe Disease In March 2015 Pompe disease was added to the HRSA Recommended Uniform Screening Panel. State coverage of this condition is not included in the 2015 Progress Report, but will be included in future iterations. Currently, only New York has implemented universal screening for Pompe disease; 3 states (Illinois, Kentucky, and New Jersey) have recently required screening, but have not yet implemented this; and Missouri is currently conducting a pilot project. Note on exclusion of non-recommended conditions Appendix II-C details state coverage of non-recommended conditions, which were not included as part of the scores for this section. In general, NORD supports state adoption of required screening for many non-recommended disorders, but considers it unfair to score states negatively on coverage of diseases that are not recommended by the Department of Health and Human Services. MN & NJ The only states to cover all Core and Secondary recommended conditions 12 States Cover all Core recommended conditions 22

24 Newborn Screening: Core Condition Definitions Each name links to the NORD Rare Disease Database page for that disorder or to the NIH Database Hearing Hearing loss HMG 3-Hydroxy-3-methylglutaryl-CoA lyase deficiency CH Congenital hypothyroidism IVA Isovaleric acidemia CAH Congenital adrenal hyperplasia 3-MCC 3-Methylcrotonyl-CoA carboxylase deficiency S/S, S/A, S/C Sickle cell disease Cbl-A,B Methylmalonic acidemia BIO Biotinidase deficiency BKT Beta-ketothiolase deficiency GALT Galactosemia MUT Methymalonyl-CoA mutase deficiency CF Cystic fibrosis PROP Propionic acidemia CCHD SCID Critical congenital heart defect Severe combined immunodeficiency MCD ASA Holocarboxylase synthetase deficiency Argininosuccinic aciduria CUD Carnitine uptake defect CIT Citrullinemia, type I LCHAD MCAD Long-chain 3-hydroxyacyl- CoA dehydrogenase deficiency Medium-chain acyl-coa dehydrogenase (MCAD) deficiency HCY MSUD Homocystinuria Maple syrup urine disease TFP Trifunctional protein deficiency PKU Phenylketonuria VCLAD Very long-chain acyl-coa dehydrogenase deficiency TYR-1 Tyrosinemia, type I GA-1 Glutaric acidemia, type 1 23

25 Newborn Screening: Secondary Condition Definitions Each name links to the NORD Rare Disease Database page for that disorder or to the NIH Database CACT CPT-1A CPT-II DE-RED CA-II MCKAT M/SCHAD SCAD 2M3HBA 2MBG 3MGA Cbl-C,D IBG MAL ARG BIOPT-BS BIOPT-RG CIT-II H-PHE MET Carnitine-acylcarnitine translocase deficiency TYR-II Tyrosinemia, Type II Carnitine TYR-III Tyrosinemia, Type III palmitoyltransferase 1A Carnitine Galactoepimerase palmitoyltransferase II GALE deficiency deficiency 2,4 Dienoyl-CoA reductase GALK Galactokinase deficiency deficiency Carbonic anhydrase II HBS HbS disease Medium-chain ketoacyl- CoA thiolase deficiency 3-Hydroxyacyl-CoA dehydrogenase deficiency Short-chain acyl-coa dehydrogenase deficiency 2-Methyl-3- hydroxybutyric academia 2-Methylbutyryl-CoA dehydrogenase deficiency 3-Methylglutaconyl-CoA hydratase deficiency Cobalamin C cofactor deficiency Isobutyrylglycinuria Mal de Meleda Argininemia Biopterin defect in cofactor biosynthesis Biopterin defect in cofactor regeneration Citrullinemia, type II Hyperphenylalaninemia Hypermethioninemia 24

26 Newborn Screening: Non-Recommended Conditions Definitions Each name links to the NORD Rare Disease Database page for that disorder or to the NIH Database HHH PRO EMA OTC,MTHFR NKH G6PD Krabbe Niemann-Pick MPS I Gaucher Fabry MPS II CPS 5-OXO TOXO ALD Ornithine transcarbamylase deficiency Prolinemia Ethylmalonic encephalopathy Ornithine transcarbamylase deficiency Nonketotic hyperglycinemia Glucose-6-phosphate dehydrogenase deficiency Leukodystrophy, Krabbe disease Niemann-Pick disease Mucopolysaccharidosis Type I Gaucher disease Fabry disease Mucopolysaccharidosis Type II Carbamoyl phosphate synthetase I deficiency Pyroglutamic acidemia Congenital toxoplasmosis Adrenoleukodystrophy 25

27 Medicaid/CHIP Eligibility Map: Medicaid Eligibility Excellent Good Satisfactory Insufficient Background In 2012, the Supreme Court decision in National Federation of Independent Business v. Sebelius enabled states to choose whether or not to expand the financial eligibility for their Medicaid program. Since the decision, a growing number of states have decided to expand their Medicaid programs to cover all individuals at or below 138 percent of the federal poverty level (FPL). States that have opted not to expand their eligibility have left approximately 5 million Americans without health insurance who would otherwise be eligible for Medicaid coverage. NORD strongly supports expanding Medicaid in every state, as it would increase access to needed health services and allow thousands of Americans with rare diseases to gain health insurance coverage. The State Children s Health Insurance Program (CHIP) is an important source of health coverage for children and families that are ineligible for traditional Medicaid. All states provide increased coverage for children and families through CHIP, but may operate the program slightly differently. For example, some states use the federal funding for CHIP to expand their Medicaid program to reach this target population (this is sometimes referred to as CHIP-funded eligibility ). Other states use these funds to operate a separate CHIP program that provides separate coverage from their Medicaid program. 26

28 Methodology See Table 5 for the complete Medicaid scoring rubric. Overall, states were scored across four subcategories: 1) eligibility for parents of dependent children; 2) eligibility for childless adults; 3) eligibility for pregnant women; and 4) eligibility for children (including CHIP-funded eligibility). Each state was scored on tiered rubric with possible scores of 0, 1, 6, 8, or. The only subcategory to include a score of zero was eligibility for childless adults because multiple states do not cover this population at all. The scores for each subcategory were combined to determine the overall category score. The scoring criteria for children and pregnant women each was determined in part by how states compare to each other. However, for the other subcategories (non-pregnant adults), the threshold for a perfect score was whether a state had expanded its Medicaid to 138% of the FPL. Sources 1. Brooks, T., Touschner, J., et al. Findings from a 50-State Survey of Eligibility, Enrollment, Renewal, and Cost-Sharing Policies in Medicaid and CHIP. Kaiser Family Foundation. Published on KFF.org State Health Facts state-survey-of-eligibility-enrollment-renewal-and-cost-sharing-policies-in-medicaid-and-chip-as-ofjanuary-2015/ 27

29 Table 5: Medicaid/CHIP Eligibility Scoring Rubric Tier Score Description Sub-Category Eligibility for Parents of Dependent Children N/A 89% of the Federal Poverty Level (FPL) or less 90% to 99% of FPL 0%-137% of FPL. 138% of FPL or greater Eligibility for Childless Adults No coverage 89% of the Federal Poverty Level (FPL) or less 90% to 99% of FPL 0%-137% of FPL. 138% of FPL or greater Medicaid eligibility of 161%- 189% of FPL; Medicaid eligibility of 190% - 219% of FPL; Medicaid eligibility of 220% of FPL or greater; Eligibility for Pregnant Women N/A Medicaid eligibility of 160% of FPL or less and no CHIPfunded eligibility No Medicaid eligibility or CHIP-funded eligibility for at least two age groups; Or Medicaid eligibility of at least 138% of FPL and CHIP-funded eligibility (or Separate CHIP) of 175% of FPL or greater Medicaid eligibility of 138%- 150% of FPL for all age groups; Or Medicaid eligibility of 161%- 189% of FPL and CHIPfunded eligibility (or separate CHIP) of 200% of FPL or greater Medicaid eligibility of 151% -199% of FPL for all age groups; Or Medicaid >200% of FPL AND CHIP-funded eligibility (or separate CHIP) of 220% of FPL or greater Medicaid eligibility of 200% of FPL or greater for all age groups; Or Or Or Or Eligibility for Children N/A Medicaid/CHIP eligibility less than 150% for all age groups. Medicaid eligibility of 0% of FPL or greater for two age groups AND CHIP-funded eligibility (or separate CHIP) of 200% of FPL or greater for all age groups Medicaid eligibility of at least 0% of FPL for all age groups AND CHIP-funded eligibility (or separate CHIP) of 200% of FPL or greater for all age groups Medicaid eligibility of 175% of FPL or greater for all age groups AND CHIP-funded eligibility (or separate CHIP) of 220% of FPL or greater for all age groups 28

30 Results (For a complete breakdown of every state, see Appendix II) The number of states adopting the ACA-funded expansion of Medicaid for adults is slowly ticking upwards. Most recently, in April 2015, Montana passed the Health and Economic Livelihood Partnership (HELP) Act expanding its Medicaid eligibility to 138% of FPL. 7 However, given that many states have kept their adult eligibility at or near zero percent of FPL, overall scores showed little variance between these two extremes. There was some variance in scores for coverage of pregnant women. State eligibility for this population varies between 138% and 380% of the FPL. The most encouraging scores were for child eligibility. All states provide traditional Medicaid for children in families making 0% of FPL or less. In addition, all states provided expanded eligibility funded through CHIP. Overall, only two states (North Dakota and Idaho) scored less than 8/ for their eligibility for children. Both of these states scored lower than the rest because of limited eligibility for children ages States Only cover pregnant women making up to 150% of FPL or less. 7 Montana lawmakers endorse Medicaid expansion. Politco.com. April 11,

31 Resources for Advocates Rare Action Network Contacts Rare Action Network is a new advocacy network working to improve the lives of the 30 million Americans living with a rare disease at the state level. Rare Action serves as a broad spectrum of stakeholders ranging from patients, to their families, caregivers, and friends; from researchers to industry; to physicians and academia. While working predominantly at the state level, the network will filter information up to NORD s national federal policy team to help address issues of national concern. Sign up for Rare Action: JOIN RARE ACTION As part of NORD s State Ambassador program, there are grassroots advocates across the country ready to engage the rare community on the issues covered in the State Progress Report: Colorado: Annie Achee (annie.achee@rareaction.org ) Connecticut: Lesley Bennett (Lesley.bennett@rareaction.org ) Florida: Fran Hokkanen (fran.hokkanen@rareaction.org ) Georgia: Beth Nguyen (beth.nguyen@rareaction.org ) Michigan: Jennifer O Connor (jen.oconnor@rareaction.org ) Oklahoma: Rachel Daniels (Rachel.daniels@rareaction.org ) Texas: Riley Lee (riley.lee@rareaction.org ) Utah: Gina Szajnuk (gina.szajnuk@rareaction.org) All other regions: Tim Boyd, NORD s Associate Director of State Policy (tim.boyd@raraction.org ) Inform Your Legislators About the State Progress Report Click HERE to share the NORD State Policy Progress Report with your local legislators 30

32 Help Us Build the Next Iteration of the State Policy Progress Report The development of this report is in ongoing process for NORD. For future iterations of the report, we plan to incorporate the following issues in some form: Coverage and reimbursement of genetic testing Coverage of medical foods for allergic disorders State policies concerning false allegations of medical child abuse pertaining to children with a rare disease State policies governing Institutional Review Boards (IRBs) for clinical trials Have an idea or input on what we should look at? Let us know! We welcome the feedback and the opportunity to work with you. Send us a note at statereport@rareaction.org 31

33 Special Thanks The National Organization for Rare Disorders (NORD) would like to offer a special thanks to following colleagues and partners who assisted in the preparation and dissemination of this report: American College for Medical Genetics and Genomics (ACMG) Beth Rader, BioMarin Michael Scott (NORD Board Member), Calcium USA Marshall Summer (NORD Board Member), Children s National Medical Center Carolyn Asbury (NORD Board Member), The Dana Foundation Ron Bartek (NORD Board Member), Friedreich s Ataxia Research Alliance Steven Grossman (NORD Board Member), HPS Group, LLC Larry LaMotte, Immune Deficiency Foundation Marialanna Lee, Leukemia and Lymphoma Society Ann Rogers, National Hemophilia Foundation Dr. Bradley Therrell, National Newborn Screening and Genetics Resource Center (NNSGRC) Christine Brown, National PKU Alliance NORD Patient Organization Members NORD Corporate Council Members 32

34 Appendix I Medical Foods Mandated Private Insurance Coverage Insurance Coverage Limitations Score Covered Disorders For Private Insurance Mandate Score State-Funded Coverage Score Covered Disorders For State Coverage Score Total Score (out of 40) % Score Statutes/New Legislation AL No No mandated coverage 0 No mandated coverage 0 Medicaid coverage is on a case by case basis. Board of Health is authorized to create regulations to provide for treatment, including but not limited to advising dietary treatment. Regulations permit the health department to inform parents about department services, which may include treatment. Coverage for WIC. 6 PKU, CH, GALT, CAH, hearing, hemoglobinathy, BD, CF, AAD, FAOD, OAD, and other heritable diseases 16 40% Alabama AC AAC et seq. AK Formula Only Health insurers except fraternal benefit societies must cover formulas. Coverage limitations are allowed. PKU is the only metabolic condition covered. 6 PKU Only 6 Medicaid coverage; The state reimburses enrolled providers for nutrition services given to Medicaid eligible patients. 8 PKU Only % Alaska AS AAC AAC AZ Hospital & medical service corporations, health care service organizations, disability insurers and health benefit plans with prescription drug benefits must cover at least 50% of the cost of medical foods to treat inherited metabolic disorders up to $5,000 annually. An accountable health plan shall cover at least 75%t of the cost of the formula. The accountable health plan may limit the maximum annual benefit for formulas under this section to twenty thousand dollars. Coverage must include at least 50 % of the cost of low protein modified foods and formula and may be limited to maximum annual benefit of $ metabolic disorders that are part of the newborn screening (NBS) program: Involving amino acid, carbohydrate and fat metabolism; have standard methods of diagnosis, treatment and monitoring; & require specially processed or treated medical foods Mandated medicaid coverage; Children s rehabilitative services program provides nutrition to treat metabolic disorders and services for CF, including nutrition taken via tube that supplies 50% of daily caloric need or nutrition taken without a tube that supplies 0% caloric need. Limited WIC coverage. 8 AAD, BD, CF, hemoglobinopathy, HCY, hypothyroidism, PKU, MSUD, OAD, storage disease and other conditions with similar treatment requirements % Statutes--ARS , , , , Ariz. Rev. Stat. Ann AR Insurance Coverage of Forumla applies after $2,400 income tax credit. Insurance Coverage of Low Protein Foods applies After $2,400 income tax credit. Metabolic Conditions Covered by Insurance: PKU,Galacto-semia, organic acidemias, amino acid disorders. 8 PKU, GALT, OAD, and AA 8 No mandated Medicaid coverage.. For PKU and GALT, rules provide for formula and foods. For other metabolic conditions, rules provide for nutritional therapy as recommended by consultants. The Health Department reimburses providers $1,000 per person treated without insurance whose expenses exceed $2,400. Tax credit applies up to $2,400 annually per child for medical food & low protein modified food. 6 PKU, GALT, and other metabolic conditions % ACA ACA et seq. AAR

35 Appendix I Medical Foods Mandated Private Insurance Coverage Insurance Coverage Limitations Score Covered Disorders For Private Insurance Mandate Score State-Funded Coverage Score Covered Disorders For State Coverage Score Total Score (out of 40) % Score Statutes/New Legislation CA No limits on Insurance Coverage of Forumla and of Low Protein Foods. PKU Genetically handicapped persons program (GHPP): CF, hemoglobinopathies, and certain metabolic disorders. California children services (CCS): inborn errors of metabolism, CF and other lung disorders from metabolic & genetic defects. GHPP provides inpatient, outpatient, & home treatment services to CCS clients under 21 with eligible conditions and GHPP clients over 21. Medi Cal covers enteral nutritional supplements and replacements if used as a therapy to prevent serious disability or death in patients with conditions that preclude use of regular food. Medi Cal: conditions that preclude use of regular food. 40 0% California Health and Safety CCR CCR CCR CCR 41848, CCR CO Insurance Coverage of formula & modular counterparts No limits on coverage of low protein foods. Metabolic Conditions Covered by Insurance All Inborn Errors of Metabolism inherited enzymatic disorders caused by single gene defects involved in metabolism of amino, organic and fatty acids, including but not limited to PKU, maternal PKU, maple syrup urine disease (MSUD), TYR, HCY, histidinemia, urea cycle disorders, hyperlysinemia, glutaric acidemias, MMA and propionic acidemia. Medicaid coverage. Under the Colorado Children s Basic Health Plan, eligible children under 19 and pregnant women may receive formula for metabolic disorders 8 Metabolic disorders 38 95% CRS 16 4 CCR CT No limit on Insurance Coverage of Forumla; no limit on coverage for low protein foods; Metabolic Conditions Covered by Insurance: all Inborn Errors of Metabolism CF, PKU and other metabolic diseases, hypothyroidism, GALT, SCD, MSUD, HCY, BD, CAH and other inborn errors of metabolism as prescribed by the health department. Medicaid coverage. Statute authorizes health department to include cost of treatment in setting fees and to purchase special infant formula, amino acid modified preparations, and low protein modified food products directly and without a purchase order. Cconditions listed for insurance except CF 40 0% Connecticut CGSA 19a 55, 59a CGSA 38a 492c CGSA 38a 518c DC Legislation introduced this session No mandated coverage 0 No mandated coverage 0 Medicaid coverage for enteral but not oral. Legislation requires the District to partially pay for treatment on a sliding scale if an infant s parents are indigent. 6 GALT, HCY, hypothyroidism, MSUD, PKU, and sickle hemoglobinopathy and other metabolic disorders identified by the committee of metabolic disorders 16 40% DCA Proposed bill: B DE Group and individual health insurance policies with pharmacy benefits must cover medical foods. Deductibles, coinsurance or other cost sharing methods may apply. PKU benefits are required through 21 years of age for men and women through age PKU and other inherited metabolic diseases caused by an inherited abnormality of biochemistry, including any diseases for which the state screens newborns. Medicaid coverage: Formulary only. Statute declares intent to provide treatment. Under regulations, specialty formula fund allows the health department to cover costs of specialty formula to treat inherited metabolic disease not covered by insurance. 6 PKU and other inherited metabolic diseases caused by an inherited abnormality of biochemistry, including any diseases for which the state screens newborns 34 85% DC DC DC DAC FL Health insurance must cover enteral formulas to treat certain inherited diseases and up to $2,500 annually for low protein modified foods to treat AAD and OAD until age 24 8 Iinherited fat, carbohydrate, amino & organic acid metabolism diseases or malabsorption as a result of congenital or neonatal defects State supplies necessary dietary treatment for PKU and other metabolic diseases as medically indicated if not otherwise available and supplemental foods to eligible families under WIC. PKU and other metabolic diseases % FS FS

36 Appendix I Medical Foods Mandated Private Insurance Coverage Insurance Coverage Limitations Score Covered Disorders For Private Insurance Mandate Score State-Funded Coverage Score Covered Disorders For State Coverage Score Total Score (out of 40) % Score Statutes/New Legislation GA No No provision. 0 No mandated coverage 0 Medicaid coverage for enteral only, not oral. Health department may use state or federal funds, if available, including Maternal and Child Health Services Block Grant to provide therapy. 6 Genetic conditions such as PKU, GALT, HCY, MSUD, hypothyroidism, CAH, and other inherited metabolic and genetic disorders 16 40% GC HI Formula and low protein foods covered up to 80% of the cost. Accident and sickness insurance, employer group health policies, and individual and group hospital plans must cover medical foods and low protein modified food products to treat inborn errors of metabolism. 8 Inborn error of metabolism caused by inherited abnormal biochemistry characterized by congenital or neonatal onset metabolic defect of amino acid, organic acid, carbohydrate or fat Medcaid coverage provides for public assistance recipients for medical foods and low protein modified food products to treat inborn errors of metabolism. Inborn error of metabolism caused by inherited abnormal biochemistry characterized by congenital or neonatal onset metabolic defect of amino acid, organic acid, carbohydrate or fat 38 95% HRS HRS 431:A 120 HRS 432:1 609 ID No No provision 0 No mandated coverage 0 Medciaid coveres only entreal. Local health agencies responsible for treatment and cure of infants. Under the Children s Special Health Program eligible patients are provided treatment services which includes provision of formula. Persons over eighteen (18) years of age with PKU may purchase formula from CSHP at CSHP s cost. 6 Local health agencies: PKU and other preventable diseases Medicaid: conditions that prevent use of traditional foods alone. State special health program: PKU only % IC 39 9 IDAPA IL Formula only Statutes and regulations: medically necessary, prescribed treatment formulas. For EE, short bowel: requires insurance coverage for amino acid based elemental formulas for treatment of eosinophilic disorders. 6 Amino & organic acid metabolism disorders, including PKU, and FAOs Limited state coverage. Statutes require the health department to, as indicated, supply otherwise unavailable formulas for some disorders & nutrition services. Regulations provide for formula to treat some conditions. 6 Same as insurance and additionally lists hyperphenylalaninemia; EE, short bowel 32 80% 4 ILCS 240/2 IAC IN Formula only Insurance: medical foods prescribed to a covered individual to treat inherited metabolic diseases. No limit on coverage. 6 Inherited metabolic diseases caused by inborn errors of amino or organic acids or urea cycle metabolism and treatable by dietary restriction of one or more amino acids State services: PKU, hypothyroidism, hemoglobinopathies, including sickle cell anemia, GALT, MSUD, HCY, inborn errors of metabolism Medicaid coverage. Covers equipment, supplies, and formula. Statute creates a NBS program that provides support for all infants and individuals identified as having a disorder. 6 Inherited metabolic diseases caused by inborn errors of amino or organic acids or urea cycle metabolism and treatable by dietary restriction of one or more amino acids State services: PKU, hypothyroidism, hemoglobinopathies, including sickle cell anemia, GALT, MSUD, HCY, inborn errors of metabolism 32 80% IC IC IC