NORD Statement Regarding State Proposals to Alter Their Medicaid Program

Washington, D.C., February 8, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement regarding recent state proposals to alter their Medicaid program by utilizing the federal 1115 Waiver process:

“In an attempt to control health care costs and improve services for Medicaid beneficiaries, states have sought Section 1115 waivers that would enable them to make substantial changes to Medicaid benefits and eligibility. We at the National Organization for Rare Disorders (NORD) understand the need for states to consider creative policy solutions to improve health care quality and access. However, we have grave concerns that many of the current state proposals will have a detrimental effect on the rare disease community.

Section 1115 Waivers enable the Federal Government to approve state-administered demonstration projects that the Centers for Medicare and Medicaid Services (CMS) determines to be of benefit to the objectives of the Medicaid program. These projects waive certain Medicaid requirements and allow a state to direct federal Medicaid funds in ways that would otherwise not be permitted. If enacted, NORD believes that several of the current proposals would restructure Medicaid benefits and eligibility in a way that undermines the purpose of the program and disproportionately affects people with rare diseases.

First, multiple states have proposed to implement “commercial-style” formulary restrictions in Medicaid programs that would limit coverage for new therapies approved by the Food and Drug Administration (FDA). NORD opposes any formulary restrictions that cut access to vital orphan therapies. Restricting drug benefits would limit the ability of providers to make the best medical decisions for their patients. This could inordinately affect rare disease patients because they disproportionately rely on the new and innovative medicines these states are aiming to restrict. Rare disease patients deserve the same access to life-improving, even life-saving, medications as everyone else, and these harmful proposes will disproportionately impact rare disease patients and their families.

Second, many states are proposing to add work requirements to their Medicaid programs. On January 11, CMS released a letter to Medicaid Directors signaling its support for work requirements. The following day, CMS approved Kentucky’s 1115 waiver, the first approved waiver to include work requirements.

NORD opposes the implementation of work requirements, as we believe the exemptions to these requirements will not adequately address the complex health challenges facing rare disease patients. With a scarcity of physicians familiar with rare diseases and the prevalence of undiagnosed conditions, it is often difficult, even impossible, for rare disease patients to convey the extent of their symptoms in a way that satisfies state requirements. Forcing patients to justify their inability to maintain a consistent work schedule before they can receive or continue to receive care could result in a devastating loss of coverage throughout the rare disease community.

Third, there are some states that have proposed tying work requirements to lifetime limits on Medicaid coverage. NORD strongly opposes lifetime limits to healthcare coverage under any circumstance. Lifetime limits disproportionately harm individuals with rare diseases due to the often genetic, life-long nature of their disease, as well as the incredibly expensive therapies and orphan drugs used to treat them. Rare disease patients who are subjected to work requirements would suddenly find themselves entirely without coverage, regardless of whether they have adhered to the work requirements.  

Finally, states are debating a number of proposals that reduce eligibility and benefits for those with incomes at or around 100 percent of the Federal Poverty Level (approximately $12,140 per year for an individual). Such proposals to weaken access to specialists or other critical services in Medicaid would be exceptionally detrimental to individuals with rare diseases, as continuity of care is essential to effective treatment. Consequently, NORD opposes any proposal that would considerably weaken healthcare coverage for any population of rare disease patients.

These concerns are not exhaustive, but they are representative of the ways in which the rare disease community might be harmed by some of the emerging proposals to control costs. Medicaid exists to be a safety net for those who cannot access other forms of health care coverage. Substantially altering the program in ways that reduces benefits for people in need is not only diametrically opposed to the purpose of the program, but it will serve to worsen heath care outcomes and increase costs for rare disease patients and their caregivers. As the leading representative of the rare disease patient community, NORD will continue to engage with states on the best way to improve health care through the Medicaid waiver process.”