Medicaid, the largest health insurance program in the United States, is a joint federal-state program that provides health insurance for low-income individuals. The federal government currently matches at least fifty percent of state Medicaid spending in every state, and states with lower per-capita incomes receive higher matching amounts. Historically, Medicaid provided coverage for children, pregnant women, and families but the Affordable Care Act allowed states to expand coverage more broadly. As much as one third of the bleeding disorders community is currently insured by Medicaid.


Section 1115 Waivers

Section 1115 of the Social Security Act gives the Secretary of Health and Human Services authority to approve experimental, pilot, or demonstration projects that promote the objectives of the Medicaid program. The purpose of these demonstrations, which give states additional flexibility to design and improve their programs, is to demonstrate and evaluate state-specific policy approaches to better serving Medicaid populations.1

Since 2017, the Center for Medicare and Medicaid Services (CMS) has attempted to expand the use of waivers to allow states to limit Medicaid enrollment. Using Section 1115 waivers, states have applied to: impose work requirements for beneficiaries, establish a closed formulary for prescription drugs, cap lifetime benefits on enrollees, or require premium payments.

In January 2020 CMS announced new guidance to states that support limiting federal Medicaid funding through block grants.

The purpose of Medicaid is to provide health care for low-income individuals and families. NBDF opposes policies that make it more difficult or expensive for patients with chronic diseases to access Medicaid.



Nineteen states have submitted waiver applications to CMS to establish work requirements. Because of legal challenges no state has implemented work requirements. CMS has rejected proposals to establish lifetime benefit caps and a closed formulary.

For more detailed information on state waiver applications please visit the Kaiser Family Foundation web site



Reimbursement (Covered Outpatient Drug Rule)

In 2016 CMS released the Covered Outpatient Drug (COD) Rule requiring states to amend their Medicaid plans to adopt a new prescription drug reimbursement method based on Actual Acquisition Cost and a professional dispensing fee.2 States were required to submit their State Plan Amendments (SPA) by April 1, 2017.

In face-to-face meetings with CMS NBDF expressed concern about the rule and the potential for states to set reimbursement rates so low that providers who dispensed anti-hemophilic therapies would lose money serving bleeding disorders patients and stop serving them. NBDF was also concerned about how the rule would impact 340B pharmacies such as Hemophilia Treatment Centers that rely on pharmacy reimbursement to provide the comprehensive care that benefits bleeding disorders patients.



In 2019, California became the last state to comply with the COD Rule. Commercial specialty pharmacies challenged and delayed the final adoption of the SPA over concern that the proposed rate was too low for them to remain profitable and serve bleeding disorders patients.

States have implemented a variety of reimbursement methodologies consistent with the rule.

NBDF remains concerned about the sustainability of some states’ reimbursement methodologies and continues to monitor their implementation.



Value-based Payments

On December 31, 2020, CMS released a proposed rule to “advance CMS’ efforts to support state flexibility to enter into innovative value-based purchasing arrangements (VBPs) with manufacturers, and to provide manufacturers with regulatory support to enter into VBPs with payers, including Medicaid.”3 The rule issues new regulatory policies and clarifications that would allow manufacturers and payers to enter into VBPs consistent with the Medicaid Drug Rebate Program.

Value-based Purchasing arrangements are considered an important innovation that will help reduce the barriers to patient access that exist for novel, high-cost durable/curative therapies.4



NBDF provided comments to CMS offering tentative support to removing regulatory barriers to manufacturers and payers entering VBPs.

NBDF urged CMS to ensure that patient input – on patient needs, priorities, and outcomes – is incorporated into any VBP framework, and that VBP strategies neither lock in individuals or prevent them from choosing the best treatment or combinations of treatments in consultation with their physicians. It is also important that VBP strategies not advantage novel therapies, leading to budgetary pressures and coverage cutbacks affecting patients who have opted to continue therapy with the existing armamentarium of products.