NHF federal priorities focus on access to affordable, quality treatments and healthcare that meet the needs of the bleeding disorders community.

Step Therapy

NHF is a member of the State Access to Innovative Medicines (SAIM) Coalition, which advocates for reasonable exceptions to step therapy. From the SAIM web site: Step therapy is an insurance protocol that requires patients to unsuccessfully try one or more insurer-preferred medications before they receive coverage for the medication that their physician recommends. This practice is also known as “fail first” and can take weeks or months. Once a patient finds a medication that does work for them, they may have to repeat the step therapy process if they switch insurance plans.

When implemented inappropriately, step therapy can result in patients not being able to access the treatments they need in a timely manner. This can lead to worsened symptoms and presents a particular challenge for patients suffering from life-threatening or chronic diseases. Physicians can request exceptions to step therapy requirements, but insurers may not respond promptly to such requests, resulting in a further delay of treatment.

The SAIM Coalition supports common-sense limits on step therapy such as establishing clear timelines for insurer responses to requests for exceptions and ensuring that patients who are successfully using a treatment do not have to switch.


H.R. 2279, The Safe Step Act, was introduced April 10, 2019 by Rep. Raul Ruiz and has 146 cosponsors. Its companion in the Senate is S. 2546, introduced by Sen. Murkowski on Sept. 25, 2019. S. 2546 has sixteen cosponsors.



Affordable Care Act

On March 23, 2010, President Obama signed comprehensive health reform, the Patient Protection and Affordable Care Act, into law.

When the ACA was passed it implemented many changes to the U.S healthcare system.

This list is meant to highlight some of the key areas as it relates to the bleeding disorders community (It does not encompass every provision within the ACA):

  • Imposed additional rules around if an employer had to offer health plans to their employees
  • Expansion of public program (e.g. Medicaid and CHIP)
  • Various financial maneuvers for both employees and employers  
  • Created various types of Health Insurance Exchanges  
  • Mandated that all ACA health plans offer coverage for essential health benefits (e.g. mental health and pediatric dental care)
  • Implemented various rules around healthcare protections and greater access to care (e.g. prohibiting pre-existing conditions exclusions, dependent care)

Prior to the ACA, people with bleeding disorders were discriminated against in the insurance market.  Insurance plans could exclude from coverage people with preexisting conditions.  NHF was also concerned about lifetime and annual limits on insurance benefits, as this was a top priority for people living with bleeding disorders due to our high medical costs. Clotting factor therapies are very effective at preventing and treating life-threatening bleeding episodes, but also are extremely expensive. Annual costs for clotting factor are typically $250,000 or more for someone with severe hemophilia and development of an inhibitor (immune response to treatment), bleeding from a trauma, surgery or other complications can raise costs in a given year to $1 million or more. As a result of these high-cost treatments, people with bleeding disorders would regularly hit lifetime and annual limits, before the ACA was enacted.

Consumer Protections

NHF estimates more than 50% of the bleeding disorders community is insured by private insurance plans. NHF supports the many private insurance consumer protections included in the ACA, which have benefited our community and others with high-cost, chronic and rare conditions. Clotting factor therapies are very effective at preventing and treating life-threatening bleeding episodes, but also are extremely expensive.  Annual costs for clotting factor are typically $250,000 or more for someone with severe hemophilia.  Development of an inhibitor (immune response to treatment), bleeding from a trauma, surgery or other complications can raise costs in a given year to $1 million or more.  As a result of these high-cost treatments, people with bleeding disorders would regularly hit lifetime and annual limits, before the ACA was enacted.

NHF seeks to ensure that any proposals to change the ACA maintain these vital patient protections:

  • Elimination of lifetime and annual limits;
  • Elimination of pre-existing conditions exclusions and rating based on health status;
  • Limits on out-of-pocket expenses;
  • Coverage for dependents up to age 26;
  • Network adequacy policies to ensure access to specialists;
  • Prohibition of discrimination based on health status, and  
  • Guaranteed issue and renewal.


Many people with bleeding disorders struggle to afford premiums for insurance coverage that is comprehensive enough to meet their complex healthcare needs. They often have significant out-of-pocket costs related to their treatment. NHF supports current ACA policies and potential future policies to address affordability, including:

  • Maintaining the current out-of-pocket maximum: The ACA limit on out-of-pocket expenses (OOP) has been extremely important to our community due to our high costs. Without this policy, individuals could be responsible for paying tens or even hundreds of thousands of dollars per year.
  • Financial assistance for low-income individuals: The ACA included provisions to help low-income individuals purchase health insurance, such as cost-sharing reductions and tax credits.
  • Reinsurance:  We recognize the need for insurers to balance the financial costs for having individuals with bleeding disorders and other high-cost, chronic conditions in their pools. Rather than the reinstatement of flawed and harmful high-risk pools that would segregate our population, NHF supports proposals to either create a federal reinsurance pool or to give states funding to create reinsurance pools and to implement other innovative ways of assisting plans with balancing their financial risks.

Section 1332 Waivers

On October 22, 2018, the Trump administration released new guidance on Section 1332 waivers established by the Affordable Care Act (ACA). This replaced earlier guidance released in 2015 and substantially changed the standards for evaluating waiver applications. While waiver activity to date has been limited and mostly used to implement state reinsurance programs to help reduce the cost of ACA-compliant individual market policies, the new guidance may encourage states to use 1332 waiver authority to make broader changes to insurance coverage for their residents, including to promote the sale of, and apply subsidies to, ACA non-compliant policies. On November 29, 2018, the Centers for Medicare and Medicaid Services (CMS) released a discussion paper outlining a set of waiver concepts designed to provide states with a roadmap for developing waiver applications that use the flexibility granted under the new guidance (Kaiser Family Foundation).

By loosening the interpretation of the statutory guardrails and encouraging states to increase access to private coverage, specifically ACA non-compliant coverage, the new guidance appears to encourage states to develop waiver proposals that would make changes to their health coverage systems that are dramatically different from that provided under the ACA today. After the guidance was published, CMS released a set of “waiver concepts” to spur ideas that states could pursue through 1332 waivers (KFF).


Since 2017 Trump Administration administrative reforms to the ACA have significantly altered the law. Those efforts include:

  • Repeal of the individual mandate
  • Ending the cost-share reductions (CSR)
  • Cutting the open enrollment period in half
  • Reducing marketing for open enrollment by 90%
  • Eliminating funding for in-person assistance with enrollment

In addition, the administration has issued guidance allowing for the creation of health insurance products that are not required to meet the standards of the ACA. These so-called “skinny” plans include Short-term Limited Duration Health Plans and Association Health Plans.

Collectively, these regulations create two separate health insurance markets — one for the healthy and the other for people with pre-existing conditions. Moreover, it further destabilizes the state health insurance marketplaces.

The ACA remains the law of the land despite efforts to repeal and replace it in 2017. It is currently subject to a legal challenge brought by the state Attorney’s General.  The Supreme Court will take up that case in 2021.

NHF continues to monitor state waiver applications and take action to prevent waivers from reducing the ACA’s key protections.



Copay Accumulator Adjustment Programs

NHF opposes the use of copay accumulator adjustment programs because they jeopardize a patient’s access to life-saving medication and may drive up healthcare costs as patients turn to more expensive emergency departments to obtain their clotting factor therapy.

Manufacturer copay assistance cards have positive benefits for patients (improves access, affordability, and compliance). However, an increasing number of health insurance plan sponsors believe they drive prescription drug costs up by assisting beneficiaries in satisfying part or all of their personal out of pocket (OOP) costs.

Their contention is that if a patient has little to no personal share of the costs, they would have no incentive to consider generic alternatives, formulary, or preferred drugs and would likely not think twice about getting unnecessary tests, procedures, and labs.  

Accumulator adjustment programs target manufacturer copay assistance programs available for specialty drugs by no longer allowing them to count towards a member’s accumulator (annual total of out-of-pocket expenses), believing that manufacturers use them to steer patients to higher cost drugs, rather than lower-cost generic equivalents.


CMS’ 2021 Notice of Benefit and Payment Parameters Rule allows health insurers to exclude manufacturer copay assistance from a patient’s annual out of pocket maximum. NHF submitted comments to CMS opposing this change in policy.


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