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

Affordable Care Act

On March 23, 2010, President Obama signed comprehensive health care reform, the Patient Protection and Affordable Care Act (commonly referred to as ACA), into law.

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

This list highlights some of the key areas as it relates to the bleeding disorders community (it does not encompass every provision in the ACA):

  • Imposed rules around if an employer had to offer health plans to their employees
  • Expansion of public programs (e.g., Medicaid and CHIP)
  • Various financial protections for both employees and employers
  • Created Health Insurance Exchanges (Marketplaces)
  • Required all ACA-compliant plans offer coverage for essential health benefits (EHB’s) including 10 broad coverage categories (e.g., inpatient and outpatient hospital care, prescription drug coverage, pregnancy and childbirth, mental health services, and more)
  • Implemented various consumer protections that promote greater access to care (e.g., prohibiting pre-existing conditions exclusions)

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.  The elimination of lifetime and annual limits on insurance benefits was a significant consumer protection for people living with bleeding disorders.   Annual costs for clotting factor are typically $300,000 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 each year to $1 million or more.  As a result of these high-cost treatments, people with bleeding disorders would routinely hit lifetime and annual limits, before the ACA.  

Consumer Protections

NBDF estimates roughly 60% of the bleeding disorders community is insured by private insurance.  NBDF supported the many private insurance consumer protections included in the ACA, which have benefited our community and others with high-cost, chronic and rare conditions.

NBDF 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.
  • Guaranteed issue and renewal.


Many individuals with bleeding disorders struggle to afford premiums for comprehensive insurance coverage and out-of-pocket expenses for medication.  NBDF supports current 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 important to our community due to our higher OOP costs.  Without this policy, individuals could be responsible for paying tens of thousands of dollars per year.
  • Financial assistance for low-income individuals: The ACA included provisions to help individuals purchase health insurance, through cost-sharing reductions and tax credits.   

Section 1332 Waivers

In 2018, the Trump administration released new guidance on Section 1332 waivers established by the ACA which substantially changed the standards for evaluating waivers.  The new guidance encourages 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.  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 encourages 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.


Since 2017 Trump Administration administrative changes 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 (STLD) and Association Health Plans.1

Collectively, these regulations create twon separate health insurance markets — one for healthy people 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 is currently subject to a legal challenge brought by state Attorney’s General.  The Supreme Court is expected to announce their ruling in late Spring/early Summer 2021.

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



Copay Accumulator Adjustment Programs

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

Copayment assistance, including manufacturer discount cards, has positive benefits for patients (improves access, affordability, and compliance). However, an increasing number of health insurance plan sponsors believe they drive up prescription drug costs 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, he or she 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 copay assistance programs by no longer allowing them to count towards a member’s accumulator (annual total of out-of-pocket expenses), believing that manufacturers use these programs 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.  NBDF submitted comments to CMS opposing this change in policy.

March 2021:  U.S. Rep. McEachin (D-VA) and Rep. Davis (R-IL) have submitted a letter with 58 other Representatives’ signatures to President Biden asking him to reverse the 2021 NBPP Rule and require insurers to count all payments made by or on behalf of a patient toward that patient’s annual deductible and out-of-pocket maximum.



Step Therapy

NBDF is a member of the State Access to Innovative Medicines (SAIM) Coalition, which advocates for reasonable exceptions to step therapy.

“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.”2


S. 464, the “Safe Step Act”, was introduced February 25, 2021 by Sen. Lisa Murkowski (R-AK) and has fifteen cosponsors. The House companion bill is H.R. 2163, introduced March 23, 2021 by Rep. Raul Ruiz (D-CA) and has fourteen cosponsors.