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The State Progress Report

NORD’s first ever State 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 to the rare disease community of where each state can improve and, ultimately, push forward the discussion on rare disease public policy.


The State Progress Report

What is RAN?

The Rare Action Network is NORD’s grassroots advocacy network that connects advocates and gives them the tools to support the rare disease community in their state. Learn more.

A Roadmap for State Improvement

Map for SPR_trans

Key Policy Issues

Below lists some key policies that are important to the rare disease community. NORD recognizes that while the issues represented in the report are critical, there may be other important topics that may not be covered. In future iterations of this report, we plan to incorporate policies concerning genetic testing and medical child abuse. 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 make sure we get it right before recommending to states how to act).

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.

“Patients living with rare, genetic diseases like me are left to navigate a state policy environment rife with loopholes in medical foods coverage that result in the discriminatory provision of costly treatment based upon age, gender, state of residence, or employer.”

Jennifer has a rare, genetic disorder called Phenylketonuria (PKU). Read her full story here.

Under the Affordable Care Act, many people with rare diseases can now access affordable health insurance. However, some insurance policies place orphan therapies on the so-called “specialty-tier” of a drug formulary. For drugs placed on this tier, enrollees often must meet cost sharing requirements that can be as much as 50% of the actual cost of the medication.

This year alone, my son’s medical bills will be over one million dollars for treatment.  Caring for my child has put a financial strain on my family, as a box of his factor ranges from $3,000-$6,000, and he is a child, his medicine is dosed by weight, so the older he gets the more it will cost for his life saving medicine.

Currently our monthly treatment bill is $69,744, as the medicine is considered a specialty tier drug.

Christina is the mother of a son with hemophilia.

States have wide discretion on which conditions newborn children are screened for, including mandated times to complete the panel analysis and uses of blood spot data following the test. NORD encourages every state to adopt the recommendations of the HRSA Recommended Uniform Screening Panel (RUSP).


For Stephen, a late diagnosis of isovaleric acidemia (IVA) at age 3 ½, altered the course of his life and redefined his milestones.

Stephen suffered a metabolic acidosis that resulted in a traumatic brain injury.  He was in a coma on life support for three weeks and was left with severe intellectual and developmental disabilities, seizures, a gastrostomy tube and cortical vision impairment.   The devastation of what Stephen endured was compounded by the realization that it could have been prevented had he been screened for IVA at birth. Unfortunately, IVA was not on the screening panel in Virginia and most states at the time of Stephen’s birth leaving all babies at risk of sharing Stephen’s fate.

Our daughter Caroline, now thirteen, was born a year after Stephen’s crisis.  Our experience with Stephen influenced our decision to have an amniocentesis.  The results indicated that she too had IVA, which was confirmed with further testing at birth.  This early diagnosis enabled Caroline to be placed on the proper diet and medications indicated for the management of IVA, avoiding the devastating consequences that Stephen experienced. Today, she is a healthy, normal young teen experiencing all the many milestones in a child’s life that Stephen missed out on.

Jana Monaco is the mother of Stephen and Caroline who were both diagnosed with isovaleric acidemia (IVA).

In 2013, 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 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.


Progress Report Action Center

What can you do to drive progress in your state and push forward the discussion on rare diseases?

  • Download this toolkit for items to share on social media to get the conversation going on #NORDReport
  • Download these images to share on social media to help make an impact and drive progress in each state
  • Contact a NORD State Ambassador to connect on state initiatives going on in your state-contact information can be found on Page 9 of the toolkit.
  • Join the Rare Action Network!
  • Click here for state-by-state information

Download our toolkit and help us drive progress!

Download the State Progress Report

You can drive progress in your state. Please fill out the form below to download the State Progress Report.