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State Report Card

In 2015, the launch of NORD’s inaugural State Policy Report altered the landscape for rare disease advocates by empowering them to analyze and advocate for health care policy decisions determined at the state level that impact their daily lives. The 2015 report called attention to the lack of policies to ensure access to care for rare disease patients in many states. The policies we focused on in the report were: prescription drug co-insurance, medical foods access, newborn screening, and Medicaid eligibility.

The 2016 State Report Card provides an update on each of these policy categories as well as several new issues that we believe are pertinent to the rare disease community. The new additions are as follows: Biosimilar Prescriber Communication, Step-Therapy Protection and Rare Disease Advisory Councils.

Below lists 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).

Learn the Key Issues

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 people with these conditions, medical foods are the only viable treatment option available. Many states have mandated the inclusion of medical foods within health insurance plans sold within their state. However, in the states which do not have medical food mandates, individuals in need of these particular treatments often require assistance in paying for medical food expenses.

Read Jennifer's Story

“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.

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).

NORD supports robust, well-funded newborn screening programs in every state, and encourages state lawmakers to prioritize the early detection of these debilitating diseases. NORD encourages every state to adopt the Uniform Newborn Screening Panel developed by the Discretionary Advisory Committee on Heritable Disorders in Newborns and Children, and will continue to advocate for this adoption in each state that currently does not screen for the disorders included within the panel.

Read Jana's Story

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).

Health insurance companies use cost sharing for drugs to encourage patients to try lower-cost medications before turning to more expensive ones. However, it is common for insurance companies to charge patients with a severe condition thousands of dollars in cost sharing each month. To assist patients who find themselves in this difficult situation, several states have passed legislation mandating a limit on out-of pocket costs for certain medications. Given that new treatments can be expensive to develop (it takes an average of 12.5 years and $1.5 billion in 2014 dollars to bring a new drug from preclinical stage through FDA approval), many manufacturers set the cost high to recoup these expenses, especially given the fact that orphan products are intended to treat small patient populations.

Read Christina's Story

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 the option of whether or not to expand eligibility for their Medicaid program (publicly-funded health insurance). As a result, someone who is eligible for health care via Medicaid in one state may not be in another.

In 2013, the Supreme Court decision in National Federation of Independent Business v. Sebeliusenabled 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.

Biologics are the future of rare disease treatment. These medicines are created from living organisms to treat rare and chronic
diseases in ways other cannot. However, not all biologics are the same, which is why it is important for doctors to be alerted whenever a pharmacy or health insurer wants to change the type of biologic dispensed to a patient.

Step therapy is a process by which insurance companies require a patient to take one or more alternative medicines before being put on the one preferred by their doctor. While this is done by insurers as an attempt to control health care costs, step therapy has been increasingly applied to patients with little regard to their medical situation or treatment history. As a result, in many cases step-therapy can delay better treatment and increase costs.

Helping the rare disease community starts with ensuring that patients and families have a voice in government. Several states have recognized this and worked with local advocates to create new Rare Disease Advisory Councils. Their purpose is to evaluate and make recommendations to the state on issues related to health care access and coverage for rare disease patients.

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