Rare Daily Staff

While many states took powerful steps forward in 2019 to help people living with rare diseases, there is still major work to be done on a number of policy issues critical to the more than 25 million Americans affected by them, according the a new report from the National Organization for Rare Disorders (NORD).

Significant progress was made on access to medications for patients and newborn screening, while further efforts are still needed on Medicaid coverage and the creation of rare disease advisory councils.

The 2019 State Report Card scores states on their policies across a range of issues relevant to people with rare diseases. This year the report evaluated states on the strength of policies in the following areas: Medicaid eligibility and section 1115 waivers, medical nutrition and newborn screening, prescription drug out-of-pocket cost protections, individual insurance market protections, protections against step therapy protocols, and the establishment of rare disease advisory councils.

“States have made a great deal of progress in supporting Americans living with rare diseases. But there is still much more that needs to be done,” said Peter Saltonstall, president and CEO of NORD. “The goal of the State of the States Report is to share concrete and current information that will empower advocates to affect change in their states.”

Significant progress was made on newborn screening with nearly all states earned an overall grade of an A or B as every state now implements newborn screening and generally adheres to federal recommendations on which diseases to screen. This compares to 2018 when half of all states failed to meet federal recommendations.

Access to medications for patients has also improved as majority of states have now enacted policies to protect patients from potentially harmful step therapy practices. In 2019, six states enacted new step therapy protection laws. But a lot of work still needs to be done on placing limits on patients’ out-of-pocket costs, with only 11 states earning a grade of A or B, and 34 states getting an F.

Comprehensive Medicaid coverage is a difficult area to grade because states have a lot of leeway in eligibility requirement. In 2019, more states made the move to expand Medicaid, but some states have pursued harmful policies that could be detrimental to the rare disease community, such as the elimination of retroactive eligibility or the addition of a work requirement.

Finally, in 2019, five states successfully enacted a rare disease advisory council that will help give the rare disease community greater representation in state government. In 2014, no state had a rare disease advisory council. Today, there are eleven states that have some form of an advisory council in place.

NORD launched its State Report Card report in 2015 to provide a roadmap for rare disease advocates and provide them with an understanding of how the various states fare with regards to policies that affect the daily lives of people with rare diseases.

NORD hopes the report can serve as a tool for patients, advocates, and policy makers as they strive to ensure that state policies best serve the needs of the rare disease community.

Photo: Peter Saltonstall, president and CEO of NORD

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