Every year for the past six years the National Organization for Rare Disorders (NORD) has published a State Report Card on the progress of rare disease policies in the states.

Both great progress and areas of improvement were found.

About the Report Card

The Report Card first came out in 2015. It ranks each state individually, including Washington D.C. A wide array of issues that may be of concern to rare disease patients are documented. The goal is to uncover what issues still need to be addressed in different states, and assess in what areas we have been making progress.

In order to make notable changes happen, this report card needs to be viewed by policymakers. But additionally, it serves to inform patients and their families how their states are doing and where they can direct their advocacy efforts.

Data was collected throughout 2020 up until November and each policy assessed was given a grade.

Areas of Progress

One area that has shown progress over the past year is newborn screening. This involves collecting a small sample of blood from children at birth to assess whether they have any serious illnesses or disorders. Many of the conditions screened for are rare.

This year’s report has shown that quite a few states have expanded the number of different conditions they test for. The earlier diagnosis can lead to earlier treatment and overall better outcomes.

Another area of progress is an increased number of Rare Disease Advisory Councils. Six new states enacted legislation in 2020 to create one of these councils. This brings the total number nationwide to 16. These councils will help rare disease communities have a say in the policymaking process.

Areas That Still Need Change

One of the negative policies discussed in this report is the elimination of retroactive eligibility in some states. Additionally, some have enacted prescription drug coverage limits.

Step therapy is also still a burden for many patients in some states. This requires patients to test many forms of therapy (with documented failure) before they can get coverage for the prescription they know they need.

Other concerns include:

• High out of pocket costs on prescription medications (although some states have enacted caps on such costs)
• Limited access to comprehensive care
• Medical nutrition
• Protections on state-regulated insurance
• Protections for Medicaid

You can read more about this report here. You can also read patient stories discussing the outcomes of this report card on the NORD website.