Rare Disease Report

Is Your State a Rare Disease Friendly State?

DECEMBER 16, 2016
RDR Staff
Want to know how your state is managing people with rare diseases?  Odds are they are not doing a great job. A new report by National Organization of Rare Disorders’ (NORD) Rare Action Network (RAN) shows 80% of the States in the United States get a F grade when it comes to prescription drug cost sharing and only 4 states have a rare disease advisory council. And that is just small portion of what the report shows on whether each state is helping or hindering the rare disease community.
The State Report looked at how each state does regarding medical food access, newborn screening, prescription drug cost sharing, Medicaid access, biosimilar policies, inappropriate use of step therapy, and whether or not they have a rare disease council.
Below are highlights from the report along with some of the figures used that NORD has generously provided us. However, readers are strongly encouraged to read the report in its entirety here.

Medical Food Access

Many rare disease patients require medical nutrition to manage their condition.
Medical food can be expensive. For example, the food need for PKU (phenylketonuria) can cost as much as $12,000. Third-party payment for medical foods is inconsistent and state reimbursement policies vary widely.
Some medical foods are included in state’s Medicaid, but not all mandate coverage.
13 states in the US received a grade of an F for medical foods while 29 scored an A or B. Scores were based on scores reaches under 4 categories: covered insurance; mandated private insurance coverage; state-funded coverage; and covered disorder for state programs.

Newborn Screening

Newborn screening programs are regulated and operated almost entirely at the state level, allowing customization of their program to the state’s specific needs.
And the good news, most states are doing very well at newborn screening. Only 8 states received an F for newborn screening programs.

Prescription Drug Cost Sharing

Some states have passed legislation mandating a limit on out-of-pocket expenses (e.g., $100 - $500 per month per medication). Unfortunately, not many states have addressed this problem and/or the state’s actions are inadequate.
Only 3 states in the US received an A for their prescription drug cost sharing. 80% of the US received an F, deeming it hard for people to pay for their orphan drug treatments.

Medicaid/CHIP Eligibility

Medicaid programs to cover all individuals at or below 138% of the federal poverty level. The states that have not expanded their Medicaid eligibility leaves approximately 5 million Americans without health insurance.
Without health insurance, those in need have to pay full price for medical treatment/health services.
32 states received an A or B while 18 states received an F on their Medicaid availability.


As of December 2016, 31 states have passed laws requiring communications with a prescriber before a biosimilar can be dispensed.
Almost 50% of the states received an F for their biosimilar prescriber communication. The report suggests that biosimilar are being viewed in a similar manner as generic drugs but NORD notes that not all drugs were the same for every patient, especially when the disease progression is unpredictable.

Step Therapy

Step therapy is a type of prior authorization used by insurers to require patients to use a cheaper drug first and only proceed to the more expensive drug if the former is ineffective. That is a great idea for common ailments that have a highly competitive drug market but is extremely inappropriate for ultra-rare conditions that have only one, albeit expensive, drug. These patients may be given a less effective steroid or other drug that has been used off label for years in order to comply with the step therapy rule. Meanwhile, the patient’s disease will progress as the step therapy plays out its course.
More than 80% of the US received an F for their step therapy. 3 states received an A.


Rare disease Council

States and advocates have joined together to create Rare Disease Advisory Councils. Their intentions are to help those with rare diseases in the state and make recommendations to suit their needs.
Want to know how many states received an A on this? Four.
Four states seem to care about rare diseases. In addition, there are no B’s, or C’s, just F’s, and there’s a lot of them. More than 90% of the US does not have rare disease councils. 


Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.