While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention.
The report card project, launched in 2015, rates each state and Washington, D.C., on key issues that directly affect the more than 25 million U.S. residents who have one of roughly 7,000 existing rare disorders.
“The state report card provides insight into the advances being made at the state level in supporting Americans living with rare diseases, and identifies where we still need to focus attention,” Peter L. Saltonstall, NORD’s president and CEO, said in a press release.
“For almost 40 years, NORD has served as the voice of the rare disease community, and we are committed to fighting for rare disease patients and policy changes that will make a difference in their lives,” Saltonstall said.
“Our hope is that patients, advocates, and policymakers find the new State Report Card site a useful tool in their work on policies to best meet the needs of rare patients and families,” he added.
The report was compiled using data current as of November 2020 and provides detailed analysis across eight major policies, grading each state on its performance.
One policy is newborn screening, a practice in which clinicians collect a small blood sample from newly born babies to test for a number of serious health conditions, many of them rare disorders. The report indicates that many states increased the number of conditions for which they screen, removing possible future guesswork about children’s health and allowing for early treatment.
NORD advocates for a strong, well-funded screening program in every state.
As for Rare Disease Advisory Councils (RDACs) — advisory bodies that help the rare disease community have a stronger voice in state governments — six states last year successfully enacted legislation to create them. New RDACs were launched in Massachusetts, New York, Ohio, Tennessee, Utah, and West Virginia. These additions bring the number of RDACs in the nation to 16.
A top NORD goal moving forward is to help more states enact RDAC legislation and to optimize existing councils.
In a key development, Oklahoma and Missouri last year expanded eligibility for their Medicaid programs, joining 38 states and Washington, D.C. in extending access to what NORD calls a “vital safety net.”
Several states, however, sought policies that could impede access to care, according to the NORD report. Those measures included the elimination of retroactive eligibility or prescription drug coverage limits. NORD wants patients to receive Medicaid services sans what it terms unnecessary and harmful barriers.
Another important policy issue for NORD concerns so-called step therapy. According to the advocacy group, health insurers use step therapy to control treatment costs by requiring patients to try one or more treatment options, and document their failure, before the patient can receive a covered prescribed medication.
Louisiana, North Carolina, and South Dakota last year passed new patient protocols requiring such documentation, adding to the list of 31 states with comprehensive step therapy protections. NORD supports policies to reform such therapy restrictions.
Regarding the issue of out-of-pocket expenses, New Jersey last year capped such costs at $150 monthly for each medication. In addition, Georgia banned co-pay accumulator programs. Such programs are implemented by insurers and pharmacy benefit managers to limit the impact of manufacturer contributions on patients’ deductibles and out-of-pocket costs. NORD advocates for policies that lower out-of-pocket expenses and maximize access to needed treatments.
Other major state policies that were graded include medical nutrition, protecting patients in state-regulated insurance, and protecting patients in state Medicaid programs.
Patient stories about the policies graded by NORD are available on the report card website.