Have you encountered opposition when advocating for Krabbe Disease
to be added to your state’s Newborn Screening panel?

Krabbe Disease Newborn Screening advocates often hear objections or excuses from their state officials regarding adding conditions like Krabbe Disease or other Lysosomal Storage Disorders (LSDs) to their Newborn Screening (NBS) panels.

Before we list some of these objections, it is important to remember that the root of these objections is typically a lack of information, or misinformation and not an adversarial one. NBS advisory boards are passionate about NBS and want to see as many conditions as possible added in their state – they may simply be unaware of the facts surrounding Krabbe Disease NBS.

To learn more about NBS Advisory Boards, check out the webinar LDNBS hosted recently!

To assist you in your advocacy, we have created a list of the most commonly heard questions/objections along with answers to each question, and journal articles when possible, as a resource.

You can learn more about your particular state and its Newborn Screening information here.

Frequent Objections and How to Counter Them: 

We don’t have the lab capacity or equipment

• • If your state is already screening for Pompe Disease and MPS-I, Krabbe can be multiplexed with these conditions.
  • Did you know you can outsource your LSD screening to Mayo Clinic or PerkinElmer? 
    • • Mayo Clinic
      • PerkinElmer

We don’t have the follow-up capacity

• • It is estimated that your program will encounter fewer than one case per year.
    • See table 1 for data from the states already screening for Krabbe Disease.

We don’t have Krabbe specialists/transplant centers in the state

• • These babies are going to be born with Krabbe Disease whether you’re screening for Krabbe Disease or not and will seek care wherever it is available.
    • Most families travel to UPMC (Pittsburgh) and Duke to be advised by experts and there are advocacy groups ready to help with financial resources. 
    • Hunter’s Hope Foundation established a Leukodystrophy Care Network around the country to provide care for children with Leukodystrophy.
  • Why not give these families the opportunity to try to save their child’s life – even if it means traveling to another state – by adding Krabbe Disease to your NBS program? 

Our state’s Medicaid program doesn’t allow for out-of-state treatment 

• • Not every baby born with Krabbe Disease will require Medicaid, and this shouldn’t be an excuse for not allowing parents to treat their child
  • There is current legislation to improve access across state lines (H.R. 3089/S. 1544 – Accelerating Kids’ Access to Care Act)
  • Perhaps this is an opportunity for your state to evaluate its Medicaid program and solve these problems to improve access to care

There are false positives and that will cause “undue stress” on the family

• • With the addition of second-tier testing for psychosine, false positives have been essentially eliminated. 
    • Second-tier testing can be performed by Mayo or Perkin Elmer if you do not have the capacity
  • Undue stress is learning that your child could have been treated if the state’s NBS panel included Krabbe Disease, but instead being left with no options for treatment. 

It’s too expensive

• • If your state is already screening for Pompe/MPS-I, the cost to add Krabbe Disease is minimal.
  • The cost to the state to cover care for a symptomatic child with Krabbe Disease is estimated to be $700,000 per year. NBS is far less expensive. 

The treatment isn’t good enough

• • The alternative is death, and no treatment for any condition is guaranteed. Parents have the right to try to save their child’s life.
  • There are two gene therapy clinical trials underway for Krabbe Disease.
  • Hematopoietic Stem Cell Transplant has been shown to improve quality of life and parents who opted for BMT have few regrets.

Did we miss any? Let us know and we will add the objections to this resource!

Table 1:

(All data is from state NBS programs as of July 2022)

Share on Facebook

Tweet