Yesterday, I (Lesa) had the opportunity to attend the quarterly meeting of Pennsylvania’s Newborn Screening (NBS) Advisory Board. These meetings are open to the public but members of the public apparently rarely attend, as was evidenced by comments made and by the fact that so many came to introduce themselves to us.

This committee was formed to advise the Secretary of Health regarding advancements in NBS and to recommend certain diseases to be added to the mandatory NBS panel. However, they have gained a lot of power and they essentially are the final say now. The Secretary of Health has told them that she will pretty much do whatever they say.

You can see a list of the members here along with their credentials.

It was an informative meeting on many levels, but here are some bullet points of what I feel are the most important points:

• Dr. Vockley – the chair of the committee – clearly doesn’t think that Krabbe should be part of the NBS program. He made that very obvious in his statements.
• Penn State Hershey Medical Center is THE ONLY hospital in Pennsylvania screening for all of the diseases in Hannah’s Law (Act 148). They began screening on March 8, 2016. Despite the fact that hospitals are telling parents that they are screening for Krabbe, the Department of Health AND the lab that processes the NBS panels confirmed that only Hershey Medical Center is testing.
• Dr. Vockley said that he “doesn’t understand” why Hershey is testing for these diseases and wants to know their thought process. Yes, I was pretty upset to hear him say something like that.
  • He seems to be basing his entire opinion on NY’s results from the first five years of testing. One state. He kept going on and on about how “unsuccessful” treatment was, which is why I said what I did in my testimony.
• The main hospitals in Philadelphia are looking to add the same five diseases from Act 148 to their panels.
• Pennsylvania’s biggest issue is to resolve how these NBS panels are funded:
  • If the state mandates them (currently 6 diseases are mandated), the state must pay for them as the law is currently written.
  • If the state moves them to the “mandatory follow-up” or supplemental panel, the hospitals absorb the cost. This is why hospitals have the choice to screen or not screen for the 27 diseases on the “follow-up” panel, even though Dr. Vockley continually used the phrase “mandatory follow-up” when describing the panel.
  • They reported that it costs about $80 per baby to screen for all 31 (or so) diseases on both panels. Only $80!
    • Mandated six diseases = $23
    • Pompe (from Act 148) = $10
    • Additional $47 per child if all were mandatory
    • Our thought was that it should just be added to the hospital bill, or split cost with parents. What parent wouldn’t want to pay $80 more to have their children fully screened? Not all could afford it, but those who could would pay.
  • If insurance would help the hospitals with cost, hospitals would be more likely to screen for all.
  • However, it is difficult to mandate insurance coverage because most providers are not based in Pennsylvania. State can’t force them to pay.
  • Bottom line: new legislation needs to be written to change how PA funds these NBS panels because children aren’t being screened equally.
• The committee admitted that they are not “on par with the rest of the country” with how we operate and fund our NBS program. It is currently a “public/private collaboration” instead of being purely a public health program.
• Dr. Vockley continued to make comments about other diseases that would apply to Krabbe but he is still opposed to Krabbe being on the NBS panel:
  • “Why do we have to wait for a baby to die before we do something?”
  • “I’d be the last person to tell you that saving two babies out of two million is not valuable…”
• The board did vote to recommend that ALD and MPS-1 be added to the mandatory panel. Next week the Secretary of Health will review the decision memo but there is no time-table for addition to the NBS panel.

After all of this discussion (and much more that wasn’t relevant to our cause), there was a time for public comments. You can hear my testimony here. It is about 5 minutes long.

After the meeting was adjourned, Dr. Vockley came up to me and thanked me for coming and then told me “I disagree with what you said…but we’re on the same page for NBS.” I smiled and held in what I really wanted to say. That he could look me in the eye after knowing who I was, and what we had just gone through, and tell me something like that was maddening.

The legislative staff from Rep. Cruz’s office were there with me and we all agreed that the main thing we learned is that there is a lot of conflicting information being given from the Department of Health about the NBS program in PA:

• They said there are two panels (mandatory and mandatory follow-up) and kept saying that both are mandatory…yet they also said that the hospitals have the ability to choose whether or not to test for the “follow-up” panel. Are they truly mandatory, then?
• The chair of the advisory panel says he is all about saving lives through NBS, but he is adamantly opposed to Krabbe and the other Lysosomal Storage Disorders being screened. His arguments FOR ALD and MPS-1 absolutely apply to Krabbe, same success rates and treatment, but he is opposed to NBS for Krabbe. It doesn’t make sense.

The next meeting is August 23, 2016. At that meeting, they hope to further resolve some of what was discussed yesterday, but I am not hopeful that much will change. The chair is very set in his ways and is unwilling to hear differing opinions about Krabbe. The other members seemed to be more agreeable to the screenings BUT they seem to be afraid to speak up because of Dr. Vockley’s intimidating personality.

After observing this advisory board in person, it is even more clear that Rep. Cruz’s legislation is necessary. Please check out our previous post about this and contact your PA representatives!

We will keep you posted as we learn more. Pennsylvania’s NBS program is broken and we won’t stop until it is fixed!