The meeting could determine whether the two young siblings would keep going blind. The doctor knew that going in, but he was feeling good. He’d negotiated huge discounts before, allowing patients to get complex surgeries or budget-busting drugs they otherwise couldn’t afford. And his last two conversations with Spark Therapeutics had been promising.

Then again, at $850,000 a person, Luxturna was more budget-busting than just about any other drug. Spark had proposed a few different ways of helping insurers to cover the gene therapy — but Dr. Kevin Strauss’ patients tend not to have insurance. As the medical director of the Clinic for Special Children, on the outskirts of Strasburg, Pa., he mostly sees members of the Plain community: Old Order Amish and Mennonite families, who believe that caring for the sick and the elderly is a community’s responsibility. Relying on governments or private companies to step in is considered a serious moral breach. They don’t want medicine for free. They just want to pay the reduced prices that government and private insurers often do.

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  • Thanks for this article.

    Seems to me that this type of problem ($$) can only be solved with government resources.

    I would expect the drug producer to petition (in this case) the FDA for $ subsidy during the product’s development to produce an affordable cost/dose. I would also expect that government NOT increase the cost/dose by negotiating discounts for larger programs like Medicaid, Medicare, etc.

  • The comments below regarding the beliefs of the Amish and some Mennonites represent outright religious bigotry and no understanding of the First Amendment and it’s religious freedom protections. I live near a large plain people population in Central PA near Geisinger Danville noted in this article. They pay for what they consume but do not believe in insurance or “mooching” off government programs. I could cite numerous immoral things done by mainline “churches”, in particular, but the Amish faith lives morality.

  • Unfortunately the gene pool is too small in the Amish community, so lots of recessive genes/abnormalities are coming to the fore.

  • Religion has not been helpful here.

    First it led the group into becoming an highly inbred community with severe rates of genetic diseases.

    Then it prohibited the most logical and useful paths to treatment.

  • The thing is, they want to do this their way. Not through Medicaid but around it, so to say. At some point they will be forced to choose between their extreme apartness from the society in which they’re emedded and the welfare of their children. My guess is the innocent kids will pay a terrible price for their elders’ beliefs which in my view will be immoral. Accepting help is not.

    • @Seth

      You are absolutely correct, Seth.

      It’s perfectly fine for any adult to say, “It’s against my religion to do certain things, so I accept going blind.”

      But it’s another matter entirely to say to a child, “It’s against my religion to do certain things, so I accept YOUR going blind.”

      What? Wait!

      I live in Boston, the location of the Christian Science mother church, and we occasionally put on trial Christian Scientists who have allowed their child to die because of the parents’ religious sentiments — even, in some instances, parents who receive medical treatments for themselves.

  • I am an MD geneticist in Canada and diagnose and treat children with rare and ultrarare genetic disorders. I found your article excellent and very informative — we face similar challenges in Canada Re reimbursement of drugs for rare and ultrarare disorders- the issues are diffrentbtgag the American scene, but equally heart-wrenching and complicated- solutions are there and the manufacturers no doubt are trying and do creatively work with our governments and private insurers in Canada to find solutions. One of the main stumbling blocks remain the rarity of these disorders and the lack of iron-clad evidence that the new therapies work or how do you know a therapy will work. We in the medical profession participating and doing clinical trials are trying to generate evidence so more drugs can get approved but many families and health care providers are not patient nor objective enough while waiting for ironclad evidence of
    effectiveness. Thank you.

  • “It’s not really fair, ethically or legally, to ask these people to break that covenant with their church and the federal government in order to pay for these extraordinarily priced therapies,” Dr. Kevin Strauss.

    sadly, there’s no such thing as fairness. is it fair these poor children are going blind through no fault of their own?

  • Great article. So frustrating. Let me ask, why does the Amish community have these genetic anomalies ? Is the incidence statistically greater than general population ?

    • Actually, yes. Because of such close intermarriage the Old Order Amish have a lot of Ellis-vanCreveld which causes polydactyly and achondroplasia (dwarfism), SCID, congenital heart disease. Dr. Holmes Morton at Harvard did a lot of research on it.

    • Chiming in from a scientific background and also a familiarity with isolated Mennonite communities in Kansas — I think genetic anomalies are more prevalent in the Amish, Old Order Mennonites, etc. because these groups because you tend to get somewhat of a “founder effect.” People within these communities have largely descended from relatively small anabaptist communities in Europe, and they often stay within small pockets in the US and usually are only allowed to marry those within their faith tradition. This results in a small gene pool without a lot of genetic diversity, increasing the chance for genetic mutations to occur. I don’t have anything solid to point to saying that their rates of genetic anomalies are higher than the general population, but I would suspect that they are. You would likely find this in other isolated communities that tend to marry within a relatively small population as well.

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