2019-08-26 Bloomberg Businessweek

(Frankie) #1
◼ TECHNOLOGY Bloomberg Businessweek August 26, 2019

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milestoneswouldcomewithtime.Theydidn’t.
Aftera misdiagnosis,monthsoftherapy,and
scoresoftestsandprocedures,thefamilywas
toldElianahadSMAinJune.Whatshedidn’thave
wastime.Zolgensmaisa potentialcure,butit’s
approvedonlyforchildrenunderage2,beforeirre-
versibleharmis done.Eliana’ssecondbirthdaywas
fiveweeksaway.Afterinsurancerefusedtocover
thetreatment,ShaniandAriel,a rabbi,turnedto
theChesedFund,a crowdfundingorganization
fortheOrthodoxJewishcommunity.
“Asa mother,I havetodoeverythingpossibleto
helpmydaughter,”Shanisaidina videothataccom-
paniedthefamily’sfundraisingplea.“Everything,
evenif it makesmeuncomfortable.”
Sotheyreachedouttostrangers,andit worked.
Morethan23,000peoplefromtheU.S.,U.K.,and
IsraeldonatedforEliana’streatment,mainlysmall
amountsrangingfrom$5to$180.Oneanonymous
donorpledged$285,000tobringthecampaignover
the$2.2milliontarget.It onlytookfivedays.
“Idon’tknowwhatthefuturewillbringfor
her,”Shanisaysinthevideo.“Idon’tknowwhat
machines,whattubes,whatanything.AllI knowis
thatI wantmydaughtertohaveanopportunityto
havea normallife,justlikeeveryotherkid.”
ElianareceivedZolgensmaonJuly19,theday
afterhersecondbirthday.

IMMIGRATING
PortfoliomanagerRajdeepPatgirihasbeenona
crusadeforhisdaughter,Tora.Shewasbornin
theU.K.inSeptember2018.ByJanuaryheandhis
wife,TaisiyaUsova,a stay-at-homemother,noticed
thatshedidn’thavemuchmovement.Hesearched
“babydoesn’tmoveherleg”ontheinternetand
thefirstfewanswerspointedtoSMA.HetookTora
totheemergencyroom,butthedoctorsaidit was
likelya developmentaldelayandsentthemhome.
Patgirikeptpursuingit,andtwomonthslatera
neurologistgavehimthedreadedSMAconfirma-
tion.Therewerenoapprovedtreatmentsforthe
conditionintheU.K.,soheaskedhisemployerif he
couldmovehisfamilytotheU.S.togetToraintoa
Zolgensmastudy.Fiveweekslater,herelocatedto
hiscompany’sNewYorkoffice,thoughhe’sbeen
workingremotelywhilethefamilyhasfocusedon
gettingToratreatmentinColumbus,Ohio.
Battling insurance wasn’t his initial plan.
PatgiritriedtogetZolgensmathroughNovartis’s
compassionate-access program, which makes
experimental drugs available for free while they’re
awaiting regulatory approval. But that was closed to
them because he wasn’t a U.S. citizen or green-card
holder. He applied for Zolgensma in June through

his UnitedHealthcare insurance plan and was
denied within days, because the insurer covered
the drug only for children younger than 6 months.
There was a wrinkle. On June 25, the day
UnitedHealthcaresentPatgirithedenial,it alsopub-
lishednewguidelinesexpandinguseofthedrugto
childrenupto 2 years old. “I actually saw the new
guidelines first, and two hours later I saw the denial,
which didn’t make sense,” he says. The insurer told
him Tora’s case reviewer didn’t have the authority
to approve her treatment. So he shared their story
withtheWashingtonPostandothermediawhilecon-
tinuingtheappealsprocess.A weekafterthestory’s
publicationinnewspapers, Tora was approved. The
family plans to move to New York in January.

COMPASSIONATE ACCESS
Sara Harlan has survivor’s guilt. The social worker
is in the SMA Facebook groups, follows the news,
and knows how much trouble some parents have
had getting Zolgensma. For her daughter, Lucy,
things weren’t quite so complicated, and she says
that weighs on her. “I have a really, really hard time
knowing how easy it was to get this stuff,” she says.
Lucy didn’t move much when she was born in
Louisville in April 2018. She was diagnosed with SMA
when she was 10 weeks old. She started on Spinraza
within days and almost immediately started kicking
her legs. “That was like hitting the lottery,” Harlan
recalls. “We just screamed.” But the doctor had told
her about Zolgensma, and she and her husband,
Danny, an accountant for health insurer Humana,
set their minds on gene therapy. The clinical trials

◀ Moore with Margaux
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