Pub­lic cash sought for $1b drug

A drug of­fers hope for free – for now, write Jac­ques Steenkamp and Imo­gen Neale.

Sunday Star-Times - - NEWS -

A drug com­pany de­mand­ing $1 mil­lion tax­payer fund­ing to treat New Zealand chil­dren with a rare ge­netic dis­or­der has al­ready been re­buffed by over­seas au­thor­i­ties, which ac­cused the com­pany of un­eth­i­cal pric­ing.

Amer­i­can com­pany Bio­gen has been sup­port­ing the Muscular Dys­tro­phy As­so­ci­a­tion of New Zealand (MDANZ) and is of­fer­ing free med­i­ca­tion to chil­dren with spinal muscular at­ro­phy (SMA) as it tries to get its drug Spin­raza pub­licly funded. That free med­i­ca­tion is likely to last only un­til Phar­mac’s rare dis­or­ders sub­com­mit­tee de­cides whether they will fund Spin­raza in New Zealand; in the UK, Bio­gen pulled free tri­als af­ter be­ing re­fused NHS fund­ing. The Phar­mac sub­com­mit­tee met this week to con­sider 13 fund­ing ap­pli­ca­tions, in­clud­ing Spin­raza.

Bio­gen, which made US$12.3 bil­lion in rev­enues last year, re­cently closed its Ex­panded Ac­cess Pro­gramme (EAP) to treat SMA type 1 pa­tients in Eng­land, Wales and North­ern Ire­land af­ter the NHS de­clined fund­ing for the drug. An EAP, sim­i­lar to the one that Bio­gen has dropped in the UK, has been run­ning at Star­ship Hospi­tal in Auck­land this year. A hand­ful of chil­dren have been re­ceiv­ing Spin­raza for free. It’s es­ti­mated that the re­tail cost is US$750,000 (NZ$1.14 mil­lion) for the first year fol­lowed by four­monthly in­jec­tions priced at NZ$135,000 per in­jec­tion.

In Oc­to­ber 2017, the au­thor­i­ties in Den­mark re­fused some Spin­raza treat­ment be­cause of its ‘‘un­rea­son­ably high price’’ com­pared to the clin­i­cal ef­fect, and the same month Nor­we­gian au­thor­i­ties re­jected the fund­ing be­cause the price of the medicine was ‘‘un­eth­i­cally high’’.

Star­ship Hospi­tal spokesper­son Sarah McMahon said the hospi­tal was treat­ing a small num­ber of chil­dren who were di­ag­nosed be­fore the EAP was es­tab­lished but ‘‘since we be­gan the EAP ear­lier this year, we have not seen any newly di­ag­nosed cases of type 1 SMA’’.

This news came as a shock to Lisa Ged­des from Taupo, whose two-year-old son Tama was di­ag­nosed with type 1 SMA sev­eral months ago and was told he wasn’t el­i­gi­ble for treat­ment. ‘‘Ev­ery­thing sounded re­ally promis­ing. And then we got told, not so long ago, that be­cause he has sat up un­sup­ported, in his life, that he was de­nied the treat­ment,’’ she said. ‘‘I just lost it. I just broke down and just said, this was the hope that we were hold­ing on to and that Tama – as well as so many – des­per­ately needs. To be told he was de­clined for it was just devastating.’’

Bio­gen’s Fiona Ti­gar has con­firmed the com­pany is pro­vid­ing Spin­raza ‘‘free-of-charge’’ to Star­ship Hospi­tal. How­ever, she said it was up to Star­ship’s clin­i­cians to de­cide whether both newly di­ag­nosed pa­tients and those liv­ing with the dis­ease were el­i­gi­ble for the treat­ment. ‘‘Bio­gen has not made any changes to the cri­te­ria for the pro­gram.’’

Ti­gar said the pric­ing of Spin­raza was com­plex and needed to re­flect the ‘‘highly spe­cialised na­ture of the prod­uct and the enor­mous in­vest­ment Bio­gen has made ... dur­ing prod­uct de­vel­op­ment’’.

‘‘We have made a sub­mis­sion to Phar­mac for re­im­burse­ment and will work closely with them to de­ter­mine an ap­pro­pri­ate ... We are com­mit­ted to tack­ling the chal­lenges of neu­ro­science medicine but need to re­coup our in­vest­ment,’’ Ti­gar said.

How­ever, US char­ity Cure SMA pro­vided the ini­tial seed money for the drug in 2003 be­fore Bio­gen’s in­volve­ment in 2012.

It was re­ported in Oc­to­ber that global sales of Spin­raza sur­passed US$1 bil­lion in an­nual rev­enue.

Mean­while, ques­tions have also been asked about the re­la­tion­ship be­tween MDANZ and Bio­gen be­cause of MDANZ’s re­peated calls for Phar­mac to fund Spin­raza. MDANZ chief ex­ec­u­tive Ronelle Baker said the re­la­tion­ship with Bio­gen is ‘‘one of many that we have with com­mer­cial or non-profit groups’’.

‘‘We have a pos­i­tive re­la­tion­ship with Bio­gen, and cur­rently a shared in­ter­est in ad­vanc­ing the well-be­ing of peo­ple with SMA – at present, this hap­pens to in­volve ac­cess to the first ever, es­tab­lished treat­ment for SMA (Nusin­ersen/Spin­raza).’’

She con­firmed that their SMA mem­ber’s ref­er­ence group has de­ter­mined ac­cess to Spin­raza as a pri­or­ity.

‘‘Our mem­bers are fully aware of the ap­pli­ca­tion made by Bio­gen for fund­ing re­im­burse­ment ... How­ever, we don’t con­trol in­di­vid­u­als and what they de­cide to do is com­pletely up to them.’’

Bio­gen did not re­spond to ques­tions on whether they be­lieve it’s eth­i­cal to do­nate money to an or­gan­i­sa­tion (MDANZ) whose mem­bers are pub­licly cam­paign­ing for their drug to be funded in New Zealand.

This was the hope that we were hold­ing on to and that Tama – as well as so many – des­per­ately needs.


Lisa Ged­des and her two-year-old son Tama Hub­bard who has SMA type 1.

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