BMJ 2017;358:j4302 doi: 10.1136/bmj.j4302 (Published 2017 September 15)
Page 1 of 1
News
NEWS US drug maker sues to force Ireland to offer expensive Duchenne’s treatment Owen Dyer Montreal
The American pharmaceutical firm PTC Therapeutics is suing Ireland’s Health Service Executive (HSE) over its decision not to fund drugs for two boys with Duchenne muscular dystrophy (DMD).
affordability of new medicines is a challenge because of the extraordinary prices proposed by drug manufacturers. The HSE must have regard to its obligations to the wider population of 4.7 million people.”
PTC’s drug, ataluren (Translarna), has been approved by the European Medicines Agency (EMA) to treat the nonsense mutation form of DMD in children aged over 5. This condition affects two boys over 5 in the Republic of Ireland and one in Northern Ireland. The boy in Northern Ireland is receiving the drug and has benefited, according to his parents. The two boys in the Republic have not.
The HSE was advised by the National Centre for Pharmacoeconomics not to fund the drug, based on an estimated cost of €414 000 (£365 000; $495 000) per child per year. The maker said that its final offer was lower than that based on a “confidential discount.”
Ataluren is not a cure for DMD but can delay the onset of paralysis of the legs, keeping children out of wheelchairs for several years.
“We feel we have no option on behalf of the two boys but to appeal the HSE’s decision,” Adrian Haigh, PTC’s European director, told the Irish Times. He noted that the legal action would probably cost more than the company stood to gain or the government stood to lose if the drug were funded.
Declan O’Rourke, consultant paediatric neurologist at Temple Street Children’s University Hospital, told the Irish Times: “The families involved are distressed. It is a time sensitive matter. Once their boys can no longer walk, they cannot avail of the treatment.”
The HSE was wrong to consider the US Food and Drug Administration’s rejection of ataluren, the company said, when Ireland’s regulator is the European Medicines Agency. PTC also argued that HSE failed to involve patients and clinicians in its decision to the extent that other countries have done.
Richard Lodge, chief executive of Muscular Dystrophy Ireland, told thejournal.ie that over 400 children in 22 European countries are now receiving the drug, 80% of the EU’s eligible patients. Any savings from not funding the drug would be mostly swallowed up by the cost of caring for paralysed children, he said.
Emotionally charged hearings were widely credited with pushing the FDA to approve an equally expensive DMD drug last year after its expert panel had urged rejection.1
In a letter to parents, the HSE’s primary care national director John Hennessy wrote: “I know this is a difficult time for patients with Duchenne muscular dystrophy, but for the HSE
For personal use only: See rights and reprints http://www.bmj.com/permissions
1
Dyer O. Muscular dystrophy drug looks set for commercial success despite clinical doubts. BMJ 2016;358:i5346. doi:10.1136/bmj.i5346 pmid:27698204.
Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/ permissions
Subscribe: http://www.bmj.com/subscribe
Page 1 of 1
News
NEWS US drug maker sues to force Ireland to offer expensive Duchenne’s treatment Owen Dyer Montreal
The American pharmaceutical firm PTC Therapeutics is suing Ireland’s Health Service Executive (HSE) over its decision not to fund drugs for two boys with Duchenne muscular dystrophy (DMD).
affordability of new medicines is a challenge because of the extraordinary prices proposed by drug manufacturers. The HSE must have regard to its obligations to the wider population of 4.7 million people.”
PTC’s drug, ataluren (Translarna), has been approved by the European Medicines Agency (EMA) to treat the nonsense mutation form of DMD in children aged over 5. This condition affects two boys over 5 in the Republic of Ireland and one in Northern Ireland. The boy in Northern Ireland is receiving the drug and has benefited, according to his parents. The two boys in the Republic have not.
The HSE was advised by the National Centre for Pharmacoeconomics not to fund the drug, based on an estimated cost of €414 000 (£365 000; $495 000) per child per year. The maker said that its final offer was lower than that based on a “confidential discount.”
Ataluren is not a cure for DMD but can delay the onset of paralysis of the legs, keeping children out of wheelchairs for several years.
“We feel we have no option on behalf of the two boys but to appeal the HSE’s decision,” Adrian Haigh, PTC’s European director, told the Irish Times. He noted that the legal action would probably cost more than the company stood to gain or the government stood to lose if the drug were funded.
Declan O’Rourke, consultant paediatric neurologist at Temple Street Children’s University Hospital, told the Irish Times: “The families involved are distressed. It is a time sensitive matter. Once their boys can no longer walk, they cannot avail of the treatment.”
The HSE was wrong to consider the US Food and Drug Administration’s rejection of ataluren, the company said, when Ireland’s regulator is the European Medicines Agency. PTC also argued that HSE failed to involve patients and clinicians in its decision to the extent that other countries have done.
Richard Lodge, chief executive of Muscular Dystrophy Ireland, told thejournal.ie that over 400 children in 22 European countries are now receiving the drug, 80% of the EU’s eligible patients. Any savings from not funding the drug would be mostly swallowed up by the cost of caring for paralysed children, he said.
Emotionally charged hearings were widely credited with pushing the FDA to approve an equally expensive DMD drug last year after its expert panel had urged rejection.1
In a letter to parents, the HSE’s primary care national director John Hennessy wrote: “I know this is a difficult time for patients with Duchenne muscular dystrophy, but for the HSE
For personal use only: See rights and reprints http://www.bmj.com/permissions
1
Dyer O. Muscular dystrophy drug looks set for commercial success despite clinical doubts. BMJ 2016;358:i5346. doi:10.1136/bmj.i5346 pmid:27698204.
Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/ permissions
Subscribe: http://www.bmj.com/subscribe
