Say it’s not true … New Zealand proposes NO access to an “effective therapy” for a rare disease

Has my Sunday morning coffee not kicked in?  Am I reading this wrong? Tell me it’s not true …

  • Alexion has an “effective therapy” for paroxysmal nocturnal haemoglobinuria
  • New Zealand is proposing to decline access to therapy for its citizens  … not because it does not work – they acknowledge it is an “effective therapy” – rather, they think it’s too expensive.
  • Less than 5% of the over 7,000 rare diseases have therapies and they want to hold one of the few back !!!!!!????

Until this morning, I didn’t even know what paroxysmal nocturnal haemoglobinuria (Marchiafava-Micheli syndrome or PNH) was – all I need to know was they are fellow brothers and sisters with rare disease.  We must come to their support.

Enough already!  Well over 95% of the rare diseases are struggling to understand their disease and develop therapies … yet here we have a disease with a therapy that some bean counting bureaucrat says $$ x number of patients = too much.  What they conveniently ignore in their public stance is that Alexion, like all rare disease drug companies, will work with countries that need help to offset the list price because they care about the patient.  PHARMAC (New Zealand’s purchasing agency) you should care too!

We must not allow any more piecemeal incursions on the progress we are making towards therapies for all rare diseases.  If your disease is lucky enough to have a therapy in the future – the PNH struggle could be yours.  We must education and inform now!

So what can or should you do?  read on – it won’t take much time… Continue reading