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…

Take a few minutes to read the PHARMAC request for public input (it’s short), the LDNZ (Lysosomal Diseases New Zealand) backgrounder and their detailed letter to PHARMAC, and the MLD Foundation letter to PHARMAC (text also below).  Take 3-4 minutes to write your own email, letter or FAX and get it to PHARMAC by the 31st (and remember that their 31st is the 30th for most of us!)

On behalf of all of us with rare diseases – Thank You!

MLD Foundation
21345 Miles Drive
West Linn, OR  97068-2878
+1 503-656-4808
FAX: +1 503-212-0159
[email protected]
A registered 501(c)(3) non-profit serving families worldwide


July, 21 2013

Sue Anne Yee

Therapeutic Group Manager
PO Box 10 254, Wellington 6143
[email protected]
Fax: 011 64 4 460 4995 

Dear Ms. Yee,

The MLD Foundation supports and provides a voice to families around the world with MLD, a rare terminal neurometabolic lysosomal disease that most often affects infants at 18-24 months. Our tenants are encapsulated in our motto: We C.A.R.E.Compassion for families, increasing Awareness, influencing & funding Research, and expanding Education for metachromatic leukodystrophy.

On behalf of the MLD patients and families in New Zealand we are opposed to PHARMAC’s proposed denial of access to eculizumab (Soliris) for the treatment of paroxysmal nocturnal haemoglobinuria, another rare disease.

We are shocked to read PHARMAC’s statement  “This proposal to decline the funding application is consistent with the clinical advice we have received, which recommended that the application be declined because although it is an effective treatment, it is extremely expensive.”

By your own analysis, the clinical conclusion is that eculizumab  “is an effective treatment.”  PHARMAC has a responsibility for “health outcomes that are reasonably achievable” and the patients have a right to access effective therapies which your experts have determined to be the case with eculizumab.

PHARMAC’s responsibility is to facilitate access to viable therapies in as cost effective manner as possible.  Alexion, like every other rare disease therapy company, has numerous fiscal programs to assist those countries unable to provide fully for families that need access to their therapies. PHARMAC knows this yet relies on list prices to argue for denial. New Zealand’s claim that they are a small country and cannot afford these relatively expensive therapies is offset by your smaller populations and resulting lower actual count of affected individuals.  The prevalence of rare disease in New Zealand is no higher or lower than any other developed country so your fiscal challenges and access to tax funds scale exactly as they do here in the United States, Japan, Europe, or even Australia. Or is PHARMAC arguing that New Zealand arguing, based solely on population, that they are the fiscally the same as other similar populated countries under-developed countries like Central African Republic, Congo, Liberia.  We think not.  You, like every other developed country are wrestling with balancing budgets and setting priorities.

PHARMAC has clearly stated that the proposed denial is one based only on cost. Just as a stool does not stand on one leg, basing life saving and quality of life treatment decision on only one parameter does not make sense.  Efficacy, accessibility to treatment, cost, quality of life, and impact on society of healthy productive individuals make a much more stable 5-legged stool.  In fact, when one leg is more challenging, such as the cost issue in this case, the other four legs can provide stability and balance. PHARMAC’s arguments about cost need to include total cost of not treating – both medical and loss of productivity to society, and they need to respect the rights of EVERY individual to be respected and equally cared for by society.

Why is an organization based in another country focused on another disease jumping in on this discussion?  Two reasons. 1) We hosted a MLD Family Conference™ in New Zealand in 2011.  We filled the meeting space at the Holiday Inn in Wellington with families of current and angel family members with MLD.  This was the first time ever to have a national gathering of MLD families in New Zealand.  Our meeting was very expensive to hold when travel expenses and time were considered, however, none of those families are less deserving than those we could get in our car and drive to. Supporting families with rare diseases requires these extraordinary efforts.  2) We, like PHARMAC and your District Health Boards, have an ethical and moral responsibility to, in a reasonable fashion, provide the services to families no matter where they live or their financial circumstances. That is why two of us, unpaid volunteers, literally traveled half way around the word to be with New Zealand families – and in adjacent years we went to Tokyo, Munich, and other cities outside the United States.  And it is why we are reaching out today … every patient matters, no matter their disease.  Less than 5% of the rare diseases have therapies – it is simply unacceptable that the handful of patients that can be treated might go without.

The number of patients with an individual rare disease is small and their individual voices may be soft, but rare disease patients have as much right to health services as those with chronic disease.  In the United States the total cost of rare disease therapies, and we are charged the full list prices that New Zealand will most likely not be charged, is but a drop in the bucket compared to what is spent on cholesterol lowering drugs.  Yes the prices are high, but the total cost impact is very low because the patient count is low. Taking drug prices out of context, and even extrapolating about patient prevalence must always be considered in context.

Further, please realize that the voice of the rare disease community is just beginning to be heard.  Just as HIV/AIDS activists organized some 40 years ago, we are doing the same in the rare disease community.  Worldwide, 1 in 10 people has one of the over 7,000 rare diseases. 1 in 10 is not something to be ignored, but consider that each of these patients has two parents, a neighbor, perhaps a teacher or a boss – that 4 or 5 in 10 that are directly impacted by rare disease.  Dismissing this community disease by disease is not a good choice.

Further, you should feel fortunate that LDNZ – Lysosomal Disease New Zealand has such talented and committed individuals in their leadership.  They literally travel the world to be informed about and influence research.  This is not your typical “I read it on the Internet” organization.  LDNZ is known, recognized, respected, active, connected, and contributing, not only on behalf of patients in New Zealand, but worldwide.  You would be wise to heed their perspective and knowledge.  We are in 100% full support of the detailed response LDNZ have provided to you with regard to the New Zealand specific issues surrounding this proposal.  We cannot speak to the intimacies of the Kiwi health system and society, but we can add a global voice to the discussion.

We DO NOT support PHARMAC’s proposed intent to decline access to eculizumab (Soliris) for the PNH group or other access for other rare disease patients where there are therapies.



Dean Suhr, President
MLD Foundation

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