Access to care for PNH patients

The media this morning, carried considerable coverage around access to Soliris, a drug  used to treat a rare and unpleasant blood condition paroxysmal nocturnal haemoglobinurea (PNH).

PNH is a very challenging blood condition, which cannot be prevented and brings with it significant loss to length and quality of life.

It concerns us that at present there are no effective treatment options available for New Zealand PNH patients. These patients are often younger, clearly a solution needs to be arrived at urgently to meet their needs, and we certainly support access to this medicine.

Frankly, there is an on-going challenge in New Zealand with the current PHARMAC assessment of high cost and highly specialised medicines. This really hits hardest when the conditions are rare, as with PNH. Each and of themselves the numbers may be small, but collectively, large numbers of people are affected and we don’t have a system that is catering well to support them. Let’s also remember these are not preventable diseases, the causes are unknown and by no fault of their own patients find themselves diagnosed with something rare, difficult and expensive to treat.

Achieving reimbursement for these medicines is problematic pharmaco-economically under the current PHARMAC process, they simply will never look cost effective compared to cheaper treatments for other conditions.  Treatment for a number of blood conditions and blood cancers is becoming increasingly specialised, personalised and yes, expensive.

However, these treatments are becoming more targeted and increasing in efficacy, meaning that only those who will gain real benefit will be treated, as in the case of patients with PNH. What is rarely mentioned are the considerable downstream costs of not treating, this likely leads to significant morbidity, blood transfusions and hospital admissions.

In addition to requiring access to this particular drug, access to drugs for rare conditions are a growing issue in New Zealand, and something that as an organisation Leukaemia & Blood Cancer New Zealand will keep engaged with.

Like others, we would welcome meaningful movement from both sides of the negotiating table to bring this drug to patients.

Please see below for media coverage on the issue below.

TVNZ Breakfast
Firstline
New Zealand Herald

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