Posts tagged ‘Pharmac’

January 24, 2013

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
New Zealand Herald

June 14, 2011

PHARMAC not always a perfect model for specialised drug funding

Nearly nine years ago, New Zealand’s drug-buying agency PHARMAC, gave the all-clear to fund Glivec (also known as imatinib).

The funding of the drug has given much hope to Kiwis diagnosed with CML (chronic myeloid leukaemia), with most patients able to achieve remission. The course of this disease was markedly different 10 years ago when other medications were the standard treatment. At that time, most patients progressed to the advanced phase of the disease and a large number died as a result of CML.

At the time, Glivec was hailed globally as a new era in drug development, the first in a new era of targeted new medicines – and all these years later that has been borne out. Nearly all these New Zealandpatients treated with Glivec are still leading active and productive lives.

Whilst we at the Leukaemia & Blood Foundation are very, very pleased that there has recently been several drugs funded for haematology patients, the approval process for funding has – as is usual – been very much delayed, commonly over a number of years.

A lot of respected voices have been heard in the media recently applauding the PHARMAC model. We agree it is certainly true that the agency serves New Zealand very well with regards to funding generic drugs; and the supply of drugs for a mass market with an eye to containing costs. We do, however, note that there is a far less rosy picture for many patients who require access to high cost and highly specialised medicines. This applies to thousands of cancer patients and others with chronic diseases. We need a system that can factor in providing access to these types of medicines as well.

We readily acknowledge there is a finite budget, and careful evaluation, drug by drug, is necessary. What we are struggling with is the review system that is deficient and fraught with delays, often for up to years on end.

Whilst our organisation has no agenda to see PHARMAC dismantled, we do believe the model lets New Zealanders down in the area of these highly targeted specialised medicines. Patients are often not well served in getting timely access to drugs in the more specialised fields, such as the one in which we operate.

We also remain concerned with the complete lack of transparency in the current funding model. We know first-hand the challenges in extreme delays with access.  In the intervening period it’s very difficult to get information with regards to the status of a particular drug’s funding status. We also see decisions are often selectively evidence-based.

The treatment for patients our foundation supports, many of whom have life-threatening illnesses, is obviously extremely time-critical and most can’t afford such delays.  There is also sometimes a false economy at play, in the case of haematology patients often the lack of access to a pharmaceutical treatment can frequently lead to even higher costs of supportive care in a hospital setting, such as intensive transfusion regimes with platelets, red blood cells, immunoglobulins and other treatments.

PHARMAC will shortly announce the results of a review into the Exceptional Circumstances scheme which is also part of the equation, and very much so for smaller patient populations. Let’s hope that a sense of fairness and equity can be brought to bear, first indications from the consultation were that this was not the case.

Nine years on from PHARMAC’s funding of Glivec, we reflect on the effectiveness of its funding model for a small population. Glivec was called the silver bullet and, for those diagnosed with CML, it certainly has been. We’d love to see this success replicated throughout the system.