WE NEED YOUR HELP
The Situation
PNH is a serious, very rare disease which affects people in their early adult life, drastically reducing both quality of life and life expectancy.
Paroxysmal Nocturnal Haemoglobinuria (PNH) is characterised by haemolysis, or the destruction of red blood cells. Excessive haemolysis in patients with PNH can lead to a variety of major health problems such as thromboses (formation of blood clots), pulmonary hypertension and damage or failure of organs such as the brain, liver, gastro-intestinal system and kidneys. PNH is extremely rare, with 22 diagnosed cases recorded on the New Zealand PNH patient registry. Of these approximately 8 are considered to have severe disease. There is no cure, but there is a life-saving treatment which is funded in more than 40 countries; Australia, Argentina, Brazil, Canada, Denmark, England, France, Germany, Ireland, Israel, Italy, Japan, Korea, Taiwan, Wales - to name just a few. That's 40 countries whose citizens have the reassurance that, if they are unlucky enough to develop PNH, they're lucky enough to have access to a treatment that can save their lives. PHARMAC, the NZ Government's medicines funding agency, has the power to provide access to the small number of people who desperately need this treatment. PHARMAC, in their own words, acknowledge that there is "no alternative" to the life-saving treatment Soliris (eculizumab) and that PNH patients have a "high clinical need." On Tuesday 21st of May PHARMAC abruptly walked away from negotiations with the supplier of Soliris and have launched a campaign to present PNH patients seeking access to a life saving treatment as an unwanted burden to the health system. PNHSANZ is appalled by PHARMAC's behaviour and ask that you express your support for PNH patients by making a submission to PHARMAC's proposal to decline funding for Soliris before 31 July. More details are available in the "What you can do" section on this page. PHARMAC have acknowledged that without the Soliris treatment, studies have shown PNH patients have a median survival of just 10 years. In their own words, PHARMAC have noted the "excellent evidence" for the effectiveness of Soliris in reducing the destructive processes of the PNH disease. PHARMAC have also accepted the "likely" survival gains offered by Soliris while simultaneously ignoring findings from the past decade of research which points to an average survival gain of 32.5 years. Most significantly, PHARMAC are claiming cost is the barrier to funding Soliris but have walked away from the negotiations which would secure a price for the treatment which NZ can afford. We simply cannot understand why PHARMAC are choosing to act in this way? The real costThe real cost of PHARMAC's proposal to decline funding for the Soliris treatment is the lost contributions of the New Zealanders affected by the PNH disease.
These are the people who, through no fault of their own, are struggling with a devastating disease and who are now being told by PHARMAC: your lives are not worth saving. 
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What you can doPlease download the below template and email submissions to PHARMAC before 31 July
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What you can do
Download this template...
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...and email submissions to PHARMAC before 31 July
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Submissions in response to the
Proposal to Decline Funding for Soliris (eculizumab) should be sent to: eculizumabfeedback@pharmac.govt.nz Submissions in response to the Decision Criteria consultation should be sent to: opp@pharmac.govt.nz |
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We need you to tell PHARMAC it is unacceptable to let people die when a life saving treatment is available.
To do this please use the above template and write an email submission in response to PHARMAC's proposal to decline funding for Soliris. We would also like you to send the same submission to PHARMAC's Decision Criteria review panel as rare disease patients should not be made to fight for access to new treatments. Submissions must be sent to PHARMAC before Wednesday 31 July. PLEASE DO IT NOW. Submissions in response to PHARMAC's Proposal to Decline Funding for Soliris (eculizumab) should be sent to: eculizumabfeedback@pharmac.govt.nz Submissions in response to PHARMAC's Decision Criteria consultation should be sent to: opp@pharmac.govt.nz |
Proposition Equity & Fairness
Treatments for rare disease are almost by definition, expensive. Research, clinical trials and regulatory approval costs hundreds of millions of dollars. The Soliris treatment is a breakthrough for medical science however the market for the treatment is a patient group which makes up around 0.0005% of the population.
Internationally, there is growing recognition of the need to assess treatments for rare disease in a way which fairly assesses the health gains offered and acknowledges the fact that per patient treatment costs are higher than for diseases which affect a greater number of people.
In New Zealand, PHARMAC have demonstrated an unwillingness to consider the principles of equity and fairness as they apply to patients affected by rare disease. We think this needs to change.
Proposition Equity & Fairness proposes that:
i) PHARMAC must return to the negotiating table with the supplier of the Soliris treatment
ii) PHARMAC must negotiate in good faith toward funding Soliris for a minimum of 8 NZ PNH patients
iii) PHARMAC must establish fair assessment criteria, based on expert advice from the international haematological community, to assess patient need for the Soliris treatment
iv) PHARMAC must amend its Operating Policies and Procedures to acknowledge the right of rare disease patients to access life restoring and life saving treatments as in the specific example of the Soliris treatment
Internationally, there is growing recognition of the need to assess treatments for rare disease in a way which fairly assesses the health gains offered and acknowledges the fact that per patient treatment costs are higher than for diseases which affect a greater number of people.
In New Zealand, PHARMAC have demonstrated an unwillingness to consider the principles of equity and fairness as they apply to patients affected by rare disease. We think this needs to change.
Proposition Equity & Fairness proposes that:
i) PHARMAC must return to the negotiating table with the supplier of the Soliris treatment
ii) PHARMAC must negotiate in good faith toward funding Soliris for a minimum of 8 NZ PNH patients
iii) PHARMAC must establish fair assessment criteria, based on expert advice from the international haematological community, to assess patient need for the Soliris treatment
iv) PHARMAC must amend its Operating Policies and Procedures to acknowledge the right of rare disease patients to access life restoring and life saving treatments as in the specific example of the Soliris treatment
Is this how a government agency should behave?
PNH patients, as with all New Zealanders, deserve to be treated fairly and with respect.
Not only have PHARMAC refused to release information in relation to the decision making process of the past 15 months, they have also failed to consult with PNH patients as part of this process. As a further show of contempt, PHARMAC neglected to provide the PNH Support Association with their proposal to decline funding for Soliris when it went out to consultation.
Having now had an opportunity to review the information released by PHARMAC, we are astounded at the extent to which PHARMAC have misrepresented the situation - keeping in mind that PHARMAC are bringing their considerable weight to bear against a vulnerable patient group contending with a serious disease.
Based on advice from the haematology community in New Zealand, the PNH patient registry and the advice to PHARMAC from their own haematology subcommittee, PNHSANZ estimate PHARMAC have inflated the estimated cost of Soliris to the New Zealand medicines budget by some $8 million dollars.
PNHSANZ are aware of a single PNH patient registry in New Zealand - this registry details 22 patients. Of these patients, 3 are receiving compassionate supply of the Soliris treatment. Based on the treatment assessment criteria of the Australian Life Saving Drugs program, an estimated 8 further NZ patients would qualify for treatment with Soliris.
PHARMAC, by contrast, calculate the estimated total cost of Soliris by simply claiming the existence of a "sub-group of 12-20 patients with more severe disease". No detail is provided for the basis of these numbers, and, given the sensitivity of this particular factor, PHARMAC would seem to have allowed an extraordinary margin of error from which to extrapolate costs.
Alongside inflated patient numbers, PHARMAC have presented an international "list" price for the Soliris treatment. This international list price is offset by discounts, rebates and other funding agreements negotiated by PHARMAC which, according to industry experts, routinely results in a true cost 20% or greater below the list price.
Clearly PHARMAC's intention is to prevent a balanced, informed and reasoned response to their proposal to decline funding for Soliris.
Not only have PHARMAC refused to release information in relation to the decision making process of the past 15 months, they have also failed to consult with PNH patients as part of this process. As a further show of contempt, PHARMAC neglected to provide the PNH Support Association with their proposal to decline funding for Soliris when it went out to consultation.
Having now had an opportunity to review the information released by PHARMAC, we are astounded at the extent to which PHARMAC have misrepresented the situation - keeping in mind that PHARMAC are bringing their considerable weight to bear against a vulnerable patient group contending with a serious disease.
Based on advice from the haematology community in New Zealand, the PNH patient registry and the advice to PHARMAC from their own haematology subcommittee, PNHSANZ estimate PHARMAC have inflated the estimated cost of Soliris to the New Zealand medicines budget by some $8 million dollars.
PNHSANZ are aware of a single PNH patient registry in New Zealand - this registry details 22 patients. Of these patients, 3 are receiving compassionate supply of the Soliris treatment. Based on the treatment assessment criteria of the Australian Life Saving Drugs program, an estimated 8 further NZ patients would qualify for treatment with Soliris.
PHARMAC, by contrast, calculate the estimated total cost of Soliris by simply claiming the existence of a "sub-group of 12-20 patients with more severe disease". No detail is provided for the basis of these numbers, and, given the sensitivity of this particular factor, PHARMAC would seem to have allowed an extraordinary margin of error from which to extrapolate costs.
Alongside inflated patient numbers, PHARMAC have presented an international "list" price for the Soliris treatment. This international list price is offset by discounts, rebates and other funding agreements negotiated by PHARMAC which, according to industry experts, routinely results in a true cost 20% or greater below the list price.
Clearly PHARMAC's intention is to prevent a balanced, informed and reasoned response to their proposal to decline funding for Soliris.
What PHARMAC have neglected to mention
Not only does the Soliris treatment restore quality of life and return life expectancy to expected norms, it has also been linked to increasing incidents of spontaneous remission in patients undergoing treatment.
While the specific cause of this effect is not fully understood - we do not claim Soliris is a cure for PNH - it is likely related to the increased survival prospects offered by the treatment.
Not only is this good news for patients, it is also in stark contrast to the claims made by PHARMAC that Soliris is a "life-long" treatment.
In fact, for a small but increasing number of patients, Soliris is simply a measure which allows the body to conduct its own healing processes and return to a disease free state.
While the specific cause of this effect is not fully understood - we do not claim Soliris is a cure for PNH - it is likely related to the increased survival prospects offered by the treatment.
Not only is this good news for patients, it is also in stark contrast to the claims made by PHARMAC that Soliris is a "life-long" treatment.
In fact, for a small but increasing number of patients, Soliris is simply a measure which allows the body to conduct its own healing processes and return to a disease free state.
PHARMAC: In their own words
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PTAC Meeting, 16 & 17 February 2012
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Haematology Subcommittee, 6 August 2012
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PTAC Meeting, 18 March 2013
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