John Murphy (Reid, Australian Labor Party) Share this | Link to this | Hansard source

Last Monday morning when I presented petitions in this House, one I presented, on behalf of the member for Deakin, was signed by 1,718 petitioners and concerned the urgent need to publicly fund the drug, Soliris, for sufferers of paroxysmal nocturnal hemoglobinuria, or PNH. Tonight I will present a further petition on behalf of 4,813 signatories in respect of the same matter and particularly on behalf of my constituent and principal petitioner, Ms Whitney Lane. These petitions have also been approved by the Standing Committee on Petitions.

Whitney is a very sweet, softly spoken, intelligent and articulate young lady. I had the great pleasure of meeting Whitney and her parents in a meeting earlier this year in my electorate office, and again here in Canberra last week, to discuss the very rare blood disease, PNH. Approximately 100 Australians suffer from this disease and Whitney is one of the youngest known sufferers. Whitney has just finished her final HSC exams and I wish her all the very best with her results. However, it is with great sadness that I report to the House that Whitney was unable to sit every exam because she was physically unable to do so.

In simple terms, PNH is the destruction of red blood cells and some white cells. Normal red blood cells contain a protective protein that prevents this occurring but in sufferers like Whitney this protective protein is absent. Extreme fatigue, chronic body pains and constant headaches are common symptoms. As the condition worsens, sufferers may experience clotting and major organ failure such as that of the liver, kidneys and lungs.

Unfortunately, science has been unable to tell us exactly why the disease starts or how we can cure it. The horrifying reality is that one in three people living with PNH will die within five years of being diagnosed if they do not receive the right treatment, and life expectancy without the drug is approximately 10 years. Soliris is the only known treatment for PNH and approximately 23 people are already receiving it under the manufacturer’s ‘compassionate program’, with around 30 other people awaiting treatment. The cost of the treatment is $480,000 a year and, obviously, this is simply beyond the reach of the average person. Further, once a patient begins treatment with Soliris, they must continue to receive it, every 12 to 16 days, for the rest of their life.

This is a treatment that requires serious consideration, not least because of the cost, but because of its ongoing requirements. Previously when the independent expert advisory committee, the Pharmaceutical Benefits Advisory Committee, or PBAC, considered a submission for the funding of Soliris through the Life Saving Drugs Program, or LSDP, the PBAC did recommend that the government consider it for inclusion under the LSDP. However, the PBAC also noted a number of uncertainties in the evidence about the drug, particularly the extent that Soliris extends life compared with the best supportive care, as well as the size of the benefit in reducing the incidence of blood clots, a major component of the disease.

In light of the doubts surrounding the effectiveness of Soliris I am pleased to note that the minister for health made an urgent request for further advice from the PBAC on the merits of Soliris for funding. This review enabled the PBAC to provide further advice to the government under the recently revised funding conditions and criteria for the LSDP. On 29 September 2010 the PBAC again recommended to the Australian government that it consider funding Soliris for PNH sufferers through the LSDP. In light of the recent PBAC recommendation I know that the minister is giving this very serious matter her utmost compassionate and expeditious attention.

Following the recommendation by the PBAC, the minister announced that there were several steps that must be completed before the government could consider the drug including negotiations with the sponsor as well as treatment, eligibility and guidelines. These are positive signs; however, no announcement has yet been made about the future of Soliris for PNH sufferers in Australia.

Whitney’s parents are keeping the faith but expressed to me the urgency of getting an answer from the government about the future funding of Soliris. They said that as each day passes, more harm is being done to Whitney’s health. I can only imagine how they must feel and I strongly urge the government to make a decision about the future of Soliris as soon as possible. Whitney is a very strong person, as is her family, and I know that she and other sufferers have much more to contribute to our community and our country, and I ask that the government provide the necessary support to ensure Whitney and others living with PNH have the opportunity to do so for many, many years to come. I therefore present the petition.

The petition read as follows—

To the Honourable The Speaker and Members of the House of Representatives

This petition of Australian Citizens, who are friends, family or supporters of Australian sufferers of Paroxysmal Nocturnal Haemoglobinuria (PNH) disease, a rare and potentially fatal disease of the blood draws to the attention of the House the need to publicly fund out of the Federal Government, the only currently available treatment for PNH sufferers, namely Soliris® (Eculizumab) as a matter of urgency.

We therefore ask the House to introduce and pass any legislation or to take any administrative action available to the House that will enable sufferers of PNH disease to urgently receive breakthrough life saving treatment for this very rare and debilitating disease.

from 4,813 citizens

Petition received.

Joanna Gash (Gilmore, Liberal Party) Share this | Link to this | Hansard source

I would like to concur with my colleague from the other side, who has just spoken, because I am going to speak about the very same subject. Jenny is a 29-year-old, is married, lives in Heathmont, Victoria, and some day wants to have children. Jenny also has a death sentence hanging over her head but with a single stroke of the pen, Jenny can be reprieved and go on to enjoy a long and full life.

Up until January this year, Jenny was employed as a weather forecaster with the Bureau of Meteorology. In January she collapsed and after extensive testing was diagnosed with an extremely rare condition, PNH, or paroxysmal nocturnal hemoglobinuria. It is a disease in which the body destroys its own red blood cells, eventually leading to organ failure due to the lack of oxygen being circulated.

Jenny’s life has been turned upside down. She is chronically breathless and has had to give up many things most of us take for granted. She has reduced her working hours, she cannot travel, and she has given up playing basketball. Even as we were speaking to her last week, we could hear she was running out of breath.

Her husband Luke is terrified. Just the other morning Jenny woke suddenly at about one o’clock with a violent episode of vomiting. Her husband had to get up, clean his wife up, change the sheets, clean up the mess and generally nurse her. Both are losing an enormous amount of sleep. Fortunately, her mother lives close by and comes to the house regularly to help clean. Jenny also has a supportive network of friends who rally to help her. Right now the only treatment available to her is to have blood transfusions every three to four weeks. Along with her health, her quality of life is slowly but surely diminishing. The point is that it does not have to be like this.

Last week I meet with Jenny and her colleagues from the PNH Support Association in my office here in parliament. Linda Charlton is the President of the PNH advocacy group and is also on the compassionate program with the drug Soliris. The group came to beg the government to sign off on the life-saving drug, Soliris, which will allow Jenny and about 50 others under the sentence of death from PNH to have a chance to live a long and full life. On that day, the group met with the Prime Minister and minister for health, who acknowledged the drug Soliris has been recommended for adoption by the PBAC for the Life Saving Drugs Program. On that day also, my colleague the member for Ryan, asked in question time:

Given that the government has wasted billions of dollars on the home insulation and school building schemes … why, 20 months after the PBAC first recommended listing of the drug, does it remain unlisted on the PBS?

The Prime Minister’s answer was disappointing. It was not incorrect but was lacking in real empathy because the core of her reply went to the fact that a price had not been set. And the Prime Minister said as justification for the rationale: ‘It happened every time under the Howard Government as well.’ What a pathetic and heartless statement that was. The Prime Minister seems oblivious to the fact of the highly shortened lifespan of PNH sufferers. The five- to 10-year survival span is not measured from the day of the question.

Indeed, two people have died since the Prime Minister first came to office three months previously. How many have died since the PBAC delivered their recommendation? And how many will die while they wait for this government to make up its mind? Surely the power is available to give interim approval and wear the cost differential. After all it is a matter of life and death and sometimes in the interests of compassion and humanity we have to put aside some of our dogmatic rules. I do not know what the cost of the drug is but I do know what the cost of doing nothing could be. Twenty months is just far too long to wait in this case and I appeal to the Prime Minister to put aside the rule book. Yes, there is a process but that should not be the be-all and end-all.

Linda Charlton, who led the deputation to the Prime Minister and minister for health, was gracious in her comments, describing the Prime Minister as ‘lovely’, and ‘she was fantastic’. Wouldn’t it be wonderful if the Prime Minister could show just how fantastic she was by approving the subsidy of Soliris. Measured against a life, it is a small price to pay. Flattery and soothing words just may not be enough.

In closing, I make mention of my constituent in Gilmore, John Beauchamp, who also has the disease. We are indebted to all who contacted me to try and assist him. I especially want to thank Glen Ellard of the South Coast Register for printing John’s story. As a result of the combined effort of everyone concerned, Alexion Pharmaceuticals has added John to their compassion program. I urge the government to sign the documents now so that Soliris can be made available to those whose lives are at risk. The process has been completed; it just needs the Prime Minister to sign off.

May I wish all in the House, the attendants, the Comcar drivers, the cleaners and the security staff all a wonderful Christmas and a safe and happy New Year. You are simply the best and we all appreciate you.