Advocacy

Our plea for life

The aPSGA, which represents two-in-one million Australians battling aHUS – an ultra-rare, life-threatening blood disease that claims the lives of 10 per cent of patients within the first year of diagnosis if untreated1,2 – is appealing to the Government to save the lives of our small but important community.

Soliris (eculizumab) – the only treatment available and clinically proven to prevent premature death and vital organ damage in those with aHUS3 – holds the key to our survival.

According to Associate Professor David Mudge, Consultant Nephrologist at Princess Alexandra Hospital who runs a national aHUS patient registry, Brisbane, more than two-thirds of aHUS patients die, require dialysis or develop permanent kidney damage within one year of diagnosis,while 32 per cent die within four years.1,2

“Soliris is the only treatment available for aHUS shown to prevent patients from dying from this fatal disease.

“Without treatment, approximately one-in-10 patients will die within the first year of diagnosis, and almost all surviving patients will experience damage to vital organs including the kidneys, heart and brain,” A/Prof Mudge said.

Soliris was approved for use by the Therapeutic Goods Administration (TGA) in October, 2012. However, patients in 30 countries — including the USA, countries throughout Europe, and Japan — have access to this life-saving therapy while Australia is lagging woefully behind.

Should an expert panel (the Pharmaceutical Benefits Advisory Committee) advise the Federal Minister against funding Soliris through the Life Saving Drugs Program (LSDP) when it meets on March 11-14, 2014, this stroke of the pen will seal the fate of our small network living with this fatal disease.

We are calling on the Government to follow the lead of more than 40 countries worldwide, by funding Soliris through the LSDP. While this treatment is currently available, its cost is beyond reach of every Australian living with, or caring for someone with aHUS.

More than 21,000 patients, aPSGA members and friends have signed a petition urging the PBAC to approve Soliris for Government funding at the March 2014 PBAC review, following its failure to recommend Soliris for funding in 2013.

Australian aHUS registries report a 17 per cent annual mortality rate for people living with aHUS.1

The majority of aHUS patients are being treated with supportive therapies which are ineffective in the long-term, life-disrupting and more often than not, fail to stop the disease from progressing. Current supportive care options for aHUS comprise plasma exchange, kidney dialysis and on rare occasions, organ transplantation.

According to A/Prof Mudge, “More than one-third of patients with aHUS will lose their kidneys after their first presentation and require chronic dialysis. For those whose kidneys retain some function, they are faced with often daily or several-times-a-week plasma transfusions. However, neither dialysis nor plasma exchange treat the underlying problem which causes aHUS.

“In addition, aHUS patients are extremely unlikely to be considered for organ transplantation due to the nature of their disease, which carries a 50-80 per cent risk of disease recurrence leading to the loss of the transplanted organ,” A/Prof Mudge said.

International research has shown supportive treatment options are clinically inadequate, life-disrupting and pose significant safety risks to patients.5 Plasma exchange is poorly tolerated and associated with major complications and death in up to 26 per cent of patients, while up to 54 per cent of patients die despite dialysis treatment within five years.5

The rarity of a disease is insufficient reason for the Government to not fund the only treatment available for aHUS. Without this treatment, Australians living with aHUS will die.

Evidence

Clinical trials have shown Soliris prevents aHUS from progressing, restores kidney and major organ function, and enables patients to receive organ transplants and return to normal, healthy lives.6,7,8,9

Those aHUS patients who are living end-stage renal disease and are dialysis dependent, will only be considered for a kidney transplant if treated with Soliris. Their overall survival and quality of life will improve if they can undergo a kidney transplant. Soliris is their last option.

Ten Australians living with aHUS have been granted temporary access to the treatment through the manufacturer’s compassionate use scheme. All Australians on Soliris have shown significant improvement in kidney function and overall health, without further need for plasma exchange or dialysis.

Kerri Grey’s son, Ashley, was Australia’s first aHUS patient to be granted compassionate access to Soliris.

“Soliris has saved my child’s life. Without access to Soliris, my beautiful baby boy would have died,” Kerri said.

Should the Government elect not to fund Soliris through the LSDP, Ashley and others receiving compassionate supply of Soliris will have no assurance of ongoing access; patients on dialysis will have no hope for transplant; while others experiencing multiple organ failure, will remain uncertain of what tomorrow holds.

About the LSDP

The LSDP is a Federal Government scheme dedicated to providing patients living with ultra-rare and life-threatening diseases with subsidised access to costly, life-saving treatments like Soliris.

The LSDP currently funds 10 medications for seven different disease states at a cost representing less than two per cent of the national medicines bill. Soliris is funded through the LSDP for another ultra-rare, life-threatening blood disorder – Paroxysmal Nocturnal Haemoglobinuria (PNH).

Funding Soliris

The cost to fund Soliris for aHUS would be comparable to other treatments currently funded through the LSDP.10

If funded through the LSDP, Soliris, would cost the Australian tax paper $2.22 per year.

References

References

1. Minkowski et al. Atypical Haemolytic Uraemic Syndrome. Update on the Complement System and What is New. Nephron Clin Practice. 2010: 114;C219-C235.

2. Loriat C, Fremeaux-Bacchi V. Atypical Haemolytic Uraemic Syndrome. Orphan J Rare Disease. 2001: 6; 1-20.

3. Vilalta R et al. Eculizumab Therapy for Paediatric Patients with Atypical Haemolytic Uraemic Syndrome: Efficacy and Safety Outcomes of a Retrospective Study. Haematologica: 2012: 97(suppl 1); 479.

4. The aHUS Patient Support Group 2014. http://apsga.org.au

5. Noris M, Renuzzi G. Atypical Haemolytic Uraemic Syndrome. N Engl J Med. 2009: 361; 1676-1687.

6. Greenbaum et al. Eculizumab Inhibits Thrombotic Microangiopathy and Improves Renal Function in Paediatric aHUS Patients. ASN Abstract 2013: Abstract SA-PO849.

7. Fakhouri et al. Eculizumab Inhibits Thrombotic Microangiopathy and Improves Renal Function in Adult aHUS Patients. ASN Abstract 2013: Abstract FR-OR057.

8. Delmas et al. Eculizumab in aHUS Patients with Long Disease Duration and Chronic Kidney Disease. Sustained Efficacy at Three Years. ASN Abstract 2013: Abstract FR-PO536

9. Gaber et al. Eculizumab Maintains Efficacy in aHUS Patients with Progressing Thrombotic Microangiopathy: Three Year Update. ASN Abstract 2013: Abstract SA-PO852.

10. PBAC Public Summary Document, July 2010.

http://www.health.gov.au/internet/main/publishing.nsf/Content/14A6BBF12A5384ADCA257BF000209D3D/$File/Eculizumab%20SOLIRIS%20Alexion.pdf

Get involved

Become an aHUS community advocate

Our aPSGA community is seeking urgent assistance from Members of Parliament and communities nation-wide to appeal to the PBAC and ultimately, the Federal Health Minister, to fund Soliris for those whose lives ultimately depend on the life-saving treatment.

To lend your support to our campaign, please:

  1. Contact your local MP;
  2. Contact us to subscribe to our news updates;
  3. Follow and support us via Facebook, Twitter, Linked In, Youtube
  4. Watch and share our videos on YouTube;
  5. Share our story.

The role of your local MP is to act as a voice of support. Please contact your local MP to inform them that you support aHUS patients and their families in their endeavours to secure urgent access to the life-saving treatment, Soliris.

If you are living with, or have a family member or friend affected by aHUS, please organise to meet face-to-face with your local MP to relay the dire predicament that our small community is currently facing.

Member of Parliament contacts list

Electorate boundaries

One thought on “Advocacy

  1. Pingback: Curious About the Hashtag #saveaHUSlives? Don’t Worry, We’ve Got You Covered.

Comments are closed.