Fund Treatment of Pompe Disease in Australia
- al: Vaughan Thomas
- destinatario: Australian Commonwealth Government Minister of Health
Pompe disease in adults causes wasting of muscles to the point where sufferers are unable to walk and require respirators to breathe. Treatment (Myozyme) is available ($400,000 per person per year) but the Government refuses to pay for it. Without the treatment the disease is eventually fatal. The Pharmaceutical Benefits Advisory Committee (PBAC) continues to reject applications for funding.
In 2010 the criteria for including any new drug on the Life Saving Drugs Program (LSDP) were changed. Since those changes came into effect no new drugs have been included on the LSDP. We believe that if the old criteria were used with the medical evidence currently available then Myozyme would be included on the LSDP. We want the criteria to be changed back to the pre 2010 position.
To the Minister of Health Canberra.
Minister. Pompe disease is eventually a fatal disease as it attacks all muscles including the heart and lungs. Treatment via infusion of a hormone is available. It costs the individual $400,000 per year. This treatment is funded for infants with the disease but not for adults. Without treatment patients require artificial respiration and cannot walk. The PBAC refuses to add the treatment Myozyme to the Pharmaceutical Benefits Scheme or the Life Saving Drugs Program. This refusal is ultimately a death sentence to adult-onset sufferers.
In 2010 the criteria for including any new drug on the Life Saving Drugs Program (LSDP) were changed. Since those changes came into effect no new drugs have been included on the LSDP. We believe that if the old criteria were used with the medical evidence currently available then Myozyme would be included on the LSDP. We respectfully request that the criteria be changed back to the pre 2010 position.
