Our 7 year old son Christian is the victim of government red tape.
We appeared on the Sunrise program on Monday 20 October. Below is the link to the story.
Just some background information about Christian and his fight for life.
Christian was diagnosed with an ultra-rare genetic condition called Hunter syndrome when he was only two weeks old. The condition attacks his entire body both physically and mentally. Life expectancy for him is around 14 to 15 years of age without treatment. I lost three brothers to the same condition so we are unfortunately well aware of its horrible effects.
There is no cure for this disease at the moment but there is a treatment called Elaprase which treats and stabilises the physical effects of the condition on the body but not the brain. Therefore the brain continues to progressively degenerate leading to dead whilst the body is stabilized.
Christian was receiving this life saving treatment for his body as part of the Federal Health Departments Life Saving Drugs Program. In January 2012 he was removed from the program for a period of about 18 months in which time my husband and I lobbied and fought to have his treatment reinstated. His treatment was reinstated in June 2013 and we then turned our attention to finding a treatment for his brain.
I learnt about a medical research being conducted in the USA and UK using a similar composition of the same drug to treat the brain with very positive results. In July 2013 we wrote to the bio-pharmaceutical company and encouraged them to consider Australia as part of their global clinical trial for phase II/III they were commencing in 2014 to 2016 to treat the disease in the brain. This is global trial involving 10 sites and 42 patients with patients having some cognitive impairment but not too severe. The bio-pharmaceutical company will fully sponsor and pay for all aspect of the clinical trial in Australia.
We have been corresponding with the bio-pharmaceutical company since our initial contact to understand the process in establishing a trial site in Australia. At the beginning there were a number of roadblocks to get over. One of which was working out the contractual side of things with Genzyme Australia who is the distributor of Elaprase within Australia.
In December 2013 the bio- company informed us that they would extend the trial to Australia and began looking at feasibility within Australia. Part of the feasibility studies was to locate a hospital that has the capacity to run the trial. With our initial letter to the pharmaceutical company we had a letter from one of the metabolic doctors that provided his support to be the lead investigator but unfortunately Westmead hospital either didn’t have the capacity or was not willing to run the trial for one reason or the other.
Given we were no longer living in Sydney we decided to focus our attention on our local hospital Princess Margaret. We wrote a letter to the medical director asking him why the feasibility assessment of his hospital was taking several months. When his response wasn’t adequate or positive we asked for a formal meeting. In that meeting he explained that the hospital currently doesn’t have the capacity but would be willing to have the trial at Princess Margaret once the new hospital currently under construction was completed. He assured us that he would be willing to facilitate the trial if it was only for Christian. He reassured us that the new hospital being built has capacity to undertake clinical trials in the future. The new hospital was about 15 months away from completion so we knew that would be too late for the trial.
We then began speaking with the Women and Children’s hospital Adelaide and got their support to run the trial. They then began the process of applying to have the trial at their hospital and since then we have been in constant communication with them. However, when they became concerned about the speed of which the trial feasibility was proceeding. We contacted the bio-pharmaceutical company and was told the trial site had to be selected by October 2014 so the trial could get on the way. They had now narrowed down their assessment to two hospitals. One will be selected as the clinical trial site. But couldn’t proceed any further as there was a government guideline precluding this from happening.
The guideline which states that patients participating in a clinical trial will no longer be eligible for subsidized Elaprase which treats the body. This treatment has already been approved and not under trial. This treatment we cannot afford as it costs about $300,000 a year. Furthermore, Elaprase is not available privately and is subsidized by governments all around the world where the trial is being studied with the exception of the USA.
We believe the guideline can be interpreted differently to facilitate the trial going ahead as the researchers are not looking to introduce a totally new drug for the same purpose which the guideline would perhaps be applicable in that case. Even though we believe the guideline was written in good faith to hopefully protect the safety of Australian patients participating in clinical trials. This guideline is also short-sighted, not taking into consideration the advancement in medical research for rare diseases. With the age of technology and social media parents like us are now able to gather credible information firsthand and are at the forefront of helping to find treatment for our son. Indeed there are times when parents have more information about particular treatment before doctors do and this was the case with this trial.
But unfortunately when we began to seek to have this trial in Australia for our son we not only face opposition from bureaucratic red tape but from some aspect of the medical fraternity that is very conservative and do not believe parents should be involved in finding treatment. We were certainly not about to leave our son situation and this trial in the hand of someone else to hopefully sort out.
In May 2014 we wrote to our local Federal MP and asked him to contact the Health Minister to intervene to remove this red tape from the Guideline. But after five months the Federal Health Minister has done absolutely nothing to resolve the issue and put the issue back to the bio-pharmaceutical company to resolve. They have stated this is a show stopper and unless the Government continues to subsidise the currently approved medicine Elaprase which treats the body they will not proceed with the trial to treat the brain in Australia. Hence Australia is about to shut the door on foreign investment into medical research due to red tape in the existing Health Department's Guideline.
We are extremely frustrated, Australia only has a very limited time (approx 1-2 months) before our son and other patients miss out on this global trial that is being fully sponsored and paid for by the bio-pharmaceutical company.
Our Son's life is at risk and without treatment he will succumb to the disease in his brain and ultimately die.
He has hope but this hope is being dashed by redtape. We would like to continue raising the profile of this issue and are seeking an urgent meeting with the Minister to ask him to remove this red tape and allow the trial to go ahead for our son and others.