Idursulfase IT trial & LSDP Australia

03/06/2014 16:51

We are very pleased to advise that all negotiations have been very positive with regards to issues raised to us by the biopharmaceutical company Shire in regards to the Intrathecal Idursulfase IT trial for Australia.

However we are unable to disclose confidential information and discussions about the trial which we feel could jeopardise goodwill. We just want to make it clear that we are not saying the trial is commencing, these issues take time and require careful negotiations and understanding of the issues. It is important that interesting stakeholders understand that as much interest as they might have in this trial, we believe it is unhelpful and unproductive to give out information prematurely. There is a lot of work that has been going on behind the scene since my husband and I commenced pioneering this trial nearly 12 months ago.  

However, three weeks ago, for more than one reason including the LSDP guidelines the trial was at risk of not going ahead in Australia. But we are now relief that we have managed to get the trial back on track.  We have been fighting to change the LDSP guidelines since we began fighting for our son nearly 2 years ago and there has been some welcoming changes.  However, there are still aspects of the guidelines that is stilll a problem for clinical trials in Australia but they are issues being worked on by others and that we are working on at the highest levels of government to resolve. It is not important to us whether the LSDP communicate with us directly on these issues or not. As far as we are concerned it is totally irrelevant who they communicate with. Indeed our only interest is to ensure that the issues are resolved and there is no unnecessary obstacle to the trial.  The fact remains there is a timeline for which the trial has to be established before Australia misses out and our only goal is to get the desired outcome of trial site in Australia .


 As parents our son desperately needs treatment. I know how deadly this condition is, I have experienced it firsthand. We could have sit back after the unprecedented decision was made to put Christian back on ERT but that’s not who we are. We have been given the very difficult challenge but also the privilege to fight for our son and in the process others. Whilst we do not take this privilege for granted it can be a challenge and unfortunately we cannot be all things to all people, We have to keep our focus on a single task.


It is also equally important that interested stakeholders understand that governments do not have the funds to invest into research and development especially for very rare conditions, as generally only a small number of the population is affected. We therefore need biopharmaceutical companies to invest their own funds in life saving medical breakthroughs and treatment. We therefore cannot give them reasons not to invest. Indeed we need to make the pathway clear so that both government and biopharmaceutical companies can work effectively together. 


We will update you when we have more information to give