Gavin Finkelstein is President, Haemophilia Foundation Australia
I was very much looking forward to the 2017 Conference in Melbourne, but unfortunately a leg injury which required surgery prevented me from travelling to Melbourne. We always think an accident like I had is stupid and avoidable, but “stuff happens”, as they say. I had just been getting into the deep end of the pool using the bars for leverage and fell in, leaving my left leg behind and thus fracturing badly. It was quite an embarrassing production for me to get out of the pool. I knew instantly something bad had happened. I am grateful to the health professionals, especially the nurses, who have looked after me from the surgery through to my rehabilitation.
If there is one thing I have learned during this experience, it is that things that impact significantly on your life happen when you least expect them to, and it makes you realise how fragile we are and how much we need to adhere to all our treatment regimens. As a person with severe haemophilia, I was fortunate to have treated just before going to the pool – if I hadn’t, things could have been much worse.
I have been at each of the Australia/New Zealand conferences over the last 20 years, and as well as always getting something out of the sessions on the program, I have really enjoyed meeting up and talking with others at the conferences. It’s always good to share experiences, build friendships and relationships and in recent years since Australians have had more access to clinical trials, it has been great to hear of the experiences of those on trials or from doctors who have been involved in these trials. We are in a time when more and more treatment products are emerging from “the pipeline”.
The feedback I have received about the 2017 Conference has been positive. You will see reports of the various sessions in this publication, and I hope you agree that the spread of topics covered everyone in our community.
I heard there was great interest in the extended half-life (EHL) factor products now available in several other countries, but are not yet funded in Australia. Published reports show improved outcomes for both haemophilia A and B patients on trials and extension studies. We have received calls from people who have been on completed clinical trials but had continued access to the products by the grace of the manufacturers, but are now worried that they might be forced to switch back to a standard half-life product if their product is not funded by Australian governments.
HFA was disappointed there was no tender for EHLs and other new products before the current contracts for standard half-life products were extended. However, the National Blood Authority has started a process for health technology assessment for these “new” products by the Medical Services Advisory Committee, and while this process will take time, we are pleased that some very limited arrangements for access to specific EHLs are being considered for the interim (for further information go to https://www.blood.gov.au/closed-public-consultations). HFA will work proactively with governments and the Australian Haemophilia Centre Director’s Organisation towards funded access as soon as possible. We want to be sure a range of products are available, including both standard half-life and EHLs and other new products so that people in our community and their clinicians can select the most appropriate treatment product for their needs.
Sharon Caris, our Executive Director, recently attended the WFH Global Forum in Montreal, and will report to Council on this soon, but her immediate comments to me were about the encouraging treatment outcomes where EHLs have been available in other countries and the importance of individualising treatment to a persons’ bleeding patterns to eliminate bleeds. There is great excitement globally about the benefits of EHLs and other new therapies such as Roche’s emicizumab which was approved in the USA for people with haemophilia A with inhibitors in November, and there are others in the pipeline. Gene therapy is doing very well, with trials likely to ramp up. Although it will still be some time before gene therapies will be fully evaluated as safe and effective, and for them to be scaled up and affordable, we are closer than ever. These new treatments present a new paradigm for doctors and their patients and for government payers. Their costs and benefits may need to be measured differently to understand their full impact and funding processes may need to adapt to accommodate them.
We are in exciting times for treatment of people with bleeding disorders and we want to work with governments towards access to the products people will benefit most from.
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