New haemophilia therapies Alex Klever Plenary Treatment landscape into the future Chair ~ David Fagan From then to now ~ Gavin Finkelstein What is precision or personalized medicine? New and emerging treatments for all bleeding disorders ~ Dr Glenn Pierce Australian experience and challenges with new and emerging haemophilia therapies ~ Dr Liane Khoo Access to new therapies – policy and regulation ~ Jo Cameron Hopes for the future: reflections from a parent ~ Claire
It was such a pleasure to have Dr Glenn Pierce giving the opening plenary at the Conference. He spoke about personalized medicine and emerging treatments, touching on the progression of treatment options for people with haemophilia over the years.
and their families to work together with their healthcare professionals to make the best informed choice about their treatment options. One issue highlighted by Dr Pierce was the ongoing health inequity for people with haemophilia in countries with limited resources and how this could be addressed by providing gene therapy. Even though currently this is a costly treatment, it has the potential to be ‘one shot and you’re done’ which may well be a more sustainable option in the long term for countries that have limited or no access to other haemophilia treatments. Dr Liane Khoo then spoke about the Australian experience and challenges with new and emerging haemophilia therapies. She spoke about the whole team approach with patients and their families at the centre. The evolution of the treatments for haemophilia patients was very interesting and it was amazing to see how far we have come. What’s even more exciting is that Australia thus far has participated in 64 clinical trials!
Conundrum, Continued
Pro
tein s
Gene Therapy Bispecific Mimetic MAbs
Personal Preferences
New Extended Half Life Current T x
Current Access and Availability Access to Future Therapies Patients Patient Advocates Heath care providers
Dr Khoo then walked through the newer therapies that are on the market now and what is coming soon and the challenges these bring to patients and clinicians. Needs of the patients will be changing as well as the needs of the Treatment Centres and their models of care. As a closing note she mentioned as we work together to improve outcomes together for a better future, which was a very appropriate statement for this year’s Conference. >>
National Haemophilia 224, December 2023
Dr Pierce discussed patient-orientated algorithms for personalised treatment choices in the current treatment landscape, which led to the development of the WFH Shared Decision-Making Tool, now available at sdm.wfh.org. This tool enables patients
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