Gavin Finkelstein is President, Haemophilia Foundation Australia
It was interesting that the World Hemophilia Congress in Orlando was held not very far from the Kennedy Space Centre, in Florida. Unfortunately I didn’t have a spare day to travel to the visitor centre there, while in Orlando, but many Congress delegates did take a day tour and have talked about their informative and fascinating visit. People involved in space flight and investigation take what most of us would see as enormous risks in their work. One of the Congress speakers was Michael Lutomski, a former employee at NASA who talked about assessing and managing risk in space. The disasters of the Space Shuttle Challenger Program and the HIV/AIDS contamination of blood products in the 1980s taught us many lessons. The catastrophic outcomes in both areas demonstrates the importance of how risk is approached, tolerated and managed. Continuous risk assessment and management is a core principle for treatment product safety.
We now have safer treatment products for bleeding disorders since those dark times. HFA will not forget the experiences of Australian men and women who continue to live with the consequences of blood safety problems in the past, Currently, we are working with the community to ensure prompt access to the new direct acting antiviral (DAA) treatments for hepatitis C. As we also look to the future, lessons from Australia and around the world continue to remind us that we should not be complacent. What will haemophilia treatment and care look like in Australia? What should it look like? These are two very different questions. Are we aspiring to world’s best practice?
Later in this publication I reflect upon the emerging new treatment products. The development of recombinant clotting factor products a generation ago marked the availability of new treatment products, that were not only considered potentially safer than their plasma alternatives because they were manufactured in laboratories but this promised increased supplies. This became true for developed countries like Australia but World Federation of Hemophilia (WFH) still estimates 70% of the worlds’ bleeding disorders population remains undiagnosed or untreated partly because of their high cost. We have a long way to go in that respect and I am deeply committed to steps HFA can take to contribute to WFH’s work.
But globally change is around the corner. Gene therapy has been 20 or more years away for most of my adult life. We are getting closer, with promising reports, but I believe it will still be some time before it is safe, effective and affordable. However extended half-life clotting factors are now used in several countries. They are not funded in Australia by governments, but the experience of people who remain on extension studies following their participation in clinical trials tell us of their experiences. Extended half-life means less infusions. This has a great impact on the lives of many who tell us it is not just a convenience factor – they already know they are having less time off from work or school. Some report more effective management of their bleeding disorder due to the way the product works in their bodies and the closer relationships and monitoring with their treaters – and their files are starting to show they have less bleeds every year. This is good for them and in the long run, must surely be good for the budget for clotting factors! HFA is very keen to make sure the Australian patient community has timely access to a range of treatment products and we will work with clinicians and governments to ensure we do not fall behind.
The recent publication of the Guidelines for the management of haemophilia in Australia by the Australian Haemophilia Centre Directors’ Organisation (AHCDO) in collaboration with the National Blood Authority (NBA) – with its focus on the importance of comprehensive care for people with inherited bleeding disorders – is a positive step towards achieving best practice for all in our community. The recommendations of this publication alongside our commitment to the Australian Bleeding Disorders Registry (ABDR) and to MyABDR, so that the data recorded by individuals about their bleeds and treatment can be used by them and their treating health professionals to better understand their treatment needs, is critical to achieving this.
Sign up for the latest news, events and our free National Haemophilia magazine