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The 8th Annual Global Haemophilia Advocacy Leadership Summit

By Sue Ellis, HNZ CEO

Along with President Deon York, I recently attended the 8th Global Haemophilia Advocacy Leadership Summit in Lisbon, Portugal. This is my second attendance of these annual summits, and this one provided even more interesting presentations and workshops over the two days. It is always a privilege to be invited to attend these annual summits, hosted by the Haemophilia Advocacy Advisors Board, Bayer, and Biomarin. Although it was a long way to travel, it was kindly funded by Bayer and co-sponsored by Biomarin

The theme for this summit was Advocacy in Action: Pushing the Access Boundary, and focused on how we as advocates can work to improve access to innovation and treatment advances.

There were panel discussions, workshops, and presentations from a number of experts. These included from industry, from academia, from other national and international haemophilia groups, including the European Haemophilia Consortium, the Australian Haemophilia Foundation, and the WFH, as well as from people with bleeding disorders.

Senior Medical Officer of the European Medicines Agency Hans-Georg Eichler gave an interesting keynote address looking at identifying decision-maker requirements from patients, and discussed what he called, “The evolution of the concept of ‘drug’”. He talked about the ‘advanced therapy medicinal products’, or ATMPs - from chemicals to biologicals to ATMPs and noted that for regulators, the problem is predictability. He believes that in the mid to long-term future 25% of all new approved drugs will be ATMPs. Currently the drivers of change are ethical concerns, and personalized treatment combinations and said enables of change are availability of patient-level randomised controlled trials (RCT) data and availability of real-world data (RWD). An OECD report has recently been produced on this called “Readiness of electronic health record system” to contribute to national health information and research.

I particularly liked Dr Eichler’s discussion on interaction between decision-makers. Historically the flow of interactions between decision-makers go from left to right - industry to regulators to HTAs to payers to clinicians to patients, which he believes are silos of collaboration. If the regulator fast tracks a product, the payer says no not enough evidence, therefore the patient misses out, which appears dysfunctional. The evolution of information needs to move from right to left. That is, start with the patient. Patient reported outcomes must be validated and methodology needs to be done to make it valid – that is: is this fit for purpose.

There was an interesting presentation on Barriers to Access: Patient-Payer Dialogue. Of importance, when we are looking at providing a submission, we must think about what do we need, what is important to us, what are our trade-offs? The submission must be quantifiable, reproducible, and transferable, not anecdotal. However, patients should stay out of the price debate, while insisting on what is important to them and what are the relevant risk/benefits.

Discussions on patient outcomes led well into an update on PROBE by Mark Skinner, President/CEO of the Institute for Policy Advancement. PROBE stands for Patient Reported Outcomes Burdens and Experiences, the global research project to enhance the direct patient voice in healthcare decision-making. PROBE is an online survey that patients with Haemophilia A and B and carriers can access through their smart phone or tablet. The PROBE study provides a way for people with haemophilia to report data on their health status and quality of life. This evidence can then be used as evidence to advocate for better care and treatment. The survey can also be completed by people who don’t have a bleeding disorder – they serve as a control group.

The App can be downloaded for free from the Apple Store for IOS and Google Play for Android - search for myPROBE - or you can complete the survey at The questionnaire measures key outcomes such as pain, mobility, health impact on work and education, ability to conduct activities of daily living and quality of life. It takes less than 15 minutes to complete.

The Australian Haemophilia Foundation have been working towards getting all their members with haemophilia A and B to complete the survey, working though the laborious process of ethics approval.

Another presentation given by Amanda Bok, CEO of the European Haemophilia Consortium, was about working with key decision makers, taking a systems approach perspective. In this, Amanda noted there are different types of personalities. As she noted with a quote from Churchman, 1968, “A systems approach begins when you first see the world through the eyes of another”, keeping in mind the different personality types we may encounter in our advocacy work. These include the ‘Analytical’ the ‘Driver’ the ‘Expressive’ and the ‘Amiable’. We spent 3 hours in a workshop looking at how we can navigate our approach with these different types of personalities and what works the best for each. This was an interesting exercise and lots of fun.

We also spent some time in another workshop planning an advocacy strategy to help a health organisation that was undergoing change, with workforce and resource issues, along with education and information requirements. It looked at organisational change and how we as advocates deal with those issues to ensure our voices and needs continue to be heard.

It was so good to meet up again with fellow advocates from the haemophilia community around the world and to meet new ones. All of us were there to engage with leading advocates, key stakeholders and industry experts, working together to learn new ways of doing things, to cement those and previous learnings that will benefit our haemophilia community.