Advocacy in Hemophilia Series: Crafting a Path that Leaves No One Behind

Dec 14, 2022 - Dec 14, 2022,

Ethical and realistic policy considerations for the future of hemophilia care

Advocacy in Hemophilia: Building the Future by Design

Creating a visionary paradigm for the future of hemophilia and rare diseases that disrupts current thinking

Advances in the treatment and care of hemophilia have accelerated in recent years. And, according to Jack Uldrich, global futurist and author, the field could be on the precipice of even more disruptive changes, coming at an accelerated pace.

Uldrich explains that multiple innovations could potentially advance the field. These new frontiers include gene therapy and many other novel treatment approaches, such as mobile and decentralized blood-testing technology, artificial intelligence, nanomaterials for the delivery of novel treatments, advancements in battery technology (enabling drone-blood-delivery services), synthetic biology, and 3D printing of organs. . 

“In 2021, venture capitalists funded to the tune of $5 billion a handful of promising blood diagnostics startup companies,” says Uldrich, “There could be ways to diagnose hemophilia better and faster—and more affordably—all across the world.” The world is changing quickly, and how we treat hemophilia in the future has the potential to differ drastically from how we treat it today.

So, which innovations could have the most significant impact on hemophilia care around the world, according to leading patient advocates? 

Innovations primed to advance hemophilia care in different communities
Decentralized diagnosis and mobile testing for hemophilia could greatly benefit remote areas and rural populations that do not have direct access to medical testing currently, explains Masood Fareed Malik, Founder & Advisor of the Hemophilia Foundation-Pakistan [HFP]. “I believe that for geographically huge countries, mobile testing will be very important. In Pakistan, we can see a huge gap between the estimated number of people with hemophilia and the number who have been diagnosed, initiatives like this can definitely close the gap.”

Taking into consideration the further three billion people globally that are estimated to come online in the next few years due to internet coverage improvements, access to information will increase exponentially, which could have significant benefits for currently undiagnosed people with hemophilia as well – and the community overall.

Healthcare professionals could also benefit from improved access to information on hemophilia and enhanced opportunities for education. Barriers to learning could be further addressed by advances in translation technologies, such as real-time translation and transcription glasses that could provide subtitles for the wearer, opening the door to a multitude of new educational possibilities. 

Emerging treatment approaches that could improve access in low- and middle-income countries include non-intravenous delivery systems that require less frequent administration, as well as rebalancing agents, which could potentially be used for more than one type of hemophilia. “Some of these subcutaneous treatments [in development] where the frequency of the treatment is maybe once per month, could be far more functional for patients than trying to get an infusion twice a week,” says Chris Bombardier, executive director of Save One Life.

Bombardier adds that technologies such as subcutaneous implants might also help to address issues around home-storage of hemophilia treatments in developing countries.

Treatment equity between developed and developing countries is a key consideration of innovation, according to Cesar Garrido, President of the World Federation of Hemophilia. “In my recent visit to Kenya, I saw what we thought ten years ago was almost an impossible dream. Today they have access to medicine with new therapies as well.” 

Garrido advises that the most effective way to advance equity of access is through scientific evidence and reliable data about the benefits of new innovations to individuals and health systems alike, coupled with flexibility to keep moving through challenges that will undoubtedly arise. “The assessment of health technologies is something that we need to learn,” says Garrido, “It will be necessary to measure and record the impact of innovations”.

Want to view the entire event recording of this discussion? Register for series access here

Strategies to advance best advocacy practices 
The core elements of patient advocacy will remain foundational as the community continues to innovate for the future, according to Brian O’Mahony, chief executive of the Irish Hemophilia Society. O’Mahony reminds fellow advocates that having a united voice between healthcare professional and patient organizations, as well as strategic objectives and a clear case with strong supporting evidence, will always be imperative when approaching decision-makers.

“Ideally, though, we need to be in the room when decisions are made,” says O’Mahony, “with concrete solutions to help co-design the future of the service. You can achieve what you want by working in partnership with the government and payers, by persuading them that what you want is realistic.” 

It’s also imperative to be organized, efficient and nimble. In 2017, a team from the Hemophilia Foundation of Uganda presented a policy on hemophilia to decision-makers in the hopes of increasing access to treatments in the country. According to Agnes Kisakye, executive secretary of the organization, they maintained a dialogue with various policymakers but experienced a setback after the 2020 elections, when a new team of decision-makers arrived.

“When you get into the [decision-making] room, it’s very important to fully utilize that time whilst the policymakers and other stakeholders still have influence,” says Kisakye, pointing specifically to the importance of a well-laid out strategy and having the right spokesperson presenting the case. “It’s very important to have a great connection with the policymakers, to utilize the data as much as possible and produce enough evidence.” 

According to Kisakye, empowering patients, families, and communities to advocate for services for themselves is also essential. “These are the beneficiaries of the services. We have seen that most patients in my own country [Uganda] don’t know the services they are entitled to or how to demand them because they don’t know the right approaches.”

Getting into the decision-making room in the first place can still be challenging, particularly if a country has little budget allocated to healthcare. This is another example of how moving from ‘traditional advocacy’ to the concept of ‘co-creation’ may help. “How do you move forward in this case?” asks O’Mahony, “You’re not going to achieve everything overnight, and you take small steps that lead to bigger steps.” O’Mahony advises looking at the government’s healthcare priorities and linking hemophilia with their focus areas.

Successful examples noted by O’Mahony include governments that aimed to improve the procurement system, the blood transfusion service, and rare disease policy—and were presented with solutions that included hemophilia. “You get in the door whatever way you can, and then once you’re in the room, you make yourself indispensable by coming up with solutions and advising them in other areas.”

Different healthcare budgets may require different approaches but being aware of the mindset of politicians and government officials in a country can help advocates choose the best strategy. “Politicians and government officials see healthcare only as expenditure,” says Baiba Ziemele, chairwoman of the Latvian Hemophilia Society.

Ziemele believes that lobbying for a change in therapies or an increase in budget, specifically for hemophilia, is more complicated than opting for a solution from a broader effort to treat rare diseases. “Collective efforts are taking place at the highest levels [in Latvia and Europe] and we are requesting faster change and a personalized approach to each person with a rare disease. That might help lagging countries to provide the best possible solutions for their people in terms of treatment and medical care.”

Watch the recordings of events 1 and 2 in the Advocacy in Hemophiliha series for free here, and sign up for event 3.

Future-proofing the hemophilia advocacy community 
“There are a lot of trends in our world that are going to double 10–20 times in the next decade ahead,” says Uldrich, “If you can start thinking exponentially, you’re not going to be caught unaware of the future.” According to Uldrich, embracing ambiguity and planning for uncertainty means staying humble enough to change tactics.

Patient advocates also point to the importance of succession planning in preparing for the future and enabling fresh, adaptive thinking to take root. This requires proactively recruiting new leaders by providing access to education, training, and motivation to upskill in various areas. “We are preparing future leaders,” says Kisakye, “Ever since we started putting them [the youth in Uganda] through school, we are seeing new doctors coming on board in the future. These future leaders are knowledgeable and will be able to speak for coming generations.”

Fareed Malik believes investing in community activists today will be critical for what happens further down the line. “At this stage, it’s very important that you invest today, and maybe in two years, or five, or ten, we’ll be able to impact the community. Because the community is the engine of the whole campaign.”

But it is not just looking inside the community that matters, according to Ziemele, who believes that increasing awareness and influence of patient advocacy groups outside the so-called bubble of hemophilia, rare disease groups, and health ministries is also essential. “We have to have conversations with startups, innovations hubs, and the entire ecosystem that surrounds us,” says Ziemele, “I went to a startup event for healthcare initiatives and met people who have fantastic ideas, and they didn’t know that patient organizations exist, what we do, or how to reach out to us to include our experiences in their solutions, which is a huge, missed opportunity.”

What comes next? Dream big, stay focused, and measure impact
 “We have to be unafraid to try new things and be willing to think big and dream big,” says Bombardier who is also an accomplished mountaineer. “The thing about climbing a mountain is that you don’t do it in a single day, you do it step by step, and you have to modify and change your path, depending on what obstacles come up.” He believes the same holds true when thinking about how the hemophilia community can achieve big goals together.

Bombardier explains that the global hemophilia community has built a strong foundation that will help them advance towards their ‘Everest’. “I think as a community, we’ve always been good at communicating with one another and sharing a common goal of equitable access for all. I think if we can continue to stay behind that mission as a community, that will help us stay ahead of the game and keep us ‘future-proofed’.”

Meanwhile, Garrido agrees, and underscores that every innovation must be backed-up by reliable and demonstrable evidence. This means measuring, recording, and robust reporting are necessary so that transformative innovations can reach the people who need them across the globe.

Want to view the entire event recording of this discussion? Register for series access here

Speakers and Panel
Jack Uldrich, Global Futurist and Author, The School of Unlearning.
Chris Bombardier, Executive Director, Save One Life.
Cesar Garrido, President, The World Federation of Hemophilia.
Kim Isenberg, Head of Global Public Affairs, Rare Blood Disorders, Sanofi.
Brian O’Mahony, Chief Executive, Irish Haemophilia Society.
Agnes Kisakye, Executive Secretary, Hemophilia Foundation of Uganda.
Baiba Ziemele, Chairwoman of the Board, Latvia Hemophilia Society.
Masood Fareed Malik, Founder & Advisor, Hemophilia Foundation-Pakistan [HFP].

Watch the recordings of events 1 and 2 in the Advocacy in Hemophiliha series for free here, and sign up for event 3.


Advocacy in Hemophilia Series: Crafting a Path that Leaves No One Behind

Dec 14, 2022 - Dec 14, 2022,

Ethical and realistic policy considerations for the future of hemophilia care