Advocacy in hemophilia: Crafting a path that leaves no-one behind
Implementing an ethical and realistic global access strategy for hemophilia so that patients everywhere can receive treatment
The global patient advocacy community for hemophilia has ambitious aspirations that include hemophilia patients around the globe having equitable access to diagnosis, treatment and care. In this third and final 2022 piece on how to make equitable access to hemophilia treatment a global reality, patient advocate leaders come together from around the world to discuss how to disrupt the status quo, support policymakers, and make progress toward a future where no patient is left behind.
Disrupting the status quo takes courage, unity, evidence and a moving narrative
Advocates challenge the status quo and draw attention to specific issues to drive positive change. Advocacy in hemophilia is no different in this regard, and according to Chief Executive of the Irish Hemophilia Society, Brian O’Mahony, the community is particularly strong thanks to a well-connected, organized approach and targeted activities. “I think we’ve come this far, not because we waited for governments to come to us but because we went to governments,” says O ́Mahony, who encourages advocates to approach decision-makers proactively and make themselves indispensable by adding value wherever possible.
Tirsa Carcamo Bonilla, Project Lead of the Hemophilia Community in Honduras backs a similar hands-on approach. Carcamo Bonilla believes that “you need to [be brave]” to be a healthcare disruptor and highlights the importance of reconvening and strategizing in the face of inevitable defeat. “You also have to know your data. There is a table and a room where the decisions are being made. We had better be there, and we [must] be prepared.”
The above sentiment is echoed by Chris Bombardier, Executive Director of Save One Life, based in the US, who also believes that when asking for change an evocative narrative enhances the probability of success. “It's about having an inspiring story to go with the data,” says Bombardier, “if they're not moved, or inspired by what you're doing it’s not going to stick in their minds.”
In addition to a compelling and proactive approach, advocates agree that a single, coordinated voice is more effective at creating change than a fragmented community. This includes organizations specifically advocating for hemophilia and larger umbrella organizations representing bleeding disorders, for example.
“One national organization and legal representative for each country is best,” says Cesar Garrido, President of The World Federation of Hemophilia. “But sometimes two or three recognized advocacy groups are operating in one region. That is a challenge because it disseminates the strength of the message. Governments are looking for a single voice and position.”
Carcamo Bonilla alludes to this well-coordinated and centralized effort: “We are not fractured in Honduras. There is only one patient organization for bleeding disorders. We work together so the efforts are not disseminated in multiple national organizations. We’re one unit, we have objectives and instructions, and then the benefits are for all.”
Supporting policymakers to advance access to treatments
Founder and Advisor of the Hemophilia Foundation in Pakistan, Masood Fareed Malik, emphasizes the importance of facing up to the realities of living within different systems. “To bring about real change, we have to be realistic, we need to understand our own scenario first, before we attempt to disrupt the system or try to influence policymakers,” says Malik, pointing out that awareness of the resources available to the government is vital to prevent over-ambitious appeals that will be denied. He argues that a clear case must be prepared in advance, and empathy and diplomacy with decision-makers are key. “If we go to policymakers with unrealistic demands, and they say no, we could react by saying they're not listening to us. But actually, the other side of the coin is very important,” says Malik.
Speaking from his experiences with The World Federation of Hemophilia, Garrido confirms that decision- makers across countries always need realistic data. “We have to create tangible, measurable objectives so that we can provide evidence on how a strategy could benefit everybody,” says Garrido, recognizing the neutral and objective position of his organization regarding which treatment options might be best for a country; a stance that aids diplomacy in countries where it is not immediately clear which treatment options are most suitable.
At the core of O’Mahony’s take on supporting policymakers is the principle of adding value and creating win–win situations. “To make it easier for governments to commit to sustainable hemophilia care you give them added value, you bring them a humanitarian aid program, training of healthcare professionals from the patient organizations. That way, for the investment the government makes, they're getting a multiplier effect for the work, and you're also giving the politicians what they really like, which is a win and a success story.”
Ethical considerations for leaders to implement an equitable design for the future
Ethical considerations surrounding access to healthcare globally are complex. Access to fundamental resources such as clean water, food, and clothing, in addition to basic services, such as education and healthcare, vary greatly by location and economic factors. As a result, many people have limited or no treatment options for hemophilia.
Assistant Professor of Medical Ethics at NYU Grossman School of Medicine, Alison Bateman, suggests that the development of new therapies for bleeding disorders should be centered around equity and justice. “We are not simply looking for new, safe, and effective treatments. Rather, we are looking for new, safe, and effective treatments that are capable of being used widely by all patients who have a condition.”
Communicating with drug developers
Focusing on equity of use in end users could impact the treatment modalities that are prioritized for development. “The entities developing new treatments make decisions that will have far-reaching impacts on access,” says Bateman. “For example, if it is decided to develop a product that can be infused at home, that product is going to be much more widely available to patients than one that needs to be infused in [high-tech] specialty medical centers.”
Following a recent trip to Cambodia, Bombardier saw firsthand how environmental factors could influence treatment decisions. “It was clear that if patients were able to access newer subcutaneous treatments, for example, [this specific modality of treatment suited] their environment and surroundings so much better than current clotting factor concentrates,” says Bombardier, concerned that the administration of clotting factors required invasive and impractical travel to specialized treatment centers. “We should be pursuing conversations with drug developers. Part of the discussion also needs to be with healthcare workers in [these] countries. Would they be comfortable with how to give new medications?” asks Bombardier.
Nonetheless, best practices for structuring advocate-driven conversations with drug developers are yet to be defined, with the panel suggesting that discussions should occur early in development and include questions on equitable pricing structures, ease of administration, monitoring and follow-up.
Collaborating across advocacy groups
Rather than carving solo paths, Bateman also suggests that advocacy groups should group together, share a common language, and make it clear to governments and stakeholders that the request of one group is in keeping and alignment with the requests of others, “All of them tied together by a vision of trying to ensure access to care in a timely manner for everyone.” Bateman makes a compelling case for strategic partnerships between advocacy groups to “amplify each other's equity-based claims instead of clawing for scraps at the expense of other groups.”
Many advocates within the panel noted, however, that collaborations could cause a loss of voice. “We have to accept the reality that we're not in an ideal world,” says Masood, highlighting that influencing policymakers can be highly challenging in emerging countries, such as Pakistan. “The very least we can
do for ourselves is to raise awareness and try to get policymakers’ attention in order to change the policy. We have to go and knock on the door.”
Similarly, Carcamo Bonilla calls for a more grounded approach. “It would be beautiful to have governments being more proactive just to make sure that the population is in a better condition: creating solutions and identifying where there are areas of opportunity to grow and giving better access to treatments. However, that is not what happens. It comes down to us.”
Of particular concern to O ́Mahony is that collaborations with large umbrella organizations could hinder the mission of equitable access to hemophilia treatments. “There are 7,000 rare diseases and hemophilia is one of them,” says O’Mahony, explaining that in a group representing numerous diseases, advocacy work for hemophilia could be diluted.
Carcamo Bonilla and Fareed Malik agree that partnerships could offer benefits but should be entered into with full awareness. “We have to be analytical and careful,” says Carcamo Bonilla. “As long as there is common ground— like between hemophilia and bleeding disorders—then by working together, the results could be mutually beneficial.” Alongside Bateman’s point regarding the competition for valuable resources, Carcamo Bonilla warns that advocates need to take stock and be aware. “If you are investing time and resources—such as staff volunteers—into something that you're not sure will yield results, and it comes to a point where you're competing [between disease states], that’s when you need to be careful.”
Speakers and Panel
Alison Bateman, Bioethicist and Assistant Professor of Medical Ethics, NYU Grossman School of Medicine
Brian O’Mahony, Chief Executive, Irish Hemophilia Society
Chris Bombardier, Executive Director, Save One Life
Cesar Garrido, President, The World Federation of Hemophilia
Masood Fareed Malik, Founder and Advisor, Hemophilia Foundation Pakistan Scott Williams, Head of Global Public Affairs, Specialty Care, Sanofi
Tirsa Carcamo Bonilla, Project Lead, Hemophilia Community Honduras