Bringing hope in hemophilia
By collaborating, gathering compelling global data and sharing knowledge hemophilia advocates are driving fairer and broader access to diagnosis and treatment
Treatment for hemophilia has come a long way in just a few decades and near-term innovations are bringing a new hope for those with clotting disorders.
But there’s still a big problem: that of access to treatment. Despite scientific innovation, many have been and continue to be left behind, says Cesar Garrido, President, The World Federation of Hemophilia.
“Three decades after clotting factor concentrates began drastically improving the life of those with access to them, we are now looking to the next wave of innovation in hemophilia treatments, diagnostics and digital tools. The problem is that more than 75% of people with hemophilia still have limited or no access to diagnosis and treatment,”
In the first of three Reuters Events Pharma events convened to collectively examine how hemophilia advocates, policy makers, payers and regulators can deliver a better and more equitable future for people with the hemophilia, a panel of advocates from around the world explores the fraught issue of lack of access to treatments: its causes and solutions.
Living with hemophilia
Panellists from Europe, Africa and the Americas outline their own experience of working in countries with limited healthcare budgets access to treatment can be scarce and inconsistent, depending on local coverage or even the whims of policy makers, since political issues such as budgetary healthcare cuts can also make access sporadic.
Cultural issues loom large in Algeria, where hemophilia is only recognised in boys, leaving women very often untreated, and where access in large rural areas is sparse at best, says Latifa Lamhene, President, Algerian Hemophilia Association.
Education, literacy and knowledge of the disease and its treatment options are another common issue, says Agnes Kisakye, Executive Secretary, Hemophilia Foundation of Uganda. “Some patients have limited knowledge about treatments and so may not even know to ask for treatment, especially for newly diagnosed patients.”
Geography can be another barrier. In Honduras, for example, only eight hospitals hold factor VIII, and only two receive factor IX. “Treatment is not always available where I am, where I need it,” says Tirsa Carcamo Bonilla, Project Lead, Honduran Society of Hemophilia.
Even in high income countries like the US, hurdles, challenges and constant worry surrounds having a bleeding disorder, says Chris Bombardier, Executive director of international nonprofit, Save One Life. “Obviously, we do have much better access to care, but there's often a large financial worry and income disparity. There's always this concern of a surprise bill or the loss of insurance which will leave you without access to your medication.”
Securing international recognition
Progressively, the community is starting to change the global conversation. Important breakthroughs are happening for people living with a rare disease at an international level, says Flaminia Macchia, Executive Director, Rare Diseases International (RDI).
A resolution addressing the challenges of the rare disease community has already been adopted by the UN General Assembly and will hopefully be follosed by a World Health Organisation (WHO) resolution on universal health coverage for rare disease. It’s making these conditions a more pressing policy priority for policy makers and other key stakeholders.
But more work needs to be done, particularly with regard to market access strategies for low- and middle-income countries, adds Macchia. “We cannot take the market access strategies that are developed for high-income countries and hope that we can transfer them into low-income countries.”
RDI is working to connect centers of expertise, clinics and experts at the national and regional level to encourage the sharing of information and pooling of resources around the world. This is part of a wider initiative, called the Collaborative Global Network, to support broader access to diagnosis and care.
“It's a complex journey, but it's a simple idea in the sense that there are pieces of expertise in different centers, in different countries, in different regions of the world,” says Macchia. “What this global network aims to do is to gather this expertise under a global network for rare diseases that would collaborate in order to benefit as many persons living with a rare disease as possible around the world.
“We need to leverage the regional and global frameworks to really increase the support to access for medicines and diagnostic tools. We also need to promote access to genomic technologies and we need to build on the digital progress that can really democratize access to expertise and knowledge.”
Building solutions together
This global approach is key to solving the access problem. Sharing best practices from other campaigners around the world not just from hemophilia patient organizations, but in other disease areas as well, is important, says Baiba Ziemele, Chairwoman of the Board, Latvia Hemophilia Society.
Solutions can also involve direct involvement in establishing access to care, says Carcamo Bonilla. Since some hospitals in Honduras have their own budget, working closely with them to help create solutions by training staff, for example is a fruitful approach. “We can equip your laboratory, we can train your hematologist. We want to give them something so they can help us also.”
But inevitably, approaches will differ depending on the local context, says Kisakye. “We need to come up with actions that are reasonable and implementable in our settings. We need to map our settings. It would be also good to conduct some research. We need to hear from the patient's perspective why they are not demanding services. Why aren't they able to receive services when they need them? What about the health workers perspective?”
Another important lesson is that advocacy groups should use meetings with policy makers to maximum effect. First-person accounts from patients and parents can be especially powerful, says Kisakye. “Every time you have a chance to utilize the person in power, please use him fully.”
But perhaps of most importance in securing access within individual countries is the value of gathering data from all over the world to inform arguments with payers and providers locally, such as that being gathered as part of World Federation of Hemophilia data collection initiatives, says Ziemele.
“This data is very helpful for showing your local Ministry what is the situation [in other countries]. For example, for Latvia, we were clearly able to show that Lithuania and Estonia, our neighboring countries, are achieving better access, even though we all started in the same place in 90s.”
Winning the argument
Knowing about and being able to point out globally established guidelines on the principles of care developed across different disorders is another way to help close the gaps in diagnosis, care and treatment by comparing efforts in other countries and showing how it has helped, adds Ziemele. “We were able to advocate for home delivery during the Covid situation because others started doing it earlier and now we also have it, nobody will take it away.”
Carcamo Bonilla agrees that such data is essential in winning the arguments for care locally. “Data is so important. It's a game changer. When you have organized data as detailed as possible, it shows us evidence and it changes your position before the authority. It empowers.
“If my neighbor country is a little better, then I can show that they're doing a little better. If they have the same system, the same demographic, the same cultural similarities also, then that's going to be impactful, since I said nobody wants to be less than or under their neighbor countries.
“We cannot come with demands or request that high income countries are having. We want the data to be real and honest because our budgets are not high income. Our budgets reflect what's going on in the country. Our requests also have to be feasible and achievable for the authority.”
Data is key to winning the important arguments, says Garrido. “It is maybe the main tool, generate evidence using that data and have the knowledge to effectively convince them with that data that a treatment they don't currently purchase is more economic, more efficient, can save them money, human resources, et cetera.”
Working together as a block, together with medical sector stakeholders, will magnify influence at each country or region level because no health authority will want to be compared unfavourably to their neighbours, says Garrido. “It's our biggest opportunity right now to create a regional, global voice of togetherness in hemophilia with many patients and many organizations.
“We have to work together, sharing ideas, best practices. Data is the currency of global access. A global data initiative can provide us with the evidence, knowledge and tools to participate and change public policy at the local level. It empowers advocacy and builds accountable access across regions. Comparison and data are our best tools today for achieving Equitable access.”
For more information, see our Reuters Events Advocacy in Hemophilia 2022 Series.