Hemophiliacs in Europe have never had so many reasons to feel hopeful about the future, thanks to significant recent scientific advances. But “vested interests, entrenched systems, and blatant, outright corruption limit access” to even basic healthcare in some Eastern European countries, says Amanda Bok, CEO of the Brussels-based European Haemophilia Consortium.
Bok spoke April 12 at the 2019 World Orphan Drug Congress USA, held in Oxon Hill, Maryland — and subsequently to Hemophilia News Today.
The consortium, founded in 1989 in the Netherlands, is based in Brussels and represents about 90,000 hemophiliacs in 46 countries. This includes every nation in the 28-member European Union except Malta, as well as non-European countries such as Israel and Kyrgyzstan.
“Our objective has always been to ensure the quality of life of people with bleeding disorders,” she said. “We have had quite a journey behind us.”
In the 1980s, people with hemophilia finally got access to adequate treatment with the introduction of factor replacement therapy produced from healthy donors. But then the HIV crisis hit hard.
“We went from no therapy at all to a death sentence,” she said. “In some communities, we lost up to 90% of our patients, due to contaminated products given to them by their governments.”
For this reason, Bok said, “safety is of utmost concern to us. We are intimately involved with the biology of all products. We were the first to put out educational therapy films. It doesn’t help us to have strong advocates today if we don’t have strong advocates tomorrow.”
The consortium has eight full-time staffers, up from one part-time employee seven years ago. Its annual budget is now €1.5 million (about $1.68 million), compared to €190,000 (about $210,000) when Bok started in 2012.
Hemophilia healthcare varies widely
Eleven years ago, the consortium published its first European Principles of Haemophilia Care. The most recent report, issued in 2015, covered 37 countries; its main findings can be viewed here.
Measured by the use of per-capita factor VIII compared to per-capita GDP for each country, hemophiliacs in Finland, Germany, Ireland, Italy, Hungary, Slovenia, Sweden and the United Kingdom have the best access to treatment, according to the consortium’s 2015 survey.
At the other end of the spectrum are Albania, Azerbaijan, Bulgaria, Georgia, Latvia, Macedonia, Montenegro, Romania, Serbia, and Turkey, where — according to the consortium — “economies have been growing but this has not resulted in similar increases in access to factor VIII products as would have been expected, and in some cases even basic on-demand care is not available.”
Factor VIII availability is highest in Hungary (9.4 international units per capita) and Sweden (9.2 units), and lowest in Albania (0.46 units) and Kyrgyzstan (0.1 units).
Overall, Bok said, the general picture for European hemophiliacs seems quite positive.
“More than 40 treatment products are now available. Plasma-derived therapies are much safer than before, and we have multiple generations of recombinant therapies behind us,” she said. “There are novel methods of rebalancing hemostasis in the body, and gene therapies are coming down the pipeline.”
Even so, she added, “we’ve seen very little improvement in factor VIII and IX in some Central and Eastern European countries. Vested interests, entrenched systems and blatant, outright corruption limit access. Some countries have made bribery legal up to a certain threshold.”
Bok declined to identify specific countries in Europe or the former Soviet Union where bribery and corruption endanger the lives of hemophiliacs, saying only that “the best way we can hope to make an impact is to be able to change the system from above.”
The promise of gene therapy
Also speaking at the WODC event was Jeff Ajer, executive vice president and chief commercial officer at San Francisco-based BioMarin Pharmaceuticals. His theme: gene therapy access and affordability.
“In many cases, gene therapy can be a quantum leap forward over standard of care,” Ajer said, noting that five diseases can now be treated with cell or gene therapy, more than 100 diseases are being explored for such treatment, and 289 cell and gene therapies are currently in development.
By 2030, Ajer predicted, “those new therapies will be treating up to 15,000 patients in the U.S., at a cost of [$8 billion to $10 billion] per year.
Yet current standards of care are quite limiting, he said. Breakthrough bleeds are common, factor augmentation is needed for less severe incidents, and quality of life is affected by the constant need for factor replacement.
This is the rationale behind BioMarin’s valoctocogene roxaparvovec gene therapy program for people with severe hemophilia A.
“Gene therapies have the potential for one-time treatment designed to maintain constant expression at normal levels, and the potential to reduce or eliminate the need for supplemental infusions,” he said.
In May 2018, the company announced that its Phase 1/2 trial (NCT03520712) had dosed its first participant with pre-existing AAV5 antibodies. BioMarin is now enrolling patients for its Phase 3 program, GENERATE 1. If that phase is successful, Ajer said, “we would have a robust set of data” to support continued development of the program.
“The question on everybody’s minds is not if there’s a clinical effect, but how long is that clinical effect going to last?” he said. “Gene therapy must deliver compelling and differentiating value for patients with significant disease burden. In the case of hemophilia A, we think the unmet need is pretty high.”
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