What the World Is Working On

Fortunately, most of the issues no longer concern our country, but some still do. The cardinal question at the last forum was: in the era of recombinant products, does the production of plasma-derived ones still have a future? A long and well-argued discussion was held on this topic. For the majority of the world's population, unfortunately still irrelevant, because only 25% of those who need it have access to adequate diagnosis and treatment of haemophilia, and many of them are limited to plain plasma or, in the better case, cryoprecipitate. Three quarters of people with haemophilia are thus completely left out.

Nevertheless — or perhaps precisely because of this — the effort to ensure the highest possible safety of plasma-derived products is warranted. Countries with large consumption and even greater needs are building their own small processing facilities with potential risk. This mainly concerns India, Russia, and China, where the WFH expresses great concern about the reliability of their viral control. In Europe, America, Australia, and similar regions, there is no need to worry about contamination of plasma derivatives — here there is certainty that the currently known viruses are caught by testing of donated plasma. However, the common and previously unusual global travel now carries a greater risk of transmitting viral diseases to places where they had not previously occurred. Therefore, manufacturing companies devote enormous attention to testing and the safety of their products. The result is that recipients of plasma-derived concentrates in developed countries — and we count ourselves among them — need not worry. The acute dangers for undeveloped markets remain primarily hepatitis E, West Nile virus, and parvovirus. In developing countries, 45 million units of plasma are collected annually, yet today no country is self-sufficient in plasma consumption.

And what about recombinants? They will likely play an increasingly important role in the near future, but will probably never fully replace plasma-derived products. Plasma sources are nearly inexhaustible, and the recombinant industry cannot yet fully saturate the market. In 2011, 8.1 billion units of Factor VIII were consumed worldwide. Recombinants produced from hamster cell lines remain more expensive than plasma-derived ones, and this plays a role not only here but in many — perhaps all — other developed countries.

The World Federation of Hemophilia is therefore convinced that the twilight of purely plasma-derived products is far from coming. The ideal situation is one in which each haemophilia patient — together with their centre — chooses the product themselves. Without the influence of regulators, meaning various institutions such as SUKL (the State Institute for Drug Control) or the Ministry and insurance companies. But that is a long way off everywhere in the world, as the cost factor plays too large a role.

The globally recognized measure of treatment quality is the consumption of clotting factors per capita. In one of the most successful countries, Ireland, it was 1.5 IU of Factor VIII per capita in 1996, 3.5 IU in 2003, and 8.12 IU in 2012. For comparison, in our country in the same year 2012, it was 4.0 IU. Consumption is therefore constantly rising sharply everywhere, and we are not doing badly. The level of education of the Czech haemophilia population and the influence of the local organization on state institutions' decisions about treatment are highly rated. The WFH considers this one of the decisive factors in product selection for each individual. Influence on choice should be regarded as one of the important components of human rights. But nothing should be overdone, and the American approach, where individual manufacturers can market directly to recipients of treatment concentrates, does not meet with great understanding.

The conclusion of all this? We are not doing badly at all in terms of treatment quality, concentrate consumption, and the influence of the broad haemophilia community on decision-making processes. The choice of each individual product should be strictly individual, which most centres in our country practise. Clients of those centres that do not honour such principles can turn to the Society.

Plasma-derived products are far from finished — they have an irreplaceable role for the future as well. Those who have not yet switched to recombinants are not being shortchanged in any way.