World-wide inequalities in Haemophilia care

Too many Haemophilia A patients still receiving inadequate or no treatment

We have been analysing the data provided by the World Federation for Hemophilia (WFH) for a number of years to develop our understanding of the market. Its Reports on the Annual Survey give details of the number of patients by country and the quantity of FVIII used. By combining these data with the sales figures reported by La Merie Publishing (2018 Sales of Recombinant Therapeutic Antibodies & Proteins), we can estimate the averageprice obtained by the six commercial producers/vendors as well as the level of treatment in different countries.

The WFH data, however, is incomplete with some countries not fully reporting number of patients or usage. But by extrapolating the data based on our experience of the data over the last ten years we estimate there was 9.6BN International Units (IUs) of recombinant Factor VIII (rhFVIII) and 3.8BN IUs of plasma-derived (pdFVIII) used. Since 2011, plasma derived product availability has consistently been in the order of 4BN IUs per annum. Over the same period, our estimated recombinant product volume has increased in availability and by a further 8% in 2018. The introduction of Elocate®, the longer acting version, and the launch of NovoEight® appear to be a big reason for the increase in availability though we also believe Recombinate® has expanded production.

Nevertheless, we believe that this analysis demonstrates the rigid nature of rhFVIII production: for plasma by the quantity of plasma donated and fractionated; and for rhFVIII by the installed capacity of production. However, it’s worth noting that the increase in product availability has allowed WFH to record more patients now receiving treatment (173,000 – 2018; 161,000 – 2017; just 54,000 in 1999 the first WFH Annual Survey).

Total sales of rhFVIII products in 2018 were $6BN but the quantity behind this number is still only enough for treating less than 25% of the estimated number of patients in the triad (USA, Japan, Western Europe).

As in many aspects of modern life, we see inequality around the world for haemophiliacs.

Based on the disease prevalence, 1 in 5,000 males, Ireland, Azerbaijan, Slovenia, United Kingdom and Macedonia have 100% diagnosis, with Slovak Republic, France, Hungary and New Zealand at over 90%. Belgium, Australia, Romania, Czech Republic, Canada and Chile appear to have over 80% diagnosis and treatment. Interestingly, this analysis suggests that the USA and Germany treat only 42% by prevalence. Nevertheless, we understand treatment generally in the Western World is adequate but wonder if there are haemophiliacs in the USA who, without insurance, don’t get treatment.

Using the WFH data we’ve calculated the average usage per patient with estimated average consumption of IUs per patient in the triad at ~150,000 at a cost, on average, of almost $100,000 per year. For the patients in the USA this is probably more than $150,000 due to higher prices. In the countries outside the triad, i.e other than USA, Japan, Western Europe, the average usage per patient is just 50,000 IUs. At 100,000 IUs per annum, our estimated 9.6BN IUs world production of recombinant Factor VIII could treat about 96,000 patients – ~50% of the 173,000 patients reported in the WFH Annual Survey 2018. The lower usage outside the triad continues to show the inadequacy of treatment in most of the world notwithstanding those getting no treatment at all.

If patients outside the triad were to get 100,000 IUs per year, we would need another 12BN IUs – that means doubling existing production from all sources.

It’s worth considering that just eight countries (United States, Japan, Brazil, United Kingdom, Italy, Germany, France and Russia) consume more than 60% of the total available FVIII. And a total of just 12 countries, representing 18% of the world population and 38% of the reported patients, consumed 80% of the rhFVIII.

People ask us why the current producers don’t just increase production? Well, they have, but, even with the new entrants, there still isn’t enough to go around. With our low-cost production process, we intend to focus on markets currently unserved in order to get even more patients treated and even-out some of the inequalities.