Recombinant and plasma product usage and cost

1
Fifth WFH Global Forum on the Safety and Supply
of Treatment Products of Bleeding
Disorders Montreal, 24 September 2007

• Recombinant and plasma product usage and cost
• throughout Europe

Wolfgang Schramm University Hospital of
Munich Dpt. of Transfusion Medicine and
Haemostasis
2
Wildbad Kreuth Initiative 1999

• Recommendation 128
• Adequate amounts of coagulation factor
  concentrates for the treatment of patients with
  haemophilia and related disorders should be
  available in each Member State. Quantities of
  both plasma-derived and recombinant products
  should be maintained, although it is recognised
  that recombinant products could gradually
  supplant plasma-derived ones. Individual patient
  preferences should be taken into consideration
  when choosing products.

Schramm W. Conference Proceedings ISBN
3-00-005705-6
3
Factor usage varies widely between European
countries F VIII units per capita
Europ social econ study / ppta. com. /
P.L.Robert MRB
4
Main aspects influencing factor concentrate supply

• Demand
• living conditions of individuals
• medical guidelines
• availability of factor products

Financial strength of health care system
The socio-economic reality and haemophilia
treatment varies in the EU other countries of
Europe.
Pattern of health care system
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Different purchasing, pricing and reimbursement
systems are determinants for hughe price
differences
(0,12 - 1,11) (0,55 - 1,02)
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
8
Factor consumption in European countries)
Europe(mio-inhab./country units/capita / PPTA
share pdFVIII rFVIII)
Europ social econ. Study, ESCHQoL, pers. com.
P.L.Robert MRB, PPTA 2005
9
The countries financial capacity is only a rough
indicator for factor usage
Factor consumption gt 5 IU
Factor consumption 2-5 IU
Germany 5.5 2247 26.686 Schweden 6.6 288 32.03
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Austria 3.8 245 29.988 Belgium 3.1 298 28.932
Czech Rep. 2.0 98 9.505 Denmark 3.2 208 38.51
9 Finland 4.0 155 29.981 France 3.7 1710 28.79
8 Hungary 3.2 88 8.687 Italy 3.1 1417 24.389
Portugal 2.1 147 14.909 Slovakia 2.6 38 6.998
Spain 3.6 904 21.889 Switzerland
2.8 295 40.301 UK 4.1 1791 29.751
GDP1
GDP/capita2
GDP1
GDP/capita2
Factor consumption lt 2 IU
Greece 1.5 181 16.455 Lithuania 0.4 21 6.176 Ro
mania 0.2 79 3.549 Poland 1.3 243
6.295 Slovenia - 27 13.500 Turkey - 291 3.836
GDP1
GDP/capita2
1 GDP 2005 data in billion / 2 GDP/capita 2005
data in Source Eurostat 2007 / P.L.Robert MRB
/ PPTA 2005
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Usage plasma derived versus recombinant factors
Interim analysis of the ESCHQoL database
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European Perspective Shared Concerns
Quality of factorconcentrates
Supply Priviliged countries
Past
Progress in quality of factor concentrates

• Supply
• for all?
• - Demand is growing in many European countries

Quality of factorconcentrates Open
question Emerging pathogenesInhibitor
development
Present / Future
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F VIIIIX Units (MM) 1990-2005 Plasmaderived
Recombinant
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
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F VIII Units (MM) Plasma-Derived
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
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Example 8 countries use three fold of pd factor
VIII units than19 other European countries
Example 3 countries (D,I,ESP) use more than 2
fold factor VIII units than19 other European
countries
p653,41
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
15
F VIII Units (MM) Recombinant
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
16
Example 4 countries (D,UK,F, I) use four fold
ofrec factor VIII units than13 other European
countries
p653,41
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
17
F VIIIIXTotal Sales [phone]
Europ social econ. Study ESCHQoL pers. com.
P.L.Robert MRB
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Summary

• Therapeutic use of clotting factor concentrates
  ranges in Europe from lt0.1 - gt6 units per capita.
• Demand of clotting factor concentrates is growing
  from an European perspective
• Both pd and rec factor concentrates are needed
• Practices vary in Europe At present, there is a
  knowledge gap as to what is actually the best
  practice for the treatment of haemophilia for a
  individual country.

Are cost-benefit-analyses the appopriate
instrument for decisions how to allocate
resources? Shared concern Supply of adequate
amounts of factor concentrates for all patients.