Understanding your treatment

Haemophilia Index 2020

To mark the 30th annual World Haemophilia Day, Sobi has released new data showing the
different opportunities available for people living with haemophilia in Europe and around the world.

As part of our Liberate Life vision, we compiled a study comprising a European survey examining quality of life for people with haemophilia in Europe, and a global index looking into the state of international care through the far-reaching effects of humanitarian aid. The result is the two tables below that give insights into treatment for people living with haemophilia around the world.

For the first part of the study, we asked specialised healthcare professionals across Europe for their opinions on the levels of physical activity, quality of life and satisfaction experienced by people with haemophilia with access to treatment in their countries. To provide context, data relating to the amount of medication and rates of haemophilia in these countries was also included. The second part of the study further focuses on the reach of humanitarian aid by showing the percentages of haemophilia medication provided to countries across the globe.

Although many challenges remain, the results show that advances in diagnosis, improved access to prophylaxis, and humanitarian aid have led to greater stability and better treatment options for people living with haemophilia across the globe. This positive news allows doctors and researchers to raise the ambition to improving physical activity and the process of ageing with haemophilia for the first time. 

Below you will find two tables. Each column in both tables are filterable, from highest to lowest, and vice versa. The full methodology explaining every indicator and how it was measured, as well as explanations of various terms in the data, can be found at the bottom of the page.

The first table shows in the first three columns key figures per country based on the WFH Annual Global Survey and in the last three columns the results of a separate survey of medical professionals from Haemophilia Treatment Centres (HTCs) across 19 European countries, in alphabetical order. The first three indicator columns are shown as numbers. The last three indicator columns are scored out of 100; 100 representing countries with the highest aggregate survey ratings. Descriptions of each indicator and the questions asked can be found in the methodology below.

The second table shows the percentages of medication for the treatment of haemophilia provided through humanitarian aid in 46 countries around the world based on the WFH Annual Global Survey, in alphabetical order. 

PLEASE NOTE: DATA INCLUDED FROM THE WORLD FEDERATION OF HAEMOPHILIA ANNUAL GLOBAL REPORTS IS SELF REPORTED FROM PATIENT ASSOCIATIONS.

 

Methodology

 

The study is divided into two sections: the results of a survey of medical professionals from European Haemophilia Treatment Centres (HTCs) in 19 countries, and an index of 46 countries showing the percentage of medication provided by the World Federation of Hemophilia Humanitarian Aid Program for haemophilia treatment across the globe. Combined, the two sections take into account a total of seven indicators:

 
Table 1:

Factor VIII IU / 1,000 capita (WFH Annual Global Survey reports)

Factor IX IU / 1,000 capita (WFH Annual Global Survey reports)

Registered People with Haemophilia / 1,000,000 capita (WFH Annual Global Survey reports)

Physical Activity Levels (survey responses)

Quality of Life (survey responses)

Satisfaction Levels (survey responses)

 
Table 2:

Factor Provided by Humanitarian Aid

% of Factor Provided by Humanitarian Aid (WFH Annual Global Survey reports 2016-2018)

 

Table 1:

European Survey Results

Medical professionals from Haemophilia Treatment Centres (HTCs) across 19 European countries were surveyed over March 2020, resulting in 280 responses. The respondents were asked to answer a number of questions regarding their opinions on their respective countries’ treatment options and ability to offer quality of life to people with haemophilia. Descriptions of each indicator and the questions asked can be found below.

The score given to each indicator is determined by calculating the Standard Score, often referred to as the Z-Score of each indicator; averaging all Z-Scores and standardizing the resulting score using a minmax normalization.

The formula for calculating the Standard Score/Z-Score is as follows:

z = (x - avg(X)) / S ; where ‘x’ is the indicator value for each country, avg(X) is the average of all indicator values in the index and S is the standard deviation of the indicator values in the index.

The formula for the minmax normalization is as follows:

Minmax = (x - min(X))/(max(X)-min(X))*99+1

 

Physical Activity Levels

The degree to which people with severe haemophilia in each country can participate in varying levels of physical activity. The question was formulated as follows:

“For persons with severe haemophilia, to what extent is a person with haemophilia able to engage in the following activities?”

Each professional was asked to rate the following activities on a scale of 1 to 7. (1=  there is no possibility for patients to engage in this type of activity, and 7 = it's possible for patients to engage in this type of activity at a competitive level):

Light exercise - (e.g. aerobics, swimming); Moderate exercise - (e.g. diving, cycling, jogging); Intense exercise - (e.g. BMX racing, powerlifting); Low-impact contact sports - (e.g. basketball, soccer, volleyball); High-impact contact sport - (e.g. wrestling, rugby, hockey)

The score for the five activities was then combined into an aggregate score which gave more weight to higher-impact activities.

 

Quality of Life

The degree to which people with severe haemophilia in each country are able to maintain quality of life. The question was formulated as follows:

“Given the medical treatment and social support available today in your country, what quality of life can a person diagnosed today with severe haemophilia before the age of 3 expect over their lifetime?”

Each professional was asked to rate perceived quality of life on a scale of 1 to 7. (1 = extremely reduced quality of life, compared to persons without haemophilia and 7 = the same quality of life compared to persons without haemophilia).

 

Satisfaction Levels

The degree to which people with severe haemophilia in each country are satisfied with the overall care they receive. The question was formulated as follows:

“Please estimate how satisfied, on average, severe haemophilia patients are in your country with the overall care they receive?”

Each professional was asked to rate perceived satisfaction on a scale of 1 to 7. (1 = very unsatisfied and 7 =extremely satisfied).

To provide context to the survey responses, additional data collected by the World Hemophilia Federation on the amount of coagulant factor available per capita and the number of persons registered with haemophilia was included.

The information displayed is based on the latest data available from the World Hemophilia Federation (WFH) Global Annual Survey report (2016-2018). Where figures are marked with an ‘n/a’, country data was not available.

 

Factor VIII IU / 1,000 Capita        

The amount of available medication used to treat and prevent bleeding in people with haemophilia A and other causes of low Factor VIII, per 1,000 citizens. 

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Factor IX IU / 1,000 Capita
The amount of available medication in each country used to treat and prevent bleeding in people with haemophilia B and other causes of low Factor IX, per 1,000 citizens. 

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Registered People with Haemophilia / 1,000,000 Capita 

The number of people with Haemophilia A and B in each country per 1,000,000 citizens.

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Table 2:

% of Factor Provided by Humanitarian Aid
The Percentage of Factor VIII or IX replacement medication made available in each country around the world through humanitarian aid.

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

Haemophilia Index 2020

To mark the 30th annual World Haemophilia Day, Sobi has released new data showing the
different opportunities available for people living with haemophilia in Europe and around the world.

As part of our Liberate Life vision, we compiled a study comprising a European survey examining quality of life for people with haemophilia in Europe, and a global index looking into the state of international care through the far-reaching effects of humanitarian aid. The result is the two tables below that give insights into treatment for people living with haemophilia around the world.

For the first part of the study, we asked specialised healthcare professionals across Europe for their opinions on the levels of physical activity, quality of life and satisfaction experienced by people with haemophilia with access to treatment in their countries. To provide context, data relating to the amount of medication and rates of haemophilia in these countries was also included. The second part of the study further focuses on the reach of humanitarian aid by showing the percentages of haemophilia medication provided to countries across the globe.

Although many challenges remain, the results show that advances in diagnosis, improved access to prophylaxis, and humanitarian aid have led to greater stability and better treatment options for people living with haemophilia across the globe. This positive news allows doctors and researchers to raise the ambition to improving physical activity and the process of ageing with haemophilia for the first time. 

Below you will find two tables. Each column in both tables are filterable, from highest to lowest, and vice versa. The full methodology explaining every indicator and how it was measured, as well as explanations of various terms in the data, can be found at the bottom of the page.

The first table shows in the first three columns key figures per country based on the WFH Annual Global Survey and in the last three columns the results of a separate survey of medical professionals from Haemophilia Treatment Centres (HTCs) across 19 European countries, in alphabetical order. The first three indicator columns are shown as numbers. The last three indicator columns are scored out of 100; 100 representing countries with the highest aggregate survey ratings. Descriptions of each indicator and the questions asked can be found in the methodology below.

The second table shows the percentages of medication for the treatment of haemophilia provided through humanitarian aid in 46 countries around the world based on the WFH Annual Global Survey, in alphabetical order. 

PLEASE NOTE: DATA INCLUDED FROM THE WORLD FEDERATION OF HAEMOPHILIA ANNUAL GLOBAL REPORTS IS SELF REPORTED FROM PATIENT ASSOCIATIONS.

 

Methodology

 

The study is divided into two sections: the results of a survey of medical professionals from European Haemophilia Treatment Centres (HTCs) in 19 countries, and an index of 46 countries showing the percentage of medication provided by the World Federation of Hemophilia Humanitarian Aid Program for haemophilia treatment across the globe. Combined, the two sections take into account a total of seven indicators:

 
Table 1:

Factor VIII IU / 1,000 capita (WFH Annual Global Survey reports)

Factor IX IU / 1,000 capita (WFH Annual Global Survey reports)

Registered People with Haemophilia / 1,000,000 capita (WFH Annual Global Survey reports)

Physical Activity Levels (survey responses)

Quality of Life (survey responses)

Satisfaction Levels (survey responses)

 
Table 2:

Factor Provided by Humanitarian Aid

% of Factor Provided by Humanitarian Aid (WFH Annual Global Survey reports 2016-2018)

 

Table 1:

European Survey Results

Medical professionals from Haemophilia Treatment Centres (HTCs) across 19 European countries were surveyed over March 2020, resulting in 280 responses. The respondents were asked to answer a number of questions regarding their opinions on their respective countries’ treatment options and ability to offer quality of life to people with haemophilia. Descriptions of each indicator and the questions asked can be found below.

The score given to each indicator is determined by calculating the Standard Score, often referred to as the Z-Score of each indicator; averaging all Z-Scores and standardizing the resulting score using a minmax normalization.

The formula for calculating the Standard Score/Z-Score is as follows:

z = (x - avg(X)) / S ; where ‘x’ is the indicator value for each country, avg(X) is the average of all indicator values in the index and S is the standard deviation of the indicator values in the index.

The formula for the minmax normalization is as follows:

Minmax = (x - min(X))/(max(X)-min(X))*99+1

 

Physical Activity Levels

The degree to which people with severe haemophilia in each country can participate in varying levels of physical activity. The question was formulated as follows:

“For persons with severe haemophilia, to what extent is a person with haemophilia able to engage in the following activities?”

Each professional was asked to rate the following activities on a scale of 1 to 7. (1=  there is no possibility for patients to engage in this type of activity, and 7 = it's possible for patients to engage in this type of activity at a competitive level):

Light exercise - (e.g. aerobics, swimming); Moderate exercise - (e.g. diving, cycling, jogging); Intense exercise - (e.g. BMX racing, powerlifting); Low-impact contact sports - (e.g. basketball, soccer, volleyball); High-impact contact sport - (e.g. wrestling, rugby, hockey)

The score for the five activities was then combined into an aggregate score which gave more weight to higher-impact activities.

 

Quality of Life

The degree to which people with severe haemophilia in each country are able to maintain quality of life. The question was formulated as follows:

“Given the medical treatment and social support available today in your country, what quality of life can a person diagnosed today with severe haemophilia before the age of 3 expect over their lifetime?”

Each professional was asked to rate perceived quality of life on a scale of 1 to 7. (1 = extremely reduced quality of life, compared to persons without haemophilia and 7 = the same quality of life compared to persons without haemophilia).

 

Satisfaction Levels

The degree to which people with severe haemophilia in each country are satisfied with the overall care they receive. The question was formulated as follows:

“Please estimate how satisfied, on average, severe haemophilia patients are in your country with the overall care they receive?”

Each professional was asked to rate perceived satisfaction on a scale of 1 to 7. (1 = very unsatisfied and 7 =extremely satisfied).

To provide context to the survey responses, additional data collected by the World Hemophilia Federation on the amount of coagulant factor available per capita and the number of persons registered with haemophilia was included.

The information displayed is based on the latest data available from the World Hemophilia Federation (WFH) Global Annual Survey report (2016-2018). Where figures are marked with an ‘n/a’, country data was not available.

 

Factor VIII IU / 1,000 Capita        

The amount of available medication used to treat and prevent bleeding in people with haemophilia A and other causes of low Factor VIII, per 1,000 citizens. 

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Factor IX IU / 1,000 Capita
The amount of available medication in each country used to treat and prevent bleeding in people with haemophilia B and other causes of low Factor IX, per 1,000 citizens. 

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Registered People with Haemophilia / 1,000,000 Capita 

The number of people with Haemophilia A and B in each country per 1,000,000 citizens.

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.

 

Table 2:

% of Factor Provided by Humanitarian Aid
The Percentage of Factor VIII or IX replacement medication made available in each country around the world through humanitarian aid.

Sources: WFH Annual Global Survey reports (2016-2018), country population statistics 2008-2018.