Haemophilia (2014), 20 (Suppl. 4), 1–3
DOI: 10.1111/hae.12430
FOREWORD
A message from WFH President Alain Weill: Broad perspectives for a new era Standing at the edge of the second 50 years of the World Federation of Hemophilia, I see the marks of a changing landscape before us, with innovation potentially heralding a new era for our community and for the bleeding disorders industry. Disruptive innovation [1] is a powerful thing. In the midst of the Digital Revolution, analyst Clayton Christensen coined the term to describe the way businesses could have far-reaching, seismic effects on customers by employing emerging technologies. Just as email effectively killed the hand-stamped letter, the mobile device has changed the way we do everything from reading books to watching sports. What will this new era hold? Some analysts see longer acting products that will alter and confront accepted methods of treatment; a potential redistribution of the existing products because of lower prices of recombinants, driven by companies having to look for new markets, with a related potential for surplus; and new players who will challenge the incumbents as they bring gene-transfer therapies and treatments to trial and begin to market them [2]. Innovation in the pharmaceutical industry will forever change the landscape in which it operates. And our community will feel the impact of that change in all its aspects. At the WFH, we are here to serve all who have bleeding disorders and we welcome all treatment products as long as they are safe and prove useful to those individuals and countries in need. And this movement in the industry seems rather positive as it indicates we may have greater quantities and variety of products with which to treat those in need. Against this backdrop of changing faces and products, we will also see a shift from an economic perspective. Developing markets – the BRICS countries (Brazil, Russia, India, China and South Africa) as well as Mexico, Turkey and Ukraine – will account for one-third of the world’s GDP by 2020 – just 6 years away [3]. Twenty years beyond that, if infrastructure, regulatory environments and distribution systems evolve and improve, developing markets will have caught up with more mature markets. While disruption will be cause for much reflection on – and re-definition of – business models, for people affected by a bleeding disorder, it heralds a time of celebration for a number of reasons [2]: © 2014 John Wiley & Sons Ltd
• • • •
Factor replacement therapy usage per patient will increase in developing markets; The improved convenience of longer acting therapies in the pipeline could begin to encourage more people, particularly adults, to consider ongoing prophylaxis therapy; We expect to see acceptance of the real possibility of lower priced recombinant and possibly plasmaderived therapies in emerging markets; We will come closer to achieving treatment for all.
These are opportunities we cannot afford to miss. Collectively, we must connect more effectively to technology and not allow ourselves to be complacent. We cannot stop advocating for better treatment, and better access to treatment, regardless of geography, the level of medical knowledge, or the wealth of a nation. The bleeding disorders community has the power, the means, and the potential knowledge to bridge, and eradicate, the divide between the developed and developing worlds we serve. As we look at what it will take to provide optimal levels of treatment across the world, we must broaden our perspective to redefine the roles each of us will play to bring that about. If we look at world demographics and the number of people with inherited bleeding disorders, we will see that reconciling the healthcare needs of the rich and the poor is one of the biggest challenges we face as a society. There is a strong correlation between socioeconomic status, availability of medicine, and access to care. The reach of communications technology in under-served areas provides a perspective on an under-utilized means to access and care: mobile technology. In Central Asia, mobile penetration stands at 90%, whereas Western Europe has 129% penetration, North America 101%, and the world average is 93% [4]. Communications technology has become the great equalizer, and has democratized access to information. It is clear that we have the technological means to reach out to the estimated seven million under-diagnosed and under-treated people in the world. And thanks to the variety of WFH programmes that seek to level the quality and access to care between the two groups, we are well positioned, better than at any time in our history in fact, to reach out.
1
2
A. WEILL
As the WFH continues to focus on advancing the best available treatment to all who need it, we firmly believe advances will be the result of innovation on all fronts, but particularly in gene therapy that offers hope for a cure; the development of longer acting factor concentrates that offer an improved quality of life and distribution methods. Innovation will continue to lead our focus and direction for the next decade, influencing how we perceive and train members for advocacy, as well as how we perceive the role of education and its inherent processes. We understand that we need to continue to evolve to meet the needs of our members, our NMOs, our partners, our staff, and most especially, the people for whom we work. Innovation was also on our mind as we looked within to determine if the WFH was structured as effectively as possible. As a result, we have realigned with an eye toward becoming a more agile and responsive organization. One of the more significant changes was the decision to bring renewed emphasis to education, web technology and communications in our strategic planning. Education for people with bleeding disorders results not only in their empowerment; it also empowers our global community. Developing a strong organization for advocacy and education is a vital component of the WFH comprehensive care programme. In fact, it is now accepted that the therapeutic education of people with bleeding disorders improves their long-term health prospects. Education is necessary and must be at the heart of our community actions. The WFH must continue to promote global programmes centred on the specific needs of people with bleeding disorders and adapted to their personal and cultural realities [5]. Education also has an important role in promoting standards of care and, with all the emerging treatment products, our community will turn to us for information about how they may change the treatment paradigm. We are also committed to developing new digital tools for both educational and communications purposes. One component of the research programme we launched last year is a data collection system, developed to increase accessibility, create a user-friendly interface, and provide live backups so the WFH can improve the quality of data it collects and thereby enhance advocacy efforts. Currently undergoing testing, the system will be ready to launch in time for the 2014 annual global survey. We also plan to collect data on specific research questions from selected NMOs or major treatment centres around the world. We may be able to address many unresolved haemophilia management issues with this programme and potentially provide better care. Along with innovation and education, solidarity among all stakeholders in its myriad forms must play
Haemophilia (2014), 20 (Suppl. 4), 1--3
a key role in helping us meet our goals. Indeed, collaboration – of a kind rich in potential but as yet untried – lies at the heart of re-defining the roles each of us plays to provide the most attainable levels of treatment to all people with bleeding disorders, regardless of socioeconomic standing and geography. One example of innovative collaboration is our recent joint agreement with the French Hemophilia Association and the National Institute of Blood Transfusion to establish basic haemophilia care programmes in French-speaking or western Africa. African countries with strained economies cannot benefit from our programmes unless they are WFH members, which requires a patient organization and, of course, identified and diagnosed patients. This new programme will create the much-needed foundation to establish basic diagnostic facilities for haemophilia and develop basic training programmes. Like other WFH programmes such as Hemophilia Treatment Centre (HTC) twinning and Hemophilia Organization Twinning (HOT), this programme, called AFATH (Franco-African alliance for the treatment of haemophilia), enables participants to share knowledge, expertise, experience and resources. The recent twinning of a South African HTC with one from Mauritius is a case in point. Not too long ago, the South African HTC was on the receiving end of knowledge transfer; today, it delivers it. However, another exciting example of innovative collaboration is Project Recovery, a humanitarian project that is finally reaching fruition after many years. Originally conceived and supported by the Canadian Hemophilia Society and Canadian Blood Services, Project Recovery will see people with haemophilia benefit from a new source of factor VIII from Canadian blood donations. This is a milestone for the haemophilia community, and the WFH is privileged to be part of the remarkable collaboration that has made it possible. Project Recovery required the negotiation of a number of complex agreements among all the parties involved, and hopefully it will serve as a model of collaboration and international solidarity to reclaim essential, life saving proteins. Project Recovery provides a new model for humanitarian aid and one that bodes well for the future. Along with our three major strategic thrusts – the ongoing Global Alliance for Progress and country programmes, the Cornerstone Initiative, and the WFH Research Program – our focus on innovation in all its guises will help ensure that our vision of Treatment for All becomes reality during our second half-century. As we exchange ideas at this 2014 World Congress, let us look for innovation in technological, scientific and clinical advances. Let us also ensure there is innovation in improving the quality of life for the many thousands with undiagnosed or undertreated bleeding
© 2014 John Wiley & Sons Ltd
MESSAGE FROM WFH PRESIDENT
disorders. Let us marshal our collective efforts to help achieve treatment for all.
Disclosures
3
Alain Weill World Federation of Hemophilia, 1425 ouest, boul. Rene-Levesque, Bureau 1010, Montreal (Quebec) H3G 1T7, Canada Tel: (514) 875-7944
The author has no interest which might be perceived as posing a conflict or bias.
References 1 Available at http://www.claytonchristensen. com. Accessed March 31, 2014. 2 Healthcare Observer Jan 2013. Available at: http://corporate.morningstar.com/us/html/pdf/ Healthcare-Observer-Jan-2013.pdf. Accessed March 31, 2014.
© 2014 John Wiley & Sons Ltd
3 From Vision to Decision: Pharma 2020. Available at: http://www.pwchk.com/web media/doc/635022359823422732_pharma 2020_vision_decision_apr2013.pdf. Accessed March 31, 2014. 4 We Are Social. Global Digital Statistics2014. Available at: http://www.slideshare.net/ wearesocialsg/social-digital-mobile-around-
the-world-january-2014. Accessed March 31, 2014. 5 Bensing JM. Doctor-patient communication and the quality of care. Soc Sci Med 1991; 32: 1301–10.
Haemophilia (2014), 20 (Suppl. 4), 1--3
DOI: 10.1111/hae.12430
FOREWORD
A message from WFH President Alain Weill: Broad perspectives for a new era Standing at the edge of the second 50 years of the World Federation of Hemophilia, I see the marks of a changing landscape before us, with innovation potentially heralding a new era for our community and for the bleeding disorders industry. Disruptive innovation [1] is a powerful thing. In the midst of the Digital Revolution, analyst Clayton Christensen coined the term to describe the way businesses could have far-reaching, seismic effects on customers by employing emerging technologies. Just as email effectively killed the hand-stamped letter, the mobile device has changed the way we do everything from reading books to watching sports. What will this new era hold? Some analysts see longer acting products that will alter and confront accepted methods of treatment; a potential redistribution of the existing products because of lower prices of recombinants, driven by companies having to look for new markets, with a related potential for surplus; and new players who will challenge the incumbents as they bring gene-transfer therapies and treatments to trial and begin to market them [2]. Innovation in the pharmaceutical industry will forever change the landscape in which it operates. And our community will feel the impact of that change in all its aspects. At the WFH, we are here to serve all who have bleeding disorders and we welcome all treatment products as long as they are safe and prove useful to those individuals and countries in need. And this movement in the industry seems rather positive as it indicates we may have greater quantities and variety of products with which to treat those in need. Against this backdrop of changing faces and products, we will also see a shift from an economic perspective. Developing markets – the BRICS countries (Brazil, Russia, India, China and South Africa) as well as Mexico, Turkey and Ukraine – will account for one-third of the world’s GDP by 2020 – just 6 years away [3]. Twenty years beyond that, if infrastructure, regulatory environments and distribution systems evolve and improve, developing markets will have caught up with more mature markets. While disruption will be cause for much reflection on – and re-definition of – business models, for people affected by a bleeding disorder, it heralds a time of celebration for a number of reasons [2]: © 2014 John Wiley & Sons Ltd
• • • •
Factor replacement therapy usage per patient will increase in developing markets; The improved convenience of longer acting therapies in the pipeline could begin to encourage more people, particularly adults, to consider ongoing prophylaxis therapy; We expect to see acceptance of the real possibility of lower priced recombinant and possibly plasmaderived therapies in emerging markets; We will come closer to achieving treatment for all.
These are opportunities we cannot afford to miss. Collectively, we must connect more effectively to technology and not allow ourselves to be complacent. We cannot stop advocating for better treatment, and better access to treatment, regardless of geography, the level of medical knowledge, or the wealth of a nation. The bleeding disorders community has the power, the means, and the potential knowledge to bridge, and eradicate, the divide between the developed and developing worlds we serve. As we look at what it will take to provide optimal levels of treatment across the world, we must broaden our perspective to redefine the roles each of us will play to bring that about. If we look at world demographics and the number of people with inherited bleeding disorders, we will see that reconciling the healthcare needs of the rich and the poor is one of the biggest challenges we face as a society. There is a strong correlation between socioeconomic status, availability of medicine, and access to care. The reach of communications technology in under-served areas provides a perspective on an under-utilized means to access and care: mobile technology. In Central Asia, mobile penetration stands at 90%, whereas Western Europe has 129% penetration, North America 101%, and the world average is 93% [4]. Communications technology has become the great equalizer, and has democratized access to information. It is clear that we have the technological means to reach out to the estimated seven million under-diagnosed and under-treated people in the world. And thanks to the variety of WFH programmes that seek to level the quality and access to care between the two groups, we are well positioned, better than at any time in our history in fact, to reach out.
1
2
A. WEILL
As the WFH continues to focus on advancing the best available treatment to all who need it, we firmly believe advances will be the result of innovation on all fronts, but particularly in gene therapy that offers hope for a cure; the development of longer acting factor concentrates that offer an improved quality of life and distribution methods. Innovation will continue to lead our focus and direction for the next decade, influencing how we perceive and train members for advocacy, as well as how we perceive the role of education and its inherent processes. We understand that we need to continue to evolve to meet the needs of our members, our NMOs, our partners, our staff, and most especially, the people for whom we work. Innovation was also on our mind as we looked within to determine if the WFH was structured as effectively as possible. As a result, we have realigned with an eye toward becoming a more agile and responsive organization. One of the more significant changes was the decision to bring renewed emphasis to education, web technology and communications in our strategic planning. Education for people with bleeding disorders results not only in their empowerment; it also empowers our global community. Developing a strong organization for advocacy and education is a vital component of the WFH comprehensive care programme. In fact, it is now accepted that the therapeutic education of people with bleeding disorders improves their long-term health prospects. Education is necessary and must be at the heart of our community actions. The WFH must continue to promote global programmes centred on the specific needs of people with bleeding disorders and adapted to their personal and cultural realities [5]. Education also has an important role in promoting standards of care and, with all the emerging treatment products, our community will turn to us for information about how they may change the treatment paradigm. We are also committed to developing new digital tools for both educational and communications purposes. One component of the research programme we launched last year is a data collection system, developed to increase accessibility, create a user-friendly interface, and provide live backups so the WFH can improve the quality of data it collects and thereby enhance advocacy efforts. Currently undergoing testing, the system will be ready to launch in time for the 2014 annual global survey. We also plan to collect data on specific research questions from selected NMOs or major treatment centres around the world. We may be able to address many unresolved haemophilia management issues with this programme and potentially provide better care. Along with innovation and education, solidarity among all stakeholders in its myriad forms must play
Haemophilia (2014), 20 (Suppl. 4), 1--3
a key role in helping us meet our goals. Indeed, collaboration – of a kind rich in potential but as yet untried – lies at the heart of re-defining the roles each of us plays to provide the most attainable levels of treatment to all people with bleeding disorders, regardless of socioeconomic standing and geography. One example of innovative collaboration is our recent joint agreement with the French Hemophilia Association and the National Institute of Blood Transfusion to establish basic haemophilia care programmes in French-speaking or western Africa. African countries with strained economies cannot benefit from our programmes unless they are WFH members, which requires a patient organization and, of course, identified and diagnosed patients. This new programme will create the much-needed foundation to establish basic diagnostic facilities for haemophilia and develop basic training programmes. Like other WFH programmes such as Hemophilia Treatment Centre (HTC) twinning and Hemophilia Organization Twinning (HOT), this programme, called AFATH (Franco-African alliance for the treatment of haemophilia), enables participants to share knowledge, expertise, experience and resources. The recent twinning of a South African HTC with one from Mauritius is a case in point. Not too long ago, the South African HTC was on the receiving end of knowledge transfer; today, it delivers it. However, another exciting example of innovative collaboration is Project Recovery, a humanitarian project that is finally reaching fruition after many years. Originally conceived and supported by the Canadian Hemophilia Society and Canadian Blood Services, Project Recovery will see people with haemophilia benefit from a new source of factor VIII from Canadian blood donations. This is a milestone for the haemophilia community, and the WFH is privileged to be part of the remarkable collaboration that has made it possible. Project Recovery required the negotiation of a number of complex agreements among all the parties involved, and hopefully it will serve as a model of collaboration and international solidarity to reclaim essential, life saving proteins. Project Recovery provides a new model for humanitarian aid and one that bodes well for the future. Along with our three major strategic thrusts – the ongoing Global Alliance for Progress and country programmes, the Cornerstone Initiative, and the WFH Research Program – our focus on innovation in all its guises will help ensure that our vision of Treatment for All becomes reality during our second half-century. As we exchange ideas at this 2014 World Congress, let us look for innovation in technological, scientific and clinical advances. Let us also ensure there is innovation in improving the quality of life for the many thousands with undiagnosed or undertreated bleeding
© 2014 John Wiley & Sons Ltd
MESSAGE FROM WFH PRESIDENT
disorders. Let us marshal our collective efforts to help achieve treatment for all.
Disclosures
3
Alain Weill World Federation of Hemophilia, 1425 ouest, boul. Rene-Levesque, Bureau 1010, Montreal (Quebec) H3G 1T7, Canada Tel: (514) 875-7944
The author has no interest which might be perceived as posing a conflict or bias.
References 1 Available at http://www.claytonchristensen. com. Accessed March 31, 2014. 2 Healthcare Observer Jan 2013. Available at: http://corporate.morningstar.com/us/html/pdf/ Healthcare-Observer-Jan-2013.pdf. Accessed March 31, 2014.
© 2014 John Wiley & Sons Ltd
3 From Vision to Decision: Pharma 2020. Available at: http://www.pwchk.com/web media/doc/635022359823422732_pharma 2020_vision_decision_apr2013.pdf. Accessed March 31, 2014. 4 We Are Social. Global Digital Statistics2014. Available at: http://www.slideshare.net/ wearesocialsg/social-digital-mobile-around-
the-world-january-2014. Accessed March 31, 2014. 5 Bensing JM. Doctor-patient communication and the quality of care. Soc Sci Med 1991; 32: 1301–10.
Haemophilia (2014), 20 (Suppl. 4), 1--3
