WFH – the cornerstone of global development: 45 years of progress

Measuring success – the WFH Global Survey

To achieve optimal results, decision-making processes for healthcare planning require information on the needs of the patient population, the necessary resources, and the expected results if resources are applied [8]. Before looking at individual country examples, it is noteworthy to look at what has occurred at the global level over the past decade. In 1998, the WFH conducted the first global survey to track progress on key indicators of the level of care and treatment. The WFH compiled the first report on these data in 1999 and has since published reports annually [9–16]. Quantifiable measures contained in the WFH Global Survey document the significant impact of the WFH development programs.

Since 1998, the number of people identified with haemophilia and other disorders worldwide has increased from 103 435 in 65 countries [9] to 208 006 in 100 countries, representing 88% of the world’s population [16]. As part of the enhanced mission of the WFH, in 2004 the survey was expanded to incorporate patients with rare factor deficiencies (Factor I, II, V, V + VIII, X, XI, XIII). In 2006, inherited platelet disorders (e.g. Glanzmann thromboasthenia, Bernard Soulier syndrome) were incorporated [9–16] (Fig. 2).

Another measurement to track the impact of WFH development programmes is the increase in availability of clotting factor concentrates. The WFH has established that one international unit (IU) of FVIII clotting factor concentrate per capita should be the target minimum for countries wishing to achieve optimal survival for the haemophilia population [8]. The 2001 survey first reported data on the usage of factor concentrates. Since 2001, the availability of FVIII clotting factor concentrates in countries with <US$2000 Gross Domestic Product (GDP) has increased from 0.012 to 0.02 IU per capita [11–16]. In countries with a GDP between US$2000 and $10 000 GDP, the IU per capita has steadily increased from 0.64 to 0.95 [11–16]. Developed countries with an IU per capita of >US$10 000 have increased from 3.3 to 4.5 IU per capita over a similar period [11–16]. Overall, worldwide factor consumption has increased in this period (2001–2006) and global per capita consumption is now above 1 IU. Although countries in the highest economic category consume 3 to 4 times the amount of factor per capita than those in the second category, the rate of increase worldwide is fairly consistent across all three economic categories. To avoid countries shifting between categories year over year and allow the observation of trends in consistent groups of countries over time, 2006 economic rankings have been applied to all years. The drop in IU per capita from 2001 to 2002 can be attributed to the worldwide shortage of clotting factor concentrates during this period (Fig. 3).

The WFH Development Model

Through many years of experience, the WFH has identified and optimized the essential elements of a model for the development of a sustainable national care program. The five elements of The WFH Development Model are integrated and interdependent:

Later sections in this article will discuss each of these elements and provide examples of proven and quantifiable outcomes.

Closing the GAP – the Global Alliance for Progress project

This year marks the fifth anniversary of the Global Alliance for Progress (GAP) project. In announcing plans for GAP in 2002, then WFH president Brian O’Mahony explained, ‘Our challenge is to close the gap: the gap between the estimated and actual number of people with haemophilia; the gap between the amount of treatment products needed versus that available, the gap between the number of people born with haemophilia and the number who reach adulthood’ [17].

The GAP project was established to introduce or improve national programmes for patients with bleeding disorders in up to 30 targeted countries over 10 years. At the halfway point, 14 countries have been selected for GAP Armenia, Azerbaijan, Ecuador, Egypt, Georgia, Jordan, Lebanon, Mexico, Philippines, Thailand and Russia. In 2008, Belarus and Tunisia joined the list, along with China in preparatory phase.

Within GAP, the WFH integrates and simultaneously addresses all five areas of the Development Model. Across the board, significant progress is already evident.

1. The importance of being counted – ensuring accurate laboratory diagnosis 

As a rare disease group, it is especially true that there is strength in numbers. In addition to the need for global comparative data such as that contained in the annual reports of the WFH global surveys, building a robust database of credible data at the national level is also essential. The primary vehicle for collecting long-term observational and planning data is a national patient registry.

The WFH Guide to Developing a National Patient Registry recommends the development of registries through collaboration between national patient organizations, healthcare professionals, treatment centres and ministries of health [18]. Due to the work of the WFH since this guide was published in 2005, the number of countries adopting a registry has increased from 41 (43% of reporting countries) to 58 (58% of reporting countries) [14,16]. The increased number of countries reporting data from a national registry has led to a continual and significant improvement in the data quality of the annual WFH Global Survey.

Data collection is not an end in itself, but a means to build, preserve and promote the overall quality of care. Registries are beneficial for patients and their quality of life, to doctors and nurses for the identification of changing care needs, and to the public health authorities for a better management of care requirements and resources [19]. A national registry will [20]:

For example, there was no national patient registry when GAP started in Mexico in 2003. The Hemophilia Federation of the Republic of Mexico developed a national registry and organized regional outreach projects to identify and register patients. Outreach projects in Mexico City and Mexico State were especially successful, identifying 406 new people with haemophilia in only 1 year. Now, the patient registry includes 3625 people with haemophilia and 67 with von Willebrand’s disease. Of these, 1527 are newly identified patients [13–16]. The registry served as a catalyst to organize care, establish a national network and now provides an important planning resource.

Closely related to the development of a national patient registry is ensuring accurate laboratory diagnosis. The WFH laboratory manual published in 2000 is the essential training and lab reference guide utilized to enhance the accuracy of laboratory diagnosis [21]. Additionally, the WFH–World Health Organization (WHO) International External Quality Assessment Scheme (IEQAS), launched in 2004, monitors and improves laboratory performance in haemophilia treatment centres (HTC) worldwide. Seventy-seven HTCs from 50 countries are registered with the scheme. Based on regular reports, the overall standard of laboratory performance has achieved a 90% accuracy rating [27].

Outreach and accurate diagnosis are the essential first steps to improving treatment. A core objective of GAP is to increase the overall number of patients diagnosed by 50 000 worldwide. Globally, since 2003, 41 395 new patients with bleeding disorders have been identified including 31 189 with haemophilia [13–16].

For each GAP country, the WFH seeks to validate the total number of diagnosed patients when enrolling a country in the project. To ensure accuracy, rediagnosis is sometimes necessary. Within GAP, over 8500 newly identified patients with haemophilia, von Willebrand’s disease and other rare factor deficiencies have been added to national registries [13–16,22] (Fig. 4).

2. Building a winning coalition – achieving government support for a national programme 

Formation of a coalition made up of patients and their families, healthcare professionals, and the Ministry of Health working in harmony with industry and other stakeholders is a key feature of the WFH Development Model. Experience has shown that a coalition under the umbrella of the WFH is the optimal pathway to achieve sustainable treatment improvements. In fact, formation of such partnerships is a founding principle upon which Mr Schnabel built his vision. ‘I urge an international declaration of war on haemophilia. Create a pact, which will enable strong leadership to utilize a strategy, which exploits openings by concentrating our limited weapons and personnel. Be ready to sacrifice sovereignty in the search of allies. Employ a criteria in deliberations and decision that gives first priority to the haemophiliac, even though the new organization and its medical advisors are indispensable’ [3].

Predicating GAP is the formation of such coalitions. In GAP countries, formal agreements committing the WFH, national patient organizations and the Ministry of Health to collaborate in developing and strengthening a national healthcare programme for patients with bleeding disorders have been signed (Armenia, Azerbaijan, Ecuador, Egypt, Georgia, Jordan, Lebanon, Thailand and Tunisia).

In such an agreement, a Ministry of Health would typically agree to establish a national programme, establish an HTC and increase the purchase of clotting factor concentrates. In exchange, the WFH would agree to implement a tailored development plan. Elements of such a plan include a commitment by the WFH to provide professional training for a multidisciplinary team of healthcare professionals, translating educational materials, enrolling a local diagnostic laboratory into the WFH–WHO IEQAS, helping to establish a national patient registry and building the capacity of the national patient organization.

With the establishment of a national programme, major breakthroughs have also been achieved in many GAP countries to increase the level of government support. For instance, the governments of Azerbaijan, Georgia, Jordan, Russia and Thailand have more than doubled their national programme budgets over the last 5 years.

3. Promoting comprehensive care – improving the care delivery system 

The collective vision of Treatment for All is not based solely on achieving improved access to clotting factor concentrates. While such access is the first thought for many, treatment means much more. Treatment for All also means proper diagnosis, management, and care by a multidisciplinary team of trained specialists within a comprehensive care setting [23].

Comprehensive care requires a broad range of healthcare providers and support systems. The improved outcomes in morbidity and mortality when comprehensive care occurs within an HTC setting are established [24]. Trained physical therapists, nurses, dentists, social workers and laboratory technicians are critical to the care team and serve as the backbone of care, especially where treatment products are limited.

Training all members of the multidisciplinary comprehensive care team is a central feature of GAP. Within GAP, the WFH has provided specialized training to 2478 care team members and general education on bleeding disorders to 4441 health professionals [22,25].

Also, the WFH International Hemophilia Training Centre (IHTC) fellowship programme has been providing intensive specialized training to members of the multidisciplinary team since its inception in 1969. The oldest WFH programme, it has been referred to as the ‘Jewel in the Crown’ of the WFH [26]. Fellows undergo 6–8 weeks of training at a WFH-designated training centre. The immense competition among applicants to earn a fellowship is a testament to the prestige and quality of the training. Postfellowship, the network of fellows provides an important base for many other WFH development programs. Since the beginning of the programme, 345 individuals from 74 countries covering all medical disciplines have been awarded fellowships to train at one of the 31 recognized training centres [26,27]. In 2006, a long-term evaluation of the impact of the IHTC programme was conducted of 109 fellows from 2000 through 2005. The study found 70.6% of the 77 respondents from 40 countries have remained associated with haemophilia care since completing their training [28].

More extensive training occurs through the WFH HTC Twinning programme. Twinning is a formal, two-way collaboration between emerging and established HTCs [29]. Each year approximately 30 twins are involved in collaborations. Twinning often serves as the catalyst for development of a national programme. For example, in 1997, the first twinning partnership was formed in China between Tianjin and Calgary, Canada. Later twins included Guangzhou – Ottawa, Canada and Shanghai – Calgary/Ottawa jointly. These three twinnings helped the WFH establish a network of Chinese medical centres equipped to manage haemophilia. This early work has led to the establishment of the China Hemophilia Treatment Centre Collaborative Network, laying the groundwork for China’s selection for the GAP project.

Each year, the WFH conducts more than a dozen national and regional workshops for members of the comprehensive care team [16]. In recent years, the WFH has focused on the importance of exercise, physical therapy and rehabilitation to improve the quality of life of patients. In Egypt during 2006, healthcare providers, the Egyptian Hemophilia Society, and the University of Cairo organized a training workshop for physical therapists. In 2007, the WFH and University of Cairo held a Train-the-Trainer workshop to teach physical therapists from around the country how to train other physical therapists on exercises and proper techniques. Now, Egypt is poised to go one step further and organize Train-the-Trainer workshops for physical therapists throughout the Middle Eastern region. WFH recently published an exercise guide as a supplement to the training curriculum [30].

The WFH has a rich multilingual library of publications to support educational, training and development needs. Over 90 treatment monographs, guidebooks, web-based training modules, fact sheets and organizational capacity-building tools are available covering all aspects of treatment for bleeding disorders and care development [31]. In 2005, the WFH published Guidelines for the Management of Hemophilia [23]. Around the world, translated and adapted guidelines serve as the standard for many national programmes.

4. Increasing the availability of treatment products

Increasing the availability of clotting factor concentrates is a significant turning point to bringing about adequate treatment. Since 2003, GAP countries have reported to the WFH a total cumulative increase of 467 million IU of clotting factor concentrates purchased [22,25]. Individually, GAP countries almost universally report an increase in FVIII consumption measured on a per capita basis when compared to the consumption level at GAP enrolment [13–16,22] (Fig. 5). The exception is Lebanon which, prior to the regional conflict, consistently reported an increased IU per capita each year.

Noteworthy is the growth of factor availability in Russia, which joined the GAP programme in 2004. At that time, treatment products were very limited and mainly available in St Petersburg and Moscow. The central government purchased 1.4 million IU of clotting factor concentrates in 2004. Over the years, the Russian Hemophilia Society, buoyed by the support of the WFH, successfully lobbied the government to purchase more factor concentrates. Today, treatment is available in every region throughout Russia and a large number of children are on prophylaxis. In 2007, the government purchased more than 200 million IU of clotting factor concentrates.

To help educate government officials, patients and healthcare professionals, the WFH has published two guidebooks on how to assess and purchase clotting factor concentrates [32,33]. Together, these guidebooks provide a foundation for the selection and purchase of products based first and foremost on product safety and efficacy.

5. Empowering patients – building a strong national patient organization 

An essential part of the winning coalition is the national patient organization. Without the involvement of a strong organization to promote care development and to educate patients and their families, success is unlikely.

Today, across nations, patient organizations have firmly established their role in decision making at the highest levels of government. Success in many countries, including within GAP, has been achieved by integrating patient organizations within a national programme, public health oversight committees and/or in the selection of treatment products.

The World Health Organization has also endorsed the participation of non-governmental organizations such as national haemophilia organizations to serve on National Committees on the Clinical Use of Blood [38].

Additionally, through GAP, the WFH in collaboration with national patient organizations has facilitated the education and training of 2974 patients, family members and organization board members [22,25]. Likewise, similar to the Twinning programme for HTCs, the WFH offers twinnings for patient organizations [39]. Each year, the WFH arranges nearly 20 twinning partnerships between established and developing patient organizations [27]. These partnerships play a crucial role in helping patient organizations become a powerful voice representing the interests of patients with bleeding disorders and as a driving force for improved treatment. The increased resources and training focused on patient organizations also ensure that treatment gains will continue long after a country development project concludes.

Tomorrow – future opportunities to develop care

While much progress is occurring through the development programmes of the WFH, other global challenges remain. To ensure the continued advance towards Treatment for All, it is vital that global collaboration occur on the research front as well. The most notable research challenges requiring global collaboration are:

Leading the list, inhibitor development has replaced pathogen risk as the most common adverse event for patients with severe haemophilia A. Clinicians, those responsible for government purchases, and patients in particular, find it very difficult to evaluate the differing inhibitor risk profiles of treatment products. They are frequently not equipped to put risk into relative context with other considerations (supply, pathogens, efficacy and cost). Answers are needed so that informed treatment decisions may be made based on facts and not from fear or marketing campaigns.

Answering these questions necessitates global collaboration due to the limited number of patients eligible for research protocols combined with the outcome data required. The answers will not come quickly. However, if we keep in the forefront of our minds the words of Mr Schnabel to ‘Employ a criteria in deliberations and decision that gives first priority to the haemophiliac’ [3] it becomes self-evident that working together is the key to finding the answers. Parochial and commercial interests should be secondary to the interest of the patient.