Ensuring maximum outcomes and benefits in comprehensive care for bleeding disorders through surveillance and data collection

Abstract

Objectives: In order to maximize the impact and outcome of comprehensive care, it is important to track the identification of people with bleeding disorders and to evaluate their health outcomes over a long-term period.

Methods: Establishment of a comprehensive care program for the care of patients with bleeding disorders is an essential feature of national health systems desiring to achieve the best health outcomes for their patients. Implementation of a surveillance system in conjunction with a comprehensive care program allows assessments to be made on data, with regards to optimizing resources and outcomes for the patients.

Results: This data provides governments with answers about the bleeding disorders population, and indicates what the trends are, what the best practices are and what interventions may be required.

Discussion: The challenges of answering government and payer demands for evidence-based medicine and cost justification for the introduction and enhancement of treatment and care are ever-present and growing. To sustain and continue the expansion of access to care globally it is critical to build the body of outcome data for individual patients, within HTCs, nationally, regionally and globally. Doing so will not only improve clinical practices and support the allocation of scare resources, but most importantly, the well-being of patients will improve as well.

Keywords:

• Comprehensive care
• Data collection
• World Federation of Hemophilia
• Surveillance
• Health economics

Introduction

The World Federation of Hemophilia (WFH) is the cornerstone of global development. For nearly 50 years, the WFH has been working to achieve ‘Treatment for All’ patients with haemophilia and related bleeding disorders, regardless of where they might live. The WFH has had official recognition and relations with the World Health Organization (WHO) since 1969. Establishment of comprehensive care programs for the care of patients with bleeding disorders is an essential feature of national health systems desiring to achieve the best health outcomes for their patients. It is well established that optimizing economic and human resources through such an integrated multidisciplinary care delivery system leads to improved patient outcome. In order to evaluate the impacts of comprehensive care, it is important to track and monitor the identification of people with bleeding disorders and to monitor their health over a long-term period. This information allows ministries of health to better prioritize resources for patients with bleeding disorders and health care in general. A surveillance system allows for assessments to be made on data, with regard to optimizing resources and care for the patients. These data provide governments with answers about the bleeding disorders population, what the trends are, what the best practices are and what interventions may be required.

Discussion

Comprehensive care

The World Health Organization (WHO) and World Federation of Hemophilia (WFH) recommend that optimum treatment for patients with bleeding disorders be provided in a specialized haemophilia treatment centre (HTC), where hematologists, nurses, orthopedists, physical therapists, psychologists, social workers, dentists, and others come together as a specialized multidisciplinary care team to comprehensively look after each patient’s unique care needs.

Achieving ‘Treatment for All’ requires proper management and care by such a multidisciplinary team of trained specialists. Given the complexity of managing bleeding disorders, one of the core steps to improving outcomes is the introduction of comprehensive care. Although access to safe viral-inactivated clotting factor concentrates is fundamentally important, it alone is not sufficient to optimize care.1^–3

Because care for these patients is very specialized and affects many other areas of the patient’s physical and mental health, it is best met through a multidisciplinary team approach. Comprehensive care ensures that the unique treatment needs of a patient are met to maintain health, including physical, emotional, psychological, social, and educational aspects.4

The comprehensive care model has been one of the most successful public health programmes in many developed countries, resulting in significantly improved health for patients with haemophilia as well as producing a reduction in healthcare utilization.5 The improved outcomes in morbidity and mortality when comprehensive care occurs within an HTC setting are well established.6 In low resourced countries where treatment is typically not immediately and consistently available, patients are at increased risk of suffering severe and permanent disability and early death.7

Surveillance and data collection

Using data collected from national patient registries, the impact, trends, and significant progress achieved through a systematic development approach can be demonstrated. Through many years of experience, the WFH identified the essential elements of a model for the introduction and development of a sustainable national care. The essential elements of the WFH Development Model are integrated and interdependent: ensuring accurate laboratory diagnosis, achieving government support for a national programme, improving the care delivery system and medical expertise, increasing the availability of treatment products, and building a strong national patient organization.8 The Model is equally applicable to developed and developing countries. More recently, a sixth critical element of the Model has emerged and is now separately recognized — the ability to track and report patient health outcomes.

To do so, the WFH recommends the development of national patient registries through collaboration between national patient organizations, healthcare professionals, treatment centres, and ministries of health.9 Registries are beneficial for patients and their quality of life, to doctors and nurses for the identification of changing care needs, and to public health authorities for a better management of care requirements and resources.10,11 A national registry will:^12,13

1. centralize all relevant clinical and diagnostic patient information;

2. establish disease prevalence and the distribution of patients;

3. allow monitoring of trends in health and provide comprehensive surveillance systems for treatment-related adverse events;

4. allow analysis of standard of care and outcomes;

5. enable identification of resources needed and facilitate priority setting for improved outcomes;

6. improve the tendering process;

7. facilitate the establishment of a better communication network.

In 1998, the WFH began collecting information on care throughout the world. This survey, called the WFH Global Survey,¹⁴ collects basic demographic information, data on resources of care and treatment products, and information on the prevalence (the percentage of the population affected) of infectious complications such as HIV and hepatitis C virus (HCV). The WFH compiled the first survey report in 1999. The Global Survey provides illustrations of the general state of care for patients with bleeding disorders on a global basis and examples of achievement with limited resources that encourage other countries to attempt the same.

Quantifiable results contained in national registries and the WFH Global Survey facilitate the measurement of the effectiveness of healthcare programs. Basic data, such as increases in the number of patients diagnosed or the life expectancy of people with bleeding disorders, are useful in evaluating the worth and success of various development programmes to improve care within countries. The need to have a surveillance system becomes more and more important as ministries of health want to see the outcomes of investing resources into haemophilia.

All countries have a limited amount of resources to spend on health care and they allocate resources based upon what they assume is the highest priority. A properly designed registry that also tracks outcomes, including complications, of treatment will allow analysis of where resources are being expended and inform future health planning. Resources should be used to achieve visible results. This is important for two reasons. First, successful healthcare outcomes (such as reduced days of hospitalization or absence from school) will increase the support and enthusiasm of the affected population, and second, the successes will demonstrate to the health authorities the usefulness of this program. Data obtained from a centralized registry are invaluable in identifying the needs for resources and the highest priorities for successful outcomes.¹⁵

To fully understand the outcomes of comprehensive care, continual monitoring is important to be aware of trends or changes. Examples of these data include the number of infections with blood-borne viruses (HIV, HCV, etc.), the number of patients with joint disease or the degree of joint disease in the patients, the number of patients that have inhibitors, liver disease, are hospitalized, or die. This information is critical in identifying the changing needs of the patients, identifying particular problems that need to be addressed, or assessing and documenting the effect that changes in healthcare delivery have made for the population.¹⁶

Through continuing research, clinical tools and knowledge are further evolving to allow treatment delivery tailored and personalized to the individual patient rather than generally treating the disease. Concepts such as personalized prophylaxis, the identification of individuals at risk of developing an inhibitor, and health indicators unique to women with bleeding disorders are moving into clinical care. Accurate and comprehensive data will accelerate these advances and optimize their utility in clinical care.

Conclusion

The challenges of answering government and payer demands for evidence-based medicine and cost justification for the introduction and enhancement of treatment and care are ever-present and growing. To sustain and continue the expansion of access to care globally, it is critical to build the body of outcome data for individual patients, within HTCs, nationally, regionally, and globally. Serial data collection will also allow comparison of patient outcomes and trends year over year within a country, as well as establish a comparative benchmark between patient outcomes in HTCs nationally and against their regional and global peers. Doing so will not only improve clinical practices and support the allocation of scare resources, but most importantly, the well-being of patients will improve as well.

The author reports no conflicts of interest. The WFH would like to thank the National Member Organizations, WFH volunteers and staff, government’s committed to building national care programmes, and WFH partners and donors for their commitment to achieving ‘Treatment for All’.