Beta thalassemia: Looking to the future, addressing unmet needs and challenges

Thalassemia management has reached new milestones, with new therapies promising the dawning of a new era. However, conventional and new approaches require accessibility, affordability, acceptability/adherence by patients, and medical expertise from healthcare providers. Current treatments still do not offer the expected duration and quality of life, and inequalities in patient care are almost a universal phenomenon. To understand the requirements to achieve improved care, including the adoption of new therapies, for the maximum number of the global patient population, it is necessary to recognize the weaknesses that are experienced in the present so that future corrective action can be taken. Deficits in service provision are due to poor political and financial support, lack of prioritization during resource rationing, and absence of epidemiological information for policy making. These system weaknesses require improved resource management and would benefit from patient support organizations, improved psychosocial support and patient welfare, and an increase in professional expertise through educational programs. Medical products and technology must also be made affordable and widely available, and the curative treatments and cheaper approaches to technology must be recognized as resource saving. Improvements in the access to innovative and quality care, and even a cure, require concerted actions by all stakeholders, including physicians and the patient community.