Global Hematological Disorders Market 2017 - Innovative First-in-Class Product Developments Remain Attractive

DUBLIN, October 18, 2017 /PRNewswire/ --

The "Frontier Pharma: Hematological Disorders - Iron Regulators and Immune Response Targeted Programs Within Anemias and other Red Blood Cell Disorders Hold Potential to Transform Therapy Area with Significant Unmet Need" drug pipelines has been added to Research and Markets' offering.

Hematological disorders market is small and represents a significant investment opportunity. Due to few treatment options, many patients have a poor quality of life and poor prognosis, especially those with more severe disease states. Severe disease phenotype in cases of genetic disease correlates with a homozygous phenotype, as opposed to a heterozygous phenotype. For example, sickle cell anemia is a homozygous disorder, and sickle cell disease is a heterozygous disorder. The lack of pharmacotherapy treatment options means that alternative treatment approaches such as chronic blood transfusions and bone marrow transplants are used.

Globally prevalent disorders such as anemias, of which the most common form - iron deficiency anemia - is estimated to affect over 13% of the world's population, represent significant investment opportunities due to the number of patients eligible for treatment. Although some forms of iron deficiency anemia are treatable with iron supplementation, other forms are hereditary in their etiology, such as iron malabsorption, and therefore require an alternative treatment approach to improve patient care. The therapy area also contains rare disorders of much lower prevalence, such as paroxysmal nocturnal hemoglobinuria, which is classified as an orphan disease and has no currently marketed treatment options.

Historically the hematological disorders therapy area has suffered from a lack of funding for research, leading to a low number of treatment options. However, first-in-class pipeline innovation is promising and 92 of 327 pipeline programs (28%) with a disclosed molecular target are first-in-class. Investment in first-in-class development is particularly important within hematological disorders as it holds the potential of achieving clinical breakthroughs through radical pharmaceutical innovation. A successful marketed product has the potential to gain a large market share due to the significant level of unmet need with regard to effective marketed treatment options.