The haematological disorders therapy area, which covers indications such as sickle cell disease, paroxysmal nocturnal haemoglobinuria, anaemia, thalassaemia and hereditary haemochromatosis, is experiencing a significant level of first-in-class innovation, with 92 of its 327 pipeline programmes with a disclosed molecular target identified as first-in-class, according to business intelligence provider GBI Research.

The company’s latest report states that the haematological disorders market is small with substantial unmet need and as such represents a significant investment opportunity.

Owing to few treatment options, many patients have a poor quality of life and poor prognosis, especially those with more severe disease states.

The lack of pharmacotherapy treatment options means that alternative treatment approaches such as chronic blood transfusions and bone marrow transplants are used.

Callum Dew, Analyst for GBI Research, explains: “Although blood transfusions can manage certain red blood cell disorders, this approach itself can lead to iron overload.”

“This is known as haemochromatosis, another haematological disorder that itself affects patients’ quality of life and prognosis because of an increase in the risk of organ failure. Similarly, bone marrow transplants, which can treat a range of haematological disorders, carry many risks, ranging from anaemia to more severe and potentially fatal disorders.”

Investment in first-in-class development is particularly important within haematological disorders as it holds the potential of achieving clinical breakthroughs through radical pharmaceutical innovation. As such, it represents the most likely method of discovering new and effective drugs for a therapy area in which there has been a lack of funding and few recent breakthrough treatment discoveries.

GBI Research believes a successful first-in-class product has the potential to gain a large market share owing to the significant level of unmet need with regard to effective marketed treatment options.

Dew continues: “There are a multitude of reasons for investing in the haematological disorders market. Aside from the small number of effective marketed products within the therapy area, there are also a wide range of indications that vary greatly in their prevalence and a number of orphan diseases.”

“Globally prevalent disorders such as anaemias, of which the most common form – iron deficiency anaemia – is estimated to affect more than 13% of the world’s population, also represent significant investment opportunities because of the number of patients eligible for treatment.”