Unmet need is highest in aggressive haematological cancers and in relapsed or refractory disease. Outcomes are particularly poor in adults with acute myeloid leukaemia (AML) and T-cell acute lymphoblastic leukaemia (T-ALL). In relapsed or refractory AML, survival is often less than one year. Patients with diffuse large B-cell lymphoma (DLBCL) who relapse after frontline treatment also face poor outcomes. In contrast, many patients with indolent lymphomas have a good prognosis, and multiple myeloma can now be considered a chronic condition for many patients. Because treatment is often ongoing, quality of life during treatment becomes a critical consideration.

Haematological cancers were worth $75 billion globally in the year to Q2 2026 at ex-manufacturer list prices. That is 26% of the $293 billion oncology market. IQVIA forecasts compound annual growth of 12% from 2026 to 2030, well above expected growth for the overall pharmaceutical market. Myelodysplastic syndromes (MDS) are forecast to be the fastest-growing segment, followed by multiple myeloma and lymphoma.

Novel modalities, including cell and gene therapies, antibody-drug conjugates and multispecific antibodies, made up 32% of blood cancer clinical trial starts in 2025, up from 17% in 2016. Promising areas of innovation include:

• New approaches to CAR T-cell therapy
• Trispecific antibodies, with two assets in phase III in relapsed or refractory multiple myeloma
• Protein degraders such as BTK degraders
• Novel antigen targets such as GPRC5D
• New combinations such as menin inhibitor, chemotherapy-free, and fixed-duration combinations

Innovation in CAR T technology may help to address the challenges currently faced by CAR T-cell therapies. Many next-generation CAR T therapies are already in development, with key areas of focus including:

• New antigen targets such as CD22, CD20 and CD7, which could extend CAR T to additional haematological cancers and provide options for patients who relapse after CD19- or BCMA-targeted treatments
• Dual-target therapies, for example directed at both CD19 and CD20, which aim to minimise the risk of antigen escape and relapse
• Advances in allogeneic (off-the-shelf) therapies, which remove the need for patient-specific manufacturing and could simplify logistics, reduce delays and potentially lower costs
• In vivo CAR T, which engineers immune cells directly within the patient's body. Although still at a very early stage, it could eliminate the need for cell collection and personalised manufacturing, typically avoids lymphodepleting chemotherapy, and could significantly improve scalability and access.

Efficacy, regulatory approval and reimbursement remain essential, but they are rarely sufficient on their own. IQVIA identifies five success factors:

• Early advocacy
• Patient centricity
• Streamlined pathways for patient identification and referral
• Demonstrated long-term value
• Healthcare system readiness

Strategies and capabilities also need to reflect the specific realities of each indication, given that haematological cancers are such a diverse group of diseases. For example, developing a therapy for relapsed AML differs markedly from developing a multiple myeloma treatment.