Hematology is the study of the blood, blood-related organs and tissues (e.g., bone marrow, spleen, and liver), and blood diseases. Blood disorders can be caused by inherited genetic mutations, environmental insults, or nutritional deficiencies. Common hematological disorders include hemophilia (impaired clotting ability), clotting disorders (predisposition to form blood clots), and blood cancers (i.e., liquid tumors) such as leukemias, lymphomas, and myelomas.
Because the pharmacological targets of blood disorders are primarily located within the blood, issues involving delivery and distribution tend to be reduced compared to diseases affecting other locations in the body. However, because the blood has access to all tissues and organs, this attribute also underlines the need for target specificity and for closely monitoring undesired effects. This usually requires an understanding of the underlying biology and the relationship between pharmacokinetics and pharmacodynamics (PK/PD), as well as close attention to potential and emergent toxicities during both preclinical and clinical development.
Given its close ties with oncology, much of the recent work in hematology has focused on treatments for blood cancers. This focus has pushed the boundaries of drug development beyond small molecules and into the realm of cellular therapies like CAR-T cells. Because of their complexity and limited regulatory precedent, CAR T cell therapies can present some unique drug development hurdles.
Careful clinical trial design, including establishing appropriate dosages and endpoints, is important for the development of hematological drugs. For newer, more complex therapeutics (e.g., CAR-T cell therapies), innovative bioanalytical procedures must also be employed, along with specialized manufacturing and distribution techniques and controls. As with any drug development program, it is vital to develop a solid clinical pharmacology and regulatory strategy to help navigate potential pitfalls and ensure the most efficient path to marketing approval.
Nuventra’s Hematology Experience
Nuventra can perform any of our wide range of services for drugs to treat Hematology indications. Some of our recent project experience in this area includes:
- Attended Pre-Submission Health Authority Meetings
- FDA and EU Rapporteur and Co-Rapporteur
- Authored 2.7.1 & 2.7.2
- Authored CSRs
- Authored Modeling Reports
- Clin Pharm/Pharmacokinetics
- Data Interpretation
- Data Management
- Labeling Language
- Modeling and Simulation Strategy
- Pediatric Study Plan (PSP)
- Pre-IND and IND
- Study Design
Visit our services page to see our full range of services and to learn more about what our drug development consultants can offer your program.
Nuventra has experience with a number of Hematology indications, including:
- Chronic immune thrombocytopenia
- Chronic Lymphocytic Leukemia
- Coagulation Deficiencies
- Non-Hodgkins Lymphoma
- Oral Thrombopoietin
- Paroxysomal Nocturnal Hemoglobinuria
- Platelet Deficiency in Chronic Liver Disease
- Radiation Induced Thrombocytopenia
- Sickle Cell Disease