Introduction
Before any potential new drug can be discovered, research must be carried out to understand as much as possible about the disease being treated and the underlying cause of it. This may include understanding which genes are altered, the effect this has on the proteins they encode, and the way this interferes with the functioning of the cells and tissues.
Myeloma is a hugely complex cancer but advances in the understanding of the biology of the disease and of the bone marrow microenvironment have led to important therapeutic advances. While overall survival of myeloma patients has improved, relapse is inevitable and the survival of patients progressing after treatment with the novel agents greatly diminishes. There is therefore a continued need to conduct research and translate it into future promising treatments.
The following sections describe the process by which new drugs are developed, approved and become accessible to patients. This is summarised in Table 1 below:
| Stage of development | What happens |
|---|---|
| Treatment development and discovery | Identifying possible new myeloma treatments, based on an understanding of how myeloma develops |
| Laboratory testing | Potential treatment is tested in the laboratory for activity and safety |
| Clinical trials | Groups of patients receive the new treatment (or combination of treatments) under carefully controlled conditions. They are closely monitored for tolerability and effectiveness of the treatment |
| Treatment licensing | Treatment (or combination) is licensed as being safe and effective |
| Treatment approval (Health Technology Assessment) | Treatment (or combination) is approved as being effective and cost-effective for use on the NHS |