What are clinical trials and why are they important for blood cancer?

People with blood cancers (leukaemias, lymphomas and Multiple Myeloma) who agree to take part in a clinical trial are contributing to the development of our knowledge about the safety and effectiveness of new blood cancer drugs. It is important to know that there are many types of leukaemia and lymphoma, all of which need to be considered uniquely when designing trials. The results of a trial on a sub-type of Acute Myeloid Leukaemia (AML) will not be applicable to Chronic Lymphocytic Leukaemia or even another sub-type of AML, for example.

Clinical trials are designed and run with the aim of improving upon the drug, or combinations of drugs, that are normally used for a specific disease in standard clinical practice. These standard treatments, known as the Standard of Care, were established as the best available treatments in earlier clinical trials.

The ultimate aim of blood cancer trials is to develop cures for these devastating diseases and there are examples of where this has already been achieved. The majority of cases of childhood Acute Lymphoblastic Leukaemia, Hodgkin Lymphoma and Chronic Myeloid Leukaemia are cured by treatments that have become the Standard of Care following successive clinical trials.

Unfortunately, for most types of blood cancer there are very few treatments that cure a majority of patients. Sequential trials that each improve upon previous standards of care have enabled cures in some patients and have improved outcomes with respect to prolonging life and improving the quality of life in all types of blood cancer.

Process of new treatments:

New treatments can only be made routinely available to patients if they are approved by the Regulatory bodies around the world. In the UK the relevant authority is the Medicines and Healthcare Regulatory Agency (MHRA), in Europe it is the European Medicines Agency (EMA) and in America the US Food and Drug Administration (FDA). Regulatory agencies may approve new drugs if they are shown to be safe and at least as effective as other drugs that are already used for the disease in question. In the UK, this does not automatically mean that newly approved drugs will be available through the NHS.

Supply of new drugs to the NHS requires approval by the National Institute for Health and Clinical Excellence (NICE) or sometimes, in the case of some new cancer drugs, by the cancer drugs fund (CDF). Clinical trials are essential because they provide the data that regulatory authorities require for them to assess the safety and efficacy of treatments for licensing. In addition, both NICE and the CDF require the data for the rigorous assessment of cost effectiveness in the context of safety and efficacy as compared to the current Standard of Care for any given disease.

There are different types of trials that contribute to developing better and, hopefully, curative treatments. New treatments are developed through different phases of trials. The early phases are mostly concerned with ensuring that the treatments are safe and will not induce intolerable side effects. If treatments get through this early safety phase, they may be used in larger trials that examine efficacy as well as safety by comparing them to the Standard of Care for the disease being investigated.