Who can enter
- Children with cancer (solid tumors) in whom the disease has returned (relapsed) or reacted insufficiently to treatment (refractory)
- All ages
Goal
In this protocol we aim to collect data from patients who, based on their cancer cell characteristics, are treated with targeted anti-cancer drugs that are not yet approved for use in the Netherlands.
Background
An increasing number of targeted anti-cancer drugs are available nowadays. Each of these drugs targets one or more features of the cancer cell; only if such a feature is present in the cancer cell can the targeted drug be given.
Certain cancer cell characteristics are common in specific tumor types. In those tumors, studies are done to determine the effect of a drug directed against that particular characteristic. If a drug treatment is found to work well, this can lead to approval and reimbursement of this drug in the Netherlands for that specific tumor type with that particular characteristic.
However, there can be quite a long time between the moment a drug is found to be working (effective) in studies and the moment it is actually available to patients. In the period between applying for approval and a drug becoming available, in certain situations a patient can sometimes still be treated with the drug through the treating physician (for example, if the manufacturer makes the drug available free of charge).
In this protocol, we want to collect data on the effect of the treatment on the disease of all these patients in a structured and controlled way. From these data we can learn a lot about the effect of the treatment. Sometimes these data are even necessary to get regulatory approval for a drug.
All targeted drugs in this protocol have proven their anti-cancer efficacy in previous research for the particular type of tumor. Therefore, these are not completely new, experimental drugs. This is why we call it a protocol and not a study. The data that are collected are general data of the patient, tumor type, effect of treatment and any (serious) side effects. Also, in some cases, data are collected on the patient's physical, psychological and social functioning.