Can anyone access medicines on a compassionate basis?

Before a medicine is authorised and placed on the market, it must be duly evaluated to learn more about its efficacy and safety. The first priority is to conduct clinical trials that will respond to these questions. Only patients who cannot be part of a clinical trial are eligible for compassionate use.

Under compassionate use, the treatment that is given to the patient is a medicine, not a placebo. The patient receiving it is expected to benefit from it. In a clinical trial, the medicinal product is tested against a comparative treatment which may be a placebo. People enrolled in clinical trials correspond to specific biomedical criteria, and people who should not be enrolled in a clinical trial are also defined (so-called exclusion criteria).

A common example involves patients who are too severely ill, with multi-organ impairment, or severe liver or renal deficiency. They are usually excluded from clinical trials. However they can receive the treatment on a compassionate basis. This ensures the optimum care for the more severely ill: they cannot take the risk of receiving a placebo or a treatment that would not help them. Even though it has not been determined whether the treatment patients receive on a compassionate use basis is useful, if there is the slightest chance the treatment may work, patients should benefit from it.

Regulators, when authorising a compassionate use, can do so on a named patient basis (the doctor makes a request for a given patient), or for a group of patients (a so-called cohort).

On a named patient basis, authorisation will be on a case-by-case basis. For a cohort compassionate use, regulators will define the patient population eligible for the programme. For example, in a disease for which only two medicines exist, regulators may decide the Compassionate Use Programme is only open for patients who have already been treated with both medicines with no response, or who cannot tolerate either product.  

Page created: 30/10/2013
Page last updated: 07/11/2014
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