FDA Focus: Life-threatening disease and the benefit-risk of treatment options

In the fifth of a monthly FDA-focused blog published exclusively by The Pharma Letter, Dr Nicola Davies looks at how the FDA weighs up the risk versus the benefits of new drugs for terminally ill patients.

It is painful and traumatic for a patient to be told that they are dying from a terminal illness. This becomes even more unbearable when faced with the discovery that there may be a treatment that could help – but could also do more harm. When faced with death or life-threatening treatment, many patients will opt for treatment, regardless of the potential harm it may cause or whether it is experimental. However, if a drug hasn’t been approved by the US Food and Drug Administration, it is for a reason – usually because it could cause more harm than the disease itself, potentially decreasing life expectancy and/or quality of life.

Benefit-risk Assessment Framework