While this new benefit-risk guideline opens a door to partner with patients for conducting preference studies and to structure patient-focused benefit-risk assessment, we cannot forget that there is a lack of obligation to put these guidelines into effect, and how exactly patient communities and their representatives can get involved in this?
There were never enough of us activists, but we seemed to be wherever a politician was making a speech or opening a new neighbourhood centre or celebrating an event, etc. We were rarely quiet and respectful. We were constantly protesting, dying in the streets, planning disruptive events. Not only were there no treatments, there was no respect towards the people with the disease. Things have changed dramatically since then.
A medicine is commonly defined as a drug or preparation that can treat or prevent disease. Medicines must be delivered into the body, where they are then distributed to the organs that need them before being broken down and excreted.
Gene therapy is a way of restoring the function in cells where genes are missing or not working properly. It is a highly experimental technique, but has shown promise in clinical trials for some diseases.
The term ‘personalised medicine’ includes both stratification and personalisation, which are often incorrectly used as synonyms. However, the two are different, as explained further below.
Stratified and personalised medicine involves tailoring treatments towards a specific group of patients or to individuals.
Personalised medicine is a developing area that faces procedural and ethical challenges in research and treatment.
There are several new research areas driving personalised medicine forward, such as molecular genetics, epigenetics, pharmacogenetics, and the development of biomarkers and biomedicines.