During research, or ‘drug discovery’ a chemical molecule can be identified as having the potential to be developed into a drug that delivers therapeutic benefit in the form of a treatment or cure for some disease or disorder.
A New Molecular Entity (NME) or New Chemical Entity (NCE) is a drug or chemical that is without precedent among regulated and approved drug products. The NME designation indicates that a drug in development is not a version or derivative of an existing and previously investigated, trialed and approved substance.
Being labeled as entirely ‘new’ or first-in-class molecule dictates that certain types of clinical trials must be run, and that particular attention must be paid to proving a drugs safety.
Drug efficacy is often demonstrated through a comparison with existing drugs and treatments, an NME being explored as a treatment for a previously untreated condition and as an NME, means there are no existing treatments, or even similar drug products, against which efficacy can be measured.
This situation requires that other measures of efficacy be located and measured; such as quality of life scores and deep, detailed attention to the direct physiological impact of the drugs mechanism of action and effect upon the symptoms and physiological attributes of the disease or disorder that is the selected clinical indication.