21 September 2012 1 © iPRI – International Prevention Research Institute www.i-pri.org

Peter Boyle

Efficacy of Medicines in Different Therapeutic Areas

modified from Spear et al., Trends in Molecular Medicine, 2001, Vol 7: 201-204

Efficacy Rates

Definition of Terms

Precision Medicine

The design and evaluation of medicines that

•  incorporate clinical and molecular information to identify potential patient populations most likely to benefit

•  inform clinical development strategies that are most likely to be successful

Personalized medicine

The use of genetic and clinical information to

•  select a medication that will have a higher likelihood of benefit in patients with those characteristics

•  avoid use of a medicine that is unlikely to be effective or more likely to cause harmful effects in a given patient

Personalized Medicine in Oncology is Not a New Idea Breast cancer

Lung cancer

Chronic Myelogenous Leukemia (CML)

1952 1973 1977

1960 1973

1960 2001

Removal of adrenal glands found to cause regression of some breast cancers by “starving” the tumor of estrogen

First reports of clinical responses to tamoxifen in women with breast cancer

Association of response to tamoxifen with ER receptor status

Lung cancer separated into “small cell” and “non-small cell”

Recognition of key differences in the outcomes of surgery and response to chemotherapy in SCLC and NSCLC

Identification of the Philadelphia chromosome as a hallmark for CML

Imatinib approved in the US for treatment of CML

Promise of Personalised Medicine

21 September 2012 5 © iPRI – International Prevention Research Institute www.i-pri.org

" Personalised medicine is the next evolution in healthcare. It will tailor healthcare solutions to the individual patient, instead of relying on the "one-size-fits-all" approach that currently predominates.

" Through the introduction of personalised medicine, healthcare services will deliver 'the right treatment to the right patient at the right time'.

" Personalised medicine will improve the outcomes for patients and cut down the use of unnecessary and expensive treatments.

" The result will secure more value from healthcare spending, to the benefit of healthcare providers and patients.