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Precision Medicine


nation of systems biology and personalised/preci- sion medicine approach to DDD.


Personalised and Precision Medicine In order to understand the concept and nomencla- ture of a ‘Precision Medicine Drug’ it is important to first appreciate the subtle but real differences between the terms Personalised versus Precision Medicine. As with any new and emerging field of endeavour, clear definitions are often a work in progress as terminology evolves and/or disappears. In the case of Personalised and Precision Medicine it is complicated by the fact that these terms are often mistakenly used interchangeably as umbrella descriptors. Hence, the terms Personalised and/or Precision Medicine and how they are practised have broad and confusing interpretations19.


Personalised Medicine Historically, Personalised Medicine emerged in the early 2000s. The Personalised Medicine Coalition (PMC), founded in 2004 to represent the interests of the then fledgling Personalised Medicine com- munity, defined Personalised Medicine as “…an evolving field in which physicians use diagnostic tests to determine which medical treatments will work best for each patient. By combining the data from those tests with an individual’s medical histo- ry, healthcare providers can develop targeted treat- ment and prevention plans”20. Other pioneers of Personalised Medicine continued to stress the importance of the individual. The belief was that “actionable understanding of disease and wellness as a continuum of [molecular] network states unique in time and space to each individual human being” is possible21. Redekop and Madsi have sur- veyed that literature and the plethora of competing definitions of Personalised Medicine. They con- cluded that the most appropriate definition for Personalised Medicine is “the use of the combined knowledge (genetic or otherwise) about a person to predict disease susceptibility, disease prognosis or treatment response and thereby improve that per- son’s health22. They reinforced the idea of specific analyses for treatment of the individual patient.


Precision Medicine The term ‘Precision Medicine’ was first coined by Clayton Christensen in his book the Innovator’s Prescription published in 200923. However, the descriptor ‘Precision Medicine’ did not gain wide acceptance and usage until a report entitled Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease was published by the US


Drug Discovery World Summer 2018


National Research Council (NRC) in 201124. The report laid out a series of recommendations for dis- ease ontology predicated on molecular information content in the form of causal genetic variants or genomic information rather than a symptom-based classification system. This prompted a firestorm of activity, and the initial focus of Precision Medicine was on genetic and genomic underpinnings of dis- ease. For example, the Institute of Precision Medicine provided an early definition that stated: “Precision medicine is targeted, individualised care that is tailored to each patient based on his or her specific genetic profile and medical history. Unlike traditional medicine where one-size-fits-all, practi- tioners of precision medicine use genomic sequenc- ing tools to interrogate a patient’s entire genome to locate the specific genetic alterations that have given rise to and are driving his or her tumour”25. This type of approach garnered significant atten- tion, but it was difficult to discern the fundamental differences practiced by the Precision Medicine ver- sus Personalised Medicine communities19.


Differences between Personalised versus Precision Medicine The NRC Council Report in 2011 attempted to define and differentiate Precision Medicine from Personalised Medicine. The report stated: “Precision Medicine is the tailoring of medical treatment to the individual characteristics of each patient. It does not literally mean the creation of drugs or medical devices that are unique to a patient, but rather the ability to classify individuals into subpopulations that differ in their susceptibil- ity to a particular disease, in the biology and/or prognosis of those diseases they may develop, or in their response to a specific treatment. Preventive or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side-effects for those who will not. Although the term ‘personalised medicine’ is also used to convey this meaning, that term is sometimes misinterpret- ed as implying that unique treatments can be designed for each individual”24. It should be noted that the word ‘precision’ in


Precision Medicine is used colloquially to include both accurate and precise scientific measure- ment24,26. However, based on the NCR definition, it is clear that the Precision Medicine approach utilises individuals and defined (sub)-population- based cohorts that have a common network of dis- ease (or health) taxonomy. In addition, it requires an integrated molecular and clinical profile of both the individual as well as the subpopulation-based cohort. Zhang has described Precision Medicine,


References 1 Kauppi, DM and Naylor, S. Therapeutic Drug Repurposing, Repositioning and Rescue: Part IV – Financial Model and Analysis. Drug Discov. World Winter Edition. 54-63 (2016). 2 Cook, D et al. Lessons Learned from the Fate of Astra-Zeneca’s Drug Pipeline: A Five Dimensional Network. Nature Reviews: Drug Discovery. 13, 419-431 (2014). 3 Paul, SM et al. How to Improve R&D Productivity: the Pharmaceutical Industry’s Grand Challenge. Nature Reviews: Drug Discovery, 9, 203-214 (2010). 4 Deloitte Centre for Health solutions. Measuring the Return from Pharmaceutical Innovation 2014. Turning a Corner? http://www2.deloitte. com/content/dam/Deloitte/uk/ Documents/life-sciences- health-care/measuring-the- return-from-pharmaceutical- innovation-2014.pdf. 5 DiMasi, JA, Grabowski, HG and Hansen, RW. Innovations in the Pharmaceutical Industry: New Estimates of R&D Costs. J. Health Econ. 47, 20-33, (2016). 6 Gaffney, A. How Often Does FDA Withdraw Drugs Using Discontinuation Petitions? Very Rarely. Regulatory Affairs Professional Society. June 15th, (2015). http://www.raps.org/ Regulatory-Focus/News/2015/ 06/15/22690/How-Often- Does-FDA-Withdraw-Drugs- Using-Discontinuation- Petitions-Very-Rarely/. 7 ProCon.Org Thirty-Five FDA-approved Prescription Drugs Later Pulled from the Market. http://prescription drugs.procon.org/view.resourc e.php?resourceID=005528. 8Vizirianakis, IS (Ed). Handbook of Personalized Medicine: Advances in Nanotechnology, Drug Delivery and Therapy. CRC Press/Taylor Francis Group. Boca Raton, FL, USA. (2013). 9 Spear, BB, Heath-Chiazzi, M and Huff, JJ. Clinical Applications of Pharmacogenetics. Trends Mol. Medicine 7, 201-204 (2001).


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