Sunday, June 05, 2016

Difference between Precision Medicine and Personalized Medicine?

A friend of mine asked me about the difference between ‘personalized medicine’ and ‘precision medicine’. Just a couple of years ago, the term ‘personalized medicine’ was everywhere. Since last January and after the president Obama’s state of union (where the term ‘precision medicine’ was used), the term ‘precision medicine’ have been replacing the term ‘personalized medicine’ in the US even though the term ‘personalized medicine’ continues to be used in EMA and other regions.
There isn’t really any difference in the true meaning of these two terms. The purpose of the precision medicine or personalized medicine is to find the right drug for the right patient at the right dose at the right time.
On January 20, 2015, President Obama announced the Precision Medicine Initiative® (PMI) in his State of the Union address. Through advances in research, technology and policies that empower patients, the PMI will enable a new era of medicine in which researchers, providers and patients work together to develop individualized care.
According to a NIH website, the reason for switching from ‘personalized medicine’ to ‘precision medicine’ is that the word "personalized" could be misinterpreted to imply that treatments and preventions are being developed uniquely for each individual. The fact is that the precision medicine or personalized medicine is to identify the sub-group of patients who may have a specific type of genetic biomarkers or genetic traits and may benefit from one specific type of drug or treatment regimen.  
There is a lot of overlap between the terms "precision medicine" and "personalized medicine." According to the National Research Council, "personalized medicine" is an older term with a meaning similar to "precision medicine." However, there was concern that the word "personalized" could be misinterpreted to imply that treatments and preventions are being developed uniquely for each individual; in precision medicine, the focus is on identifying which approaches will be effective for which patients based on genetic, environmental, and lifestyle factors. The Council therefore preferred the term "precision medicine" to "personalized medicine." However, some people still use the two terms interchangeably.
In an article on RAPS.ORG, the difference between ‘precision medicine’ and ‘personalized medicine’ is described as:
The basic premise of personalized medicine is defined in layman's terms as "Providing the right treatment to the right patient, at the right dose at the right time." The difference between the terms "personalized medicine" and "precision medicine" is subtle and was previously used interchangeably. "Precision Medicine (PM) comprises specifically customized diagnostic and therapeutic strategies targeting a patient subpopulation with susceptibility to a distinct disease condition based on the individualized discrepancies of select criteria, including genomic profiles, environmental influences, lifestyle habits and family history. Whereas, personalized medicine, specifically refers to the individualized treatment regimen tailored for the benefit of a single patient.
In FDA’s Precision Medicine website, the term ‘precision medicine’ and ‘personalized medicine’ are used interchangeably.
Precision medicine, sometimes known as "personalized medicine" is an innovative approach to disease prevention and treatment that takes into account differences in people’s genes, environments and lifestyles.

What is the relevance of the precision medicine to the statisticians? American Statistical Association Listed the Statistical Objectives in Precision Medicine and Roles of Statisticians on Precision Medicine Teams
Statistical Objectives in Precision Medicine
  • Develop methods to incorporate rigorous statistical analyses of large datasets into decision analyses tailored to the individual patient.
  • Develop better methods for validation of risk prediction models, and facilitate sharing of data so that investigators can validate their models.
  • Develop solid foundations for drawing rigorous inferences from electronic health records and other large datasets.
  • Develop data sharing approaches that facilitate learning from the small and highly dispersed set of cases similar to the patient for whom a decision is to be made.
  • Develop methods to efficiently monitor and validate the precision of laboratory diagnostics and diagnostic imaging.
  • Develop statistics curricula that teach the modeling and analysis procedures specifically addressing patient care and broader health outcome issues under increased emphasis on individual patient data.

Roles of Statisticians on Precision Medicine Teams
  • Statisticians develop approaches to optimally collect and use data to inform which treatment is best for a given individual at a given time under the given circumstances.
  • Statisticians translate a medical question into a precise data-based question. This includes carefully describing data structure, the underlying system that generated the data and what the team is trying to assess or predict.
  • Statisticians develop data analytics for the health scientists to test and further develop theories in order to develop more informative diagnostics and more effective interventions.
  • Statisticians enhance communication across disciplinary boundaries such as the computational, health, and behavioral disciplines.

In reality, statisticians will be involved in the precision medicine by doing more than what was listed by ASA. For example, statisticians are proposing the innovative clinical trial designs to accommodate the need of the precision medicine. The recent discussions of umbrella design, basket design, master protocol, Bayesian adaptive design,... are driven by the concept of the precision medicine.  

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