Preparing for personalised medicine in the US

5 November 2013

The US medical regulatory body the Food and Drug Administration (FDA) has released a new report on personalised medicine.
The FDA, which defines personalised medicine as ‘the tailoring of a medical treatment to the individual characteristics, needs and preferences of a patient’, often using genomic information, says that the new report sets out their unique role and responsibilities in furthering the field in the US – specifically, in supporting the development and introduction of new medical diagnostics and therapeutics. The FDA’s role is to protect and promote health by assuring the safety, efficacy and security of medical products and devices, as well as foods and other products.
Paving the Way for Personalized Medicine emphasises that the concept of personalised medicine itself is not new, but scientific advances are offering new potential to predict the best treatment choices and produce targeted therapeutic products, resulting in improved patient care. The report cites examples of new treatments targeted to specific genetically defined tumour or other disease sub-groups, and the need for companion diagnostic tests as an integral part of the treatment.
It sets out ways in which the FDA has developed regulatory processes appropriate for the new generation of personalised medical products. Examples relevant to genomics and related fields are largely collaborative research initiatives, including into genomic features and evolution of cancers, the genetics of vaccine responses and the interaction between genetic and environmental factors in influencing cardiovascular risk. Evaluation methods are also discussed, such as work to adapt clinical trial design to appraise new targeted therapeutics, the development of genomic reference materials for evaluating whole genome sequencing (WGS) and statistical methods to analyse genomic data.
The report underline’s the FDA’s on-going commitment to personalised medicine in terms of ‘advancing the science and tools that will help drive innovation, collaborating with scientists worldwide in important research activities, and providing guidance to industry to help shepherd new products through regulatory review’.

Comment: The FDA is right to be preparing for personalised medicine, and in recognising that different regulatory approaches will be required to appraise tailored treatments and associated diagnostics, as opposed to more general tests or therapeutics. Other agencies of the US Department of Health and Human Services such as the NIH and CDC are also aware of the potential; NIH Director Dr Francis Collins recently said at an event on personalised medicine that the US medical community needed to plan ahead to keep pace with the rapid expansion in genomic medicine, predicting that WGS would rapidly outstrip more targeted forms of genomic sequencing and analysis. 

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