January 04, 2016
Author(s): Valerie Breslin Montague
Copyright 2015, American Health Lawyers Association, Washington, DC. Reprint permission granted.
The Food and Drug Administration recently launched precisionFDA, a web-based platform intended to further the president’s Precision Medicine Initiative. This alert discusses what scientists, researchers and other interested parties need to know.
On December 15, the U.S. Food and Drug Administration (FDA) launched precisionFDA, a web platform to allow scientists and other stakeholders to come together to continue the development of Next Generation Sequencing (NGS). This online, cloud-based portal is intended to further President Barack Obama’s Precision Medicine Initiative.
NGS is a method that allows scientists to compile data on an individual’s order or sequence of DNA. This allows for studies to determine differences that can indicate risk of disease, response to treatment or current health. Analysis of these factors can allow physicians to tailor medical treatments to the specialized needs of a particular individual.
The precisionFDA community platform  allows all types of scientists, researchers and other interested parties to further the science of “reading” DNA through NGS. The platform will allow individuals from academia, health care, government and other sectors to evaluate NGS assays with the intent to advance precision medicine. The platform currently references more than a dozen governmental and private community participants, ranging from the Centers for Disease Control and Prevention to individual medical professionals.
The precisionFDA site allows users to draft notes describing their work and theories, attach files or apps, generate content through use of apps on the site, compare sets of variants and read the posts and documents of other users. It also allows users access to reference genomes, including the National Institute of Standards and Technology’s “Genome in the Bottle,” which is a reference sample of DNA that can be used to validate human genome sequences. Users can test new approaches and cross-validate results against posted reference materials.
As seen particularly in the comparisons section of the platform, precisionFDA’s platform is in beta. FDA states that, within the next year, it should have recommendations or software solutions to allow for comparing variants (VCF files) and for reporting the results. In the interim, the platform provides for an initial VCF comparison framework.
Precision medicine, also referred to as personalized medicine, approaches disease prevention and medical treatment in a manner that takes into account differences in a person’s genetic makeup, environment and lifestyle. For example, a cancer patient who undergoes molecular testing may benefit from focused treatments that address the genetic profile of the patient’s tumor.
To advance the development of precision medicine, Obama announced his Precision Medicine Initiative in his 2015 State of the Union Address.  This Initiative provided funding to the National Institutes of Health, the National Cancer Institute, the U.S. Department of Health and Human Services Office of the National Coordinator for Health Information Technology, and FDA. The funds allocated to FDA were targeted toward the development of a new approach for the evaluation of NGS technologies. One result is the creation of precisionFDA.
FDA states that it welcomes feedback and questions on precisionFDA at email@example.com.
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