Obama’s precision medicine initiative: the future of healthcare?
2 February 2015
US President Barack Obama has launched a new research programme in precision medicine, also known as stratified and personalised medicine, to include creation of a national research bank of at least a million people.
What’s in a name? Precision, participation and personalised medicine
The alternative terms are often used interchangeably, although each reflects different aspects of the new possibilities arising from genomic medicine: increasingly tailored or precise diagnostics, therapeutics, monitoring and screening. Stratified medicine refers to the capacity to subdivide patient populations into groups based on their (genetically determined) response to a given type of drug. Personalised medicine encompasses both the ability to deliver individualised medical care and treatment choices; this is arguably already the aim of medicine, but there is new capacity to include genetic information alongside lifestyle and environmental data in refining these choices. Precision medicine reflects the increasing capacity to deliver the optimal dose of the right drug to a specific patient for a given condition. This is particularly well advanced in oncology (cancer medicine) where clinicians can analyse the genomic nature of a tumour to select the best treatment, including new biologically targeted treatments, and monitor their effects.
Both personalised and precision medicine are also terms frequently used to imply increased, active patient empowerment and participation in their own healthcare, including disease prevention, diagnosis and treatment; the new US programme is no different. Launched as part of the US President’s 2016 Budget, the new Precision Medicine Initiative will receive $215 million funding to ‘pioneer a new model of patient-powered research that promises to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients’.
A ‘million genome’ project?
The programme will encompass public and private research initiatives to combine and analyse genomic and other health data using new and emerging methods for analysing ‘big data’ to accelerate biomedical discovery and development. In particular, one key aim is to generate a national ‘cancer knowledge network’ to help identify genomic drivers of cancer and deliver new, genetically targeted cancer treatments. It is not specified whether the genomic data will involve whole genome sequencing or will instead be more focused.
Creation of a new national cohort of a million or more volunteers to underpin research will form the core of the precision medicine effort. This massive group of Americans will have the opportunity to feed into the design of the initiative and to contribute not only health, environmental, lifestyle and genomic data but also information on their metabolites, microbiome and from personal devices and sensors. It is emphasised that privacy will be ‘rigorously protected’ via engagement with not only participants but also ‘bioethicists, privacy, and civil liberties advocates, technologists, and other experts’.
Promised funding will include $130 million to the National Institutes of Health (NIH) to develop the national research cohort ; $70 million to the National Cancer Institute (NCI); and $5 million to Office of the National Coordinator for Health Information Technology (ONC) for the development of data standards to ‘address privacy and enable secure exchange of data across systems’.
The vision is that open, responsible data sharing and strongly engaged patient participation will ‘set the foundation for a new way of doing research’ as well as generating crucial information about health and disease. Importantly, this includes plans for the Food and Drug Administration (FDA) to develop new expertise and supporting regulatory structures for the new databases, not only for the effective evaluation of Next Generation Sequencing (NGS) technologies but also to ensure that participants can access their own health data and analytical tools. The purpose of this is to ‘empower individuals and families to invest in and manage their health’.
A commentary by Francis Collins and Harold Varmus in NEJM sagely observes that the new initiative ‘taps into converging trends of increased connectivity, through social media and mobile devices, and Americans' growing desire to be active partners in medical research’. What is not also articulated is that there is a simultaneously growing need for new approaches to healthcare to deliver cost-efficiencies.
Whilst the US has a primarily insurance-based healthcare system - as opposed to the UK’s taxpayer-funded National Health Service (NHS) - the Obama administration has been anxious to reduce the disparities this can create, leading to the development of so-called ObamaCare, a federally-regulated, subsidised form of health insurance that extends the pre-existing Medicaid system.
Nevertheless, like many other economies (including our own) the US is facing a massive challenge as the population ages and medical costs soar – in part thanks to the rise of high-tech personalised treatments for cancer and rare diseases. It is widely hoped that harnessing existing social and technological trends and developments for personal monitoring and connectivity alongside new tools for better disease prediction, prevention and treatment, may lead to a sustainable model for healthcare provision driven by increased patient participation and responsibility for their own health.
The new US precision medicine initiative, whilst not necessarily delivering even the 100,000 full genome sequences of the UK counterpart, is cleverly combining the development of genomic and health data collection and analysis capacity with a model for even greater patient involvement. In this, it potentially goes much further than the 100,000 Genomes Project, but with one caveat. The latter is intended to be firmly rooted within the NHS and clinical practice, as a test-bed for integration of genomic medicine throughout the health service. Whether the US equivalent will be able to make a prompt leap from research into everyday practice remains to be seen.