Precision Medicine Initiative
What it does
Aims at customizing patient care by taking into account an individual’s genetics, environment, and lifestyle to develop personalized treatment strategies.
The Precision Medicine Initiative (PMI) is a $215 million project that seeks to improve medical care by increasing research, resources, and infrastructural support for tailored healthcare. The initiative is based on the premise that taking into account personalized information about genetics, environment, and lifestyle while making treatment decisions will improve the precision and success of patient care.
According to the White House, the official PMI mission is “to enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized treatments.”
The initiative has five main objectives:
- More and better treatments for cancer – The National Cancer Institute, allocated $70 million of the PMI funding, is focusing on four broad areas:
- Expanding Precision Medicine Clinical Trials by assigning patients to therapy based on the genetic alterations that are thought to be driving their cancer. NCI-Molecular Analysis for Therapy Choice (NCI-MATCH) is exploring treating adult patients based on the molecular profiles of their tumors, regardless of the type of cancer they have. The NCI Pediatric MATCH trial is expected to open in late 2016.
- Overcoming Drug Resistance by using tumor profiling and laboratory models to better understand how and why tumors begin to grow again after initially responding to therapy.
- Developing New Laboratory Models for Research by increasing the number of human cancer cell lines and patient-derived tumor samples available for researchers to study.
- Establishing a National “Cancer Knowledge Network” by creating a national database of genomic tumor information, clinical data, and outcomes. This resource will be available for use by scientists, health care professionals, and patients alike.
- Creation of a voluntary national research cohort – The National Institutes of Health (NIH) will use $130 million in PMI funding to launch a research cohort of one million participants who will contribute diverse sources of data—including medical records, profiles of the patient’s genes, metabolites (chemical makeup), and microorganisms in and on the body, environmental and lifestyle data, patient-generated information, and personal device and sensor data. The project seeks to take advantage of existing research and clinical networks and to be broadly accessible to researchers from multiple disciplines. The Office of the National Coordinator for Health Information Technology (ONC) is also allocated $5 million to support the development of standards and requirements to ensure the privacy and security of data exchanged across systems.
- Commitment to protecting privacy – The White House is working with HHS and several other Federal agencies to identify and address legal and technical issues related to the privacy and security of patient data. Privacy and Trust Principles have been developed that encompass the following categories:
- Inclusive, collaborative, and adaptable governance,
- Transparency toward participants and the public,
- Respect of participant preferences,
- Empowerment of participants through access to information,
- Ensuring appropriate data sharing, access, and use, and
- Maintaining data quality and integrity.
- Regulatory modernization – The Food and Drug Administration (FDA) will use $10 million of PMI funding to promote development of high-quality, curated databases to support the regulatory structure needed to advance innovation in precision medicine and protect public health. As part of this effort, the agency has created precisionFDA, a community research and development portal that enables testing, piloting, and validating bioinformatics approaches to processing next generation sequencing. This initiative will facilitate faster and more reliable generation of knowledge about genetic information important to patient care.
- Public-private partnerships – The Obama Administration will use existing research cohorts, patient groups, and the private sector to develop the infrastructure needed to advance the Initiative. The Administration will call on academic medical centers, foundations, researchers, privacy experts, medical ethicists, and medical product innovators to lay the foundation for this effort, including developing new approaches to patient participation and empowerment
Initially referred to as “personalized medicine”, the term “precision medicine” has gained increasing acceptance following a National Academy of Sciences report in 2011. The report called for combining genetic and other molecular data with medical records for large groups of people into a single “knowledge network” that would be used to better understand disease and tailor treatments.
Though biomedical researchers have applied considerable effort in the last few decades to investigate the relationship between specific genetic alterations and epidemiology and progression of disease, recent scientific and technological advances (including a dramatic decrease in genome sequencing costs) have set the stage for the PMI. Similar, albeit smaller initiatives are already underway in the U.S. (Resilience Project and Framingham Heart Study) and abroad (United Kingdom - 100,000 Genomes Project, Japan - DNA Data Bank of Japan, and Iceland - deCODE Genetics).
Endorsements & Opposition
President Obama stated that the promise of the PMI is "...delivering the right treatments at the right time, every time, to the right person." Understanding the genetic underpinnings of disease on an individual level would allow physicians to prescribe treatments with the best predicted outcomes. Although enhanced funding and support for genomic research is welcomed by many researchers, clinicians, and patients, some have begun to voice concerns.
- The majority of biomedical researchers, medical professionals, and pertinent advocacy groups such as the American Association for Cancer Research and Research America! have supported the PMI.
- NIH Director Dr. Francis Collins has been one of the PMI’s most vocal supporters. He has produced testimonies to the Senate and House and participated in numerous interviews (PBS, New England Journal of Medicine (NEJM)) and commentaries (The Journal of the American Medical Association, NEJM) to advocate for the necessity and benefits of this initiative.
- Critics have voiced concerns over a number of issues, including the level of funding, feasibility, unrealistic public expectations, and matters of privacy.
- Other critics have commented that physicians are not qualified or properly trained to interpret complex genetic testing and/or communicate the results to their patients.
- Some have speculated that a focus on health disparities and social determinants of health would be more beneficial to public health