Cancer Pharmacoepidemiology and Pharmacogenomics
Pharmacoepidemiology uses observational methods to study the benefits and risks of drug therapy in clinical and population settings. In the field of cancer, pharmacoepidemiology examines the effects of medications on cancer risk, disease prevention, and response to treatments, as well as any adverse and/or long-term effects of chemotherapeutic and other pharmacologic agents used to treat cancer.
Pharmacogenomics focuses on understanding how variability in genes encoding for drug-metabolizing enzymes, drug transporters, drug receptors, and proteins involved in pathway signaling influences individual differences in adverse effects and treatment efficacy and effectiveness. Together the evolving and emerging fields of pharmacoepidemiology and pharmacogenomics, along with the expanded development and approval of new, targeted cancer therapies, are key trends converging to create important new opportunities in cancer research and control and provide the basis for personalized cancer therapy.
NCI has an increasing focus on pharmacoepidemiology related to pharmaceutical use and cancer risk, recurrence and survival, as well as pharmacoepidemiology related to treatment response and adverse outcomes from chemotherapeutic agents and other medications used to treat cancer. As more treatment options become available, it becomes increasingly important to identify patients at the highest risk for both short- and long-term adverse events and to identify treatment regimens that may reduce such risks and improve quality of life.
In January 2008, NCI established the Trans-NCI Pharmacogenomics and Pharmacoepidemiology Working Group (PPWG) to support development of a comprehensive and interdisciplinary pharmacoepidemiology and pharmacogenomics cancer research program. The PPWG published recommendations for future research directions in the November 10, 2010 issue of the Journal of the National Cancer Institute: Cancer Pharmacogenomics and Pharmacoepidemiology: Setting a Research Agenda to Accelerate Translation. Key recommendations included:
- Supporting the routine collection of germline and tumor biospecimens in NCI-sponsored clinical trials and in some observational and population-based studies;
- Developing and supporting opportunities to identify clinical, sociodemographic, lifestyle, and genomic markers related to treatment response and/or adverse events in NCI-sponsored clinical trials;
- Supporting observational studies that identify clinical, sociodemographic, lifestyle, and genomic factors of treatment response and adverse events;
- Incorporating pharmacogenomic markers into clinical trials;
- Addressing the ethical, legal, social, and biospecimen- and data-sharing implications of pharmacogenomic and pharmacoepidemiologic research; and
- Establishing partnerships across NCI, with other federal agencies, and with industry.
There are not currently any specific NCI Requests for Applications (RFAs) or Program Announcements (PAs) for cancer pharmacoepidemiology and pharmacogenomics research; however, related NIH Funding Opportunity Announcements (FOA) are available:
- Enabling Resources for Pharmacogenomics - expires September 26, 2016, unless reissued
- PAR-14-185 (R24)
- Research Centers for Pharmacogenomics in Precision Medicine - expires September 26, 2016, unless reissued
- PAR-14-075 (P50)
EGRP joins with other NCI Divisions, Offices, and Centers and other Institutes and Centers at the National Institutes of Health (NIH) to fund grant applications submitted in response to FOAs. View the full list of EGRP FOAs.
EGRP also encourages investigator-initiated grant applications on pharmacoepidemiology and pharmacogenomics topics.
NCI's Division of Cancer Control and Population Sciences (DCCPS) has sponsored the following workshops related to cancer pharmacogenomics and/or pharmacoepidemiology research:
- Cancer Pharmacogenomics: Setting a Research Agenda to Accelerate Translation (July 2009)
- Cancer Pharmacogenomics: Integrating Discoveries in Basic, Clinical and Population Sciences to Advance Predictive Cancer Care (October 2010)
- Methodological Considerations in Evaluation of Cancer as an Adverse Outcome Associated With Use of Non-Oncological Drugs and Biological Products in the Postapproval Setting (September 2014)
- Cancer Epidemiology Cohorts
Cohorts are powerful tools with the potential to address scientific questions not amenable, or unlikely to be addressed, by other study designs like prevention or clinical trials. Evidence generated has also been useful in providing the basis for the design and testing of many preventive, therapeutic, and health policy interventions.
- Cancer Registries
This compilation of resources provides links to information on cancer statistics, such as incidence, mortality, and the prevalence of certain risk factors. It may be possible to link these statistics to national- or state-specific pharmacological databases.
- Cancer Research Consortia
EGRP supports a growing number of national and international research consortia focusing on intradisciplinary and translational research on common and rare cancers.
- Pharmacoepidemiology and Healthcare Databases
These databases can be used to access national and international data that can help answer some of the outstanding questions of cancer-related outcomes (positive or negative) associated with pharmaceuticals.
- Pharmacogenomic Resources
Links to pharmacogenomics collaborative opportunities, consortia, and networks; databases related to pharmacogenomics research; knowledge synthesis resources; reports; and toolkits.
- Research Studies and Networks
These large epidemiological studies contain detailed information on pharmaceutical use and disease risk, including cancer. These studies may assist in evaluating associations between certain pharmaceuticals and disease risk.
Links to surveys that may be useful for pharmacogenomics research.
- Andrew N. Freedman, Ph.D., Chief, Clinical and Translational Epidemiology Branch