Pharmacogenomics and Health Policy Amalia M. Issa, PhD, MPH Founding Director, Program in Personalized Medicine & Targeted Therapeutics Professor and Chair Department of Health Policy and Public Health University of the Sciences Philadelphia, PA
Disclosures I didn t do it. Nobody saw me do it. You can t prove anything.! None related to this presentation.! No Commercially Relevant Financial Relationships
What this talk is about What could we learn now that would help establish the potential utility of pharmacogenomic products? Provide an update on policy-relevant questions in pharmacogenomics Discuss some methods to investigate policyrelevant questions in pharmacogenomics
Successes of Genome Wide Association Studies (GWAS) Celiac disease Atrial fibrillation Colorectal cancer Breast cancer Prostate cancer Diabetes Gallstones Asthma Multiple sclerosis Rheumatoid arthritis Crohn disease Age-related macular degeneration Manolio TA. N Engl J Med 2010;363:166-176.
How is Pharmacogenomics being used? Drug development Drug product labels Large-scale global studies Utilization by pharmacy benefit managers Improvements in technology
Clinical Applications of Pharmacogenomics
Drug Labels and Pharmacogenomics Drug Safety " HLA-B*5701 for abacavir " CYP2D6 in nursing mothers taking codeine Drug Efficacy " CYP2C9 and VKORC1 for warfarin " CYP2C19 for clopidogrel " CYP2D6 and tamoxifen
Some Current Pharmacogenomic Based Drugs Available Today
Coming to a clinic near you "
What Do We Do With Genes When We Find Them? Key Stakeholders in PGx
Factors Driving Personalized Medicine Forward # State of the science and information systems now evolved to enable personalized medicine # Regulatory agencies have taken steps to integrate biomarkers into drug development # Payer struggles to balance quality and costs # Limit access to responders/subpopulations effectiveness & safety # wastage and unnecessary costs # Manage increasingly expensive drug categories (e.g., oncology) # Aggregate affordability of health services is a significant concern in many nations # Manufacturers recognize challenging reimbursement landscape and emphasis on PM is one tool to support market access
The Goal of Health Policy Research in Pharmacogenomics: Bridging the Research Application Gap Goal: To develop an evidence-base to maximize Impact, Adoption, Reach Science Documenting, improving & communicating discovery and knowledge Delivery Capacity Building the capacity of relevant systems to apply the knowledge Market pull and demand
Policy A set of principles guiding decisionmaking Public Policy: policy of governments Health Policy: health promotion, health protection, health services (plus ) Clinical Policy: policy related to the functioning of clinical environments
Why Conduct Policy-Relevant Research? To provide rigorous evidence to appropriate stakeholders (clinicians, patients, government regulatory agencies, payers, etc.) To impact decision making
Policy-Relevant Research Questions in Pharmacogenomics
Policy-Relevant Concerns about Pharmacogenomics: Stakeholder Perspectives
Policy Analysis Process Evaluation Policy Issue Implementation Issue Adoption Issue Identification
Research Tool Box Surveillance Quantitative Risk Assessment Economic Evaluation Expert Panels Systematic Reviews Meta-Analysis & Evidence Synthesis New Methods
Relationship between Clinical Epidemiology and Policy Basic (Bench) Research - Uncovers Promising Intervention Epidemiology Assesses Efficacy and Effectiveness Economic Evaluation Assesses Efficiency Health Policy Assesses Implementation Clinical Policy Consensus Statements; Formulary Decisions; CPGs Health Policy Evaluation of the use of research findings & determinants of that use
Example of Research to Inform Policy
Markov decision analytic modeling
Oncotype DX is dominated Why this study is interesting:
What does this mean for pharmacoepidemiologists? Opportunities to get involved in pharmacogenomic and drug safety studies Opportunities for development of methods to ensure analytical and clinical validity of genotype-phenotype associations Opportunities to help manage the data deluge (databases) Health policy has to be informed by evidence and good data!