IMPACT OF PHARMACOGENOMICS ON THE PHARMACEUTICAL INDUSTRY

Viktoriya BuchkoAmerican Institute of Chemical Engineers

August 6, 2008

Pharmacogenomics = drug therapy + genetic diagnostic

test

OVERVIEW

Relevance: realization in 5-10 years Especially because of the passage of Genetic

Information Non-discrimination Act (GINA) 2008 Objectives:

Increase safety and effectiveness of drugs Efficiency and economical concerns Do not exacerbate health disparity concerns

METHODOLOGY

0.1% genetic difference Limit market size Use biomarkers or other diagnostic tests

BENEFITS

Short-term Reduce side effects Reduce trial-and-error dosingLong-term Improve health care system Reduce health disparities

CURRENT PHARMACEUTICAL DRUG DEVELOPMENT MODEL

2-3 years 2-3 years3-5 years 3-7 years

Compound ID’ing

Clinical TrialsI: 20-30 healthy volunteers safety & dosageII: 100-300 patient volunteers efficacy & side effectsIII: 1000-15000 patient volunteers long-term use & ADRs

Preclinical Studies-lab & animal testing-toxicity research-safety

Examination-New Drug App.

DIFFICULTY IN DRUG DEVELOPMENT

Increased R&D costs, yet fewer new drugs approved

9% of candidates filed for NDA 1/10 on market considered market success Competition from generics, “me-too” drugs

PHARMACOGENOMIC SOLUTIONS

Decrease development time Faster approach to drug candidate

identification Methods for approval

New niches, such as anti-obesity drugs Rescue drugs

Less risk

CONCERNS WITH PHARMACOGENOMIC-BASED BUSINESS MODEL

High-risk for large pharmaceutical companies Identifying genetic basis Post-marketing surveillance studies

POLICY ACTION TO FACILITATE PHARMACOGENOMICS

Genetic test standardization Laboratory Test Improvement Act (S. 736)

Integration of clinical and genomic data Genomics and Personalized Medicines Act (S.

976) Mitigate ethical concerns

Distributive justice Selection & prioritization of drugs Enact the Institutional Review Board

Incentive for coupling diagnostic and drug treatment S. 976 calls for Nat’l Academy of Sciences for

analysis Mandate Phase IV clinical trials

POLICY ACTION TO FACILITATE PHARMACOGENOMICS

KEY FINDINGS

Segmenting drug development industry Cooperation of large pharmaceutical

companies with smaller biotech companies Immediate relevance in Mendelian one-gene-

based diseases Genetic tests as diagnostics most pertinent

in all-or-nothing benefit Genetic test to be first used in complement

to a drug already on the market New uses for current drugs

CONCLUSIONS

With passage of GINA 2008, greater security in genetic testing will allow for the realization of personalized medicine.

Must increase regulatory control to keep pace with the developing pharmacogenomic-based drugs.

Increase public awareness, including that of physicians, of the possibilities of safer, more efficacious drug treatments.

QUESTIONS

BIDIL: TO REDUCE HEART FAILURE IN SELF-IDENTIFIED BLACK INDIVIDUALS

Twice the fatality rate in black individuals than other Americans

Reduce disparity through minority recruitment

Reduce mortality rate by 43%, 1st hospitalization rate by 33%

Statistic valid for 45-64 year olds, where only 6% of mortality population is accounted for

Commercial tactic to obtain new patent

Self-identification is not proof of genetic basis

Assessment of disparities in population targeting

Level of significance to determine variation within group

Off-labeling present? Follow-up genetic studies

PROOF CRITICISM

IMPLICATIONS OF GENETIC TESTING

Access predictive info Constraints Issues with direct-to-consumer genetic tests Method of administration