Abstract
Pharmacogenomics (PGx) is often considered "the great new wave" in medicine,
promising better, safer, and more affordable healthcare. Yet its adoption and
widespread use in the clinic is up against some tremendous challenges. This
report considers:
- Drivers of PGx
- Applications of PGx in drug development
- Industry experiences with PGx and lessons learned
- Type of evidence needed to establish clinical utility
- Challenges to moving PGx forward
- Survey results and expert views on utilization of PGx
Pharmacogenomics: Delivering on the Promise explores the pharmacogenomic (PGx)
realm of personalized medicine, analyzing current R&D and market trends
related to the use of genetic information to predict how well patients will
respond to certain drugs. Pharmacogenomics is an extremely difficult business,
one for which there are no easy answers. Even companies manufacturing and
marketing already successful pharmacogenomic drug-test combinations continue
to face difficulties. This report examines how these and other companies have
(or are) navigating through the scientific, statistical/experimental design,
and "clinical utility" landscape of PGx.
Not the least of the challenges facing pharmacogenomics is scientific.
Separating a consistent, predictive association between a SNP or other genetic
marker and a drug response phenotype from all the other variables that play
into drug response can be next to impossible. Once candidate associations are
identified, knowing how to design clinical trials capable of teasing out these
associations in the clinic and aligning those trials in preparation for
regulatory review create another set of challenges. Pharmacogenomics:
Delivering on the Promise considers prospective versus retrospective clinical
trial design and when, if ever, the FDA might allow the latter for regulatory
decision-making purposes.
Moreover, validating an association in a carefully controlled clinical setting
is different than knowing whether or not that association is truly clinically
useful. The risk of a product not being adopted as the standard of care
(either by prescribers, payers, or patients) because of insufficient evidence
regarding its "clinical utility" is clearly a disincentive. Varying opinions
raise questions about the nature of the evidence necessary for establishing
the clinical utility of PGx tests. We consider the growing skepticism about
the universal nature of the randomized controlled trial (RCT) gold standard
and the usefulness of non-RCT experimental designs with respect to genetic
testing.
Challenges examined include economic, reimbursement, regulatory,
technological, and those related to a lack of physician education and
awareness of pharmacogenetic testing. Overcoming these challenges could enable
physicians to engage in smart PGx prescribing, leading to increased efficacy
and reduced adverse drug reactions. PGx also has important applications in
drug development, both with new drugs or new indications and as a way to
"rescue" failed drugs; these various uses of PGx are explored in detail.
Pharmacogenomics: Delivering on the Promise also includes comprehensive
interviews with experts in the field and results from a qualitative survey of
individuals involved with pharmacogenomics.
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