OUR VALUE PROPOSITION
How well can we predict the right medicine for the right patient?
The Cabernet value proposition responds to the demands of regulatory authorities, payers, physicians, and patients for greater predictive power in the use of medicines. Pharmacogenetics (PGx)—the study of the genetic basis of variation in individual responses to medicines—addresses these demands from early clinical testing through full clinical development, regulatory approval, and market surveillance.
The potential benefits of incorporating PGx in clinical development include:
Improving R&D Pipeline Efficiency
When genetic makeup underlies response to a drug in development, PGx data inform the search for the right patient population. Efficacy that fails to show across the whole of a broad study population may become clear within a subpopulation defined by PGx analysis, and adverse events that initially appear random may be found limited to a PGx-definable subgroup. Rather than contribute to pipeline attrition, the drug would now have an opportunity to progress, and its market potential could be better assessed. Subsequent studies may then enroll patients according to whether they match the favorable benefit-to-risk profiles that have emerged through the PGx component of the clinical program. This “genetic enrichment” of study populations further raises the chance of success in more expensive, later-phase studies.
Decreasing the Cost and Time of Development
The magnitude of a beneficial drug effect can increase with the homogeneity of the study population. To the extent that PGx measures that homogeneity, it enables the design of smaller and more economical yet still adequately powered studies. Smaller studies may also be faster studies, shortening time to submission, regulatory approval, launch, and peak sales.
Strengthening Regulatory Filings
A regulatory framework is rapidly taking shape to foster the inclusion of PGx data in regulatory submissions and product labeling. The Food and Drug Administration is evaluating a growing number of Voluntary Exploratory Data Submissions. Globally, each region represented by the International Conference on Harmonization has published PGx guidelines or concept papers, and more are to come.
Enhancing Market Acceptance
The predictive power of PGx promises to assume growing importance among physicians and payers vis a vis the conventional product differentiators of chemical structure and mode of action. Hence also the promise of more confident prescribing, better formulary acceptance, and pricing that rewards a more certain clinical return on expenditure. Important to maximizing value will be convenient prognostic tests that determine which patients fit the genetic profile for which a medicine has been designed and thus inform benefit/risk decisions. Ideally, prognostics and therapeutics, developed together, will enter the market simultaneously.
Amid intensifying concerns about drug-related risks, PGx brings additional rigor to the analysis of adverse-event reporting. Just as in development leading to product approval, the application of PGx following approval may determine whether an adverse event signal, if real, arises only in a subpopulation that can be genetically defined. Accordingly, PGx may enable appropriate prescribing and prevent withdrawal from the market, thus preserving useful medicines and corporate assets. It can also support the development of appropriate risk-management programs.