Zuckerman and Milne at the Tufts Center for the Study of Drug Development (CSDD) have published results from interviews conducted in 2009 – 2010 with 20 individuals from 13 biopharmaceutical companies to understand the current and future state of personalized medicine. The definition of personalized medicine used in those interviews was: “A medical intervention (i.e., drug, biologic or vaccine) tailored by the physician to the genetic, genomic or proteomic characteristics of individual patients, or subpopulations, drawing on data gathered from a variety of sources (including individual genetic variation, differences in molecular-level and cellular-level disease processes, health states, behavioral and environmental determinants, and response to treatment).
The personalized medicine will contain such information on the label. It is possible the approved labeling for such a product may suggest the use of a diagnostic to select patients for treatment or to monitor efficacy or safety.” It certainly is a comprehensive definition that is slowly but surely moving towards realization. One of the observations from the CSDD study was that on average up to half of a company’s pipeline projects had associated biomarkers, but only less than 10% of projects had identified specific target populations or companion diagnostics. Also, all respondents interviewed noted that biomarker research is done for compounds before they enter clinical development, and that the primary intent of biomarker development was to provide more information about products internally, not for prescribing or monitoring a marketed product. These observations expose a very apparent gap in the necessary incorporation of biomarkers in clinical trial design and implementation. Beyond the scientific and cost problems, respondents of the interview considered that utilizing pharmacogenomic data was challenging because of a lack of regulatory guidance. Although progress has been made, such as the release of a draft guidance by the FDA on pharmacogenomics in early-phase clinical studies, many companies felt that they were unable to use pharmacogenomic data in a regulatory approval package until pathways are better defined.
Given the scientific barriers, economic toll, regulatory evolution, and commercial uncertainties associated with personalized medicine, what are the incentives for biopharmaceutical companies to develop personalized medicines as defined above?
The obvious invitation is for pharmaceutical and biotechnology companies to take a long-term investment view. And, importantly, to refocus on creating differentiated clinical outcome value during the drug discovery and clinical trial process. The drugs in development today will be entering markets with more competitors, more pricing pressures as well as higher standards for better clinical outcomes. The driving force should be clinical value, supported by practical innovative models for risk-sharing along the way.
The expectation is that over the next few decades, more personalized medicines will be created. In order to support this and reap the benefits of personalized medicine, all stakeholders should work together to help re-shape and create well-aligned incentive structures.
Rachael Zuckerman & Christopher-Paul Milne. Market watch: Industry perspectives on personalized medicine. Nature Reviews Drug Discovery 11, 178 (March 2012).
By Mirella Zuleida