Abstract
s2-1 Scientific understanding of the genetic aberrations driving disease is advancing at a rapid pace, heralding the advent of the long awaited era of personalized medicine. For patients, personalized medicine promises the opportunity to benefit from the most effective treatment that targets the fundamental driver of their disease, while also potentially avoiding toxicity. For payers, personalized medicine is attractive as a mechanism to control usage of expensive drugs, and avoid wasteful expenditure on treatments that are ineffective. For the pharmaceutical industry personalized medicine presents both challenges and opportunities. Many pharmaceutical companies have committed to the vision of ‘right drug, right patient, right time‘, particularly in therapeutic areas such as oncology and neuroscience. Pharma recognizes that this strategy provides the opportunity to achieve substantial clinical advances in specific patient populations, compared with currently available “non-specific” medications which is expected to create a compelling value proposition and to facilitate reimbursement by payers. Drug development timelines could be accelerated, and success rates improved by conducting trials in molecularly selected patient populations that result in more rapid proof of concept and more robust clinical outcomes, allowing smaller Phase 3 trials. However, there are also significant challenges. Most importantly, the drug developer must be able to achieve the requisite return on investment despite the restricted market size. In addition, drug development costs may be increased due to the complexities of biomarker analysis and diagnostic development. Molecular profiling is an emerging science, and several large and expensive drug development programs have faltered due to the selection of the wrong biomarker to guide patient selection. Trials involving biomarkers are attracting high interest from researchers, but require new competencies in trial design, data analysis and investigator expertise in sample collection and management. Greater statistical and computing power is necessary to mine data for complicated relationships between genetic, biological and environmental factors. Additionally, if the right biomarker was not identified prospectively, there is currently no regulatory pathway for approval via a retrospective analysis. Regulatory approval pathways for therapeutics and their companion diagnostics are not well established. In the US, the diagnostic and therapeutic are reviewed by different agencies, and the requirements of each are not completely aligned, creating additional complexity. Global regulatory processes for companion diagnostics are even more diverse and challenging. Additionally, the companion diagnostic needs to be available for validation during the clinical development phase, but with the risks inherent in drug development, and limited reimbursement, diagnostic manufacturers have little incentive to develop such tests “at risk”. Despite the challenges, personalized medicine is widely believed to offer the best prospect of effective treatment and cure for patients with serious diseases. The relevant stakeholders – Pharma and biotech, diagnostic companies, regulatory agencies, payers and policy makers, must be committed to working together to provide incentives and remove obstacles so that this goal can become a reality.