Genome-Based Therapeutics Targeted Drug Discovery and Development: Workshop Summary (2012) / Chapter Skim
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5 Evolving Paradigms
5 Evolving Paradigms
Pages 37-46
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From page 37... ...
· The blending of pre- and postmarket environments could com bine considerations of safety and efficacy with considerations of clinical effectiveness. The fourth session of the workshop featured case studies of organizations and initiatives that have furthered genomic-based approaches to drug discovery and development.
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From page 38... ...
This effort in turn has supported the Multiple Myeloma Personalized Medicine Initiative, which seeks to more fully characterize the range of disease subtypes to enable the development of targeted therapies and potentially curative approaches for patients. Spread across 50 centers and including industry partners, the project combines a 1,000-patient longitudinal study with a companion genomics study that will comprehensively assess the molecular profiles of patient's tumors throughout disease progression and be correlated to clinical interventions, including treatment regimens.
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From page 39... ...
compel and initiate scientific discoveries that are not possible today." In the same manner, by mobilizing the multiple myeloma community through a dedicated online portal, the MMRF aims to accelerate and enable personalized therapies. Key features of the online community include groups based on common molecular profiles, the ability to connect with similar patients, a health metrics tracker, tools to help manage the disease, access to educational materials and data, live Web discussions, and clinical trial recruitment tools.
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From page 40... ...
One early way in which CDER has stimulated innovation in the genomic sciences is through the Voluntary Exploratory Data Submission program. This program allowed companies to share data informally without regulatory consequences; to obtain feedback on trial designs, methodologies, and data interpretation; to gain insights into evolving regulatory practices; to provide experience to facilitate policy development; to discuss data elements used to streamline new drug applications; to educate FDA scientists on emerging data and innovative approaches; and to forge partnerships among scientists from different sectors.
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"It is part and parcel to rational and sound drug development and will probably be applied in almost every scenario in the coming decades." PHARMACY BENEFIT MANAGEMENT AND PHARMACOGENOMICS In the current paradigm of drug discovery and development, the premarket environment and the postmarket environment are separate and distinct (Figure 5-1)
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From page 42... ...
The demand for more safety data cannot be met entirely by randomized controlled trials, so regulators in the United States and Europe have set up sentinel networks to assess postmarket data. Reimbursement bodies are calling for value-based pricing that is tied to the demonstration of comparative effectiveness in the real world.
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From page 43... ...
All of this information can also be important for drug developers who need to make decisions about whether and how to proceed with the development of a particular compound. Companies could utilize a personalized medicine methodology to identify an unmet medical need, for example.
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From page 44... ...
is a comprehensive resource of 3,800 approved and investigational medicines that was designed to facilitate the repurposing of medicines by the scientific community.1 As a recent paper states, the NPC is "a definitive, complete, and non redundant list of all approved molecular entities as a freely available electronic resource and a physical collection of small molecules amenable to high-throughput screening" (Huang et al., 2011)
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From page 45... ...
One particular drug called Auranofin was originally approved for the treatment of rheumatoid arthritis in 1984. Reverse pharmacology revealed the mechanism of action of the drug, and three clinical trial sites are now active.
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From page 46... ...
46 GENOME-BASED THERAPEUTICS mental testing, and when phenotypic screens are done without a prospective plan for translating the results to humans. Repurposing generic drugs provides a tremendous opportunity to improve human health without great additional costs, Austin concluded.
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