Life Science Insights TelebriefingLife Science Insights Telebriefing
Molecular Biomarkers: The Elusive and Expensive Molecular Biomarkers: The Elusive and Expensive Search for Powerful Tools That Could Redefine Search for Powerful Tools That Could Redefine Drug Discovery and DevelopmentDrug Discovery and Development
Biomarkers Defined Types of Biomarkers Being Used Current Biomarker Areas of Focus
Pharma Challenges in Drug Discovery and Development ROI Benefits for Utilizing Biomarkers Use of Biomarkers in Development Use of Multiple Biomarkers Forecasted Investment in Biomarkers for 2004 - 2007 FDA Critical Path Initiative Risks of Reporting Toxicogenomics Data Conclusions
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Global market research and advisory firm in the life sciences,subsidiary company of IDC
Work closely with IDG and its life sciences publication, Bio-IT World Staff comprised of life science specialized analysts, researchers & consultants Provide market research and strategic advice on the convergence of IT and
the biopharmaceutical value chain Offer consulting services to solve your unique business challenges
Molecular Biomarkers Research AnalysisMolecular Biomarkers Research Analysis
Initial study sponsored by Applera and Abbott as part of “Targeted Medicine” Scientific American Conference in NYC last November Phone interviews with opinion leaders and researchers in
the field
LSI research focuses on biomarkers as part of our clinical trial research agenda Contact with industry leaders Following FDA Critical Path Initiative
A biomarker is a characteristic that can be objectively measured as an indicator of normal biologic processes, pathogenic processes or pharmacological responses to therapeutic intervention.
Types of biomarkers currently used:
•Proteins: Applicable throughout the drug discovery and development process; longest history as biomarkers
•RNA Expression Profiling: Gene arrays used in discovery phase and clinical trials
•SNPs (Single Nucleotide Polymorphisms): Large patient populations required has hindered use but should increase in the future
•Small Molecules: Used the least; may increase with the successful application of metabonomics
Pharma Challenges in Drug Discovery & DevelopmentPharma Challenges in Drug Discovery & Development
Escalating Cost of Bringing Drugs to Market Estimates range from $800M to $1.7B
Emergence of New and Expensive Technologies and Screening Platforms Risk/Benefit decisions needed, e.g.which discoveries will most likely
succeed
Estimated Drought Within Company Pipelines for New Drugs Drug development has not kept pace with new discoveries FDA has seen a dramatic decrease in the number of NDAs filed Increased Need to Partner/Acquire or In-License
Competitive Positioning
New Initiatives in a Post-Vioxx World New FDA Drug Safety Oversight Board to monitor safety of marketed drugs Public perception of prescription costs, safety issues
ROI Benefits for Utilizing BiomarkersROI Benefits for Utilizing Biomarkers
Throughout drug discovery and development value chain, molecular biomarkers can play a key role in decision making, most important is the ability to kill drugs earlier. Early Drug Discovery
Elucidation of disease mechanism, target validation Determination of efficacy and toxicity in in vitro testing
Preclinical Development Determination of efficacy and toxicity in in vivo testing Finding key relevant biomarkers to move into clinical testing phases
Clinical Development Determination of efficacy and toxicity in man
Potential for prospectively testing patients for entry into trials, reducing trial size and drop out rates
Monitoring outcomes of disease Providing better, safer therapies for patients
Use of Multiple vs. Single Biomarkers Use of Multiple vs. Single Biomarkers
Multiple (62%)
Single (38%)
Multiple markers and panels (e.g., metabolites, genes, and proteins) are considered of greater relevance to drug development than single markers. Of those who stated that they are currently using single markers, most shared the opinion that the future will focus on multiple markers.
n=17 Source: Life Science Insights, Applera, and Abbott's Biomarker Survey, November 2004
Innovation or Stagnation? Challenge and Opportunity on the Critical Path to New Medical Products [online], <http://www.fda.gov/oc/initiatives/criticalpath/whitepaper.html> (2004)
FDA Critical Path InitiativeFDA Critical Path InitiativeConcern:
•One half of all drugs moving to Phase III fail to emerge as new therapeutics •Drop in number of NDAs being filed
Goal: •Move drugs more quickly and safely through clinical trials•Increase the predictability of drug success
Potential for Biomarkers in Clinical Trials:•Change the risk/benefit ratio for patients•Reduce number of patients enrolled•Enrich the population of patients who will benefit •Reduce the likelihood of adverse events
FDA is establishing an Interdisciplinary Pharmacogenomics Review Group and a pharmacogenomics advisory subcommittee to assess data and correlations between biomarkers and clinical outcomes
FDA is encouraging submission of unvalidated biomarkers with filings
Submission of Unvalidated Biomarker Data to FDASubmission of Unvalidated Biomarker Data to FDA
n=21Source: Life Science Insights/ Applera/ Abbott Biomarker Survey Nov 2004
Key Points:
•Still considerable resistance until regulatory issues are defined. •Pharma and biotechs are aligned in their approach to voluntary submission. •Some want to be seen as leaders; some identify themselves as conservative.
Factors Preventing Wider Adoption of BiomarkersFactors Preventing Wider Adoption of Biomarkers
Factors preventing the wider adoption of biomarkers focused on two areas -- internal and external problems:
Internal: The lack of a defined strategy to date The need for organizational communication and coordination across departments The difficulty in identifying biomarkers The difficulty in finding the right balance of effort between the new approaches and
the more traditional approaches and then driving those changes throughout the organization
External: Regulatory climate and uncertainties of future FDA requirements The consequent impact on internal conflicts about whether companies want to
have information that they cannot explain No payback for the effort (due to the state of regulation and the state of the
Key Market Trends and DriversKey Market Trends and Drivers
Drug companies will continue to increase biomarker investments primarily focused at early discovery stage through Phase I/IIa
Key drivers for increased biomarker investment include: Increased need in data integration and data mining tools for real-time use of
information during trial Ability to make earlier go/no go decisions Accelerated time to market Bringing safer and more efficacious products to patients
In addition, companies are starting to voluntarily submit data to the FDA, but some want more clarity on requirements
Key trends include: Toxicology biomarkers will be regulated and required Single markers are seen as clinical ideal; however, companies will continue
to pursue panels of multiple markers. Firms will aggressively plan a strategy for companion diagnostics that may
Investment in biomarker research has dramatically grown and will continue to grow as companies realize the potential for savings at all phases of drug development.
Systems biology and new technologies, including new nanotechnology platforms, will continue to evolve, driving additional spending as novel approaches move into the predictive and preventive medicine arena.
Companies are alert that their competition is actively involved in the same activities, and there is a recognized need for identification of validated biomarkers.
Investment payoff will be earlier go/no go decisions and quicker downstream returns.
Biomarkers will enable segmentation of patient populations during clinical trials; hence, the clinical trials of the future will be smaller and safer.
