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Clinical Trials

A New Paradigm for Clinical Development

The Clinical Trial in 2015

Publication Date   November 2005
Publisher   Cambridge Healthtech Advisors
Product Type   Report
Pages   95
ISBN Number   not applicable
Product Code   CHA026
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Summary


A New Paradigm for Clinical Development: The Clinical Trial in 2015 outlines an innovative and imaginative strategy for reinventing clinical development, and demonstrates why a complete overhaul of the clinical trials process is feasible from a conceptual, technical and logistical point of view.

The current clinical evaluation process is fraught with inefficiencies, resulting in numerous compound failures and exploding development costs. Until recently, the industry has reacted to the clinical evaluation problem essentially by "streamlining" the existing processes and by introducing information technology in a cautious and evolutionary fashion. While the FDA’s Critical Path Initiative of 2004 showed that the agency is willing to take the lead in working with representatives from industry and academia towards a remedy, this report suggests that a more radical solution is needed.

A New Paradigm for Clinical Development: The Clinical Trial in 2015 proposes a bidirectional approach to accelerate the clinical process and make it more effective. These two avenues, which can be summarized as revamping trial design and as truly pervasive modelling and monitoring driven by information technology, are fundamentally different from each other but need to be implemented in a closely linked fashion. Though radical in effect, none of these changes would involve concepts or technologies that are unknown today.

According to the strategy laid out in the report, the following changes are required:

  • Phase I will assume a new role as a brief confirmatory testing stage for the model for drug-human interactions that the sponsor has proposed.
  • Phases II and III will merge into a single advanced-stage human testing phase involving fewer patients than today, relying on relatively small patient populations that are highly homogenous with respect to key criteria of pharmacological response.
  • Systematic post-marketing studies and a significantly improved and extended post-marketing surveillance system that goes far beyond adverse event reporting will be integrated into a post-marketing monitoring phase that documents real-life use of the newly licensed drug.

    These new processes will be made possible through holistic mathematical models such as the virtual patient, extensive biomarker monitoring, and pervasive computing. With a full implementation of all envisaged changes by the year 2015, the stage would be set for a new world of drug development
  • The pre-approval clinical trial phase might be shortened to about three years and 40-50 percent of all candidate compounds that enter this stage could complete it, with the majority of the failures occurring in the early human validation phase.
  • The crucial function of the advanced-stage human testing phase will be to determine whether efficacy is sufficiently superior over the established standard of therapy to warrant the cost of launch and the mandated post-marketing monitoring.
  • Developers recoup development costs earlier and enjoy a longer life cycle under patent protection, but also benefit from more and closer attention to real-life use of the newly licensed drug.

Content


Chapter 1. Clinical Trials Today

1.1. More Targets, More Research and Development Spending, More Candidate Drugs—and Fewer New Products
1.2. The Clinical Trial Process: From Phase I to Phase IV
1.3. A Strategic Problem Analysis
Why Do Phase III Trials Fail So Frequently?
High-Profile Market Recalls: The Worst-Case Scenario Enacted
Approved but Not Effective in All Eligible Patients

Chapter 2. Current Strategies for Clinical Streamlining

2.1. Cost-Effective Solutions for Clinical Go/No-Go Decisions
Better Disease Models that Are Predictive of Human Exposure
Human Microdosing: "Phase Zero"
2.2. Optimized Project Planning
2.3. Recruiting the "Right" Patients More Quickly—and Keeping Them
Maximizing Outpatient Compliance
"Offshoring" Clinical Trials
2.4. "Information-Rich" Trial Design and Biomarkers
Pharmacogenomics
Biomarkers for Clinical Monitoring
2.5. Electronic Data Capture: Heading for the "E-Trial"
Digitizing the Case Report Form
Interactive Voice Response Systems and Web-Supported Trials
The E-Trial: A "Revolutionary Evolution"
2.6. Mining Clinical Databases

Chapter 3. Forces Shaping Future Clinical Trials

3.1. The Confounding Mega-Trends
3.2. Paradigm Changes Rather than Technological Leaps at the Clinical Inflection Point
3.3. Systems Biology as a Key to Understanding Disease and Patients’ Reactions to Drugs
The Virtual Patient: A "Crash Dummy" for the Pharmaceutical Industry
3.4. Beyond Today's Biomarkers
Molecular Fingerprinting and Metabolomics
Functional Endpoints Defined by Molecular Imaging
Theranostics: The Co-Evolvement of Drugs and Diagnostics
3.5. Pervasive Computing: Can a New Type of Information Technology Bring Trials to New Shores?
Clinical Data Management Systems (CDMS)
The Omnipresent Radiofrequency Tags
Grid Computing, Virtual Trial Organizations, and Data Interchange

Chapter 4. Regulatory Agencies in an Era of Change

4.1. The FDA Takes the Initiative
The FDA's "Exploratory Investigational New Drug Application" Guideline
The FDA's Critical Path Document
The Biomarker Bootstrap Situation
The FDA and the Emerging E-Trial Modalities
4.2. The Thorough QT Trial: An Example for International Coordination of Clinical Study Reform
4.3. The Dwindling Role of the Placebo
4.4. Focused Postmarketing Surveillance Instead of Megatrials

Chapter 5. A Scenario for Clinical Trials in the Year 2015


Chapter 6. Corporate Profiles

Clinphone Group
Compugen
Entelos, Inc.
eTrials Worldwide, Inc.
Gene Network Sciences
IBM Healthcare and Life Sciences
Lifetree Technology LLC

References


Index


List of Figures



Figure 1.1. New Active Substances (NASs) Launched Worldwide, 1995–2004
Figure 1.2. The Drug Development Process
Figure 1.3. Investment Escalation per Successful Compound
Figure 1.4. Attrition during the Clinical Development Process
Figure 2.1. Comparison of the Conventional and the Microdose Approach to Candidate Selection
Figure 2.2. The Radiofrequency Indentifier–Based Med-ic™ ECM™ Smart Package for Clinical Supplies and Output from Med-ic™ Certi-Scan™ Software
Figure 2.3. Data Flow and Processing in a Typical E-Clinical Trial
Figure 3.1. Projected Schematic Development of Medicine and Healthcare toward Personalized Medicine
Figure 3.2. Representation of Raw Data from a Clinical Trial that Allows Patients to be Clustered Based on Drug Response
Figure 3.3. Schematic Representation of the VOTES Clinical Trial Grid Computing Study
Figure 4.1. The FDA’s Concept of the Three Dimensions of the “Critical Path” in Drug Development

Table 2.1. F eatures of Typical Electronic Patient-Reported Outcome Information Technology Tools