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Market Research Report

Accelerating Drugs to Market - Despite Challenges, Adaptive Clinical Trials Reduce Drug Development Costs and Time to Market

Published by GBI Research Product code 250519
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Accelerating Drugs to Market - Despite Challenges, Adaptive Clinical Trials Reduce Drug Development Costs and Time to Market
Published: September 11, 2012 Content info: 63 Pages
Description

Summary

GBI Research's new report, "Accelerating Drugs to Market - Despite Challenges, Adaptive Clinical Trials Reduce Drug Development Costs and Time to Market", presents various tools and strategies which can accelerate a drug to the market. In this report, GBI Research has studied various hurdles at different stages of drug development that can halt a drug's development. The report provides detailed information about the need for accelerated drug development. Declining R&D productivity is highlighted as one of the major needs to be addressed. The report outlines misconceptions regarding accelerated drug development; one such major misconception is the cost of development. The cost of an accelerated development program can be effectively managed by implementing a structured and complete program. The report highlights major strategies adopted by pharmaceutical companies to accelerate drug development. The adoption of the latest technologies in lead generation, preclinical stages, and the use of adaptive designs in late phase studies are regarded as tools to accelerate drugs through these stages of development.

This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research's team of industry experts.

The process of drug development starts from the initial discovery and ends with a final medication. This is an expensive, lengthy and incremental process. The main objective of the process is to identify a molecule with the potential for producing the desired effect in the human body, and to establish the quality, safety and efficacy of the molecule for treating patients. In the present scenario drug development takes about 12 years, for a molecule to progress from the laboratory and enter the pharmaceutical market. It is estimated that out of 5,000 compounds which enter the preclinical stage of development, only five compounds will be successful enough to be tested on humans, and only one among them will be approved. The slow pace of drug development greatly affects the pharmaceutical industry and patients who are in need of new therapeutics to treat their illness.

The current process of drug development begins with the synthesis of molecules, which targets specific proteins in living cells. It is followed by in vitro tests to identify any specific toxicity associated with the synthesized molecules. The compounds which make it through this stage will go further and will be tested for in vivo toxicology studies. The information gathered from these studies is utilized for planning and conducting clinical trials in human subjects.

It is important for biopharmaceutical companies to launch their products more quickly in the market, as this will lead to early revenue generation from the product. As a large number of drugs fail at the later stages of drug development, pharmaceutical companies try to maintain their revenues by launching new drugs at the earliest possible time. The decline in the total number of new drug approvals by the regulatory bodies and the patent expirations for major blockbuster drugs are forcing pharmaceutical companies to consider ways in which the time and cost of clinical trials can be reduced without affecting their quality.

Scope

  • The report presents various tools and strategies which can help to accelerate a drug to market.
  • The report provides detailed information about the need for accelerated drug development.
  • The report outlines misconceptions regarding accelerated drug development.
  • The report provides information on trends in drug transition and strategies and models adopted to accelerate drug transition through the various stages of development.
  • Description of the methods for optimum patient recruitment and retention in a clinical trial.
  • Analysis of efficient clinical trial site management so that completion of a trial is done on time.

Reasons to buy

  • Develop strategies to implement the use of various technologies for advancing drug discovery and development in an efficient manner.
  • Understand the use of biomarkers and surrogate endpoints, improvised clinical trial designs and better recruitment and retention of subjects in clinical trials, in order to avoid drug lags in various phases of development.
  • Prioritize design elements of study protocols and balance the overall protocol.
  • Ensure efficient clinical trial outcomes by implementing CDISC standards.
  • Understand the utility of operationally seamless Phase II/III design for instantaneous transition of the drug from Phase II to Phase III.

Executive Summary

Headache for Pharma as R&D Spending Climbs While Number of Approved Molecules Falls

Low research and development (R&D) productivity, combined with the patent cliff and a stringent US Food and Drug Administration (FDA) approval process, is resulting in climbing expenditure without the corresponding output, states the latest report from industry experts GBI Research.

The company's new report* says that, despite efforts made by pharmaceutical firms to cut down on costs, R&D expenditure expanded at a Compound Annual Growth Rate (CAGR) of 6% from $26 billion in 2000 to $50 billion by the end of 2011. Conversely, the number of new molecular entities (NME) approved during this same period has dropped on average, decreasing at a CAGR of 1%.

R&D is a core and integral part of the pharmaceutical industry, but poor productivity means there may soon be a drought in the R&D pipeline. GBI Research estimates that currently as much as 55% of the entire late stage pipeline is made up of life cycle management (LCM) projects, while a 28% share of the industry's top 20 companies' pipelines is devoted to LCM research.

To compound the frustrations of the industry, the FDA has adopted a stricter drug approval policy. Following controversies regarding products such as Vioxx and Exubera, as well as product recalls for 'Made in China' drugs, the agency has been more cautious in analyzing the risks and benefits of drugs before approving them, slowing down or cutting out potentially marketable treatments.

Additionally, the patent cliff has damaged company revenues, severely curbing the selling power of blockbuster medications and opening the market to generics. The reduction in NME approvals has meant the big pharmaceutical firms are less able to offset the loss of revenue resulting from patent expirations, with new drugs offering therapeutic superiority to the generic versions of their predecessors.

Accelerating Drugs to Market - Despite Challenges, Adaptive Clinical Trials Reduce Drug Development Costs and Time to Market

In this report, GBI Research has studied various hurdles at different stages of drug development that can halt a drug's progress. The report provides detailed information about the need for accelerated drug development.

This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by GBI Research's team of industry experts.

Table of Contents

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Introduction

3. Accelerating Drugs to Market - Overview

  • 3.1. Drug Development Process
    • 3.1.1. Early Stage Drug Discovery
    • 3.1.2. Clinical Development Time and Protocol Amendments
    • 3.1.3. Phase I
    • 3.1.4. Phase II
    • 3.1.5. Phase III
    • 3.1.6. Registration
    • 3.1.7. Phase IV
  • 3.2. Need to Accelerate the Drug Development Process
    • 3.2.1. Strict Regime in Regulations of Drug Approvals
    • 3.2.2. Declining Returns on R&D Investment
    • 3.2.3. Low R&D Productivity
  • 3.3. Misconceptions of Accelerated Drug Development
    • 3.3.1. Completeness of the Drug Development Process
    • 3.3.2. Cost of Accelerated Drug Development
    • 3.3.3. Quality of Study in Accelerated Drug Development
  • 3.4. Causes of Delay in Drug Development
    • 3.4.1. Delay during the Nonclinical Stage of Drug Development
    • 3.4.2. Delay During the Clinical Stage of Drug Development

4. Accelerating Drugs Through the Lead Generation Phase

  • 4.1. Process of Lead Generation
    • 4.1.1. Pre-Discovery
    • 4.1.2. Target Identification
    • 4.1.3. Target Validation
    • 4.1.4. Lead Identification
    • 4.1.5. Early Safety Tests
    • 4.1.6. Lead Optimization
  • 4.2. Traditional Strategies of Lead Generation
    • 4.2.1. High Throughput Screening
    • 4.2.2. In Vitro Studies of Drug Absorption
    • 4.2.3. In Vitro Studies of Protein Binding
    • 4.2.4. Fragment-Based Lead Discovery
    • 4.2.5. Antisense Technology
    • 4.2.6. Iterative Focused Screening
  • 4.3. Emerging Strategies of Lead Generation
    • 4.3.1. Improving High Throughput Screening
    • 4.3.2. Improving In Vitro Assays for HTS
    • 4.3.3. Whole Animal Imaging and Microscopy
    • 4.3.4. Computerized Combinatorial Chemistry and 3D Molecular Modeling
    • 4.3.5. Molecular Bioimaging
    • 4.3.6. Omics-Technology and Bioinformatics
    • 4.3.7. Outsourcing of Lead Generation

5. Accelerating Drugs Through the Preclinical Stage

  • 5.1. Preclinical Studies - An Overview
  • 5.2. Preclinical Study Design and Planning
    • 5.2.1. Preclinical Study - Strategic Planning
    • 5.2.2. Key Considerations During Preclinical Study Design
  • 5.3. Strategies and Models to Accelerate the Transition from Preclinical Phase to Clinical Phase I
    • 5.3.1. In Vitro ADMET Screening Models
    • 5.3.2. In Vivo ADMET Screening Models
    • 5.3.3. In Silico ADMET Screening Models
    • 5.3.4. Accelerating Drugs to Market through Effective Documentation in the Preclinical Phase
  • 5.4. Recent Technology Developments
    • 5.4.1. Biomarkers
    • 5.4.2. Nanotechnology
    • 5.4.3. In Vivo Imaging
  • 5.5. Accelerating Drugs to Market - Preclinical Models: Case Studies
    • 5.5.1. Apredica's Customized In Vitro ADMET Screening Assays
    • 5.5.2. AVEO Pharmaceuticals' Breakthrough with Transgenic Mouse Model for Human Breast Cancer
    • 5.5.3. Preclinical Models of Hepatocellular Carcinoma and Biomarker Strategy by Pfizer
    • 5.5.4. Simulation Modeling to Treat Spinal Cord Injuries by Novartis

6. Accelerating Drug Transition in Phase I Studies

  • 6.1. Phase I Clinical Studies - An overview
  • 6.2. Phase I Study Design and Planning
    • 6.2.1. Standard Design
  • 6.3. Strategies and Models to Accelerate Transition from Phase I to Phase II
    • 6.3.1. Site Selection and Management
    • 6.3.2. Strategies to Minimize Site Initiation Delays
    • 6.3.3. Role of the Site Management Organization in Decreasing Timelines
    • 6.3.4. Optimizing Clinical Trial Supply through a Clinical Trial Management System
    • 6.3.5. Patient Recruitment Strategies

7. Accelerating Drug Transition in Phase II Studies

  • 7.1. Phase II Clinical Studies - An Overview
  • 7.2. Phase II Study Design and Planning
  • 7.3. Strategies and Models to Accelerate Transition from Phase II to Phase III
    • 7.3.1. Selection of Primary Endpoint
    • 7.3.2. Randomization of Phase II Trials
    • 7.3.3. Use of Biomarkers
    • 7.3.4. Statistical Designs in Phase II Trials
    • 7.3.5. Increasing Patient Recruitment through the Use of Social Media
    • 7.3.6. Seamless Phase II/III Designs

8. Accelerating Drug Transition in Phase III Studies

  • 8.1. Trends in Drug Transition from Phase III to NDA Filing
  • 8.2. Issues and Challenges in Drug Transition
  • 8.3. Strategies and Models to Accelerate Transition from Phase III to NDA Filing
    • 8.3.1. Adaptive Trial Designs
    • 8.3.2. Recruitment and Retention of Patients in Phase III Clinical Trials

9. Accelerating Drugs to Market - Appendix

  • 9.1. Market Definitions
  • 9.2. Abbreviations
  • 9.3. Bibliography
  • 9.4. Research Methodology
    • 9.4.1. Coverage
    • 9.4.2. Secondary Research
    • 9.4.3. Primary Research
    • 9.4.4. Expert Panel Validation
  • 9.5. Contact Us
  • 9.6. Disclaimer

List of Tables

  • Table 1: Accelerating Drugs to Market, NME Approvals By The FDA, 2004-2011
  • Table 2: Accelerating Drugs to Market, Pharmaceutical and Biotech R&D Expenditure ($bn) versus Number of NME/BLA Approvals, the US, 2004-2011
  • Table 3: Accelerating Drugs to Market, Comparison of Microdialysis with Ultrafilteration
  • Table 4: Accelerating Drugs to Market, Factors for Minimizing Time Delays in Site Initiation, 2010

List of Figures

  • Figure 1: Accelerating Drugs to Market, Development Timeline for New Drugs
  • Figure 2: Accelerating Drugs to Market, Stages of Drug Development
  • Figure 3: Accelerating Drugs to Market, Amendments per Protocol and Changes per Amendment
  • Figure 4: Accelerating Drugs to Market, NME Approvals By The FDA, 2004-2011
  • Figure 5: Accelerating Drugs to Market, Pharmaceutical and Biotech R&D Expenditure ($bn) versus Number of NME/BLA Approvals, the US, 2004-2011
  • Figure 6: Lead Generation Strategies and Technologies in Drug Discovery, Drug Discovery Process
  • Figure 7: Lead Generation Strategies and Technologies in Drug Discovery, Applications of Lead Generation Process
  • Figure 8: Accelerating Drugs to Market, Pathways For Optimizing Lead Through Screening
  • Figure 9: Lead Generation Strategies and Technologies in Drug Discovery, HTS Absorption, Distribution, Metabolism and Excretion Assay
  • Figure 10: Lead Generation Strategies and Technologies in Drug Discovery, Evotec's Approach to Identifying Novel Beta Secretase-1 Inhibitor Using the Fragment-Based Approach
  • Figure 11: Accelerating Drugs to Market, Benefits And Challenges In Imaging Technique
  • Figure 12: Accelerating Drugs to Market, Service Providers in Combinatorial Chemistry and 3D Molecular Modeling
  • Figure 13: Accelerating Drugs to Market, Service Providers of Omics Technology and Bioinformatics
  • Figure 14: Accelerating Drugs to Market, Strategic or Tactical Advantages in Early Stage Drug Development Outsourcing
  • Figure 15: Accelerating Drugs to Market, Preclinical ADMET Studies - Current System
  • Figure 16: Accelerating Drugs to Market, Site Initiation Process, 2011
  • Figure 17: Accelerating Drugs to Market, Key Factors Responsible for Study Timeline Reduction by SMOs
  • Figure 18: Accelerating Drugs to Market, Clinical Trial Supply Management System
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