ROI in Pharmaceutical R&D: How to Halt the Decline

Report Highlights:

The global orphan drugs market should reach $315.6 billion by 2024 from $127.5 billion in 2017 at a compound annual growth rate (CAGR) of 13.8% for the forecast period of 2017 to 2024.

The global infectious disease treatments market should reach $117.2 billion by 2024 from $64.8 billion in 2017 at a CAGR of 8.8% for the forecast period of 2017 to 2024.

The global cardiovascular drugs market should reach $153.7 billion by 2024 from $141.1 billion in 2017 at a CAGR of 1.2% for the forecast period of 2017 to 2024.

Report Scope:

The scope of this study begins with the challenges facing biotech and pharmaceutical companies today. It goes on to discuss regulatory approval processes in various countries, how these are changing, and the knock-on effects which the pharma/biotech companies will have to address. The report then reviews several key financial ratios, then discusses how to maximize clinical development success rate. We then go into how machine learning (ML) and artificial intelligence (AI), along with Big Data, are disrupting the pharma/biotech industry, in a positive way. The report then reviews the global markets for treatments for selected cancers, chronic diseases, cardiovascular disease, neurological diseases, infectious diseases and rare diseases. Chapter 15 discusses how companies need to deeply integrate ML, AI and Big Data into drug discovery and clinical testing-or risk falling behind the competition. The report wraps up with summaries of selected companies active in these spaces and beginning to utilize digital tools in R&D.

Report Includes:

• 49 data tables and 30 additional tables

• An assessment of return on investment (ROI) in R&D by top pharmaceutical companies within the industry

• Analyses of the global market trends for various chronic disease areas, with data from 2016 to 2017, and projections of compound annual growth rates (CAGRs) through 2024

• Pipeline analysis of various therapeutic drugs with oncology being the largest, and coverage of ongoing clinical trials and promising forthcoming therapies in late stage etc.

• Outlining details of many factors involved in calculating ROI in R&D in pharma and how some of those factors are adjusted/developed to improve R&D ROI

• Discussion of the competitive landscape and key mergers and acquisition deals, partnerships, collaborations during the timeframe, 2014-2024

• Profile descriptions of top 15 pharmaceutical companies, their sales data, market capitalization and areas of R&D etc., including 23andMe, AstraZeneca PLC, Celgene Corp., Eli Lilly and Co., GlaxoSmithKline PLC, Merck & Co. Inc., Pfizer Inc. and Sanofi

Table of Contents

Chapter 1: Introduction

• Study Goals and Objectives
• Reasons for Doing This Study
• Scope of Report
• Information Sources
• Methodology
• Analyst's Credentials
• BCC Custom Research
• Related BCC Research Reports

Chapter 2: Summary

• ROI and R&D Outlays
• Using Technology to Weed Out Losers, Focus on Potential Winners
• Identifying Biomarkers
• Revolutionizing Clinical Research with AI, ML and Patient Centricity
• Global Oncology Markets by Region and Selected Types
• Global Chronic Disease Drugs Market by Region
• Global Cardiovascular Drugs Market by Region
• Global Neurology Therapies Market by Region
• Global Market for Infectious Disease Treatments by Region
• Global Market for Orphan Drugs by Region
• Wanted: Digitally Literate Employees Open to Continuous Learning

Chapter 3: Challenges Facing Pharma and Biotech Companies

• Strong Revenues, but Decreasing ROI
• Tracking the Decline in ROI
• Reasons that IRR Has Been Exceptionally Low
• The Competitive, Expensive Search for Novel Drugs
  • Eroom's Law
  • Emerging Global Players from India, China, Russia
• Expiring Patents
• Complying with Changing Regulatory Environments
• Pricing Pressures in the US and Elsewhere
• Traditional Structure is Blurring
• Increasing Complexity of Clinical Trials
  • Oncology Clinical Trials Remain Complex
  • Is Oncology Research Showing the Way?
  • Patient Recruitment Consumes a Major Part of Trial Duration
  • Is the Pool of Viable New Drug Targets Diminishing?
  • The Malfunctioning Clinical Trial System
  • Solutions are Out There, and Already Working
  • The 21^st Century Cures Act of 2016
  • The US FDA's New Population Pharmacokinetics Guidance Document
• Pharma Market Update
  • M&A Activity
• Biotech Market Update
  • Next-Generation Biotherapeutics Pipelines Have More than Doubled
  • Biotech Pricing Pressure
  • What's Ahead

Chapter 4: Regulatory Overview and New Drug Approvals

• Improving Regulatory Compliance
  • Drug Development Continues to be Slow Process
• Record New Drug Approvals in 2018
• US FDA Selected Drug Approvals
• Drug Approval Processes in the US, EMA, China, Japan
  • US FDA and the EU's EMA
  • China's Drug Regulatory Process
  • Japan's Drug Regulatory Process

Chapter 5: Key Financial Ratios for Pharma and Biotech Companies

• Return on Investment and Project-wise ROI
• Ensuring Financial Accountability for R&D Outlays
• Return on Research Capital (RORC)
• Profitability Ratios
• Liquidity and Debt Coverage Ratios
• Return on Equity (ROE)

Chapter 6: Maximizing Clinical Development Success Rates

• Driving Change in Clinical Development
  • Utilizing Mobile Health Technologies
  • Ramping Up Real-World Data and Real-World Evidence: the FDA's New RWE Framework
• Maximizing Immuno-Oncology Clinical Trial Success with Immune-Related Response Criteria (irRC)
  • irRC More Useful Than RECIST for immune-oncology drugs
  • Case Study: Ipilimumab
• Identifying Biomarkers
  • Challenges and Hurdles to Biomarker Detection and Usage
• Use of Patient-Reported Outcomes and Pools of Prescreened Patients
• Predictive Analytics
• Revolutionizing Clinical Research with AI, ML and Patient Centricity
  • One Example: Patient Centricity Enabled by the Medidata Patient Cloud
  • 23andMe Wants to Solve the Patient Recruitment Problem

Chapter 7: The Advantages of Being-or Acting-Small

• Breaking Down the Silo Mentality
  • Just Say No
  • Turn on a Dime
• Small Companies with Big Visions
  • Virgo Surgical Video Solutions
  • Ovid Therapeutics
  • Melinta Therapeutics
  • Applied Genetic Technologies
  • BioCryst Pharmaceuticals

Chapter 8: The Digital Transformation of Biopharma Companies

• Robotic Process Automation (RPA)
• Machine Learning (ML)
  • MELLODDY (Machine Learning Ledger Orchestration for Drug Discovery) project
• AI Lighthouse Projects: Digital Transformation Continues Apace
  • Evidation, Apple, Eli Lilly and Digital Alzheimer's Biomarkers
  • Accelerating Therapeutics for Opportunities in Medicine (ATOM) Consortium
  • DeepMind Technologies, AI and Protein Folding
  • AI For Drug Discovery and Accelerating Clinical Trials
  • Alliance for Artificial Intelligence in Healthcare (AAIH)
  • MIT's Machine Learning for Pharmaceutical Discovery and Synthesis Consortium
• The Challenges Found in Deploying AI
  • Healx Uses AI to Discover Rare Disease Treatments
• Business Strategy Should Include Technology Investment
  • Empower a Cultural Change: From "Doing Digital" to "Being Digital"
  • The Implementation of Digital Trials
  • The Tortoise and the Hare
• The Time is Now

Chapter 9: Leveraging Open Innovation to Solve Development Challenges

• Collaboration/Partnering
• Centers of Innovation
  • J&J and Pfizer: Global Centers of Innovation
• Germany Fosters Biopharma Innovation
• Co-location
• Crowdsourcing Technical and Scientific Ideas
• Other Examples of Open Innovation in Pharma
  • Otsuka's Collaboration with the Riken Center for Developmental Biology
  • AstraZeneca and its Center for Innovation
• How Takeda Chose to Move Forward
  • Internal Crowd Sourcing Through Entrepreneurship Venture Program
  • External Crowd Sourcing: COCKPI-T
  • Hybrid model: Innovation Park "Shonan iPark"

Chapter 10: The Global Pharma and Biotech Market for Oncology

• Challenges in Treating Cancer
• Clinically Significant Types of Cancers
  • Lung Cancer
  • Breast Cancer
  • Ovarian Cancer
• Smarter R&D Leads to More Oncology Drug Approvals
• Global Oncology Market by Region and Selected Types
  • Global Market for Lung Cancer Chemotherapy by Region
  • Global Market for Breast Cancer Therapy by Region
  • Global Market for Ovarian Cancer Therapy by Region

Chapter 11: The Global Pharma and Biotech Market for Chronic Diseases

• Chronic Kidney Disease (CKD)
  • Background to CKD
  • Renal Disease
  • Causes of Chronic Kidney Disease
  • Incidence of End Stage Renal Disease by Country
  • Prevalence of End Stage Renal Disease by Country
  • Pharmaceutical Drugs
  • Global Market for Chronic Kidney Disease Stage 1-5 Drugs
• Chronic Respiratory Disease Market
• Global Diabetes Therapy Market
• Global Chronic Gastrointestinal Market

Chapter 12: The Global Pharma and Biotech Market for Cardiovascular Disease

• Global Cardiovascular Drug Market by Region

Chapter 13: The Global Pharma and Biotech Market for Neurological Diseases

• Parkinson's Disease
  • Overview
  • Prevalence
• Alzheimer's Disease
  • Overview
  • Prevalence
• Schizophrenia
  • Overview
  • Prevalence
• Bipolar Disorders
  • Overview
  • Prevalence
• Depression
  • Overview
  • Prevalence
• Epileptic Disorders
  • Overview
  • Prevalence
• Autism Spectrum Disorders
  • Overview
  • Prevalence
• Global Market for Neurology Therapies by Region

Chapter 14: The Global Pharma and Biotech Market for Infectious Diseases

• Infectious Diseases
  • Classification of Infectious Agents
  • Management of Infectious Diseases
  • Bacterial Infectious Diseases
  • Viral Infectious Diseases
  • HIV/AIDS
  • Influenza A and Influenza B
  • Risk Factors
  • Etiology and Symptoms
  • Socioeconomic Burden
• Global Markets for Infectious Disease Treatments
• Global Market for Infectious Disease Treatments by Region

Chapter 15: The Global Pharma and Biotech Market for Rare Diseases

• Legal Definitions of Orphan Diseases
• Playing Games with Orphan Drug Approvals?
• Genetically Based Tools for Rare Disease R&D

Chapter 16: The Changing Future of Biopharma Work

• Wanted: Digitally Literate Employees Open to Continuous Learning
• Acquiring AI Capabilities Through Cloud-Based Enterprise Software with Integrated AI

Chapter 17: Company Profiles

• 23ANDME
• ABBVIE
• ALLERGAN PLC
• AMGEN INC.
• ASTELLAS PHARMA INC.
• ASTRAZENECA PLC
• BAYER AG
• BIOGEN IDEC
• BOEHRINGER INGELHEIM GMBH
• BRISTOL-MYERS SQUIBB CO.
• CELGENE CORP.
• CSL BEHRING
• ELI LILLY AND CO.
• EXSCIENTIA
• GILEAD SCIENCES INC.
• GLAXOSMITHKLINE PLC
• JOHNSON & JOHNSON INC.
• MERCK & CO., INC.
• NOVARTIS AG
• NOVO NORDISK A/S
• NUMERATE INC.
• PFIZER INC.
• ROCHE (F. HOFFMAN-LA ROCHE AG)
• SANOFI
• VERTEX PHARMACEUTICALS

Chapter 18: Appendix: Acronyms

List of Tables

• Summary Table: Global Market for Lung, Breast and Ovarian Cancer Therapies, by Region, Through 2024

• Summary Table A: Global Chronic Disease Drugs Market, by Region, Through 2024

• Summary Table B: Global Cardiovascular Drugs Market, by Region, Through 2024

• Summary Table C: Global Market for Neurology Therapies, by Region, Through 2024

• Summary Table D: Global Market for Infectious Disease Treatments, by Region, Through 2024

• Summary Table E: Global Market for Orphan Drugs, by Region, Through 2024
  • Table 1: Likely Applications of AI in Pharma and Biotech Companies
  • Table 2: Selected M&A Deals, 2018-August 2019
  • Table 3: Selected US Highest-Priced Drugs
  • Table 4: US FDA Expedited Pathways and Other Attributes, 2017 and 2018
  • Table 5: US FDA Selected Drug Approvals, 2017 and 2018
  • Table 6: Selected FDA Orphan Drug Approvals, 2017 and 2018
  • Table 7: Clinical Trial Challenges and the Role of AI
  • Table 8: Selected Pharma Companies Utilizing AI
  • Table 9: Global Cancer Incidence (Both Sexes)
  • Table 10: Global Markets for Lung, Breast & Ovarian Cancer Therapies, by Region, Through 2024
  • Table 11: Global Market for Lung Cancer Chemotherapy, by Region, Through 2024
  • Table 12: Global Market for Breast Cancer Therapy, by Region, Through 2024
  • Table 13: Global Market for Ovarian Cancer Therapy, by Region, Through 2024
  • Table 14: Incidence of ESRD, by Country, 2015
  • Table 15: Prevalence of ESRD, by Country, 2015
  • Table 16: Global CKD 1-5 Stage Drugs Market, by Region, Through 2024
  • Table 17: Global Chronic Respiratory Disease Therapies Market, by Region, Through 2024
  • Table 18: Global Diabetes Therapy Market, by Region, Through 2024
  • Table 19: Global Chronic Gastrointestinal Therapy Market, by Region, Through 2024
  • Table 20: Global Cardiovascular Drug Market, by Region, Through 2024
  • Table 21: AstraZeneca PLC: Cardiovascular Pipeline
  • Table 22: Amgen Inc.: Cardiovascular Pipeline
  • Table 23: Sanofi: Cardiovascular Pipeline
  • Table 24: Novartis AG: Cardiovascular Pipeline
  • Table 25: Merck and Co., Inc.: Cardiovascular Pipeline
  • Table 26: Bristol-Myers Squibb Company: Cardiovascular Pipeline
  • Table 27: Global Market for Neurology Therapies, by Region, Through 2024
  • Table 28: Global Health Estimates, Leading Causes of Death, Infectious Diseases, by WHO Region, 2015
  • Table 29: Prevalence and Incidence of Hepatitis B, 2015
  • Table 30: Prevalence and Incidence of Hepatitis C, 2015
  • Table 31: Global Prevalence and Incidence of Genital Herpes, 2005-2015
  • Table 32: Prevalence of HIV, Adults (15-49 years old), by WHO Region, 2015
  • Table 33: New HIV Infections (per 1,000 uninfected population), Adults (15-49 years old), by WHO Region, 2015
  • Table 34: New HIV Infections, 2000-2030
  • Table 35: Years of Life Lost (YLLs), Pandemic Influenza A H1N1, by WHO Region, 2009
  • Table 36: Global Market for Infectious Diseases, by Product Type, Through 2024
  • Table 37: Global Market for Infectious Diseases, by Region, Through 2024
  • Table 38: Selected Country/Regional Definitions of Orphan Disease
  • Table 39: Description of Selected Orphan Diseases
  • Table 40: Global Market for Orphan Drugs, by Region, Through 2024
  • Table 41: Applications of AI in Pharma and Biotech Companies
  • Table 42: Details of Allergan's Late-Stage Pipeline, 2019
  • Table 43: Astellas Pharma Inc.: Selected Financials, 2016-2018
  • Table 44: Astellas Pharma Inc.: Marketed Products, 2018
  • Table 45: AstraZeneca PLC: Selected Financials, 2016-2018
  • Table 46: AstraZeneca PLC: Selected Marketed Products, 2018
  • Table 47: AstraZeneca PLC: Selected Global Product Sales, 2017 and 2018
  • Table 48: Bayer AG: Selected Financials, 2017 and 2018
  • Table 49: Biogen: Selected Financials, 2017 and 2018
  • Table 50: Boehringer Ingelheim GmbH: Selected Financials, 2017 and 2018
  • Table 51: Boehringer Ingelheim GmbH: Top 4 Human Pharmaceutical Product Sales, 2018
  • Table 52: Bristol-Myers Squibb Co.: Selected Financials, 2017 and 2018
  • Table 53: Bristol-Myers Squibb Co.: Selected Marketed Products, 2018
  • Table 54: Celgene Corp.: Selected Financials, 2017 and 2018
  • Table 55: Celgene Corp.: Selected Marketed Products, 2018
  • Table 56: CSL Behring: Selected Financials, 2017 and 2018
  • Table 57: Eli Lilly and Co.: Selected Financials, 2017 and 2018
  • Table 58: Eli Lilly and Co.: Selected Products
  • Table 59: Gilead Sciences Inc.: Selected Financials, 2017 and 2018
  • Table 60: Gilead Science: Selected Products and Indications
  • Table 61: GlaxoSmithKline PLC: Selected Financials, 2017 and 2018
  • Table 62: GlaxoSmithKline PLC: Selected Marketed Products
  • Table 63: Johnson & Johnson Inc.: Selected Financials, 2017 and 2018
  • Table 64: Johnson & Johnson Inc.: Selected Marketed Products, 2018
  • Table 65: Merck & Co., Inc.: Selected Financials, 2016-2018
  • Table 66: Merck & Co., Inc.: Selected Marketed Products, 2018
  • Table 67: Novartis AG: Net Revenue, 2017 and 2018
  • Table 68: Novartis AG: Selected Marketed Products, by Indication, 2018
  • Table 69: Novo Nordisk A/S: Net Revenue, 2017 and 2018
  • Table 70: Novo Nordisk A/S: Selected Products and Indications, 2018
  • Table 71: Pfizer Inc.: Net Revenue, 2017 and 2018
  • Table 72: Pfizer Inc.: Selected Products and Indications, 2018
  • Table 73: Roche: Net Revenue, 2017 and 2018
  • Table 74: Roche: Revenues from Top 10 Pharma Products, 2018
  • Table 75: Sanofi: Net Revenue, 2017 and 2018
  • Table 76: Sanofi: Selected Products and Indication, 2018
  • Table 77: Vertex Pharmaceuticals: Net Revenue, 2017 and 2018
  • Table 78: Acronyms Used in This Report

List of Figures

• Summary Figure: Global Market for Lung, Breast and Ovarian Cancer Therapies, by Region, 2016-2024

• Summary Figure A: Global Chronic Disease Drugs Market, by Region, 2016-2024

• Summary Figure B: Global Chronic Disease Drugs Market, by Region, 2016-2024

• Summary Figure C: Global Chronic Disease Drugs Market, by Region, 2016-2024

• Summary Figure D: Global Chronic Disease Drugs Market, by Region, 2016-2024

• Summary Figure E: Global Chronic Disease Drugs Market, by Region, 2016-2024
  • Figure 1: Selected Challenges
  • Figure 2: Annual Revenues of Leading Pharmaceutical Companies, 2010-2018
  • Figure 3: Annual Revenues of Leading Biotech Companies, 2010-2018
  • Figure 4: Selected Pharma Companies' Return on R&D Spend, 2010-2018
  • Figure 5: Selected Pharma Companies' R&D Spend, 2010-2018
  • Figure 6: Selected Biotech Companies' R&D Spend, 2010-2018
  • Figure 7: Pharma/Biotech Drug Development Cycle
  • Figure 8: Likelihood of Approval from Phase I, by Disease Area
  • Figure 9: Complexity of Oncology Clinical Trials, 2010-2018
  • Figure 10: Number of Next-Generation Biotherapeutic Pipeline Products in Late-Stage Pipeline, 2009-2018
  • Figure 11: Drugs Approval Process, US FDA and EU
  • Figure 12: China CFDA Drug Regulatory Approval Process
  • Figure 13: Japan PMDA Drug Regulatory Approval Process
  • Figure 14: AI for Clinical Trial Design from Methodology to Improved Outcomes
  • Figure 15: Percentage of US and EU5 Patients Tested for Biomarker, by Cancer Type
  • Figure 16: Build Digital Capabilities Now
  • Figure 17: Annual Incidence and Mortality Data for Lung Cancer in the U.S., 1975-2015
  • Figure 18: Annual Incidence and Mortality Data for Lung Cancer in Five EU Countries, 1975-2012
  • Figure 19: Annual Incidence and Mortality Data for Breast Cancer in the U.S., 1975-2015
  • Figure 20: Annual Incidence and Mortality Data for Breast Cancer in Five E.U. Countries, 1975-2012
  • Figure 21: Annual Incidence and Mortality Data for Ovarian Cancer in the U.S., 1975-2015
  • Figure 22: Annual Incidence and Mortality Data for Ovarian Cancer in Five EU Countries, 1975-2012
  • Figure 23: Number of New Active Substance Approvals in Oncology, by Indication, 2019
  • Figure 24: Global Market for Lung Cancer Chemotherapy, by Region, 2016-2024
  • Figure 25: Global Market for Breast Cancer Therapy, by Region, 2016-2024
  • Figure 26: Global Market for Ovarian Cancer Therapy, by Region, 2016-2024
  • Figure 27: Global Cardiovascular Deaths, by Region, 2015 and 2030 (Forecast)
  • Figure 28: Global Health Estimates, Leading Causes of Death, Infectious Diseases, by WHO Region, 2015
  • Figure 29: Global Market Share of New HIV Infections, by WHO Region, 2015
  • Figure 30: Number of Deaths due to 2009 Pandemic Influenza A H1N1, by WHO Region, August 2009-August 2010
  • Figure 31: Global Market for Infectious Diseases, by Product Type, 2016-2024
  • Figure 32: Top 10 Pharmaceutical Companies, by Global Orphan Drug Projected Revenues, 2024
  • Figure 33: Global Market for Orphan Drugs, by Region, 2016-2024
  • Figure 34: AbbVie's Pipeline, at the Time of Proposed Merger with Allergan, June 2019
  • Figure 35: Allergan Developments, 2018
  • Figure 36: Astellas Pharma Inc.: Revenue Share, by Region, 2018
  • Figure 37: Astellas Pharma Inc.: R&D Expenditure, 2017-2019 (forecast)
  • Figure 38: AstraZeneca PLC: Revenue Share, by Region, 2018
  • Figure 39: AstraZeneca PLC: Revenue Share, by Business Segment, 2018
  • Figure 40: AstraZeneca PLC: R&D Expenditure, 2016-2018
  • Figure 41: Bayer AG: Regional Sales Share, 2018
  • Figure 42: Boehringer Ingelheim GmbH: Revenue Share, by Region, 2018
  • Figure 43: Boehringer Ingelheim GmbH: Revenue Share, by Business Segment, 2018
  • Figure 44: Boehringer Ingelheim GmbH: R&D Expenditure, 2016-2018
  • Figure 45: Bristol-Myers Squibb Co.: Revenue Share, by Region, 2018
  • Figure 46: CSL Behring: Sales Share, by Region, FY2018 and FY2019
  • Figure 47: Eli Lilly and Co.: Revenue Share, by Business Segment, 2018
  • Figure 48: Eli Lilly and Co.: Revenue Share, by Region, 2018
  • Figure 49: Gilead Sciences Inc.: Revenue Share, by Region, 2018
  • Figure 50: GlaxoSmithKline PLC: Revenue Share, by Business Segment, 2018
  • Figure 51: GlaxoSmithKline PLC: Revenue Share, by Region, 2018
  • Figure 52: Johnson & Johnson Inc.: Revenue Share, by Business Segment, 2018
  • Figure 53: Johnson & Johnson Inc.: Revenue Share, by Region, 2018
  • Figure 54: Merck & Co., Inc.: Revenue Share, by Business Segment, 2018
  • Figure 55: Merck & Co., Inc.: Revenue Share, by Region, 2018
  • Figure 56: Novartis AG: Revenue Share, by Region, 2018
  • Figure 57: Novartis AG: Revenue Share, by Business Segment, 2018
  • Figure 58: Novo Nordisk A/S: Revenue Share, by Region, 2018
  • Figure 59: Novo Nordisk A/S: Revenue Share, by Business Segment, 2018
  • Figure 60: Pfizer Inc.: Revenue Share, by Region, 2016
  • Figure 61: Pfizer Inc.: Revenue Share, by Business Segment, 2018
  • Figure 62: Roche: Revenue Share, by Business Segment, 2018
  • Figure 63: Roche Pharmaceuticals: Revenue Share, by Region, 2018
  • Figure 64: Sanofi: Revenue Share, by Country/Region, 2018
  • Figure 65: Sanofi: Revenue Share, by Business Segment, 2018