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.
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
Summary
This report investigates recent years of performance of pharmaceutical and biopharmaceutical companies with respect to return on investment (ROI) in research and development (R&D), and to discuss ways that companies are trying to improve ROI through 2024.
ROI and R&D Outlays
Many factors enter into the calculation of ROI; this is not a straightforward calculation, especially if one does not have access to the company’s internal accounting books. However, the fact that many factors enter into the calculation, also may be taken to mean that a number of factors might be adjusted or developed to improve ROI. Laying off employees is one way, but this is not effective longer-term unless the company is at the same time reorganizing for greater efficiency. Sanofi, for example, hit hard by pricing pressure in diabetes, started laying off 400 US sales employees in June 2019 but states that it is also undergoing a reorganization to its salesforce.
Global research and development spending is forecast to grow by REDACTED in 2019 to a total of REDACTED in purchasing parity values for the more than 110 countries that have significant R&D investments of over more than $100 million, according to the 2019 Global R&D Funding Forecast, an annual compilation produced by R&D Magazine. The amount of investment in R&D is heavily influenced by a country’s economic power, plus the importance that the country places on research. China continues to increaseits global share of R&D spending with an increase of REDACTED, as other countries lose share in the R&D pie. The US will increase its R&D spending by about REDACTED in 2019 over 2018.
Using Technology to Weed Out Losers, Focus on Potential Winners
The most significant route to decreasing costs and improving failure rates is to cut off the development of a failing drug as early as possible in order to focus on potential winners. Pretty obvious—but how best to do this?
Many pharma and biotech companies are exploring the use of technology to make the most of clinical development and clinical trials. In the next three to five years:
- Robotic automation will streamline resources across the clinical trial value chain.
- Use of AI, ML and IT with Big Data will enable researchers to see larger trends and patterns in data, and to act on those trends earlier in the process.
- Clinical trials will become open, and trials will be better understood by the public.
- Clinical trials will become patient-centric and better integrated into medical systems, thereby becoming an integral part of health care.
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 21st 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
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