Clinical Trials – Precision and Personalized Medicine

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This report provides analysis of clinical research involving precision and personalized medicines and identifies trends throughout the years.

The terms precision and personalized medicine are often used interchangeably as they refer to similar types of treatment. They both define the stratification of patients within a disease segment using their genomic and phenotypic variations to find targeted therapies. This is an alternative to the traditional process of drug design, which typically follows a one-size-fits all approach. Precision and personalized medicine is an emerging field that contrasts with the historic concept that patients who present similar symptoms may respond differently to the same medical intervention. Technological advancements over the last two decades have revolutionized the treatment landscape for various therapy areas, particularly oncology indications. This success has encouraged substantial investment in this space as sponsors expand beyond oncology with the intention of replicating this success in other diseases with heterogenous patient populations. This report aims to provide analyses of clinical research involving precision and personalized medicines and identify trends throughout the years.


The clinical trial data used for these analyses were extracted from GlobalData’s Clinical Trials Database. The report focuses on precision and personalized medicine trials captured in the database as of June 20, 2023. The data has been analyzed by year, phase, status, therapy area, indication, geography, sponsor type (industry versus non-industry sponsor), reasons for discontinuation, and use of virtual components. An additional analysis on marketed drugs has also been included.

Key Highlights

1. Oncology has dominated the research landscape. Companies have witnessed this success and begun utilizing the approach more frequently in other areas such as immunology and CNS, both of which have highly heterogenous patient populations, making them ideal candidates for a precision and personalized approach.

2. Several countries within APAC have dedicated precision and personalized medicine initiatives that have substantially contributed to the region’s dominance. China’s PMI was introduced in 2016 and is responsible for launching APAC ahead of North America.

3. In 2020, infectious diseases hit a record high in precision and personalized medicine trials with 86% concerning COVID-19. Oncology precision and personalized medicine trials hit a record low at that time, indicating that funding was being allocated to COVID-19. Phase I trial initiations also saw an increase as they are cheaper and require the smallest patient cohort, which was desirable during the pandemic.

Reasons to Buy

Identifies and dissects trends across clinical trial phases and statuses, as well as regions, countries, sponsors, therapy areas, and indications.

Explores record highs and lows across years, regions, and therapy areas, and offers reasons for this.

Highlights leading therapy areas, indications, geographies, sponsors, and marketed cell and gene therapies.

Spotlights main reasons for trial terminations and emphasises main trial phases terminated.

Discusses the prevalence of decentralised clinical trials and virtual components.

National Cancer Institute US
Fred Hutchinson Cancer Research Center
University of Texas MD Anderson Cancer Center
Masonic Cancer Center
University of Minnesota
Memorial Sloan Kettering Cancer Center
Mayo Clinic
Baylor College of Medicine
Stanford University
City of HopeMesoblast
University of Texas MD Anderson Cancer Center
Dendreon Pharmaceuticals
Sangamo Therapeutics
Lisata Therapeutics
Sarah Cannon Research Institute
Medpace Holdings
IQVIA Holdings
Reliance Life Sciences
FGK Clinical Research
Syneos Health

Table of Contents

1 Table and Figures

2 Executive Summary

3 Introduction

– Report Scope

– Methodology

4 The Clinical Trial Landscape of Precision and Personalized Medicine

– 2020 Served as The Leading Year in Precision and Personalized Medicine Trials

– The Record Trial Count In 2020 Was Due to The Increase in Infectious Disease Trials In Response to COVID-19

– Phase II Trials Dominate Precision and Personalized Medicine Clinical Research

– Phase I Trials Are Most Successful at Achieving Their Endpoint Status

– Non-Industry Sponsors and Industry Sponsors Lead in Different Phases for Achieving Trial Endpoints

– Non-Industry Sponsored Trials Lead in Precision and Personalized Medicine

– National Cancer Institute US is the Leading Non-Industry Sponsor

– Mesoblast Is the Leading Industry Sponsor

– Masonic Cancer Center Has the Most Terminated/Suspended/Withdrawn Trials of Any Sponsor

– Emmes Is the Top CRO Supporting Precision and Personalized Medicine Clinical Trials

– Asia-Pacific Leads in Precision and Personalized Medicine Trials From 2016 Onwards

– Single-Country Clinical Trials Dominate in Every Region with North America Leading the Way

– The US Leads Year-On-Year in Precision and Personalized Medicine Clinical Trials

– Low Accrual Is the Leading Cause of Trial Termination and Phase II Trials Are Most Frequently Terminated

– Mobile HealthCare is The Leading Virtual Component in Precision and Personalised Medicine Trials

– Oncology Is the Leading Therapy Area in Precision and Personalised Medicine Trials Across All Phases

– Proportion of Precision and Personalised Medicine Trials for Oncology Has More Than Halved Since 2003

– AML is The Most Researched Indication in Precision and Personalized Medicine Clinical Trials

5 Analysis of Cell and Gene Therapies in Precision and Personalized Medicine Clinical Trials

– Top 10 Companies Involved in Upcoming Catalytic Trial Events Using Gene Therapies In Precision and Personalized Medicine

– Top 10 Companies Involved in Upcoming Catalytic Trial Events Using Cell Therapy in Precision and Personalized Medicine

– Leading Marketed Drugs by Clinical Trial Count

– Marketed Gene Therapies Have More Clinical Trials Than Marketed Cell Therapies

– Gene Therapies Have a Higher Phase Transition Rate Than Cell Therapies

– Gene Therapies Have a Higher Likelihood of Approval Score Than Cell Therapies

6 Key Findings

7 Appendix

– About GlobalData

– Contacts

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