Source Global Data

Gene therapy involves the delivery of complex treatments to patients. In recent years, there have been several promising clinical trial results within this field targeting an array of inherited neurodegenerative disorders, genetic diseases, and various cancers. Approvals based on these clinical trials have consequently encouraged interest in gene therapy from both industry and non-industry sponsors, and have boosted confidence in the therapeutic delivery of genetic material. This white paper investigates the changing clinical trial landscape for gene therapy clinical trials.

GlobalData performed multiple analyses of gene therapy clinical trials spanning Phases I–IV in all trial statuses from January 1, 1995 to December 31, 2019.

The majority of gene therapy trials are early phase studies (90.6%) with 51.0% of trials in Phase II and 40.5% of trials in Phase I.

The biggest year for gene therapy trials was 2018 whereas the biggest increase in the number of initiated trials in a one-year period was seen in 2017 from 2016. This rise in gene therapy trials may be due to the first EU and US regulatory agency approvals of gene therapy agents, giving sponsors increased confidence that they can bring gene therapy products to market. The top therapy area in gene therapy trials is oncology, followed by genetic disorders, cardiovascular disorders, hematological disorders, and central nervous system disorders. The top five indications are acute lymphocytic leukemia, multiple myeloma, solid tumor, pancreatic cancer, and melanoma. Industry sponsors outnumbered non-industry sponsors across all phases except Phase I.

Following the FDA’s approval of Kymriah, the proportion of industry sponsors rose by 15.6%, indicating renewed industry confidence in gene therapy clinical trials. Even though industry-sponsored trials outnumber non-industry trials, the top five sponsors include four non-industry institutions and only one industry sponsor.

North America dominated the gene therapy research space with the highest number of trials, and the US had the most clinical trials, followed by China and the UK. Ongoing and planned trials make up the highest proportion of gene therapy trials (62.4%), followed by completed trials (28.4%). Trials with a withdrawn or terminated status account for the same proportion of trials (4.9%).

In the last five years, four of the top five drugs targeted CD19, demonstrating the significant research geared towards gene therapies against CD19.

Single-country gene therapy trials strongly outnumber multinational trials. This is likely due to the lack of Phase III trials, which are more likely to require being conducted in multinational studies. The most common reason for trial termination is low accrual rate, followed by financial reasons. The surge in the number of gene therapy trials since 2014 highlights the increased interest within this sector, with every breakthrough and product approval offering new avenues for research.

GlobalData expects this trend to continue to rise, as 2019 is on course to be the biggest year for gene therapy clinical trials yet.