While the number of approvals has been slower than originally anticipated, CGT development has successes to build upon for the future. NEWDIGS recently completed an analysis which compares the clinical trial success rates and overall likelihood of CGTs for orphan diseases and hematological cancers to traditional drug development programs with data from years up to 2020. The comparison used the NEWDIGS FoCUS Pipeline Analysis Model (PAM) for durable cell and gene therapies and recently published BIO and IQVIA estimates based on clinical trials from all therapeutic modalities. This comparative analysis suggests durable CGTs for orphan diseases and hematological cancers are 2-3.5X more likely to succeed than other therapeutic modalities for similar conditions or compared to the entire drug pipeline. These results illustrate the power of compounding incremental success across nearly every phase for all CGTs. Details can be found within the research brief NEWDIGS-Success-Rate-Comparison-2023F210v056.pdf (tuftsmedicalcenter.org)

A strong commitment to cell and gene therapy development by the FDA will continue to enhance the potential for CGT approvals.The FDA has released multiple guidance documents to support CGT clinical development, including use of the accelerated approval pathway specifically for gene editing therapies. Advancements with groundbreaking approvals such as the first CRISPR-based therapeutic approval in December 2023, also serves to further use of the innovative technology. The approval pipeline for CGT holds immense potential for patients and the boarder healthcare landscape.