Companies currently developing in or planning to enter the cell and gene therapy (CGT) space must prepare for the market shift from targeted niche development to the broader pursuit of non-orphan indications. Commercializing CGTs in non-orphan indications will be fundamentally different than the experience to date. In our recent white paper, Key Considerations for Cell and Gene Therapy Commercialization in Non-Orphan Indications, we focus on the expansion of CGTs into non-orphan indications outside of oncology. Get a sneak peek inside the white paper below.

Non-Oncology CGT Development Is Expanding Beyond Ultra-Orphan Indications

Outside of oncology, early movers in the CGT space have heavily focused development on ultra-orphan conditions. While non-oncologic successes have thus far been limited to ultra-orphan and orphan indications, near-registration and Phase III programs suggest a trend toward the development of CGTs in broader indications. Atherosclerosis, osteoarthritis, diabetic macular edema, and wet age-related macular degeneration are among those nonorphan indications seeing early CGT development.

CGTs Face Commercial Challenges in Non-Orphan Indications

To date, CGTs have used the orphan disease model to fit into the healthcare ecosystem for a few main reasons. A small addressable patient population with limited or no access to treatment creates an environment of incredibly high unmet need that justifies high drug pricing to payers and can offset the cost of development and supply for manufacturers. As CGT development moves away from rare disease and into mainstream indications, companies must adopt new commercial and organizational strategies to find continued success within the established healthcare ecosystem.

• CGT Pricing Considerations – CGT pricing to date has followed orphan disease price justification where high unmet need and small patient populations, coupled with durable response, allow for significant one-time spend. In the US, payers accept these drug prices because the populations are rare, have significant unmet need, and in some cases, treatment has the potential to offset patients’ expensive chronic therapies.
• CGT Delivery Ecosystem Considerations – CGTs typically require complex delivery systems to administer therapies to patients. CGTs often have strict storage and handling requirements, such as ultra-cold chain storage and very short shelf lives. Additionally, administration often requires specialized management. Due to these factors, CGTs are commonly delivered only in select treatment centers, typically associated with academic medical centers. Manufacturers will need to consider the challenges of scale. CGTs for broader populations would necessitate administration in wider settings of care to ensure that there is sufficient facility capacity, and that treatment is accessible to patients.

Key Questions for CGT Development in Non-Orphan Indications

Access our white paper Key Considerations for Cell and Gene Therapy Commercialization in Non-Orphan Indications for further insights into pricing and delivery ecosystem considerations, as well as key questions developers and manufacturers should ask themselves when considering investment in CGTs in larger patient populations.