Key Considerations for Cell and Gene Therapy Commercialization in Non-Orphan Indications

The field of cell and gene therapies (CGTs) presents significant transformative potential across a wide range of diseases including cancer, genetic disorders, and autoimmune conditions. Outside of oncology, CGT development has mainly focused on orphan and ultra-orphan indications. One reason is that while more common diseases have diverse and often complex etiologies, many ultra-orphan diseases result from clearly defined monogenetic mutations or cellular flaws; developers can design therapies to address the condition at the genetic or cellular level. Additionally, gene therapy pricing has rested on rare disease price justification where high unmet need and small patient populations, coupled with durable response, allow for significant one-time spend.

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.

Commercializing CGTs in non-orphan indications will be fundamentally different than the experience to date. It is essential for companies developing (or planning to develop) CGTs in broader disease populations to understand the key differences between niche markets and broader indications.

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. There are two important challenges for companies aiming to develop CGTs in mainstream indications:

1. Reconciling orphan and non-orphan disease pricing models
2. Scaling complex distribution and delivery to meet broader patient populations

As manufacturers move toward developing CGTs in broader indications, the established pricing model and treatment paradigm break down. The medical and economic system-wide impacts of non-orphan diseases are often much larger than orphan diseases, even if the per-patient impacts are lower. To be viable in more common diseases, manufacturers need to develop new business models. Neither the current CGT model nor traditional specialty models are likely to work in their present form.

Download the white paper “Key Considerations for Cell and Gene Therapy Commercialization in Non-Orphan Indications” by Alira Health to learn more about the expansion of CGTs into non-orphan indications outside of oncology. This white paper will examine the viability of developing a CGT in a non-orphan indication and address key considerations when evaluating the business case for a successful CGT product in a mainstream market.