Gene therapy aims to replace a mutated gene that causes a disease with a healthy, good copy of the gene. Gene therapy is highly disruptive one-time, curative therapies and eliminate the need for repeated drug administration that are often burdensome for patients. Gene therapy continues to advance with several programs demonstrating proof of concept across multiple therapeutic categories.
Top trends driving the gene therapy market:
- Biopharma Major’s appetite for gene therapy assets continues to expand: To boost pipelines and offset revenue declines due to patent expirations, large biopharmaceutical companies source innovation from small biotechnology companies. This is evidenced from the commercial approval of Roche/Spark’sLuxturna and Novartis/AveXis’ Zolgensma
- Partnerships accelerate the speed to market: A number of frontier companies have emerged as important players while addressing the limitations inherent in the current approaches driving numerous partnerships in the development of the next generation technology platforms
- The evidence of success hinge in manufacturing expertise: The key challenges in gene therapy include capacity and standardization. While larger players are investing in-house manufacturing capabilities, smaller companies turn to CDMOs for contract manufacturing
- Rare diseases represent sizable market opportunity: Current clinical development of gene therapy is predominantly centered on rare and ultra-rare diseases with the prospect for multiple approvals a real possibility over the next few years
- Viral vectors are where everything starts: Viral vector manufacturing is central to gene therapy development and investor focus is increasingly shifting to manufacturing as more clinical stage candidates are nearing commercialization
- A growing universe of gene editing companies: On the gene-editing front, companies such as Editas, Intellia, CRISPR Therapeutics and Sangamo continue to make promising advances with platforms including CRISPR/Cas-9, Zinc Finger Nuclease, Meganucleases (MNs), and Mega-TALENs (MegaTALs)
Changing commercial strategies:
Addressing access barrier through novel payor programs
- Rising access barrier and ongoing payer pushback due to prohibitive costs of gene therapy has resulted in companies to adopt novel strategies and approaches to drive adoption. Amid an intensified pressure to define a sustainable business model, companies will need to provide reimbursement flexibility to payers such as innovative contracting model and outcomes based rebate arrangement for successful market launch.
Patient identification holds the key
Given the rarity of target patients and very low diagnosis rate, a large focus of the launch effort center around patient identification screen and converting screened individuals into patients.
Stakeholder value creation through an innovative patient engagement model
Unlike traditional buy-and-bill approach, gene therapy will require players to deliver value in every step of the way, right from 1. Finding new eligible patients, 2. Drive genetic testing 3. Enable treatment of identified patients, 4. Educate patients on clinical value 5. Leverage patient support program 6. Operationalize treatment centers 7. Secure coverage through novel model and 7. Robust supply chain to drive patient engagement and deliver clinical value.
In addition, the players also need to sell directly to the payer and specialty pharmacy and ensure product coverage for rapid patient benefits.
- Viral vector manufacturing is central to gene therapy development and investor focus is increasingly shifting to manufacturing as more clinical stage candidates are nearing commercialization.
- In addition, companies with proprietary manufacturing capabilities will have significant competitive advantages over their rivals due to potential improved quality control and reduced risk of delay in product launches.
- The pricing and reimbursement model in gene therapy is still to be fully established. While the field continues to evolve, questions remain around the financial risk of payers’ and the durability of a high-cost, one-time treatment.
- However, continued innovations and more product approvals are expected in the coming years supported by the broader thesis of improved treatment outcome.
