Cell & Gene Therapy
Cell & gene therapies (CGTs) represent the forefront of clinical innovation and a dynamic challenge to payers and manufacturers that struggle to adapt historic practices to novel economic and regulatory barriers to access. The market expansion opens significant opportunities for new entrants and has the potential to benefit over 3.5 million patients by 2030
Growing opportunities — but added complexity
Indistinct reimbursement models with payers remains a challenge for cell & gene therapies companies. Drug manufacturers can actively collaborate with payers to develop sustainable solutions that ensure broader availability and enhance accessibility.
Cell and gene therapies can help beyond the rare disease population. They can address more prevalent conditions, including neurodegenerative disorders and cardiovascular diseases, opening significant market opportunities.
A rapidly expanding market
Cell & gene therapies have seen exponential growth in product approvals, diversification of indications, and potentially eligible patients.
70%
FDA approvals of CGT therapies increased 70% in 2023 compared to the preceding two years
80%
Over 80% of approved CGTs in the past decade have spearheaded progress in rare diseases through Orphan Drug designations — a trend expected to continue
4x
Exponential growth is anticipated for CGT approvals, projecting a 4x increase in 2024 compared to 2023 and soaring to 10x in 2027
When CGTs first emerged there was a lot of excitement, but the first treatments were in disease areas with small populations. In many ways we are still waiting for the larger wave of CGTs to come before we make any firm decisions on the category.
Regional MCO
We weren’t initially sure how to approach this new CGT category. Given that CGTs are commonly administered in an inpatient setting, we involve our medical policy teams in the review process. However, these teams historically focus on procedures & devices and aren’t accustomed to contracting.
National Insurer
The early CGTs in rare diseases had initial N sizes in the literal single digits for our plan, so we were able to handle those requests with individual letters of agreement. But you can’t do that if you suddenly have dozens of CGTs across larger patient populations.
National Insurer
Payers are the upfront price and durability of effect. You are bottling up 20 years of medical costs into one treatment, but what happens when the patient switches plans after 3 years or if re-treatment is required?
National Insurer
How can Acquis help?
Acquis understands the complexities of the development lifecycle. Thoughtful planning in the early stages of the product lifecycle is critical to reaching the right patient at the right time with the right drug, optimizing downstream peak revenue post-launch.
Collaboration with Research Entities
To facilitate better collaboration between payers and research entities, enabling efficient trial design and execution and quicker development and delivery of breakthrough treatments to patients
Compliance Strategies
To ensure adherence to regulatory requirements by assisting in the development and implementation of compliance strategies for both payers and manufacturers
Contingency Planning
To prepare for unexpected changes in the treatment landscape, ensuring quick adaptation to new data, regulatory shifts, or market dynamics, thus maintaining continuity and efficiency
Engagement with Regulatory Bodies
To foster better collaboration by developing strategies for enhanced interaction between payers, manufacturers, and regulatory bodies
Financial Modeling and Analysis
To understand the financial impact of CGTs on payer budgets, helping in better resource allocation and budgeting decisions
Method Transfer
To ensure regulatory compliance and smooth integration by aligning testing methodologies with Health Authority requirements and assessing site capabilities
Outcome-Based Agreements
To link reimbursement to clinical results by advising on the creation of outcome-based agreements between payers and manufacturers
Patient Journey Mapping and Engagement Strategies
To develop robust patient engagement strategies by analyzing ATUs, ensuring comprehensive support throughout the patient journey
Payer Cultural Competency
To improve health literacy and address diverse patient perspectives by helping payers enhance their cultural competency
Performance Metrics and KPIs
To measure the effectiveness of strategies and programs, ensuring continuous improvement and goal achievement
Post-Market Surveillance Strategies
To ensure safety and efficacy by developing frameworks for generating real-world evidence
Protocol Optimization
To help payers streamline clinical trial protocols, which in turn ensures faster and more cost-effective research outcomes without compromising scientific integrity
Real-World Evidence Generation
To enhance payer decision-making by incorporating real-world evidence, including patient-reported outcomes
Regulatory Landscape Analysis
To understand and interpret the implications of new guidelines by analyzing the evolving regulatory landscape related to CGTs
Thought Partnership & PMO
To enhance cross-functional collaboration and improve efficiency by aligning organizational structures, goals, and processes, and identifying bottlenecks
Value-Based Pricing Strategies
To ensure pricing aligns with the clinical and economic benefits of therapies, supporting sustainable healthcare funding and patient access
Curious to learn more?
Get in touch with the Acquis team to help drive CGT impact