Cell and gene therapies (CGTs) represent a new frontier in healthcare. These treatments aim to modify or replace cells and genes to treat or cure diseases that were previously untreatable.
While their promise is enormous, launching them is not simple. Unlike traditional drugs, CGTs are highly specialized, expensive, and often given only once.
This means companies must plan carefully to ensure patients can access them while payers and regulators trust their value. Successful launches need customized strategies that balance science, logistics, and economics.
What is cell and gene therapy?
Cell therapy involves using live cells to restore or repair damaged tissues in the body. Gene therapy involves adding, removing, or altering genes to correct the root cause of disease. Both therapies can be life-changing, with many designed to work after a single dose.
Examples include CAR-T cell therapies for cancer and gene therapies for rare genetic disorders.
1. Early Stakeholder Engagement
Early stakeholder engagement means involving healthcare providers, regulators, payers, and patient groups from the very beginning of therapy development.
By listening to their needs and expectations, companies can design clinical trials and treatment protocols that address real-world challenges.
For example, payers want proof that the therapy improves outcomes compared to existing treatments. Doctors want simple and safe procedures for their patients. Patients want financial and emotional support. By aligning with these groups before launch, companies reduce the risk of delays and resistance later.
Advantages of Early Stakeholder Engagement
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Ensures clinical trials collect the right endpoints for payer approval
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Builds trust with doctors and hospitals who will deliver the therapy
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Reduces uncertainty about reimbursement and market acceptance
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Helps patient groups feel included, which increases advocacy and adoption
Disadvantages of Early Stakeholder Engagement
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Requires heavy investment of time and resources during early development
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Conflicting opinions from different stakeholders may slow decision-making
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Involving too many groups early can complicate trial planning
2. Evidence and Data Strategy
Evidence and data planning are critical because CGTs are costly and aim to be curative. Payers and regulators require strong proof that these therapies deliver long-lasting results. This strategy involves building robust clinical trial evidence and continuing to collect data after launch through registries and real-world evidence.
For example, companies must track whether patients remain disease-free for years after a single dose. This long-term data not only reassures payers but also supports future approvals and coverage expansion across regions.
Advantages of Evidence and Data Strategy
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Provides solid proof of durability and effectiveness over time
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Builds confidence with regulators, doctors, and insurers
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Helps justify high pricing by showing long-term value
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Supports international expansion by meeting different regional evidence needs
Disadvantages of Evidence and Data Strategy
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Collecting long-term follow-up data is costly and time-intensive
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Evidence gaps may create delays in reimbursement decisions
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Managing and analyzing large datasets requires advanced systems and expertise
3. Cross-Functional Teams
Launching a CGT is not the responsibility of a single department. It requires coordination across research, medical affairs, regulatory teams, commercial functions, and supply chain. Cross-functional teams ensure that every part of the launch moves in sync.
For example, while clinical teams test therapies, manufacturing must scale production, and access teams must prepare payer discussions.
Without this alignment, therapies risk approval delays, supply bottlenecks, or poor adoption. Many experts describe the CGT launch as a “team sport” where success depends on integration across all functions.
Advantages of Cross-Functional Teams
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Promotes better coordination between scientific, commercial, and operational units
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Reduces risks of misalignment that could delay launch activities
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Encourages shared accountability and faster resolution of challenges
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Ensures patient needs are addressed from multiple perspectives
Disadvantages of Cross-Functional Teams
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Complex team structures may create confusion without clear leadership
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Coordination requires frequent meetings, which can slow down decisions
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Higher operating costs due to the involvement of multiple departments
4. Commercial and Operational Readiness
Commercial and operational readiness prepares the market, supply chain, and support systems before launch. Since most CGTs are high-cost and one-time treatments, companies must ensure smooth manufacturing, controlled distribution, and patient-support programs.
For oncology CGTs, this may include certifying cancer centers to deliver therapies. For rare diseases, it may involve genetic testing and patient identification programs.
Financial support, travel logistics, and long-term monitoring systems also fall under this strategy. By addressing operational and patient needs in advance, companies can ensure smooth access from day one.
Advantages of Commercial and Operational Readiness
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Ensures therapies reach patients without supply or logistics delays
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Improves patient access with financial aid and support services
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Allows for scale-up in larger disease areas and precise targeting in rare diseases
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Builds provider confidence by training treatment centers in advance
Disadvantages of Commercial and Operational Readiness
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High upfront investment in manufacturing, logistics, and training
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Cold chain and identity tracking make distribution very complex
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Patient-support programs increase ongoing operating costs
Final Words
Launching cell and gene therapies is unlike launching traditional drugs. These advanced treatments require early alignment with stakeholders, strong data to prove long-term benefit, well-integrated teams, and careful operational readiness.
When done correctly, these strategies help overcome barriers of cost, logistics, and payer acceptance. With the right approach, CGTs can move from scientific breakthroughs to real-world cures that transform lives.
FAQs
What is the difference between cell therapy and gene therapy?
Cell therapy uses living cells to restore or repair tissues. Gene therapy changes or replaces genes to correct the cause of the disease.
What are examples of approved cell and gene therapies?
Examples include Yescarta (CAR-T therapy for lymphoma), Zolgensma (gene therapy for spinal muscular atrophy), Kymriah (CAR-T for leukemia), and Elevidys (gene therapy for Duchenne muscular dystrophy).
How do gene and cell therapies work?
Cell therapies insert healthy or engineered cells into the body. Gene therapies insert, delete, or modify genetic material to fix defective functions.
What are the main risks of cell and gene therapies?
Risks include immune responses, infections, insertion errors in genes, and uncertainty about long-term durability.
What challenges are faced in launching cell and gene therapies?
The main challenges are high costs, complex supply chains, strict regulations, payer hesitancy, and the need for long-term evidence.
