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Three questions biopharma companies must ask when building a cell and gene commercialization strategy

By AmerisourceBergen

Anticipating barriers helps the transition to market 
Breakthroughs in cell and gene therapy (CGT) are accelerating rapidly with more therapies entering the market every year, but the commercialization of these innovations is highly complex. Emerging biopharma companies have to develop a commercialization strategy early in their planning to ensure success at launch and beyond. To that end, biopharma companies must ask themselves the right questions while creating their CGT strategy. 

1. What are potential barriers during the CGT commercialization journey? 

To ensure success, many CGT commercialization decisions need to be addressed early on during clinical development. When examining the complex treatment journey for cell and gene therapies, a patient-centric approach serves as a useful blueprint. Biopharma companies need to focus on three key areas to overcome potential hurdles: patient support and services; supply chain; and market access. 

A CGT journey generally includes the following patient operations, support, and coordination activities: 

  • Patient identification and pre-screening 
  • Patient referral 
  • Patient enrollment 
  • Obtaining insurance coverage 
  • Purchase order and credit check with a distributor 
  • Transporting the product to the site of care 
  • Treatment administration 
  • Long-term monitoring and outcomes tracking 


Here are some potential barriers along the journey:


Focusing on patients:

  • Patient identification: Identifying appropriate patients can be challenging for clinical development and commercialization, especially for rare or ultra-rare conditions.
  • Care coordination: Patients expect excellence in care and seamless coordination and connection. Patients can face transportation challenges when they need to travel to qualified treatment centers to receive care. It is critical to reduce the burdens on patients and their caregivers throughout the journey by enhancing connectivity.
  • Long-term follow-up: Long-term tracking of safety and outcomes data requires careful planning, but also provides an opportunity to demonstrate value to patients, providers, and payers while generating real-world evidence. 

Developing a tailored supply chain:

  • Distribution network design: Distribution network design can impact the patient journey and logistics needs. Requirements to onboard a clinical site versus a commercial site can be different. Scalability is another important consideration even at initial launch.
  • Logistics and storage: The range of temperatures required for transport and storage necessitates tailored solutions. Flawless execution is expected but is sometimes complicated because of the need to ship materials or products globally and promptly.
  • Data orchestration: Build-as-you-go may not be a suitable option for the large amounts of data generated during the patient and product journey. Laying a foundation for data orchestration as early as possible helps enable commercial success. 
Market access professionals

Securing marketing access:

  • Payer evidence requirements: Value assessments can be challenging when data is limited, especially with single-arm pivotal trials. The need to bridge data gaps for different payers across the globe can be a daunting task.
  • Willingness to pay: Willingness to pay varies depending on payers and geographies. The impact on the healthcare budget is a concern for payers, especially in larger patient populations.
  • Innovative contracting: Innovative contracting needs to fit the purpose, require early planning, and minimize the administrative burden on stakeholders. 

2. When should a biopharma company start to think about CGT commercialization? 

It is never too early to start thinking about the CGT commercialization strategy. Some critical decisions are made during preclinical or Phase 1 studies. When such decisions are made without forward-thinking or commercial input, companies may find themselves struggling to get the product to patients later on.  

To better understand if there is a commercially viable opportunity for the therapy, biopharma companies should work with key stakeholders early and often. This means engaging with health authorities, payers, patient advocacy groups, providers, and patients. 

3. What does the right team look like for CGT commercialization? 

Having the right team is essential for successful CGT commercialization. By defining the key players at the beginning, biopharma companies can build a strong foundation for their teams and engage with the correct stakeholders. In addition, aligning with the right partner from the start gives access to crucial know-how. 

Biopharma companies can choose to allocate their resources to build a team in-house or search for the right partner to coordinate all the different stages and stakeholders for them. The right partner provides integrated solutions and delivers the right resources at the right time, including helping with:

  • Market access 
  • Quality, regulatory, and pharmacovigilance
  • Transport, storage, and logistics
  • Supply chain and distribution
  • Patient support programs 

Does your commercialization partner offer a collection of resources to strategically guide, deliver, and support therapies in the market? Are they positioned to securely and compliantly shepherd therapies through the complex patient journey and connect with core stakeholders? 

The advantages of partnering with a full-services commercialization partner include: 

  • Supply chain strategy expertise spanning clinical and commercial phases, including complex storage, distribution, and transportation expertise across global markets 
  • Real-world evidence generation strategy and long-term outcome insights from clinical development through treatment and beyond 
  • Established relationships across sites of care that offer real-time visibility to provider site activity and patient access barriers to reduce unique patient challenges pre- and post-treatment 
  • Proven patient services expertise guiding and supporting individual patients and their caregivers throughout their unique treatment journeys 

Biopharma companies who are considering CGT commercialization journeys must start planning right away. By anticipating potential hurdles, they can expedite the transition to commercialization faster. 

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