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Navigating the complexities of launch preparation for Cell and Gene Therapy

- PMLiVE

- PMLiVE

Cell and gene therapies (CGT) provide transformative treatment that can treat conditions that were previously considered untreatable. As described in the previous article in this series, most CGTs target patients with very rare diseases, requiring a more tailored approach in key areas like patient support and supply chain models, but there are signs that these treatments will start moving into larger population groups. Uniphar provides programs that can be designed around the patient and healthcare professional and navigate treatment pathways, offering guidance on therapy administration and post-treatment care.  

Accessing patients with CGT requires more than just innovation, it requires a trusted partner who can mitigate the complexities. Uniphar supports companies by streamlining regulatory navigation and ensuring that every step of the CGT journey is aligned to one goal: getting life-changing treatments to the patients who need them most. 

The need for early planning 

Launching a CGT demands not only scientific excellence but also early, strategic planning across the entire access pathway. With therapies often targeting small, geographically dispersed patient populations, it’s essential to begin engaging early with regulatory experts, map the patient journey, and understand the total pathway. Identifying barriers to access – clinical, logistical, or regulatory – can significantly influence the success of launch sequencing and market uptake.  

One of the most impactful tools in this early phase is the establishment of expanded and early access programs (EAPs), which allow patients without treatment alternatives to receive life-changing therapies ahead of full commercial availability. These programs also generate real-world insights for healthcare providers and help manufacturers refine access strategies in preparation for launch. Uniphar supports companies by integrating these critical elements early, ensuring therapies can reach the right patients at the right time. 

Regulatory compliance in CGT approvals 

The regulatory landscape for CGT is both intricate and evolving. Approvals often hinge on groundbreaking evidence and precise compliance with region-specific requirements. In the European Union, CGTs are classified as Advanced Therapy Medicinal Products (ATMPs) and undergo rigorous evaluation by the European Medicines Agency (EMA). In the United States, the FDA has established frameworks to expedite approvals while ensuring safety and efficacy. In the Middle East and North Africa (MENA) region, regulatory frameworks for CGTs are evolving, with increasing efforts to align with international standards and implement reliance-based pathways to facilitate patient accessi.Uniphar has significant expertise in working collaboratively with regulators and pharmaceutical companies to navigate these complexities. By aligning with global and regional regulatory frameworks, Uniphar streamlines approval and distribution processes, enabling timely patient access. 

Addressing cost and affordability
CGT therapies come with high price points, which can make it challenging for healthcare systems to absorb costs and maintain equitable access. Innovative payment solutions are increasingly being explored to ease this burden. Uniphar supports its partners with expertise in pricing and payment strategies and market-specific funding models, helping to ensure that high-cost therapies remain financially viable for both payers and manufacturers. 

Managing complex supply chains
Many CGT products require time sensitive cross-border movement for each patient order, involving meticulous coordination across customs regulations, temperature-sensitive logistics, and distribution models – whether implementing a centralized distribution strategy or adapting to localized needs. With deep experience in cold chain logistics, customs clearance, and regulatory compliance, Uniphar ensures that therapies reach patients safely, efficiently, and on time. 

Real-world evidence  

In CGT, where traditional clinical trials may be limited – especially for rare diseases – real-world evidence (RWE) plays a vital role in demonstrating long-term safety, efficacy, and value. Collected through early access programs and post-treatment follow-up, RWE helps fill clinical gaps and strengthens submissions to Health Technology Assessment (HTA) bodies. 

Uniphar works with manufacturers to build robust data collection strategies that support reimbursement decisions and enhance clinical confidence. Integrating RWD early in the launch process can accelerate access, inform ongoing development, and ensure therapies reach more patients effectively. 

Enhancing Medical Affairs Capabilities
Medical affairs play a pivotal role in the successful launch of cell and gene therapies, encompassing stakeholder engagement, scientific education, and real-world evidence (RWE) generation. Uniphar supports healthcare professionals (HCPs) from the earliest stages of Expanded Access Programs (EAPs), ensuring they are thoroughly trained, supported, and prepared to deliver complex therapies effectively. 

Uniphar’s experienced and versatile medical team offers both local and international expertise, providing tailored, discreet services that help overcome barriers to launch and accelerate patient access. Their capabilities span strategic and tactical medical planning tailored to European markets, including: 

  • Local brand team support, therapeutic area training, and congress reviews 
  • KOL engagement at national and European levels, along with advisory boards and scientific symposia 
  • Evidence generation and outcome research, including clinical evidence gap analysis, integrated RWE planning, and support for abstract and manuscript publication 
  • Medical education and scientific communication, through webinars, speaker meetings, podcasts, and more 
  • Patient support and medical information services, such as Patient Support Programmes and patient advocacy group (PAG) engagement 

Through this integrated and collaborative approach, Uniphar enhances medical affairs strategy to not only support launch execution but also build long-term clinical confidence and adoption. 

Pharmacovigilance, medical information, and launch excellence
Pharmacovigilance is the ongoing monitoring of therapy safety and is essential in the CGT space, where long-term data is still emerging. Uniphar’s pharmacovigilance services ensure timely identification and management of adverse events, fostering trust with regulators, clinicians, and patients alike. 

Equally critical to a successful launch is a well-defined launch plan -underpinned by clear, consistent messaging and fully briefed sales teams – is vital to accelerating market uptake and ensuring appropriate product positioning. Uniphar supports clients with brand optimisation services that help unlock the full potential of brands. Through flexible partnership models, Uniphar can support brand commercialization, including optional integration with end-to-end supply chain management. 

Conclusion 

The successful launch and delivery of cell and gene therapies requires a detailed approach that addresses regulatory, logistical, and clinical complexities. Uniphar’s extensive experience in navigating these challenges positions it as a vital partner in bringing CGT innovations to market. Through its commitment to early access, regulatory collaboration, cross-border logistics, medical affairs, patient support, and pharmacovigilance, Uniphar is constantly demonstrating the value of their expertise by delivering impact that is transforming lives worldwide.

Read our next article in this series, where we explore how early, strategic planning is critical to the successful launch of cell and gene therapies. From mapping the patient journey to implementing early access programs, we’ll examine how proactive preparation can overcome barriers and accelerate time to treatment. View this here.

This content was provided by Uniphar

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