Maximizing Potential: Essential Steps for Successful Commercialization of Cell and Gene Therapies

Introduction

Cell and gene therapy (CGT) products have significantly enhanced the quality of life for millions of patients impacted by medical conditions that are untreatable with traditional medicines or surgeries such as certain cancers, inherited and rare diseases, and intractable conditions. As these therapies continue to propel groundbreaking advancements in remedying previously incurable diseases, the complexities of properly commercializing them and ensuring ample patient access can present significant challenges for biopharma manufacturers.

The FDA and EMA are forecasted to approve and license approximately 30 new CGT products annually by 2025, and the global cell therapy market is projected to reach $247 billion by 2028. This creates tremendous potential for manufacturers, but only if their commercialization efforts go beyond conventional tactics often associated with more traditional therapies.

CGTs require a more intricate set of steps to prepare for market readiness than less complex therapies. Securing real-world evidence, robust patient engagement and thorough pricing and reimbursement strategies are only a few areas manufacturers must invest more resources into than they are likely accustomed to when launching more traditional products.

Potential vs. Reality

Despite the promising prospective for CGT to expand into broader patient populations and revolutionize healthcare systems by reducing the corrosive burden of chronic conditions, an alarmingly small amount of these therapies are brought to market successfully. In fact, experts predict up to 40% of CGT products on the verge of approval will be entirely unviable due to manufacturers’ unsuitable commercialization proficiency.

Lack of adequate preparation and understanding of complex commercialization needs, manufacturing logistics, high development costs and regulatory hurdles are often leading contributors to the disappointing performance of multi-faceted, multi-stakeholder CGT products.

Another common factor worth highlighting is that a substantial number of companies pioneering novel CGTs are start-ups or medium size enterprises that do not have sufficient resources, and quite possibly commercial expertise, available to larger and more established organizations.

The total cost of care associated with delivering and managing CGT treatments can balloon up to $750,000 to $1 million per patient. Because of the exorbitant cost, demonstrating true value in a real-world setting for these treatments is critical to provide sufficient evidence that not only supports pricing expectations, but also provides further clarity on patient populations that will respond disproportionately better than others.

Unlocking Success for CGT

Manufacturers of CGTs must think differently about their approach to and corresponding capability investments in commercialization and market readiness. This requires leveraging best practices from traditional commercialization tactics while also thoughtfully crafting novel approaches and keys to success that address the more intricate challenges specific to launching these complex products.

Featured below is a commercialization checklist that includes vital courses of action all manufacturers of CGT products should prioritize when preparing to launch a new therapy into the marketplace. While these steps require more time and resources than traditional products, they are definitively necessary to find success with these highly involved therapies.

The risk of not following these important steps is a drastic increase in the probability for failure or, at the very least, not meeting full potential. The reward will be an immediately stronger ROI for the manufacturer and, most importantly, greater access to life-altering treatments for patients with chronic conditions that require CGTs.

A Comprehensive Commercialization Checklist for CGT Manufacturers

✔ Patients

• Identify and understand the patient journey. Have a mission of “No patient left behind”
• Work with treatment centers to help their accreditation and to understand logistical issues
• Connect with and support patient groups early to understand how the condition affects their lives and demonstrate how the therapy can improve them
• Use real-world and socio-economic evidence from patients to demonstrate broader and enduring benefits

✔ Planning

• Invest time and effort in planning details
• Ensure commercial alignment by function – marketing, field sales, access and reimbursement, trade and distribution, and patient support
• Partner with outsourcing specialists to upscale potential
• Do not rely on copy-and-paste launch templates or practices from mainstream drug launches
• Invest in details and nuances of how and why institutions prioritize their decisions, investments in the willingness to prescribe, and a payer’s willingness to pay

✔ Advocacy

• Source and develop KOLs to underpin the case for the therapy and make them part of the journey to improve high-level advocacy
• Engage in media analysis to understand resistance to cost and to articulate the benefit in different geographies

✔ Medical Affairs

• Focus on MSLs to make the case and liaise with HCPs before turning up sales efforts
• Weave the economic and scientific case together to demonstrate long-term efficacy for the patient and the health system
• Make a clear case of the differences between existing therapies and the improvements on infrastructure, outcomes and economics that switching to a CGT will bring

✔ Data

• Invest in clinical and economic evidence generation before taking therapies to patients
• Commit to continuing to collect data and real-world evidence after approval
• Understand what performance details matter to stakeholders
• Have robust collections systems that eliminate duplications
• Build capabilities to track patients who move or change circumstances over time
• Make that data available as part of a running dialogue with healthcare systems to demonstrate long-term benefits and support innovative payment strategies

✔ Manufacture

• Start early with supply and capacity analysis
• Invest in understanding the differences in Chemistry Manufacturing and Controls (CMC) in different countries
• Consider partnering with established contract manufacturing organizations with specialized experience to offset high resource costs

By diligently following this comprehensive commercialization checklist, manufacturers of cell and gene therapy products can overcome the unique challenges they face and pave the way for successful market entry, ultimately providing life-altering treatments to patients in need while achieving a stronger return on investment.