Cell and gene therapies (CGTs) have significant potential to revolutionize treatment but face challenges in commercial viability due to high costs, small patient populations, and reimbursement issues. The global market is projected to grow at a 46% compound annual growth rate, reaching nearly $37 billion by 2028, with an increasing number of approvals and technological advancements like CRISPR gene editing. However, most CGTs have underperformed revenue forecasts due to approval delays, supply constraints, and unfavorable reimbursement, exemplified by Roctavian's low sales. Prices of CGTs are rising, with eight out of ten of the most expensive drugs in the U.S. being CGTs. Outcome-based agreements (OBAs) are being explored to secure reimbursement, but adoption varies by country, with Europe being more advanced. Improving operating models is critical, particularly through cost optimization in manufacturing. For cell therapies, reducing costs by at least 50% is necessary for commercial viability, while gene therapies face challenges in recouping R&D investments due to small patient populations and complex manufacturing. Advancing manufacturing technologies at a pace equal to scientific progress is essential to avoid falling short of commercial success.
