Gene therapies and genetically modified cell therapies have generated enormous interest among scientific researchers, manufacturers, investors and patients alike over the last decade.
For pharma, which has been underperforming other sectors in relative shareholder returns, the breakthrough potential of these novel medicines is attractive for both patients and companies. But the path to success has been challenging. The promise of one-time, revolutionary treatments for patients suggested that a massive disruption to the life science industry was imminent. However, as it turned out, manufacturing and commercial challenges slowed the development of the cell and gene therapy (CGT) market.
Sales of CGT therapies were lagging behind projections, and these products were available in only a handful of US ZIP codes, according to a PwC report on the market in 2019. We have updated our analysis of the market and found a resetting of expectations for these paradigm-shifting brands. Developers are working through the logistical issues, hundreds of treatment sites are open, and some products are performing well.
Manufacturing of cell and gene therapies is still a very customized, complex and expensive process but the clinical promise is very real and the pipeline of new therapies is abundant. In this report, we highlight seven key challenges in the CGT space and provide recommendations on how organizations can overcome these barriers to achieve success.
Despite the enthusiasm for innovative cell and gene therapies, the market has taken longer to develop than initially expected. Some gene therapies with blockbuster potential didn’t launch as expected, and some fell short of projections at the time of launch, while others have far exceeded forecasts.
Most healthcare facilities are unable to serve cell therapy patients because they lack the capabilities to perform apheresis and infusion on a carefully controlled schedule. Treatment today must be offered at specialized academic centers, limiting access for patients in rural communities. The complexity of administration has relegated the treatment to an inpatient setting, where costs are bundled and less lucrative for providers.
The continued excitement in this space has launched an industry-wide “war for talent,” pitting companies against each other as they fight to attract the small number of individuals with the appropriate skills. The greatest areas of concern have been related to manufacturing and quality, followed by supply chain, logistics and process development.
Allogeneic therapies represent a particularly disruptive advance on the horizon for the CAR-T market. These “off-the-shelf” treatments use donor cells or induced pluripotent stem cells to create a standardized target cell therapy for the general population, instead of a highly individualized batch made from a patient’s own cells. Allogeneic therapies have the potential to significantly improve costs with shorter treatment timelines, simplified supply chains and economies of scale during the manufacturing process.
Strict quality controls are necessary to ensure patient safety, but navigating the quality challenges for CGTs will be critical to ensure timely access to these novel therapies. Doctors also have concerns about long-term safety and efficacy, issues that can dampen interest in trying complex new therapies.
The complexity of the supply chains required for autologous cell therapy manufacturing needs to be simplified. Every step in the process provides abundant opportunities for human error, timeline delays and quality disruptions that could impact the efficacy and safety of the product.
The most important raw ingredients required to manufacture cell and gene therapies are viral vectors. Although CDMOs have expanded capacity in recent years, they have yet to meet the demand for this critical input. Viral vectors are required for a wide range of therapeutic products beyond CGT — including COVID-19 vaccines — so it is a competitive marketplace for companies in both the short and long term.
As interest in the CGT market continues to grow, companies throughout the industry must find solutions to address these challenges. Anticipating and creating strategies to prepare for these challenges can help ensure that biopharma companies are able to go to market, drive pipeline growth and ultimately find success.
Pharmaceutical and Life Sciences Transformation Leader, PwC US
Partner, Strategy&, PwC US
Managing Director, Strategy&, PwC US
Director, Strategy&, PwC US
Senior Manager, PwC US
Manager, PwC US