3 trends shaping commercialization in the cell and gene therapy sector

Disharee Nath, PhD, Associate Medical Director at Mind+Matter, attended the two-day Cell and Gene Therapy USA (Sep ’21) conference organized by Reuters. The event showcased presentations and panel discussions from key players in the biopharmaceutical industry, including leaders from pharmaceutical companies, CROs, and regulatory bodies.

The discussions focused on the key challenges to CGT commercialization and their potential solutions, nuances of manufacturing processes, roles of specific stakeholders in the process and the ongoing and upcoming trends shaping the CGT field.

Cost and time

Cost and time are key considerations for the complex autonomous manufacturing workstreams required for CGTs.

CGTs are expensive, with companies managing their own workstreams and often incurring duplicative costs. The highly-specialized and often individualized processes involved in manufacturing also mean that CGTs often take a longer time to reach patients once approved than with small molecule and biologic therapies.  The increased costs affect affordability and can potentially reduce access for patients.

Participants at the event highlighted several solutions that could help mitigate these challenges:

• Creating an agile global supply chain that can support CGTs within their own sustainable and resilient ecosystems.
• Moving from a competitive to a collaborative mindset – companies should share their strengths and resources for mutual benefit. This could be achieved by assembling an “industrialization collaboration” playbook and sharing data in a secure fashion using new advanced digital technologies.
• Companies should look for opportunities for iterative improvements, such as building new products on existing platforms, which can reduce manufacturing costs overall.

Long-term patient follow-up

CGTs are mostly functionally curative, one-time therapies and require significantly longer periods of patient follow-up compared to traditional pharmaceuticals. Most require post-treatment engagement over 10-15 years, which involves educating patients, healthcare providers (HCPs), and treatment centers, as well as optimizing processes for data collection to ensure the durability of the treatment and to better understand the risks of long-term side-effects. However, it’s very difficult to ensure follow-up with patients for that length of time so companies are adopting “decentralized” or “hybridized” models which leverage full- or part-digital and virtual engagement approaches to improve convenience for patients, facilitate easier data collection, and improve patient/HCP compliance. Examples include gathering patient generated data via online tools such as consent forms, apps, website patient portals and phone apps to track patient health and what happens between on-site visits.

Social media listening can also provide valuable insights on how to achieve proper education of patients and caregivers regarding CGT.

Collaboration and transparency

Viral delivery methods in CGT are effective, but when associated with heightened safety considerations they can cause adverse events such as toxicity, immunogenicity, and insertional mutagenesis.

One proposed solution was to establish collaborations with academic research labs can promote development of safer and more efficient vectors. This may reduce the amount of virus necessary for therapeutic effect, which will lead to easier manufacturing, improved patient safety, and better outcomes.

Further downstream, CGTs significantly differ from small molecules or biologics, and manufacturers need to collaborate with a variety of stakeholders early in the process to ensure successful commercialization. The commercialization process needs to start 24 to 36 months before launch and involves working with HCPs to identify the appropriate patient, government regulators (FDA), payers for their buy-in, and treatment centers.

Attendees at the event noted that agility is key, as roles and responsibilities can change throughout the lifetime of a product, and manufacturing process needs to be adaptable to scale up as well as scale down due to the likely fluxes in demand.

It’s also the case that regulatory bodies are proposing new regulations, pre-submission support, and specialized submission and approval pathways for CGT products so manufacturers will need to engage with regulatory bodies early in the process to ensure they can navigate the route to market.

Further insight

The concept of CGT isn’t new, but the sector is just now starting to deliver on the promises of these innovative technologies. These therapies have lifesaving curative and/or long-term life-enhancing potential but the biopharma industry still has work to do to ensure success.