Q&A: The evolution of the cell and gene therapy field

What was your overall impression of the Pharma USA conference?

Lung-I Cheng: It was exciting to feel the energy in person after so many years and to be able to connect with the leaders in the cell and gene therapy space. We discussed a variety of topics, ranging from commercialization and manufacturing to regulatory and policy, to market access. The content was so rich, and the discussion was so lively. I came away from that meeting feeling very energized.

What were some of the key takeaways on cell and gene therapies you hope the audience gained from the discussions?

Lung-I Cheng: That's a good question. I would say there were a couple of common themes. One is early commercial preparation. And by commercial, I mean broad commercial, including market access. There are many learnings from the pioneers who've launched cell and gene therapies in the last few years, and people have the shared goal of ensuring patients have access to these transformative therapies.

So, for companies launching soon, how do they build upon what's been done before or improve existing models? Thinking about that in early planning stages is critical. The takeaway for me was stakeholder engagement. This speaks to many stakeholders, but I would highlight regulatory and payers. We had a fantastic session led by Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) at the FDA. He shared his vision and hope to accelerate the approvals of cell and gene therapy. It was encouraging to see a regulator's perspective on wanting to help bring these therapies to the market.

He highlighted a couple of programs and changes the FDA made recently. INTERACT is a program that allows biopharma companies in the cell and gene space to engage with the agency earlier. Through that program and other mechanisms, he's encouraging companies to reach out to the FDA as early and as frequently as possible. I think that is an opportunity that everybody should take advantage of if they can.

Payer engagement really came through from several discussions we had around value-based contracting. Payers and therapy developers discussed their learnings, challenges, and opportunities. At the end of the day, we learned that value-based contracts are not a one-size-fits-all solution, and they must be tailored to your product profile and your patient population. Biopharma companies must carefully consider endpoints collected in the clinical trial. How will the data be collected? Because of the complexity of implementing these programs, the importance of engaging with payers cannot be understated.

Were there any new technologies or approaches discussed at the conference that you think have the potential to significantly impact the field of cell and gene therapy in the near future?

Lung-I Cheng: I think that this year alone, gene editing medicine will likely be approved in the US for the first time.¹ That's very exciting. The other thing that came up repeatedly from the conference was the desire and excitement to go beyond hematology in the cell therapy space. Solid tumors account for 90 percent of all cancers, so unmet need is exceptionally high.²

There are many shots on goal. Many different technologies and platforms are being explored right now, and that's fantastic. To see the field continuing to move in new directions is just great. I'm excited to see what the next few years will bring patients.

Were there any presentations or panel discussions that challenged your existing assumptions or provided a new perspective?

Lung-I Cheng: Going back to value-based contracting, we had a robust discussion around some of the gene therapies that were launched or planning to launch this year. It was informative to understand approaches to these different contracts and how they must be fit for purpose. We heard that companies rely on claims or rely on provider reported data. It’s clear data or data collection is a very important yet challenging aspect of the contracts. The sophistication and thoughtfulness that we're seeing in this space is something that I find fascinating. And it's great to see how the industry is trying to move forward with more ideas.

 

How do you see the field of cell and gene therapy evolving in the coming years, and what role do you believe conferences like this play in shaping that evolution?

Lung-I Cheng: Excellent question. Cell and gene therapy is only going to grow, and I think we are at the very beginning of a very long arc of scientific innovation and therapeutic transformations. Just this year alone, we are looking at potentially up to 13 cell and gene therapy approvals between the US and Europe.³ So there's much to be done, and there's the need to really share learnings and listen.

I think Reuters Pharma USA is a great forum where leaders and experts can exchange ideas. It's cross-functional, covering key components of any successful launch for cell and gene therapy— commercial, market access, regulatory, manufacturing, and so on. Cross-pollination of ideas across those different functions is so critical. I hope this conference will keep growing so we continue to have this opportunity to come together as a community and bring access to more patients.

 

As an expert in this field, what advice would you give to those who are just starting to think about commercialization for cell and gene therapy?

Lung-I Cheng: Build on the shoulders of the giants that came before you. I think the exciting part of cell and gene therapy is that there are many first-in-class medicines for diseases that have never been treated before. Just consider the patient benefit or even the societal benefit. It can be hard to map out your playbook without help from your internal colleagues or other experts outside your company, or even other experts that have launched cell and gene therapies. The stakes are so high the more conversations you can have with experts with the depth and breadth of expertise is critical to any success in the future.

References
^1. Liu, Angus. Vertex teases launch plans for first CRISPR gene editing therapy ahead of FDA decision. Fierce Pharma. 8 Feb 2023. Accessed 31 Mar 2023. Available at: https://www.fiercepharma.com/marketing/vertex-teases-launch-plan-first-crispr-gene-editing-therapy-ahead-fda-decision
^2. Solid tumor research. ThermoFisher Scientific. Accessed 31 Mar 2023. Available at: https://www.thermofisher.com/us/en/home/life-science/cancer-research/solid-tumor-research.html
^3. Hunt, Timothy. The Cell and Gene Therapy Sector in 2023: A Wave is Coming – Are We Ready? In Vivo Pharma Intelligence. 9 Jan 2023. Accessed 31 Mar 2023. Available at: https://invivo.pharmaintelligence.informa.com/IV146781/The-Cell-And-Gene-Therapy-Sector-In-2023-A-Wave-Is-Coming--Are-We-Ready