Discussing the ThinkLive Cell and Gene Therapy Summit

Pharmaceutical Commerce: Cencora’s ThinkLive Cell and Gene Therapy Summit was an inaugural biopharma event dedicated to exploring the future of cell and gene therapies (CGTs). Could you expound on the goal of this event, and what excites you the most about it?

Lung-I Cheng: At Cencora, we really believe in cell and gene therapy. There’s so much promise for patients around the world, and we’re just thrilled by what has been achieved in the last five years. There are approvals in the US and Europe, there's a lot more in the pipeline that are coming. From what we can see, we're not entering a new era, where you have new modalities, or they're reaching the markets, and you have other pipeline assets that are targeting larger patient populations. If you think about the green pipeline and the potential, there is so much that's out there. At the same time, the path to commercialization is quite complex. There’s a lot of barriers and challenges that can really impact the success of the products and ultimately, patient access. So, at Cencora we partner with cell and gene therapy companies to provide what we call end to end solutions to help them bring their therapy all the way from discovery through commercialization.

The goal of the summit was to bring our leaders together from across the industry. We had representatives from biopharma companies, patient advocates, advocacy groups, trade associations, and the FDA to really foster that collaboration. I think best practices that will really support a lot of companies that are out there developing and trying to figure out how to really focus on their commercialization. I am most excited by the level of engagement, and the knowledge sharing that has occurred. I think all of this will ultimately support the growth of the sector and ensure more patients can benefit from these groundbreaking therapies.

PC: What specific trends in the space have been instrumental in driving the commercial success of CGTs?

Cheng: The short answer is early planning. So, I’m a music fan. If you think about the commercialization process, that is cell and gene is like conducting a Mahler symphony, you have a lot of players. The music score is complex, there's so many moving parts. If you put that framework within the context of drug development, commercialization, similarly, you need to work with a lot of different stakeholders in that planning and execution process. I would say there's a couple of critical areas including patient access and support, regulatory strategy, supply chain design, market access, and then the overall stakeholder engagement. When we were putting together our meeting, we're very intentional in building out an agenda that really reflected all these crucial areas, and that we incorporated a different perspective. I know we talked about from pharma companies that participated, we made sure that we have leaders who've kind of been there. They’ve navigated through that so that they can share their perspectives.

We continue to really focus on best practices. We talked to a lot of companies. If you think about one example, and that is payer engagement, it is something that really needs to occur way early in that development cycle, understanding the payers’ expectations so that your evidence package can ultimately showcase the therapies benefit, and to justify the cost and to address the payer concerns and priorities.

The last piece I'll say as part of the early planning and going back to my analogy, being able to work cross functionally, being able to have that perspective as you develop your strategy is critical. That means you need to have crucial conversations around the different parts of the organization and an understanding the interdependencies between research development and commercialization.

Part 2 of this article was originally published on pharmaceuticalcommerce.com

PC: Could you describe some common pain points in the space that the industry has been experiencing?

Cheng: I will build off of what I just mentioned. If you think about patient access, if you think about the regulatory requirements, if you think about market access as it relates to pricing and reimbursement, one of the concrete examples I could offer is your clinical trial design. Obviously, the endpoints election is the most critical aspect of that exercise. From a drug developer’s perspective, how do you arrive at an endpoint or several endpoints that meet the regulatory requirements that are patient relevant and meet the payers’ expectations? It’s not easy. I've been in those meetings multiple times, trying to get that collective and cross functional perspective and make sure that your strategy is going to really work out for you three, five years down the line. That’s a great example of how you really need that early planning and dialogue and cross functional perspective to make sure that you can bring those drugs to market.

PC: How to you envision this market progressing in terms of its technological capabilities? At the summit, you had participated in a fireside chat with the FDA, so perhaps you could also share any valuable insight that the agency provided?

Cheng: The future is really bright. If you think about just five years ago, back then the FDA commissioner predicted that by 2025, the FDA will be approving between 10 to 27 gene products a year. We're now in 2024 and we’re on track to hit that goal. Based on what we can see from what's in the clinical development between the US and Europe just in this year, we are really hitting that target. Obviously, there's a lot more in the pipeline. There’s between 2000-3000 clinical trials, depending on how you count it around the world. So, the momentum is there. With all these different trials and all these different approvals, what was great to hear in the fireside chat at the summit, is the excitement and commitment that the FDA has and the competence that they have in the future growth of this sector. They’re really trying to help companies to shorten that development timeline to bring the markets or to bring the drugs to the market faster, and to be able to bring these transformative therapies to patients. I think one thing that came through very clear from that fireside chat, again, was early planning. The FDA is really encouraging developers to come and talk to them as early as possible. I think the learning process goes both ways. The FDA also has a lot of initiatives to make sure that they stay ahead of the technology. I don't want to speak on behalf of them, because that’s not my role, but it was encouraging to just see how much work that's being put behind cell and gene sector by the agency.

PC: From inception to commercialization, how long is the entire process timewise?

Cheng: Some of your decisions need to be made pre-clinical, before we even get into the first human. Then you have phase one, phase two, and phase three. You’re looking at 5-10 years or longer depending on where you are in that development journey. The key is to be able to bring the right subject matter experts along that journey, so that you can future proof your strategy and also keep in mind that things change very quickly in drug development. They change especially quickly in cell and gene arrays. I think that flexibility and adaptability are critical in that process as well.