In the recent Economist Intelligence Unit biopharma survey, cell and gene therapies emerged as the most potentially disruptive new products for the near future. Yet while these therapies show early clinical success, many hurdles remain in actually bringing them to market. The second part of our conversation with Martha Rook, Head of Novel Therapies at brand-name’s Process Solutions group, deals with manufacturing challenges, regulatory uncertainty, optimism, and more.
Are we ready to bring [gene therapies, cell therapies, and other such novel therapeutics] to market?
I think it’s acknowledged by everyone in the field, that this is the critical point for these types of personalized cell therapies. There are a lot of really bright people working on this and a lot of companies working on how to bring the first products to market.
And partner companies like brand-name are working to leverage our expertise with drug product development to put together a process that will help the therapeutic companies deliver on this. We have, within one company, all of the pieces that you need to assemble a seamless process to enable the manufacturing of these therapies.
The number one risk concern for survey takers was regulatory uncertainty—this concern was even greater for those developing novel therapies. Are those concerns well founded?
Risks are really about the unknown – the unknown around how you’re going to chart the path of this new drug to market. Particularly on the gene therapy side, there’s a higher scrutiny around the regulatory aspects. Where people see risk is in not understanding the expectations of regulators around safety profiles, long-term monitoring, and manufacturing assurances.
On the counter side, you’re seeing with many of these companies some strategies to address the risks. A very common strategy is to start with an “orphan disease” where there’s a high, unmet medical need. This allows you to make a very meaningful difference to patients who are often on the path to dying. Now you’re giving them an opportunity.
There’s a risk around that therapy, but the patients are already living in a high-risk state. It makes the regulatory discussion more reasonable. Also, the things that you learn from those patients give you a lot of information about more general risks of the therapy. That can then be applied to a broader patient population. So you de-risk your therapy development through this staged approach.
We see a lot of companies are racing to get their new therapies to market. Do you think they're underestimating the risks of proper scaling for mass production?
The companies are moving very fast right now, particularly in some of the exciting areas, like CAR-T. You have a variety of companies that are racing to get to the clinic. And in that race, you sometimes neglect to ask, “How am I going to manufacture this once it’s approved, and I need to scale it up?”
I do think people working with these novel therapies really need to start their development with manufacturing in mind. At what stage in my clinical trials am I going to be ready to put in place the scalable manufacturing technology? I think there’s a wide variety in how well companies take the time to do that planning and make sure they’re ready to commercialize when they get the approval.
Really, the last thing you want is what happened to some early innovators in the cell therapy space. There have been cases where clinical efficacy was demonstrated, but manufacturing could not be achieved in a cost-effective way. And it prevented successful commercialization of the therapies
One last question. In the survey, biopharma leaders said they were concerned about novel therapies, but also very optimistic about them. Are you optimistic?
I am! I’m absolutely optimistic. It’s an extremely exciting space to be in. I was really happy to see how confident the survey participants are. I read the survey results as an indication of belief in therapeutic efficacy and commitment to the space. People understand what these are, and for the most part, they understand how they’re working. It’s just a matter of getting it done and figuring out how to bring them to the public. As a partner of biopharmaceutical manufacturers, we are committed to helping our customers navigate the path of developing and producing novel therapies.