From The Editor | March 17, 2021

Regulatory Approval Trends In The Age Of COVID-19


By Matthew Pillar, Editor, BioProcess Online

Drawing Vaccine

The FDA is approving more therapies at a faster pace, but it’s not all COVID, all the time. Allan Shaw helps us expose regulatory approval trends, and the implications of those trends on the biotech business.

Allan Shaw is one of a handful of the self-made authorities in the biotech business who’s managed to hold on to his independence while providing service critical to emerging life sciences companies. On paper, he’s a finance and capital guy, having held several CFO and finance consulting positions—in addition to board seats—with dozens of emerging biotechs over the years. But Shaw’s experience is so deep and wide, he can’t be pigeonholed. His unique perspectives on finance, capital, and growth are well-served by a seemingly innate ability to connect the dots among the fiscal, regulatory, personnel, and scientific forces that are busy at work determining winners and losers in this wild age of biotech. We know better than to assume he was born with it, though. Shaw’s been around the block a few times. That’s one reason I was eager to get his take on the uptick of regulatory activity we’ve seen over the past 12-18 months, and his thoughts on the implications of that activity for new and emerging biopharmas. We discussed the topic on episode 32 of The Business of Biotech, which you can listen to in its entirety here.

BioProcess Online: How would you characterize or describe the current U.S. biopharma regulatory environment?

Allan ShawShaw: There’s an expression that I’ve been using since my formative years. Why did the auditor cross the road? Because the auditor did it last year. Historically, the regulatory environment is no different. People are very comfortable and familiar with our time-tested approach to drug approval, and the emphasis on safety and protecting patients while trying to advocate innovative medicines. But recently, and to the FDA's credit, they’ve tried to be adaptive. That began under Scott Gottlieb's regime, which I think was really an enlightened period. We’ve really seen that adaptability come to fruition over the past 12 months, as the agency has approved a series of vaccines and drugs—and new modalities, no less—in record time.

BioProcess Online: Even pre-COVID-19, hadn’t the pace of FDA action quickened, and if so, why?

As a headline number, that’s undeniable, and it’s encouraging. When you start peeling the onion back on some of those things, I think it tells a story. When you look at the calls for greater clinical efficiency, particularly as trials become more costly and complex, that's something to be mindful of. The FDA is trying to align on ongoing modernization efforts to base regulatory decisions on informed assessment of better benefit/risk balance. But that also requires a deeper understanding of those risks, a comprehensive assessment of the benefit, as well as the patient's perspectives and preferences.

When you continue peeling the onion back, you see trends in what's been approved. There’s a high concentration in orphan diseases, and there's a high concentration in oncology, and rightfully so—cancer affects us all, and we’ve made great progress in taking it from a death sentence to something more akin to a chronic condition.

But as it relates to the business side, orphan and oncology are capital-efficient programs to develop. You need smaller clinical sizes because the patient groups are smaller. Because you’re not talking about a primary care product, you have fewer considerations regarding the population mass and attributes that need to be incorporated. There’s not a large sales and marketing expense, as the drugs’ target markets are small and their price tags very, very large.

In oncology, specifically, we’re talking about life and death issues and unmet medical needs, so the risk balance is a little bit different. The FDA has demonstrated more lenience in terms of thresholds for oncology drug approval.

When I take a step back and look at primary care diseases that affect larger patient populations, like cardiovascular disease, diabetes, and neurological conditions, I don't necessarily observe as much development, and thus, approval, in those areas. I would attribute it to the fact that it's costly to develop and run big studies. I think that's the opportunity, to figure out how we make those clinical efforts more cost effective and more targeted to patient populations in the context of regulatory standards.

BioProcess Online: There’s been quite an influx of new modalities and therapeutic platforms over the past couple of years. Is FDA keeping pace?

Shaw: Generally speaking, I would say yes. I think if there's a feedback loop so we can learn, you're going to see more collaboration as you move forward. Regulatory activity needs to be as flexible and sophisticated as the science that's driving it. The speed of the approval process for messenger RNA products speaks a bit to the Agency’s criteria though. There seems to be a high level of safety associated with it. It’s on target and it offers wider therapeutic windows. As certain platform technologies like this progress, comfort levels might improve, and you might be able to lower the bar a bit as you move forward.  

In contrast, I'd say the FDA seems to be raising for gene therapies. They're putting a lot more emphasis on manufacturing, and rightfully so. Gene therapies and manufacturing are really an engineering exercise, one size doesn’t fit all, and it’s a very fluid situation.

That’s but a small snippet from the Business of Biotech Podcast episode 32: The FDA’ Progressive Appeal featuring Allan Shaw. Be sure to subscribe to the podcast for weekly insight from the leaders of emerging biotech.