Heard on the pipeline: R&D in the pharma industry

In the fast-paced world of healthcare, it can take a lot of work to keep up-to-date with the numerous innovations, trends, and opinions that emerge each year.

And so, to cut through the noise of information, when the Deep Dive team caught up with attendees at the J.P. Morgan Healthcare Conference, we kept things simple.

One question: What are the biggest pharma R&D trends to watch in 2023?

Here’s what some of the best minds in healthcare had to say:

Raj Chopra

Acting CEO, Aethon Therapeutics

There’s been a huge explosion of cancer discoveries, particularly in drug discovery. We’ve had small molecule inhibitors, we’ve now had targeted protein degradation, and then we’ve got the revolutionary use of antibody, both bispecific antibodies – we’ve just seen Genentech register a bispecific in lymphoma – and we’ve also seen antibody-drug conjugates coming through. I think this [our technology] is yet another pillar of the armamentarium against cancer.

Adi Hoess

CEO, Affimed

We have been talking about cellular therapies for many years. And despite the fact that there were some initial successes – we always have some low-hanging fruits – we’re now in the phase of optimisation. Usually, that looks like: ‘Here was a one-off success, but you have to wait another five or ten years to see the fruits coming through’, and it takes a while until it’s ripe. I think out of all the diseases out there, the most important one to address is cancer.

I can imagine that the trend in the future will be you bring together bispecifics, checkpoints, and cellular therapies as they become cheaper, but also the mRNA vaccines and technologies. Then you have a really good service for the patient. It can take care of the tumour by itself, and also, the threat is taken away from the family, where you don’t know if the cancer is coming back.

Alexandre LeVerte

Chairman and CEO, Osivax

One of the trends is that the funding market is putting a lot of pressure on biotech, so we need to be creative about how to ensure that we can still aggressively push forward on the projects that are needed. There’s a lot of money around, but it’s just not allocated as it used to be. I think 2022 and 2023 have been really different, with the historical shareholders supporting their companies in a much stronger way and probably M&A coming into play to balance this. The market relied on new rounds of funding two years ago. Now, it’s time for the maturation of this whole ecosystem.

Michelle Longmire

CEO, Medable

We’re in a golden age of science. When you look at the progress of mRNA – this is a technology we’ve been working on since the 60s, but suddenly its application has become something so profound, with so much potential. As we know, though, the clinical trial remains the bottleneck. Thirty-seven drugs were approved last year. The average is around 50. First, why fewer? And second, why have we only had the same rough number of approvals for ten years? Clearly, in a golden age of science, we should be able to see more shots on goal, ideally, and therefore more approvals without lowering any standards.

I see the awesome leverage of technology and innovation in basic science and pre-clinical development. But I don’t see that same innovation and technology applied to clinical development. I think the data we’re showing to bend the cost curve is really suggestive of a new era, but we need that same kind of leverage of technology that we see in pre-clinical to be applied to the clinical process. That’s how we unlock not 50 but 500 drugs approved.

As awesome as the science is, we don’t get cures until we solve this problem.

Gerry Chile

Newell Health (Healthware)

If we want to talk trends, almost like fashion trends or the coolest, hottest thing around, it’s hard to get away from ChatGPT and what everyone’s talking about. But certainly, artificial intelligence is going to be jumping out of the gate. It already has, probably three to five years sooner than I was expecting at this level. The floodgates have been opened with this public trial of ChatGPT; there are already health applications, it passed the medical exam, it passed the bar exam, it passed everything. When you sit down and play with it, you realise in your areas of expertise, it just cannot be trusted. But that’s only scratching the surface.

While we’re not doing anything with ChatGPT, certainly data science is a huge component of the Soturi project. It tries to build algorithms with predictive modelling, trying to make sense of data points from many different types of data sources. So, I think we are coming to a point where algorithmic, machine learning, data science-driven healthcare is really going to come of age starting this year into the next two to three years.

Robin Roberts

Novartis Biome

The pandemic created an interesting scenario where digital health, in general, became incredibly important. It allowed folks from across our industry to overcome this learning curve that they wouldn’t have necessarily wanted to push against, which got them to the point where they’re using these digital health solutions and now are seeing the value of utilising them. I think 2023 is going to be one of the first years where we are going to have to do everything we can to keep as much of that momentum as we possibly can within our industry and across the healthcare system.

When we deliver healthcare, telehealth is just a part of the way we do business. What other value-add can we incorporate into that capability that makes it even more valuable? It’s one thing to be able to talk to your physician on video. It’s another thing if they collect the metrics they need during that video call and make diagnoses. The technology is out there. It’s not a technology issue. We’re talking about using technology that has been available for decades and applying it to the right use cases in the healthcare space.

Gene Kinney

President and CEO, Prothera Biosciences

We’re very focused on these protein-based diseases, and we think the field, in particular in Alzheimer’s disease, is at a very exciting inflexion point, and it’s happening right now. We’re seeing the first two anti-amyloid treatments receive accelerated approval, first Biogen’s molecule and then the Eisai molecule.

We have more data sets coming. We’ll have another accelerated approval decision coming from the FDA around Eli Lilly’s molecule in the space. And then we’ll see, we’ve already seen Phase 3 data sets at scientific conferences, but now we’ll see the review of that from the FDA and then reimbursement decisions coming from CMS and others. So, we’re excited to see how this space continues to evolve, and there are a lot of inflexion points around that.

Jim Joyce

CEO and co-founder, Health Beacon

For me, the two R&D trends that we want to try to figure out are this GLP-1 market, so this whole market where a broader scale of consumers that suffer from weight and underlying metabolic issues are now being introduced to this really exciting new class of medications, GLP-1s, and we don’t know how that’s going to impact. And the other one is what’s happening in the cancer space around mRNA vaccinations and targeting. I think we’re going to see continued trends there.

Joe Spinelli

SVP product and strategy, AccessDx Laboratory

Generally speaking, pharma programmes increasingly do not live in a silo. It’s more about understanding underlying disease conditions, looking through risk analysis, and then helping to effectively manage and improve insights for how these things can be delivered effectively.

That last mile concept of how therapies can actually be effectively used and utilised is something that’s really important and continues to be a big opportunity for both proactive identification for where a therapy could be beneficial, but also to see it through to understanding all the other extenuating circumstances.

Lisa Yañez

Chief operating officer, Aerami Therapeutics

What I’m seeing in particular, given the nature of what’s happening right now in the markets, I think people are doubling down on what they know. From an R&D perspective, we’ve probably taken less of a risk in terms of the opportunity that we see with this [Aerami] programme. We’re probably going to hold off a little on some of our earlier potential platform opportunities to make sure that we get it right.

It really forces you to prioritise and right-size, which is good. It’s good for shareholders, and it’s good for patients. I hope that the markets get better and that, looking forward, we can be a little nimbler and a little more provocative and things like that. But as a realist, I think it’s a little different than the last 10-15 years that I’ve been working in clinical development within rare disease. It’s basically more choiceful.

James D. Lechleiter

Co-founder and chief scientific advisor, Astrocyte

I actually think neuroscience, in general, is an area where no one’s found any good treatment options. Opportunities in that space, neurodegenerative disease, stroke, trauma, that’s actually a really incredible opportunity because there’s been so little that’s been successful. That’s a big trend.

Now, how that’s going to be done – that’s another question. We think the trend is going to be taking a second look at this if you haven’t been. If you’ve had it on the back burner, let’s take a look at the neuroscience space and see whether or not there’s something we can actually work with there.

Ravi Anand

Chief medical officer, Newron

With the seriousness of the health crises, I think we’ll see many trends with regulators, academia, industry, citizens, and disease awareness groups, all collaborating very closely and helping expedite the procedures by which new treatments can be developed marketed.

For instance, I think you see a great collaboration in the ELS space. You see it in multiple sclerosis. Groups are being formed, and new treatments are being evaluated much more rapidly than ever before. So, I think we’re not going to make a fundamental breakthrough in 2023 in the kind of medication, but more in the way we cooperate with each other.

Eloise McLennan is the editor for pharmaphorum’s Deep Dive magazine. She has been a journalist and editor in the healthcare field for more than five years and has worked at several leading publications in the UK.

Jonah Comstock is a veteran health tech and digital health reporter. In addition to covering the industry for nearly a decade through articles and podcasts, he is also an oft-seen face at digital health events and on digital health Twitter.

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