Andres Sirulnik, SVP, Hematology Clinical Development Unit Head, Regeneron
The biggest bottleneck slowing progress in drug development is the competition for, and successful recruitment of, patients for participation in clinical trials, while also ensuring participants reflect real-world populations.
To fix this critical issue, our approach is about removing unnecessary burden – not lowering standards. We work to optimize clinical efficiencies and reduce the burden of participation. This means designing trials that balance scientific rigour with practical considerations so that high-quality science can move forward more efficiently and responsibly. The future of clinical trials is one in which participation is easier, protocols are streamlined, and studies are brought to patients rather than patients to studies.
Daniel Vitt, Chief Executive Officer, Immunic Therapeutics
Rather than a single bottleneck, drug development faces increasing complexity. Clinical trials are naturally becoming more complex and resource-intensive. Patient recruitment, in particular, has become increasingly difficult as inclusion criteria grow more specific and studies expand globally. At the same time, regulatory and legal requirements continue to evolve, adding additional layers of cost and time. While these safeguards are important, they also contribute to longer development timelines and, ultimately, higher costs and drug prices.
To address this, we need to find ways to make clinical development more efficient without compromising scientific rigor or patient safety. That includes smarter trial design, better use of data and closer alignment between regulators and developers to streamline the path from innovation to patients.
Ali Pashazadeh, CEO, Treehill Partners
Drug development experience at the decision-making level. The complexity of bringing a drug to market has increased enormously, and the competitive landscape – especially from South-East Asia – has intensified exponentially. But too many boards and C-suites are still operating as though what worked five-to-ten years ago still works today. It does not. You cannot hand a program to a CRO and expect that everything will be taken care of strategically. The CRO will execute your study. They will not tell you it is the wrong study. The competition for capital is more fierce than it has ever been, and the investors who are deploying it can now compare your program against a Chinese company that achieved proof of concept for a fraction of the cost and in a fraction of the time. Boards that do not understand this are the single biggest hindrance to their own companies getting funded.
Renee Aguiar-Lucander, CEO, Hansa Biopharma
I believe that we need to get better at running clinical trials in a timely manner. This means looking further afield for patients, improving oversight of contract research organizations, and complementing that with internal resources where possible. I also think that there is quite a lot of improvement which could be made on the regulatory side. There has been long discussion about making it easier to bring drugs to market – however, in reality, there is little advancement. Conservatism, preference for written responses and rules driven decision-making still dominate. With fewer – and arguably more junior – resources at the FDA, this is concerning. How we address this, I am not sure.
Piet Wigerinck, Chief Scientific Advisor, Fibrocor Therapeutics
The biggest bottleneck is accurately measuring target or pathway engagement in the tissues of interest. Too often, we rely on plasma-based assays that only become informative once the biological problem has already been solved. They are useful for demonstrating success but rarely help teams solve the underlying challenge.
A sensitive and versatile technology capable of measuring biomarker changes directly in tissues would dramatically accelerate translational research, which underpins almost every breakthrough medical innovation.
Rab Prinjha, Chief Research and Development Officer, Curve Therapeutics
At the preclinical stage, one of the biggest bottlenecks I see is translating potent, cell active molecules into demonstrated in vivo efficacy. Optimising for bioavailability and distribution against both human and rodent systems can present many challenges for novel target and modalities. Combined with our reliance on translational models, which have their acknowledged limitations but still define so many investors' expectations of fundable evidence, this slows the industry down. Sadly, I have yet to find a solution.
Bari Kowal, Senior Vice President, Development Operations and Portfolio Management, Regeneron
One of the biggest bottlenecks is that many of the workflows supporting clinical development have not kept pace with the complexity and scale of modern clinical trials. Science has advanced faster than infrastructure to support it. To fix this, the industry needs to fundamentally rethink how trials are designed and executed with greater focus on operational feasibility, participant experience, and site realities. That means reducing unnecessary complexity and modernizing the underlying processes rather than layering new tools onto outdated ways of working.
Jonny Wray, Chief Technology Officer, Nucleome Therapeutics
The biggest barrier to progress in drug discovery is the complexity of human biology. This complexity is a major reason why progress has been slow in developing treatments for complex, polygenic diseases and why clinical trial failure rates remain high.
The key to fixing these issues is the identification of the causal drivers of human disease and using those drivers as the basis of target selection and drug discovery. Starting a drug discovery programme from the solid foundation of causal human biology, as opposed to a non-predictive proxy, leads to drugs with a much higher likelihood of clinical success.
Genetic variation can be viewed as a natural perturbation study in humans and provides an ideal base to unlock causal human biology – provided the right tools are available to decode the functional consequences of those genetic changes. Nucleome has built such a toolset combining advanced experimental functional genomics techniques with AI/ML approaches to elucidate causal biology signposted by human genetics. Application of the technology is building a pipeline of first-in-class drugs spearheaded by NTP464, our lead program, promoting inflammation resolution in a broad range of inflammatory diseases.
Anders Nykjaer, Chief Scientific Officer, Vesper Bio
The biggest bottleneck remains translational predictability: our ability to determine early whether a therapeutic hypothesis will work in humans. This is particularly true for neurodegenerative diseases. We still rely too heavily on model systems that do not adequately capture human disease biology. The solution is not a single technology, but a combination of better human-relevant models, earlier biomarker integration, richer longitudinal datasets, and tighter feedback loops between preclinical and clinical research. We also need stronger incentives for data sharing – especially around failed programs – because the industry collectively learns too slowly from negative results.
Ali Tavassoli, Professor of Chemical Biology, University of Southampton; and Former Chief Scientific Officer and Co-founder, Curve Therapeutics
The mismatch between scientific timelines and funding cycles. Drug discovery operates on a decade-plus timescale; venture capital typically operates on a three-to-five-year horizon to the next financing event. That tension shapes everything from which programs get started, which get killed, how much risk a company can credibly carry, and how honest leadership can be about timelines. A number of the companies bringing genuinely game-changing medicines to market this year have been going for ten years or more, and that is the reality of what it takes. Genuinely transformative science rarely fits neatly into the milestones the next round demands, and good programs get reshaped or abandoned not because the biology failed but because the cycle did.
The fix is a more diverse capital stack for biotech. Traditional VC will and should remain central, but we need more patient capital alongside it; sovereign wealth, strategic pharma investment, evergreen funds, and government-backed translational funding that can carry programs through the valleys of death between scientific milestones and commercial inflection points. The UK and Europe in particular have made progress here but still lag the US in depth of capital and appetite for long-duration risk. Closing that gap is at least as important as any scientific advance, because without it, the science never gets the runway it needs.
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