Written by Desiree Beutelspacher, MBA
In ophthalmology, clinical trials that come in on budget but are also scientifically rigorous are often considered mutually exclusive. Twenty-five years of drug development experience has shown me that this is not the case. The key lies in thoughtful study design — one that prioritizes the scientific questions that truly matter for the stage of development, without layering in complexity that adds cost but not clarity. When protocol decisions are made with both the data package and the overall program budget in mind from the outset, rigor and efficiency stop being competing priorities and start reinforcing each other. The most successful trials I have been involved with were not the most elaborate ones; they were the ones where every design element earned its place.
Endpoint Selection
Endpoint selection is arguably where the greatest leverage exists in balancing scientific ambition with budget discipline. Primary endpoints should be chosen with the regulatory finish line clearly in view — not simply because they are established convention, but because they will compellingly tell the story of efficacy and safety that regulators and clinicians need to see. Secondary endpoints deserve particularly careful deliberation as each addition carries both a cost and a risk. Underpowered secondaries can mask a meaningful efficacy signal at a critical decision point. When secondary endpoints are designed to validate an emerging biomarker, the stakes are even higher. Done well, this can differentiate a product and open doors to future indications; done poorly, it generates data that is neither definitive enough to act on nor easy to set aside. The key is not in selecting fewer endpoints, but in selecting the right ones with a clear-eyed view of what each is powered to show.
Some Budget Decisions Create the Most Expensive Problems
Some of the most expensive problems in clinical development are created by decisions that appear to save money at the outset. Clinical operations is where this dynamic plays out most visibly. Site selection is a prime example. Fewer well-chosen sites will almost always outperform a sprawling network of sites with limited trial experience. Experienced, high-performing sites generate fewer protocol deviations, maintain better patient compliance, and produce cleaner data, all of which reduce costly downstream remediation. Enrollment assumptions deserve equal scrutiny. Overly optimistic timelines are one of the most reliable drivers of budget overruns, which is why building in realistic projections from the start is crucial. The same logic applies to vendor selection. High-quality CROs, central labs, and reading centers bring expertise that prevents problems rather than simply responding to them. In clinical research, the places where budgets are cut most aggressively are often the places where the greatest risk accumulates.
Successful trials treat operational realism and scientific rigor in the same conversation, held early in development, with the right operational and scientific expertise at the table. This is precisely the role that iuvo’s Clinical Advisory Board was built to play. Comprised of experienced ophthalmic drug developers, clinicians, and trial operationalists, the board works directly with sponsors at the design stage — stress-testing endpoint selection, grounding enrollment assumptions in operational reality, and ensuring that every protocol decision reflects both scientific intent and execution feasibility. iuvo’s exclusive focus on ophthalmology means that this expertise is deep, current, and purpose-built for the unique demands of this therapeutic area.
About the Author
Desiree Beutelspacher is an accomplished biotech executive with over 25 years of strategic drug development experience, specializing in ophthalmology. As a consultant she works with small and emerging companies to grow their ophthalmology pipeline. Most recently, she served as SVP of Development Operations at Eyebiotech. Prior to that, Desiree served in executive Clinical and Program Lead roles at Iveric, Kodiak Sciences, and Ophthotech. Desiree received a B.S. in biology from the University of California, Los Angeles and an MBA from the University of California, Irvine.