A cornerstone of clinical development is looking far ahead. You can’t focus only on the next study; you also have to ensure it will properly serve what comes later in development. I’m asked whether I could design the next phase’s studies. The answer is yes—provided we also map the road beyond that. Every stage of development becomes the foundation for the next ones, and the only way to avoid wrong choices is to look far enough ahead.
All prior data and results are, of course, highly decisive—they form the up-to-date knowledge used to weigh opportunities and risks. Based on knowledge and theory, we arrive at the assumptions on which the next plan is built. The theory consists of the scientific literature related to the specific drug target, which must be continually updated through discussions with experienced experts.
The second key foundation for plans is understanding the environment—what treatments are already available for the disease in question, what others are developing for the same disease across all relevant mechanisms, and what the unmet need is for new therapies.
Based on all the information gathered, you must be able to assume that the new therapy being developed will stand out in a positive way. The clinical program has to prove that assumption while also ensuring that the product’s formulation and dosing work in practice.
What’s challenging is looking all the way to the time when your own product could reach patients. Depending on the stage, we’re easily talking about 5–10 years ahead. Forecasting requires, at a minimum, clinical and drug development understanding, interpretation of regulatory requirements, discussions with patient organizations, and competitor analyses.
In addition to the product’s target profile, it’s worth outlining the clinical development program from start to finish right at the beginning—preferably before a single study has started. At first, the outline is very incomplete and contains only assumptions. Little by little, as studies progress, assumptions turn into knowledge, and the direction of the development program may also shift accordingly in a controlled way.
The story of a new, innovative medicine begins the moment the first idea exists. The story is managed through far-reaching plans, careful data collection, and analysis of results. And everything is examined in relation to existing knowledge and assumptions about the future.
Only after the large Phase III clinical trials do most assumptions turn into knowledge, and the therapy’s benefit–risk profile is sufficiently verified to obtain marketing authorization. The remaining assumptions become knowledge only once patients have used the therapy in real life—and data is actively collected from that as well.
In the beginning, assumptions are full of opportunities and risks—and our challenge is that the odds are on the side of risks materializing. Our work as drug development professionals is to build a story on the assumption that, as knowledge increases, risks will be managable and benefits are proven. If the developers’ own belief in this runs out, the progress must be stopped.
Then, hopefully, there is already another story alongside it—one we can focus on building, while creating new ones at the same time.