Behind the scenes: Study design provides the roadmap for developing new substances

The study design usually provides for double blinding: i.e. neither the physicians nor the patients know whether the new drug or a placebo is used.

On the long road from an active substance to an effective drug, Global Clinical Development at Bayer HealthCare, dealing with the design and conduct of studies makes sure that the right studies are being conducted. What are the goals of a certain study, what are the conditions, who can take part? The colleagues in study design find the answers to all these questions.

A lot of work has already been done once a new drug ends up in the hands of doctors and patients. It all starts with the discovery of an active substance. But how to find out how this substance really works, how effective it is for countering which types of disease, what potential side effects are and how it can be turned into an actual drug? This requires numerous studies and study design is the professional field in charge of setting up the right studies. People working in study design define the structure of a study down to the smallest detail and lay down overall conditions such as its duration, the number of subjects or patients and the criteria for participation. The overarching objective is to design the study in such a way that it provides solid, reliable data on the safety, efficacy and tolerability of the active ingredient.

Developing a study design means teamwork – and such a team consists of many different experts from clinical development, statistics and data management as well as regulatory affairs to make sure that the study complies with national legislation in various countries. After all, a study is work-intensive and costly – it needs to be set up correctly to prepare the approval process for the drug candidate.

All studies usually comprise three phases: In phase I, the active substance is tested on humans for the first time. In phase II, it is used on patients with the respective diagnosis. There is usually a control group of people who take a placebo or an already approved drug for comparison. Phase III comprises major field trials with several hundreds or thousands of patients that are designed to prove efficacy and safety of the drug. The length of a study depends on the drug. If it’s a drug for longer application or even chronic diseases, the study will also take longer. Development programs for certain Bayer HealthCare drugs, for instance, required the participation of over 75,000 patients at 1,100 locations in more than 50 countries.

Study design thus provides the roadmap for proving beyond doubt that a new drug represents a medical advance and should therefore be made available to patients. It’s an exciting field of work and a decisive element on the path towards better treatment options for doctors and patients.