Clinical trials are time-consuming, costly and often do not reach their intended goals – but when a trial does have a positive result it can be life-changing for patients. As Head of Late Stage Clinical Development for Cardiometabolism, Professor Martina Brueckmann knows all about that, because she and her team design them.
She’s pragmatic about her role. “I just help my colleagues overcome problems in clinical development,” she explains. “We organize a Patient Advisory Board to assess patient needs, design the trials, write the study protocols and ask the regulatory authorities for approval. Then we run the studies – sometimes we have to modify them as we go. After that, we do the main data readout, submit the results and ask for regulatory approval.”
It doesn’t sound like “just” anything. It takes time, patience and a clear vision of what needs to be done. “My core role is very technical – I basically get the trial run and approved. But I’m not just driven by the daily work I do. It’s the vision behind it: that drive to improve patients’ lives. That is my true motivator – to get the drug to the patient.”
“I’m not just driven by the daily work I do. It’s the vision behind it – that drive to improve patients’ lives”
The scale of the endeavour
The trials Martina runs span continents and can take place in 20-plus countries at one time. That takes a lot of organisation, so making sure she has enough team members and investigators working on a single project is important. “You have to staff your trial accordingly,” she says. “First, you have to calculate how many patients, investigators and study centres you need. That depends on factors like how long you want the trial to run and how effective you expect your medication to be.”
“First, you have to calculate how many patients, investigators and study centres you need.”
“We also need to talk to our statisticians where we can find the number of patients we need. It´s important to make them representative of the global dispersion of any illness. You don't want to recruit in just one country because then everyone will say, ‘Oh, yes it worked in Germany. But what about Japan? What about India?’ You have to spread yourself around.”
Communication is key
When a trial has a positive result, a new set of processes starts to take the medication to market. That means among other things, obtain approval by regulatory authorities. For this to be achieved all data need to be compiled in a formalised way for the authorities to be reviewed and evaluated for their approval decision.
“After the medication is approved in its intended indication, we need to think about communication. To enable patients to benefit from a new treatment option the treating physicians need to be made aware of this treatment and the underlying evidence.
So, we start by training our internal people in Medical Affairs to enable them to inform the physicians in a clear, concise and compliant way.”
Finding the right words
But it’s not just about communicating to the doctor. Martina knows that it is also important for the patient to understand the implications of their disease and treatments in order to be committed to the treatment plan.
“If a doctor talks to a patient using medical jargon, it might sound good, but it won’t ultimately mean anything to them. Therefore, we are also developing disease information in lay language for patients which can also help the physicians to discuss disease and optimal treatment with their patients.“