Why transparency drives science forward

Science - including pharmaceutical science - advances step by step, building on a trial & error principle. Researchers set hypotheses and challenge them in experiments. Each experiment has to be carefully designed to be able to address the challenges, and at the same time to minimise the risk of a flawed conclusion.

Let’s take an example. One hypothesis could be that a new medicine under development is providing a good outcome for patients and that the side effects associated with its use are rare. In this case, the experiment is a clinical trial. It must be carefully planned and conducted to ensure that the measured outcomes, known as endpoints, are relevant from both a clinical and a patient perspective, and that the risk of flaws and systematic distortion, the so-called bias, is minimal. The experiment needs to be conducted according to a preplanned protocol - which will become the ‘cookbook’ to be used by other researchers should they wish to redo the experiment.

Life is unpredictable. And science is part of life. Thus, we expect that sometimes the outcome of some well-planned and well-conducted experiments fail to support the hypothesis the researchers were testing. But this is what drives science forward! Because such ‘negative’ experiments will lead to adjusted hypotheses and new research questions which can then be tested in new experiments.

No scientific area will prosper and become fruitful in isolation. Therefore, researchers share their hypotheses, research questions, and experimental results at conferences and in scientific journals. This transparency is a crucial part of good scientific practice. It enables other researchers to redo experiments in the exact same way to confirm that the findings – either positive or negative - were indeed not by chance. It saves researchers spending resources on experiments that have been shown to fail. Transparency also enables other interested people to scrutinise the results and put them into the right context.

We at the European Medicines Agency are aiming to enable access to as much information as possible without compromising personal and commercially confidential data. This allows patients, healthcare professionals and other stakeholders to scrutinise and better understand why – and on what basis - we decided to approve - or not approve - a new medicine.

This is why EMA was the first regulatory authority in the world to give open access to clinical reports for new human medicines authorised in the EU. Clinical data publication was halted as part of EMA’s business continuity plan during Brexit and COVID-19. But in the pandemic, EMA continued to provide broad online access to clinical data supporting the marketing authorisations and safety monitoring of COVID-19 medicines and vaccines. We are very happy that Health Canada has joined these efforts.

EMA has resumed the publication of clinical data for all medicines with a new active substance evaluated by our human medicines committee, the CHMP, from September 2023 onwards, regardless of the CHMP’s recommendation being positive, negative or concerning a withdrawal of application.

Going one step further, we are now giving access to protocols of all approved clinical trials and their results via the EU Clinical Trial Information System.

Being unapologetically committed to transparency day by day means being committed to advancing science for the benefit of our health.

This article is authored by Steffen Thirstrup , EMA's Chief Medical Officer