Patient science: patients research cystic fibrosis

In this interview with MEDICA-tradefair.com, Dr. Nils B. Heyen talks about patient science, explains how it is applied in a very open project and describes what it could offer patients compared to professional science.

Dr. Heyen, what is patient science?

Dr. Nils B. Heyen: Patient science is based on the established concept of citizen science, that being scientific research that involves citizens. For our patient format, we have chosen a special group of citizens we want to actively involve in research.

You recently launched a research project on patient science that is also led by you. What is the objective of this project?

Heyen: First, we want to study a key problem pertaining to the everyday lives of cystic fibrosis patients and their family members. The goal here is to help improve their daily lives and manage the disease. This is aimed at the group of patients that actively participates in this research endeavor.

Secondly, we want to identify the potential and limitations of patient science to broaden the methodology spectrum of citizen science. Maybe it lends itself to an increased use in the health sector. Having said that, this endeavor might also reveal obstacles that make this particularly difficult.

What advantage could this have for medicine?

Heyen: At the moment, citizen science garners increased international attention in terms of what it might bring to the table for the research sector. This varies from specialty to specialty. There is little experience in health research when it comes to involving patients in this manner. Needless to say, there are clinical trials but patients are not nearly as actively included as is the case in the patient science format.

One of our objectives with this project is to explore the potential of this format. For example, you could use it to address concerns where professional research is not aided by public funding or industry support. What’s more, unlike professional research, the active participation of patients could give us a different perspective and thus deal with questions that are especially useful in managing the daily activities of patients.

What made you choose the subject of rare diseases and cystic fibrosis?

Heyen: Rare disease research is often more difficult due to medical and even economic reasons because there is a limited number of patients and not much industry interest. Hence the potential to meaningfully complement professional research is especially great in this instance. Having said that, we need a field where we can recruit patients by using established structures. There are powerful self-management structures set up in Germany that we can utilize to elicit participation and communication.

How do you plan to structure your project?

Heyen: We have chosen a very broad participation approach, which is why we also include the patients in determining research topics. First, we have to recruit patients who join us in defining these research problems and the actual research design. This is likely followed by an empirical study of the predetermined research problem.

We subsequently have to validate the findings before we publish them – this is where we also include external experts. All this is aimed at translating the findings to the group of patients that is meant to benefit from all this in everyday life. The other target audience is the citizen science community, which would learn what we found out by applying this format.

What channels do you plan to use to include patients?

Heyen: The University Hospital Frankfurt is one of our key partners. It features the Frankfurt Reference Center for Rare Diseases where patients are being recruited, especially to set up the first steps. In addition, the German Cystic Fibrosis Association (Bundesverband Mukoviszidose e. V.) is also involved in the project, allowing us to access a broad range of patients. This might become necessary if we need to conduct a survey during the empirical phase.

What are your hopes after you complete the study in December 2019?

Heyen: In the best-case scenario, the patients who are involved in the research will discover things that are truly helpful in managing the lives of all cystic fibrosis patients. That would be amazing, especially since professional research is often unable to address this aspect. Having said that, this might also be something that is more difficult for us to achieve. Since we have only just begun, at this point it is not clear yet, what our research problem will be and whether it will yield something that can truly help patients.

But in any case, we will find out whether and to what extent the patient science format is suited for specific purposes. It would be great to ultimately discover aspects that would otherwise get lost in general academic activities. That being said, we might also realize that we encounter obstacles that remain general to where they will render patient science a very specific and unique format that is only used in rare cases.