A qualitative study on the facilitators and barriers to adopting the N-of-1 trial methodology as part of clinical practice: potential versus implementation challenges

ABSTRACT

Purpose

To investigate opinions among healthcare stakeholders whether implementation of the N-of-1 trial approach in clinical practice is a feasible way to optimize evidence-based treatment results for unique patients.

Methods

We interviewed clinicians, researchers, and a patient advocate (n = 13) with an interest in or experience with N-of-1 trials on the following topics: experience with N-of-1, measurement, validity and reliability, informally gathered data usability, and influence on physician-patient relationship. Interviews were analysed using qualitative, thematic analysis.

Results

The N-of-1 approach has the potential to shift the current healthcare system towards embracing personalized medicine. However, its application in clinical practice carries significant challenges in terms of logistics, time investment and acceptability. New skills will be required from patients and healthcare providers, which may alter the patient-physician relationship. The rise of consumer technology enabling self-measurement may leverage the uptake of N-of-1 approaches in clinical practice.

Conclusions

There is a strong belief that the N-of-1 approach has the potential to play a prominent role in transitioning the current healthcare system towards embracing personalized medicine. However, there are many barriers deeply ingrained in our healthcare system that hamper the uptake of the N-of-1 approach, making it momentarily only interesting for research purposes.

Highlights

Key findings

• The potential merits of adopting N-of-1 trials into clinical practice (in terms of efficacy and participation) was acknowledged by all participants.

• The trade-off between methodological rigidity and practical application for the patient was mentioned by clinicians as an important barrier for the use of N-of-1 trials in clinical practice.

• There appears to be substantial dissensus on the usefulness of “informal/pragmatic” N-of-1 trials in clinical practice; clinicians appear the strongest advocates for strict methodological rigour.

What this adds to what is known

• Previous research suggests that lack of knowledge by researchers, clinicians, and patients on the topic of N-of-1, operational complexity, and costs are primary barriers for adoption of N-of-1 trials in clinical practice.

• Our work confirms the abovementioned barriers and adds to this list: the current design of the healthcare system and the lack of consensus on methodological requirements.

• The Quantified Self movement as well as the advances in the wearable technology were mentioned by (patient)researchers as facilitators for the adoption of N-of-1 methodologies in clinical practice.

What is the implication, what should change now

• Education on N-of-1 trials need to be included in the medical (and thus not only the biomedical sciences) curriculum.

• The N-of-1 approach might help promote shared decision making in which patient can lead using their own data.

• Best practices of N-of-1 adoption in clinical practice need to be identified and used as examples to further inform communication between medical stakeholders and policymakers.

KEYWORDS:

• N-of-1 trials
• individual point of care studies
• single case designs
• single subject research
• personalized healthcare
• personal science

Disclosure statement

No potential conflict of interest was reported by the author(s).

Dataset considerations

In line with ethical considerations and privacy concerns, the qualitative data presented in this study, due to its sensitive nature and inability to be fully anonymized, cannot be made publicly available. However, we are committed to promoting transparency and facilitating scholarly discussion. Researchers interested in discussing strategies to share or access our data are encouraged to contact us at [email protected]. We are willing to engage in conversations regarding potential methodologies, anonymization techniques, and collaborative approaches to ensure data integrity while respecting participant confidentiality.

Additional information

Funding

This work was supported by Regieorgaan SIA/NWO under Grant [number KIEM.LSH.03.014].

Notes on contributors

Ilona Wilmont

Dr. Ilona Wilmont, PhD is a researcher at HAN University of Applied Sciences and Radboud University Nijmegen, with a background in Information Sciences. Her specialty is qualitative research, with a focus on how personalized healthcare and citizen science methodologies can help patients support their quality of life. She collaborates with Stichting Je Leefstijl Als Medicijn, one of the largest Dutch patient communities, as well as with an outpatient epilepsy clinic, on empowering epilepsy patients to use lifestyle interventions and self-measurement to improve quality of life. She also works with Leiden University Medical Centre and Erasmus University Medical Centre on lifestyle interventions for migraine patients. Furthermore, Dr. Wilmont teaches Research Methods and Academic Writing at Radboud University Nijmegen.

Mark Loeffen

Mark Loeffen, PT is an experienced physical therapist and manual therapist specializing in treating long-term back, neck, and shoulder pain. Apart from his work with clients he participates in scientific research projects to improve interdisciplinary collaboration within physical therapy, as well as to improve outcomes for patients by using personalized approaches.

Thomas Hoogeboom

Dr. Thomas Hoogeboom, PT, MSc, PhD is trained physical therapist and human movement scientist. He currently holds an assistant professor position at IQ healthcare department of the Radboud university medical center. Dr. Hoogeboom specializes in implementing and evaluating personalized interventions, and in the methodologies to facilitate this. He has participated in the development of nationwide medical guidelines, and collaborates with several other universities, including the University of Montana and the University of Utah, on the topic of shared decision making between patients and healthcare providers and how clinicians can integrate physical activity in the lives of medically fragile patient groups. Dr. Hoogeboom has a particular interest in meta-research and is currently setting up the Meta-Phit group together with researchers from the University of Technology Sydney.