Student projects in the area of N-of-1 trials and digital health

This page describes multiple student projects that are currently available. The following projects use a range of different methodologies including quantitative methods, qualitative methods, and evidence synthesis.

Project 1: Making it Personal: Understanding chronic symptoms using scientific insights from N-of-1 studies

Fields:

Neuroscience
Digital health
Data analytics

Location: General Practice Clinical Unit (Herston)

Type of student:

Type of work:

Clinical work
Literature review
Qualitative methods
Statistical analysis

Brief synopsis:

This project involves applying digital N-of-1 methods to understand chronic symptoms in a range of chronic diseases. Two patient populations are currently being recruited for this project.

Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS): Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) is characterised by the cardinal symptom of post-exertional malaise (PEM). PEM is experienced as marked, rapid physical and/or cognitive fatigue, or exacerbation of other symptoms such as musculoskeletal pain, headaches, sleep problems and impaired cognitive function, in response to minimal exertion. Individuals with ME/CFS vary widely in regards to the severity and particular cluster of symptoms they experience. However, research in this area has typically used a ‘group-based’ research design to study ME/CFS, which pays less attention to individual differences in symptomology, severity and experience. N-of-1 methods involve the repeated, quantitative and prospective measurement of an outcome in an individual over time. N-of-1 methods can be used to study PEM at the individual level. This project will involve a series of N-of-1 observational studies to explore patterns and predictors of ME/CFS symptoms over time. Daily data about fatigue and potential predictors will be collected three times per day for 6-12 weeks using an electronic diary with integrated accelerometer. Participants will receive detailed feedback about their data, which may help patients with self-management of their condition.

Multiple Sclerosis: Fatigue is the most commonly reported, most disabling but least understood symptom experienced by patients with Multiple Sclerosis (MS). Patterns and predictors of fatigue may differ considerably from one individual to another but these differences are concealed in research using group-based designs (e.g. randomised controlled trials) that focus on average results for the group studied. Tools for assessing fatigue symptoms typically involve self-reported retrospective recall which can be subject to a number of memory biases. Furthermore, fatigue assessment is typically conducted on only one occasion, resulting in a limited understanding about fluctuations over time. N-of-1 methods involve the repeated, quantitative and prospective measurement of an outcome in an individual over time. N-of-1 methods can be used to identify patterns and predictors of fatigue that are unique to the individual. This project will involve a series of N-of-1 observational studies to explore fluctuations in fatigue and potential predictors (e.g. activity, sleep, mood) over time in MS. Daily data about fatigue and potential predictors will be collected three times per day for 6-12 weeks using an electronic diary with integrated accelerometer. Participants will receive detailed feedback about their data.

Learning objectives for students: Students will learn about an innovative research method. N-of-1 trials and single-case designs play a key role in the movement towards personalised medicine, patient-centred healthcare, and shared decision-making. Students will also gain experience and skills in conducting research involving patients with chronic disease who may have a high symptom burden.

Study website: Making it Personal: Understanding chronic symptoms using scientific insights from N-of-1 studies

Project 2. Exploring stakeholders' perspectives about using N-of-1 trials and single-case designs in healthcare

Fields:

General practice
Health services
Internal medicine
Pharmacology
Primary care

Location: General Practice Clinical Unit (Herston)

Type of student:

Type of work:

Literature review
Qualitative methods

Brief synopsis:

Background: N‐of‐1 trials are randomised controlled trials of treatment versus a placebo (or another treatment) within an individual patient to determine how an individual patient responds to treatment. N-of-1 trials provide the highest level of evidence for guiding treatment decisions for individual patients. N-of-1 trials are one type of single-case design. Despite the potential value of using N-of-1 trials and other single-case designs in healthcare, there has been limited uptake to date.

Aim: This study aims to explore stakeholders' perspectives about using N-of-1 trials and single-case designs in healthcare. Stakeholders include health care professionals (e.g. general practitioners, psychologists, physiotherapists), patients, trialists/methodologists, biostatisticians, health care funders, Institutional Ethics Review Board members, regulators and journal editors.

Methods: One-to-one, semi-structured interviews are conducted with people in each stakeholder group. The interview schedule is based on the Theoretical Domains Framework (TDF; Michie et al., 2005). The TDF provides a broad and inclusive approach to understanding theory-based determinants of clinical and health-related behaviour. The TDF was developed by an expert consensus group, which reviewed 33 behavioural theories and their associated 128 theoretical constructs, identifying 12 theoretical domains based on commonalities between constructs. Therefore, the TDF broadly covers all scientific explanations of behaviour based on current theorising and has been used widely to understand the perceptions and behaviours of health professionals and patients. Interviews will be audio-recorded, transcribed and analysed using the framework approach.

Implications: The syntehsised data about stakeholders’ perspectives will be used to develop a range of theory-based strategies designed to increase the uptake of N-of-1 trials and single-case designs in healthcare.

Learning objectives for students: Students will learn about an innovative research method. N-of-1 trials and single-case designs play a key role in the movement towards personalised medicine, patient-centred healthcare, and shared decision-making. Students will also gain experience and skills in relation to conducting qualitative research with different population groups.

Project 3: The International Collaborative Network for N-of-1 Trials and Single-Case Designs

Field:

Data analytics

Location: General Practice Clinical Unit (Herston)

Type of student:

Type of work:

Literature review
Statistical analysis

Brief synopsis:

The International Collaborative Network for N-of-1 Trials and Single-Case Designs (ICN) aims to advance the science of individualised research. The ICN has over 650 clinician, academic and researcher members from more than 42 countries around the world who are using N-of-1 trials and single-case designs.

Learning objectives for students: Students will learn about the key activities involved in running an international research network. Students will gain experience and skills in a range of activities including developing strategic research priorities, writing short scientific blogs on topical issues, summarising recent scientific papers, and managing a website and social media.

Website: The International Collaborative Network for N-of-1 Trials and Single-Case Designs

Project 4: Applying N-of-1 trials and single-case designs to personal science in the health domain: A systematic review

Fields:

Digital health

Location: General Practice Clinical Unit (Herston)

Type of student:

Type of work:

Literature review

Brief synopsis:

Personal science is defined as the practice of using empirical methods to explore personal questions. Personal science involves five key activities - questioning, designing, observing, reasoning and discovering - that lead an individual to answers to health questions in their daily life. N-of-1 trials are rigorous methods that have a long history of use in medicine to determine how an individual patients responds to a treatment by using them as their own control. Although there are similarities between the field of personal science and N-of-1 science, they are not the same. Applying N-of-1 trial designs to the field of personal science could improve the rigour of practices. This study aims to review the literature to identify the extent to N-of-1 designs have been applied to self-tracking and self-experimentation studies.

Learning objectives for students: Students will learn about how to plan and conduct a systematic review of the literature. Students will gain experience and skills in relation to developing a comprehensive search strategy, searching electronic databases, screening search results against eligiblity criteria, extracting data from eligible studies, and synthesising and interpreting data across studies to draw conclusions.

Please contact the supervisor if you are interested in any of the above projects. Suggestions for new projects in the area of N-of-1 trials and/or digital health are welcome.

Supervisor

Dr Suzanne McDonald

General Practice Clinical Unit,

The University of Queensland