N-of-1 trials and Single-Case Designs (also known as personalised clinical studies) are uniquely suited to identify individual responses to treatments. N-of-1 trials and Single-Case Designs are personalised clinical trials in which an individual is their own control. They may use multiple cycles, randomisation and blinding, depending on the clinical context. While traditional clinical trials provide valuable data on general trends and average responses, they often fail to capture the nuanced variations among individuals. This is where personalised clinical trials have advantages, offering insights into how treatments affect each person differently.
The Unique Role of N-of-1 Trials in Complementary and Alternative Medicine
Complementary and alternative medicine, a diverse spectrum of practices from acupuncture to herbal remedies that focuses on individual care, often accompanies conventional treatments. This field has broad therapeutic potential but also poses challenges in building a solid evidence base. These challenges include the mixtures of compounds, unknown or variable half-lives and the highly individualised approach required – the dose needs to be titrated individually. The individualised approaches of complementary medicine are well-suited to evaluation in personalised clinical studies such as N-of-1 trials. This is because, while traditional clinical trials offer invaluable data, they often overlook variations among individuals.
N-of-1 Trials in Action
Examples of complementary medicines that have been tested using N-of-1 trials include amino acids (e.g., L-arginine) [1], plants and herbal materials (eg spirulina) [2], lipids (e.g., linoleic/linolenic acid, ultra-micronized palmitoylethanolamide) [3-5] and minerals (e.g., magnesium) [6].
At N-of-1 Hub, we recognise the transformative power of personalised approaches in healthcare. Our ethos resonates deeply with the principles underlying personalised clinical trials, which prioritise the individualised response of each person. By tailoring personalised clinical trials to specific needs and characteristics of the individual —considering genetic predispositions, factors such as outcomes of importance to the individual, and more — we not only enhance the relevance of trial findings for individuals but also empower them to make informed decisions about their health.
Complementary Medicine and SCEDs
Moreover, N-of-1 trials and Single-Case Designs in complementary medicine provide a unique opportunity to track treatment dynamics over time. Longitudinal assessment can uncover patterns of response over time, identify potential side effects, and optimise treatment regimens, thereby fostering a deeper understanding of how complementary medicines interact with individual biology and environment.
These trials also hold promise for bridging the gap between conventional and complementary medicine. Results of a series of N-of-1 trials or Single-Case Designs can be pooled to provide group results with equivalent rigor to, but with far fewer participants needed, than standard large clinical trials. By focusing on individual responses, N-of-1 trials and Single Case Designs provide a robust framework for evaluating the efficacy of complementary medicines. This approach not only helps in fine-tuning treatments for better patient outcomes but also can contribute significantly to the evidence base of complementary therapies. As personalised medicine continues to evolve, these trials will play an increasingly vital role in ensuring that complementary medicine treatments are both effective and tailored to individual needs.
N-of-1 Hub is a consulting company that specialises in designing, conducting and analysing personalised clinical studies using single-case designs. N-of-1 Hub provides consulting and collaborative services to companies, clinicians, researchers, and healthcare consumers who wish to conduct personalised clinical studies using single-case designs. N-of-1 Hub also offers data management and analysis services and customised workshops and courses. Additionally, our training programs are tailored to equip researchers and clinicians with the skills needed to excel in personalised clinical studies.
For more information on how N-of-1 trials can revolutionise your clinical research and improve patient outcomes, we invite you to contact us for a free consultation. Don’t forget to follow us on LinkedIn for the latest updates and insights in the field of personalised clinical studies.
References
Baicus C, Baicus A. Spirulina did not ameliorate idiopathic chronic fatigue in four N-of-1 randomized controlled trials. Phytother Res. 2007 Jun;21(6):570-3.
Hackett A, Gillard J, Wilcken B. n of 1 trial for an ornithine transcarbamylase deficiency carrier. Mol Genet Metab. 2008 Jun;94(2):157-61.
Germini F, Coerezza A, Andreinetti L, Nobili A, Rossi PD, Mari D, Guyatt G, Marcucci M. N-of-1 Randomized Trials of Ultra-Micronized Palmitoylethanolamide in Older Patients with Chronic Pain. Drugs Aging. 2017 Dec;34(12):941-952.
Lewis SA, Shetty S, Gamble S, Heim J, Zhao N, Stitt G, Pankratz M, Mangum T, Marku I, Rosenberg RB, Wilfong AA, Fahey MC, Kim S, Myers SJ, Appavu B, Kruer MC. Intrathecal magnesium delivery for Mg++-insensitive NMDA receptor activity due to GRIN1 mutation. Orphanet J Rare Dis. 2023 Aug 3;18(1):225.
Marcucci M, Germini F, Coerezza A, Andreinetti L, Bellintani L, Nobili A, Rossi PD, Mari D. Efficacy of ultra-micronized palmitoylethanolamide (um-PEA) in geriatric patients with chronic pain: study protocol for a series of N-of-1 randomized trials. Trials. 2016 Jul 29;17:369.
Santos C, Weaver DF. Topically applied linoleic/linolenic acid for chronic migraine. J Clin Neurosci. 2018 Dec;58:200-201.