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Research method

Randomized Controlled Trial

A randomized controlled trial assigns people by chance to an intervention or a comparator, then measures the same outcomes in both arms. Randomisation is meant to balance known and unknown confounders at baseline so that later differences can be attributed to the assigned treatment rather than to who signed up. The result is a contrast — a difference in minutes walked, a quality score, a PHQ-9 change — whose credibility still depends on retention, blinding, and whether the outcome is a behaviour people report or an event the trial was powered to count.

Clinicians reach for an RCT when they need to know whether a programme, drug, app or training actually changes outcomes, not merely whether people who already use it look healthier. It answers 'does this assignment cause a difference over this follow-up?' Its main limitation in this library is that many trials are short, rely on self-report, and lose people unevenly, so a statistically significant contrast is not the same as a durable hard-endpoint benefit.

Evidence

What the evidence shows

Drawn from 33 studies in this library. Each finding starts with a plain-language takeaway, then the denser detail. Supports means evidence for a finding; Challenges means evidence against a stated position; Qualifies marks scope with a short note on each study’s contribution. Challenged positions are labeled — they are not findings.

  • Behaviour-change RCTs in this set often show a clear advantage while the intervention is running and a weaker or gone contrast once it stops. Insufficiently active adults randomised to a 50-day Facebook walking programme increased walking by about 155 min/week during the programme, but between-group advantages were not maintained three months after the app ended; Text4Heart raised lifestyle adherence at 3 months (AOR 2.55) without a significant 6-month difference.

    2 studies
    1. 1Facebook team walking RCT
    2. 2Do heart texts improve lifestyle adherence?

    Study comparison

    StudyRoleDesignNPopulationOutcome
    Facebook team walking RCT2015SupportsRCT50-day Facebook app + pedometer vs wait-list; teams of friendsN=110 · 51 intervention, 59 controlInsufficiently active adults organized in Facebook friend teamsSelf-reported MVPA/walking at 8 and 20 weeks
    Do heart texts improve lifestyle adherence?2015SupportsRCTText4Heart 24-week mHealth cardiac rehabilitation plus usual care vs usual care aloneN=123 · 61 intervention, 62 controlNew Zealand adults with coronary heart diseaseAdherence to recommended lifestyle behaviours at 3 and 6 months
  • Some service-delivery trials are designed as non-inferiority tests rather than superiority tests. Orthopaedic patients randomised to videoconference versus usual outpatient visits had quality-score differences whose 90% CI (0.05–0.17) stayed inside the pre-specified non-inferiority margin, with all video visits completed as planned.

    1 study
    1. 1Video orthopaedics non-inferior to clinic
  • Adjunctive digital CBT can move a validated symptom score without proving remission or long-term relapse prevention. Adults with antidepressant-refractory major depression randomised to add a smartphone CBT app scored about 2.5 PHQ-9 points lower at week 9 (SMD 0.40) and were more likely to meet response criteria; remission and side-effect differences were not significant.

    1 study
    1. 1Can a CBT phone app help stubborn depression?
  • Intention-to-treat mixed models can still leave a trial hard to interpret when dropout is large and unequal. NHS Choices users randomised to MoodGYM versus wait-list showed adjusted WEMWBS advantages of about 2.5 points at 6 weeks and 2.9 at 12 weeks, but attrition was 73.5% in the intervention arm versus 26.9% in control.

    1 study
    1. 1MoodGYM improves population wellbeing
  • Not every paper that uses randomisation is an intervention RCT. A Copenhagen Mendelian-randomisation analysis of BMI and ischaemic heart disease uses genotype as an instrumental variable for BMI rather than randomly assigning a diet or drug, so the causal claim is about genetically influenced BMI, not about a treatment protocol.

    1 study
    1. 1Does higher BMI causally raise heart disease risk?

Open questions

Tensions and limits

Some items are genuine disagreements on the same question. Others mark different assays, populations, or outcomes — limits on how far one study travels — not a forced fight between papers.

  • Scope / different questions

    Trials disagree on what a 'positive' result is allowed to be. The orthopaedic telemedicine trial treats staying inside a non-inferiority margin as success, whereas the Facebook walking, Text4Heart, Kokoro-app and MoodGYM trials are powered to show the intervention beating control on a change score. A student who reads only superiority language will misread a successful non-inferiority trial as 'no difference, so it failed.'

    3 studies
    1. 1Video orthopaedics non-inferior to clinic
    2. 2Facebook team walking RCT
    3. 3Can a CBT phone app help stubborn depression?

    Study comparison

    StudyRoleDesignNPopulationOutcome
    Video orthopaedics non-inferior to clinic2016SupportsRCTVideoconference orthopaedic consultations vs usual hospital outpatient visits; non-inferiorityN=389 · 389 patients; 559 consultations analysedOrthopaedic outpatients referred for specialist consultationSpecialist-rated consultation quality (non-inferiority)
    Facebook team walking RCT2015SupportsRCT50-day Facebook app + pedometer vs wait-list; teams of friendsN=110 · 51 intervention, 59 controlInsufficiently active adults organized in Facebook friend teamsSelf-reported MVPA/walking at 8 and 20 weeks
    Can a CBT phone app help stubborn depression?2017SupportsRCTKokoro-app smartphone CBT plus antidepressant switch vs medication change aloneN=164 · ITT; primary outcome at week 9 in 163/164 (99.4%)Adults in Japan with antidepressant-refractory major depressionPHQ-9 at week 9
  • Scope / different questions

    Unblinded patient-reported outcomes and attention effects pull in the opposite direction from hard clinical events. Phone telemonitoring improved MLHFQ in a 100-person heart-failure trial that was not powered for hospitalisation or death, while Text4Heart's lifestyle gains were self-reported and LDL differences were only borderline.

    2 studies
    1. 1Phone telemonitoring in heart failure
    2. 2Do heart texts improve lifestyle adherence?

    Study comparison

    StudyRoleDesignNPopulationOutcome
    Phone telemonitoring in heart failure2012SupportsRCTMobile telemonitoring plus usual care vs standard care for six monthsN=100Adults with heart failureMinnesota Living with Heart Failure Questionnaire (MLHFQ) quality of life
    Do heart texts improve lifestyle adherence?2015SupportsRCTText4Heart 24-week mHealth cardiac rehabilitation plus usual care vs usual care aloneN=123 · 61 intervention, 62 controlNew Zealand adults with coronary heart diseaseAdherence to recommended lifestyle behaviours at 3 and 6 months

Common misconceptions

Exam-style questions

Short-answer questions that ask you to explain or compare, not recall.

Why can a trial that finds 'no significant difference' still be a success, and which paper in this set is built that way?

When the scientific claim is that a new service is not worse than usual care by more than a pre-specified margin, staying inside that margin is the success criterion. The orthopaedic videoconference trial analysed quality scores that way: the 90% CI for the difference stayed within the non-inferiority margin and every video visit ran as planned.

Using the Facebook walking and Text4Heart trials, explain what 'the intervention worked' does and does not mean once follow-up continues after the programme ends.

Both show a behavioural advantage while the programme is on (about 155 extra walking minutes per week; lifestyle-adherence AOR 2.55 at 3 months). Neither maintains a clear between-group lifestyle contrast after the stimulus fades (three months off-app; non-significant 6-month lifestyle difference). That is evidence about a time-limited behaviour change, not about a self-sustaining habit or fewer cardiac events.

How should a student read MoodGYM's ~2.5–2.9 point WEMWBS advantage together with its attrition figures?

The mixed-model ITT estimate favours MoodGYM, but 73.5% of the intervention arm dropped out versus 26.9% of wait-list controls. Differential attrition can bias even an ITT model if missingness is related to outcome, so the point estimate is not a clean causal effect for everyone assigned.

Distinguish an intervention RCT from the Copenhagen BMI–IHD Mendelian-randomisation paper. What is randomised in each case?

In the walking, telemedicine, Text4Heart, Kokoro-app and MoodGYM papers, investigators assign a programme or usual care by chance. In the BMI paper, nature has already assigned FTO, MC4R and TMEM18 alleles that shift BMI; the analysis uses those alleles as instruments. That supports a causal reading of BMI, not a claim that a particular weight-loss programme was tested.

The studies

33 studies in this library bear on Randomized Controlled Trial, ordered by citations. The first 8 are shown.

  • SMS coaching for weight loss

    Daily tailored text/MMS coaching produced about 2 kg more weight loss than print materials over 4 months in overweight adults.

    Journal of medical Internet research · 2009 · 384 citations

  • SASA! cut partner violence in Kampala

    A Kampala cluster RCT found community mobilization (SASA!) associated with roughly 50% lower past-year physical IPV and male partner concurrency.

    BMC medicine · 2014 · 340 citations

  • Interdisciplinary frailty RCT

    A 12-month multifactorial interdisciplinary program reduced frailty prevalence and improved mobility versus usual care in frail older adults in Sydney.

    BMC medicine · 2013 · 304 citations

  • Did India’s toilet campaign improve child health?

    A village-randomized sanitation program raised latrine access and cut open defecation modestly but did not improve child diarrhea, parasites, anemia, or growth.

    PLoS medicine · 2014 · 266 citations

  • Same-day ART start improves HIV outcomes

    Rapid ART initiation raised 90-day treatment start and 10-month viral suppression versus standard multi-visit initiation.

    PLoS medicine · 2016 · 261 citations

  • Deprexis online therapy reduced depression scores

    An integrative online program (Deprexis) improved BDI depression symptoms versus delayed treatment in a large German internet RCT.

    Journal of medical Internet research · 2009 · 240 citations

  • Mediterranean diet and depression risk

    Overall Mediterranean diet assignment did not significantly reduce depression; a nuts arm suggested benefit in diabetes subgroup analyses.

    BMC medicine · 2013 · 232 citations

  • Does higher BMI causally raise heart disease risk?

    Genetic instruments for BMI suggest a causal increase in ischemic heart disease risk larger than simple observational estimates.

    PLoS medicine · 2012 · 231 citations

Show 25 more studies
  • Literacy-friendly HF self-care trial

    A literacy-sensitive outpatient heart failure education program improved knowledge and self-efficacy but did not clearly cut hospitalizations or quality-of-life scores versus usual care.

    BMC health services research · 2006 · 231 citations

  • Phone telemonitoring in heart failure

    Mobile telemonitoring improved heart-failure quality of life over six months with strong adherence.

    Journal of medical Internet research · 2012 · 228 citations

  • Tele-orthopedics costs less than hospital visits

    In a Norwegian RCT, remote orthopedic video visits cost less than hospital outpatient visits with similar QALY gains.

    Journal of medical Internet research · 2019 · 225 citations

  • Glucose Buddy app associated with improved type 1 HbA1c in a trial

    Adults with poorly controlled type 1 diabetes using Glucose Buddy plus weekly educator texts lowered HbA1c versus usual care.

    Journal of medical Internet research · 2013 · 225 citations

  • Do heart texts improve lifestyle adherence?

    A text-and-internet cardiac rehab program improved short-term lifestyle adherence after CHD, but the benefit faded by 6 months.

    Journal of medical Internet research · 2015 · 207 citations

  • Video orthopaedics non-inferior to clinic

    Remote video orthopaedic consultations met the trial’s non-inferiority margin versus standard outpatient visits.

    BMC health services research · 2016 · 194 citations

  • PrEP adherence in Partners PrEP

    In a Partners PrEP adherence substudy, median pill-count and MEMS adherence exceeded 97%, and counseling restored most low adherers to ≥80%.

    PLoS medicine · 2013 · 192 citations

  • Phone telemonitoring after heart-failure flare

    Home mobile-phone telemonitoring after acute heart-failure decompensation cut primary events and hospital days versus usual care.

    Journal of medical Internet research · 2009 · 185 citations

  • TB pillbox reminders beat SMS for doses

    In a China cluster RCT, medication-monitor reminders cut poor TB adherence 40–50%, while text messages mainly reduced loss to follow-up.

    PLoS medicine · 2015 · 168 citations

  • Digital pills cut blood pressure faster

    Patients with uncontrolled hypertension and type 2 diabetes using digital medicines had larger 4-week SBP drops than usual care.

    Journal of medical Internet research · 2017 · 154 citations

  • Doctor communication training helps hypertension care

    Training physicians in communication skills improved hypertensive patients’ health literacy, blood pressure control, adherence, and self-efficacy in an Iranian RCT.

    BMC health services research · 2020 · 151 citations

  • Does wireless BP monitoring boost patient activation’s benefits?

    In hypertensive adults, a wireless self-monitoring program strengthened links between rising patient activation and better behaviours/BP control.

    Journal of medical Internet research · 2016 · 127 citations

  • MoodGYM improves population wellbeing

    In a large UK web RCT, automated MoodGYM raised mental well-being versus wait-list control despite high intervention attrition.

    Journal of medical Internet research · 2012 · 113 citations

  • Web program increased older adults’ measured activity in a trial

    A Dutch RCT found a web-based intervention raised daily activity ~46% and improved weight and HbA1c markers in inactive 60–70-year-olds.

    Journal of medical Internet research · 2013 · 108 citations

  • Can a CBT phone app help stubborn depression?

    Adding a smartphone CBT program to a medication switch improved depressive symptoms more than switching antidepressants alone.

    Journal of medical Internet research · 2017 · 106 citations

  • Facebook team walking RCT

    A 50-day team social-network pedometer program boosted walking/MVPA during the intervention, but gains were not maintained 3 months later.

    Journal of medical Internet research · 2015 · 96 citations

  • Child-care diet/activity RCT and zBMI

    A seven-month child-care nutrition and activity intervention significantly reduced children's zBMI versus control centers.

    BMC public health · 2014 · 92 citations

  • Coaching vs recommendations in IntelliCare

    In a factorial RCT, coaching lowered anxiety more than self-guided use, while weekly app recommendations boosted depression improvement and app sessions.

    Journal of medical Internet research · 2019 · 87 citations

  • Fitbit wear during an activity trial

    Survivors wore Fitbits most days and roughly doubled ActiGraph MVPA over 12 weeks in the intervention arm.

    JMIR mHealth and uHealth · 2018 · 85 citations

  • iCBT for depression in diabetes

    Unmodified clinician-supported iCBT beat treatment-as-usual on depression, anxiety, distress, and diabetes-specific distress in adults with diabetes.

    Journal of medical Internet research · 2017 · 79 citations

  • Dementia-carer internet support forum

    Over 12 weeks on Alzheimer’s Society’s Talking Point forum, caregiving relationship quality improved but anxiety and depression scores did not.

    Journal of medical Internet research · 2014 · 78 citations

  • Automated e-therapy for anxiety disorders

    People finishing Anxiety Online’s disorder-specific e-therapy programs showed significant pre–post gains on severity, quality of life, and confidence measures.

    Journal of medical Internet research · 2011 · 70 citations

  • Join2move usage drop-off

    Patients with knee/hip OA used Join2move less than intended: 94% started, 46% hit 6/9 modules, only 19% finished all nine.

    Journal of medical Internet research · 2013 · 69 citations

  • Audio vs text produce-app RCT

    A tailored mobile app’s audio messages raised fruit intake more than text or control, while vegetable intake showed no overall main effect.

    Journal of medical Internet research · 2016 · 45 citations

  • Propofol vs sevoflurane and breast cancer survival

    In the CAN randomised trial, five-year overall survival after primary breast cancer surgery was essentially the same with propofol or sevoflurane maintenance anaesthesia.

    EClinicalMedicine · 2023 · 37 citations

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