Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (817 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
Evaluation undertaken to assess the results or consequences of management and procedures used in combating disease in order to determine the efficacy, effectiveness, safety, and practicability of these interventions in individual cases or series.
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Outcome, Treatment; Treatment Efficacy; Efficacy, Treatment; Clinical Efficacy; Efficacy, Clinical; Rehabilitation Outcome; Outcome, Rehabilitation; Clinical Effectiveness.
Where it fits in the MeSH classification
- Broader topics
- Prognosis; Outcome Assessment, Health Care
- Narrower topics
- Disease-Free Survival; Early Termination of Clinical Trials; Pathologic Complete Response; Progression-Free Survival; Response Evaluation Criteria in Solid Tumors; Sustained Virologic Response; Therapeutic Index; Treatment Expectations; Treatment Failure; Treatment Effect Heterogeneity
Research landscape
PubMed indexes 1,441,883 records tagged with the MeSH heading “Treatment Outcome” (counts retrieved 2026-10-29). 387,091 of them (26.8%) were published from 2020 onward, and the busiest period so far is 2010-2019 with 621,712 records.
Reviews account for 202,995 records (14.1%) and meta-analyses for 46,822. Randomized controlled trials number 173,154 (12.0%) as a share of all records on the topic. Randomized trials make up a comparatively large share of this literature, which means many treatment questions have been tested directly.
Show the data
| Period | Records |
|---|---|
| through 1999 | 76,885 |
| 2000-2009 | 356,195 |
| 2010-2019 | 621,712 |
| 2020 onward | 387,091 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 202,995 | 14.1% |
| Meta-analyses | 46,822 | 3.2% |
| Randomized controlled trials | 173,154 | 12.0% |
Method: PubMed query "Treatment Outcome"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-29.
Reading the evidence in clinical research
Research methods determine how much a result can be trusted. Randomized trials guard against confounding, systematic reviews and meta-analyses combine many studies, and observational designs are valuable but can show only association. Reporting standards, trial registration and pre-specified outcomes reduce bias. When reading about a method, ask what problem it is designed to solve and which problems it does not solve.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Refining the impact of genetic evidence on clinical success | 2024 | Research article Design not recorded in structured metadata | 364 | cc by PMID 38632401 · DOI · PMC11096124 |
| Virological Failure After Switch to Long-Acting Cabotegravir and Rilpivirine Injectable Therapy: An In-depth Analysis | 2024 | Research article Design not recorded in structured metadata | 53 | cc by PMID 38207125 · DOI · PMC11259215 |
| Toward Overcoming Treatment Failure in Rheumatoid Arthritis | 2021 | Review article Design not recorded in structured metadata | 46 | cc by PMID 35003068 · DOI · PMC8732378 |
| Predictors of failure to rescue in surgical rescue | 2026 | Research article Design not recorded in structured metadata | 0 | cc by PMID 41731099 · DOI · PMC12987867 |
Refining the impact of genetic evidence on clinical success [1]
Abstract reading summary — source excerpts
- Opening excerpt
- The cost of drug discovery and development is driven primarily by failure1, with only about 10% of clinical programmes eventually receiving approval2-4.
- Closing excerpt
- These results indicate we are far from reaching peak genetic insights to aid the discovery of targets for more effective drugs.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (137 words)
The cost of drug discovery and development is driven primarily by failure1, with only about 10% of clinical programmes eventually receiving approval2-4. We previously estimated that human genetic evidence doubles the success rate from clinical development to approval5. In this study we leverage the growth in genetic evidence over the past decade to better understand the characteristics that distinguish clinical success and failure. We estimate the probability of success for drug mechanisms with genetic support is 2.6 times greater than those without. This relative success varies among therapy areas and development phases, and improves with increasing confidence in the causal gene, but is largely unaffected by genetic effect size, minor allele frequency or year of discovery. These results indicate we are far from reaching peak genetic insights to aid the discovery of targets for more effective drugs.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 38632401 · DOI · PMC11096124
Virological Failure After Switch to Long-Acting Cabotegravir and Rilpivirine Injectable Therapy: An In-depth Analysis [2]
Abstract reading summary — source excerpts
- Opening excerpt
- BackgroundLong-acting (LA) injectable therapy with cabotegravir (CAB) and rilpivirine (RPV) is currently used as maintenance treatment for human immunodeficiency virus type 1, and has a low risk for virological failure (VF).
- Closing excerpt
- Our observations stress the need to be aware for (evolving) risk factors and the yield of a comprehensive clinical, virological, and pharmacokinetic approach in case of failure.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (251 words)
BackgroundLong-acting (LA) injectable therapy with cabotegravir (CAB) and rilpivirine (RPV) is currently used as maintenance treatment for human immunodeficiency virus type 1, and has a low risk for virological failure (VF). Although the risk is low, the circumstances and impact of VF in the real-world setting merit further evaluation.MethodsWe performed an in-depth clinical, virological, and pharmacokinetic analysis on the reasons behind and the impact of VF during LA CAB/RPV therapy in 5 cases from the Netherlands. Genotypic resistance testing was performed after the occurrence of VF, and drug plasma (trough) concentrations were measured after VF was established and on any other samples to assess on-treatment drug levels. CAB and RPV drug levels that were below the first quartile of the population cutoff (≤Q1) were considered to be low.ResultsFive cases who were eligible for LA CAB/RPV experienced VF despite a low predicted risk at baseline. Genotypic resistance testing revealed extensive selection of nonnucleoside reverse transcriptase inhibitor-associated mutations in all cases, and integrase strand transfer inhibitor mutations in 4 cases. All cases displayed low drug levels of either CAB, RPV, or both during the treatment course, likely contributing to the occurrence of VF. In 3 cases, we were able to identify the potential mechanisms behind these low drug levels.ConclusionsThis is the first in-depth multiple case analysis of VF on LA CAB/RPV therapy in a real-world setting. Our observations stress the need to be aware for (evolving) risk factors and the yield of a comprehensive clinical, virological, and pharmacokinetic approach in case of failure.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 38207125 · DOI · PMC11259215
Toward Overcoming Treatment Failure in Rheumatoid Arthritis [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Rheumatoid arthritis (RA) is an autoimmune disorder characterized by inflammation and bone erosion.
- Closing excerpt
- In this review, we also discuss the potential of precision medicine for overcoming RA treatment failure, with the introduction of various cutting-edge technologies and big data.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (183 words)
Rheumatoid arthritis (RA) is an autoimmune disorder characterized by inflammation and bone erosion. The exact mechanism of RA is still unknown, but various immune cytokines, signaling pathways and effector cells are involved. Disease-modifying antirheumatic drugs (DMARDs) are commonly used in RA treatment and classified into different categories. Nevertheless, RA treatment is based on a "trial-and-error" approach, and a substantial proportion of patients show failed therapy for each DMARD. Over the past decades, great efforts have been made to overcome treatment failure, including identification of biomarkers, exploration of the reasons for loss of efficacy, development of sequential or combinational DMARDs strategies and approval of new DMARDs. Here, we summarize these efforts, which would provide valuable insights for accurate RA clinical medication. While gratifying, researchers realize that these efforts are still far from enough to recommend specific DMARDs for individual patients. Precision medicine is an emerging medical model that proposes a highly individualized and tailored approach for disease management. In this review, we also discuss the potential of precision medicine for overcoming RA treatment failure, with the introduction of various cutting-edge technologies and big data.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 35003068 · DOI · PMC8732378
Predictors of failure to rescue in surgical rescue [4]
Abstract reading summary — source excerpts
- Opening excerpt
- AIM: To determine the current status of surgical rescue (SR) and examine the predictors of failure to rescue (FTR).
- Closing excerpt
- The SRSS stratified the short- and long-term prognoses of SR and predicted prognoses preoperatively.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (246 words)
AIM: To determine the current status of surgical rescue (SR) and examine the predictors of failure to rescue (FTR). In acute care surgery, predictors of FTR have only been reported in patients with trauma, not in SR. METHODS: This retrospective study included 142 patients who underwent emergency surgery at our institution between April 2019 and March 2023. The primary outcome was the identification of the FTR predictors using logistic regression analysis. The secondary outcome was the development of a Surgical Rescue Severity Score (SRSS) using these SR predictors and long-term outcomes. RESULTS: The patients were divided into survival and mortality groups (122 and 20, respectively). American Society of Anesthesiologists Physical Status (ASA-PS) and quick sequential organ failure assessment (qSOFA) scores were higher in the mortality group. In contrast, the survival group included more cases of SR necessitated by surgical treatment. In multivariate analysis, ASA-PS ≥ 3 (odds ratio [OR], 5.3; 95% confidence interval [CI] 1.2–36), complications caused by non-surgical therapy (OR, 5.5; 95% CI 1.8–19), and qSOFA score ≥ 2 (OR, 4.5; 95% CI 1.3–17) were independent prognostic factors. The total risk factors for FTR obtained in the multivariate analysis were calculated and established as the SRSS. The long-term prognosis of SR was examined and stratified by the SRSS (P = 0.0001). CONCLUSION: Predictors of FTR in SR were ASA-PS ≥ 3, qSOFA score ≥ 2, and complications caused by non-surgical therapy. The SRSS stratified the short- and long-term prognoses of SR and predicted prognoses preoperatively.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41731099 · DOI · PMC12987867
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article A minimal common outcome measure set for COVID-19 clinical research [5]
- Research article The association of registered nurse staffing levels and patient outcomes: systematic review and meta-analysis [6]
- Research article Update on treatments for nonmotor symptoms of Parkinson's disease-an evidence-based medicine review [7]
- Research article mRECIST for HCC: Performance and novel refinements [8]
- Research article Efficacy of screening mammography. A meta-analysis [9]
- Research article Response, remission and drop-out rates following high-frequency repetitive transcranial magnetic stimulation (rTMS) for treating major depression: a systematic review and meta-analysis of randomized, double-blind and sham-controlled trials [10]
References
- Minikel EV, Painter JL, Dong CC, Nelson MR. Refining the impact of genetic evidence on clinical success. . 2024. PMID 38632401 · DOI · PMC11096124
- van Welzen BJ, Van Lelyveld SFL, Ter Beest G, Gisolf JH, Geerlings SE, Prins JM, et al.. Virological Failure After Switch to Long-Acting Cabotegravir and Rilpivirine Injectable Therapy: An In-depth Analysis. . 2024. PMID 38207125 · DOI · PMC11259215
- Wang Z, Huang J, Xie D, He D, Lu A, Liang C. Toward Overcoming Treatment Failure in Rheumatoid Arthritis. . 2021. PMID 35003068 · DOI · PMC8732378
- Ogawa K, Miyamoto Y, Hisano Y, Maeda Y, Ohuchi M, Hiyoshi Y, et al.. Predictors of failure to rescue in surgical rescue. . 2026. PMID 41731099 · DOI · PMC12987867
- WHO Working Group on the Clinical Characterisation and Management of COVID-19 infection. A minimal common outcome measure set for COVID-19 clinical research. Lancet Infect Dis. 2020. PMID 32539990 · DOI · PMC7292605
- Kane RL, Shamliyan TA, Mueller C, Duval S, Wilt TJ. The association of registered nurse staffing levels and patient outcomes: systematic review and meta-analysis. Med Care. 2007. PMID 18007170 · DOI
- Seppi K, Ray Chaudhuri K, Coelho M, Fox SH, Katzenschlager R, Perez Lloret S, et al.. Update on treatments for nonmotor symptoms of Parkinson's disease-an evidence-based medicine review. Mov Disord. 2019. PMID 30653247 · DOI · PMC6916382
- Llovet JM, Lencioni R. mRECIST for HCC: Performance and novel refinements. J Hepatol. 2020. PMID 31954493 · DOI · PMC12452114
- Kerlikowske K, Grady D, Rubin SM, Sandrock C, Ernster VL. Efficacy of screening mammography. A meta-analysis. JAMA. 1995. PMID 7799496 · DOI
- Berlim MT, van den Eynde F, Tovar-Perdomo S, Daskalakis ZJ. Response, remission and drop-out rates following high-frequency repetitive transcranial magnetic stimulation (rTMS) for treating major depression: a systematic review and meta-analysis of randomized, double-blind and sham-controlled trials. Psychol Med. 2014. PMID 23507264 · DOI
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-29.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.