医療介入の非常に大きな治療効果の経験的評価
Tiago V Pereira1, Ralph I Horwitz, John P A Ioannidis
1Health Technology Assessment Unit, Institute of Education and Sciences, German Hospital Oswaldo Cruz, Sao Paulo, Brazil.
JAMA
|October 25, 2012
まとめ
医学におけるほとんどの大きな治療効果は,小規模な研究から生じ,追加の試験によって減少する. 十分に検証された大きな効果は稀であり,通常は非致命的な結果を含みます.
科学分野:
- 医学研究 医学研究
- 臨床試験の分析 臨床試験の分析
- 根拠に基づいた医学は,エビデンスに基づいた医療です.
背景:
- ほとんどの医療介入は,控えめな効果をもたらします.
- 時々,臨床試験では,実質的な利益や害が報告されています.
研究 の 目的:
- 医学における非常に大きな治療効果の頻度と特徴を決定する.
- これらの効果が後の研究でどのように変化するかを分析する.
主な方法:
- 系統的レビューのコクランデータベース (2010年) から85,002の森林プロットの分析.
- 最初の出版物またはその後の出版物における統計的に有意な非常に大きな効果 (odds ratio ≥5) に基づいた試験の分類.
- 各カテゴリーからランダムに抽出された250人のトピックの深層評価.
主要な成果:
- 最初の試験の9.7%と,その後の試験の6.1%で,非常に大きな効果が示されました.
- 非常に大きな効果は,死亡率よりも,実験室で定義された有効性アウトカムにおいてより一般的であった.
- メタアナリシスでは,効果の大きさは通常大幅に減少し,90~98%が小さくなりました.
結論:
- 非常に大きな治療効果は,しばしば小さな研究から生じ,より多くの研究によって減少する傾向があります.
- 確認された大きな効果はまれで,主に非致死的な結果と関連しています.
関連する概念動画
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
01:52Regression Toward the Mean
Regression toward the mean (“RTM”) is a phenomenon in which extremely high or low values—for example, and individual’s blood pressure at a particular moment—appear closer to a group’s average upon remeasuring. Although this statistical peculiarity is the result of random error and chance, it has been problematic across various medical, scientific, financial and psychological applications. In particular, RTM, if not taken into account, can interfere when researchers try to extrapolate results...
01:16Clinical Trials
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
01:20Comparing the Survival Analysis of Two or More Groups
Survival analysis is a cornerstone of medical research, used to evaluate the time until an event of interest occurs, such as death, disease recurrence, or recovery. Unlike standard statistical methods, survival analysis is particularly adept at handling censored data—instances where the event has not occurred for some participants by the end of the study or remains unobserved. To address these unique challenges, specialized techniques like the Kaplan-Meier estimator, log-rank test, and Cox...
01:12What is an Experiment?
An experiment is a planned activity carried out under controlled conditions. The purpose of an experiment is to investigate the relationship between two variables. When one variable causes change in another, we call the first variable the explanatory or independent variable. The affected variable is called the response or dependent variable. In a randomized experiment, the researcher manipulates values of the explanatory variable and measures the resulting changes in the response variable. The...
01:09Bioequivalence of Drugs: Drugs with Multiple Indications
The concept of therapeutic equivalence (TE) in drugs with multiple indications is complex. A generic drug may be therapeutically equivalent to a brand-name product for one specific indication, but this doesn't necessarily mean it's equivalent for all other indications. Evidence of TE in one patient group and bioequivalence shown in healthy volunteers can support—but not confirm—TE for other indications. However, definitive proof requires individual clinical studies for each indication due to...