The complete, transparent, and standardized reporting of the outcome of a clinical trial is a key factor in ensuring the practicality, reproducibility, and transparency of the trial, and reducing bias in selective reporting. The consolidated standards of reporting trials (CONSORT) 2010 statement provides normative guidelines for reporting clinical trials. In December 2022, JAMA released the guidelines for reporting outcomes in trial reports (CONSORT-Outcomes) 2022 extension, aiming to explain the entries related to trial outcomes, sample size, statistical methods, and auxiliary analysis in the CONSORT 2010 statement, to further improve the standards for outcome reporting in clinical trial reports. This article combines research examples to interpret the CONSORT-Outcomes 2022 extension, in order to provide normative references for domestic scholars to report clinical research results.
The patient, as the person who experiences the disease first-hand, has the most direct and accurate experience of the pain of the disease and the most accurate need for health products. Although there is a vast array of technological means to combat disease and maintain health, the human burden of disease has not been reduced and the health needs of patients have not been fully met. Therefore, "patient-focused drug development" is imperative. Gathering comprehensive information from patients through multiple channels and incorporating this information into the entire drug development process can help ensure that patients’ experiences, perspectives, needs and priorities are taken into account and valued. This article will introduce the concept, development process and the specific problems it faces in patient-focused drug development.
In order to better incorporate patient input in clinical trials, the US Food and Drug Administration has included "patient-focused drug development" in the selection and development of clinical outcome assessments, and formulated a series of guidelines. Based on the third guiding principle, "Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments", this article summarizes the clinical outcome assessments from five aspects: concept, development process, scoring mechanism, interference factors and sensitivity, and introduces four different types of clinical outcome assessments, providing new ideas for "patient-focused drug development" efficacy evaluation in clinical trials.
As subjects in drug clinical trials and participants in medical practice, patients can best understand their own conditions and needs. With this in mind, the FDA proposed "patient-centered drug discovery" and issued a set of guidelines to incorporate patient experiences, perspectives, needs, and preferences into the drug development and evaluation process. Guideline (2), methods for identifying important patient information, mainly describes methods and precautions for collecting and extracting patient experience data. This paper will focus on the characteristics, common methods and precautions of qualitative, quantitative and mixed research methods in the collection of patient experience data, in order to provide help for the comprehensive collection of patient experience data.
To ensure the scientific and recognized evaluation of patient-centered clinical research outcomes, the drug development tool qualification certification website of the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research have disclosed the information of clinical outcome assessment (COA) submitted for certification, and encouraged the use of certified COA. This paper searched all COA and approval decisions on the website, analyzed the submitted information and certification status of COA, and interpreted the certification technology and process of COA, so as to provide references and suggestions for the development of patient-centered clinical research outcome evaluation.