Selecting and developing clinical outcome assessments in patient-focused drug development_Chinese Journal of Evidence-Based Medicine

Authors：

ZHANG Chenyao ¹ , WU Xiaolei ¹ , SHI Menglong ¹ , MA Yucong ¹ , PANG Wentai ¹ , DU Liang ² , LIU Binglin ³ ,  CAO Lujia ¹ ,  ZHANG Junhua ¹

1. Evidence-based Medicine Center, Tianjin University of Traditional Chinese Medicine, Tianjin 301617, P. R. China;
2. Chinese Evidence-based Medicine Center, West China Hospital of Sichuan University, Chengdu 610041, P. R. China;
3. Center for Drug Evaluation, China Food and Drug Administration, Beijing 100022, P. R. China;

Corresponding author：

CAO Lujia, Email: caolujia0825@163.com; ZHANG Junhua, Email: zjhtcm@foxmail.com

Keywords：

Patient-focused; Drug development; Clinical outcome assessments

DOI：

10.7507/1672-2531.202307067

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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.

Citation： ZHANG Chenyao, WU Xiaolei, SHI Menglong, MA Yucong, PANG Wentai, DU Liang, LIU Binglin, CAO Lujia, ZHANG Junhua. Selecting and developing clinical outcome assessments in patient-focused drug development. Chinese Journal of Evidence-Based Medicine, 2024, 24(4): 472-477. doi: 10.7507/1672-2531.202307067 Copy

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1. FDA. CDER patient-focused drug development.
2. Perfetto EM, Burke L, Oehrlein EM, et al. Patient-focused drug development: a new direction for collaboration. Med Care, 2015, 53(1): 9-17.
3. FDA. FDA patient-focused drug development guidance series for enhancing the incorporation of the patient’s voice in medical product development and regulatory decision making.
4. FDA. Patient-focused drug development: collecting comprehensive and representative input guidance for industry, Food and Drug Administration staff, and other stakeholders.
5. FDA. Patient-focused drug development: methods to identify what is important to patients guidance for industry, Food and Drug Administration staff, and other stakeholders.
6. Schultz-Knudsen K, Sabaliauskaite U, Hellsten J, et al. New drug and biologics approvals in 2019: a systematic analysis of patient experience data in FDA drug approval packages and product labels. Ther Innov Regul Sci, 2021, 55(3): 503-513.
7. FDA. Patient-focused drug development: incorporating clinical outcome assessments into endpoints for regulatory decision-making.
8. FDA. Clinical Outcome Assessment (COA) qualification program.
9. FDA. DDT COA #000009: non-small cell lung cancer symptom assessment questionnaire (NSCLC-SAQ).

Chinese Journal of Evidence-Based Medicine

Selecting and developing clinical outcome assessments in patient-focused drug development

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