2024年4月13日发(作者：)

罕见疾病药物临床研发技术指导原则

Rare Disease Drug Clinical Development Technology Guidance Principles

一、药物研发的原则

I. Principles of Drug Development

1.药物研发应坚持以病人为中心，以病人的安全、有效性和可及性为核心，以满足病人

的需求为目标，以提高病人的生活质量为出发点。

1. Drug development should adhere to the patient-centered principle, with the safety,

efficacy and accessibility of the patient as the core, to meet the needs of the patient as the

goal, and to improve the quality of life of the patient as the starting point.

2.药物研发应坚持科学、合理、可行的原则，以确保药物研发的质量和可行性。

2. Drug development should adhere to the principles of science, rationality and feasibility

to ensure the quality and feasibility of drug development.

3.药物研发应坚持安全、有效、可及性、可行性的原则，以确保药物研发的安全性、有

效性和可及性。

3. Drug development should adhere to the principles of safety, efficacy, accessibility and

feasibility to ensure the safety, efficacy and accessibility of drug development.

二、药物研发的技术指导原则

II. Technical Guidance Principles of Drug Development

1.药物研发应坚持以病人为中心，以病人的安全、有效性和可及性为核心，以满足病人

的需求为目标，以提高病人的生活质量为出发点。

1. Drug development should adhere to the patient-centered principle, with the safety,

efficacy and accessibility of the patient as the core, to meet the needs of the patient as the

goal, and to improve the quality of life of the patient as the starting point.

2.药物研发应坚持科学、合理、可行的原则，以确保药物研发的质量和可行性。

2. Drug development should adhere to the principles of science, rationality and feasibility

to ensure the quality and feasibility of drug development.

3.药物研发应充分考虑疾病的特点，采用合理的研发模式，确保研发的安全性、有效性

和可及性。

3. Drug development should take into full account the characteristics of the disease,

adopt a reasonable development model, and ensure the safety, efficacy and accessibility of

the development.

4.药物研发应结合现有科学技术和研究方法，采用科学、合理、可行的药物研发模式，

以确保药物研发的质量和可行性。

4. Drug development should combine existing scientific and technological and research

methods, adopt scientific, reasonable and feasible drug development models, to ensure the

quality and feasibility of drug development.

5.药物研发应采取有效的技术措施，以确保药物研发的安全性、有效性和可及性。

5. Drug development should take effective technical measures to ensure the safety,

efficacy and accessibility of drug development.