The "CARE Plan" is a pilot work in the field of rare disease drug development. It has been launched to promote patient-centered drug development and to implement the work deployment of the NMPA in accelerating the development and availability of drugs for rare diseases. The "CARE Plan" aims to uphold the concept of "patient-centered" drug development and encourages drug development entities to listen to patients' voices and pay attention to their experiences and perceptions throughout the development process for rare disease therapies.

I. Background

To date, more than 7,000 rare diseases have been identified worldwide. Rare diseases are characterized by extremely low prevalence, complex clinical manifestations, and limited understanding, all of which contribute to significant challenges in the development of drugs for rare diseases.

In recent years, the concept of "patient-centered" drug development has been increasingly recognized and widely adopted. In terms of drugs for rare diseases, implementing the "patient-centered" approach during the research and development process, with a focus on the patient perspective and careful attention to patients' voices, can assist drug development entities, researchers, and regulatory authorities in gaining a better understanding of rare diseases and patient needs. This, in turn, contributes to the optimization of scientific processes and technical requirements for the development of these drugs.

Against this background, the CDE has launched a pilot work on patient-centered drug development for rare diseases, known as the "CARE Plan" (patient-Centered Action for Rare diseases Encouragement).

The "CARE Plan" is intended to guide applicants throughout the entire drug development process in incorporating the perspectives of patients with rare diseases. It aims to enhance the scientific rigor, standardization, and rationality of integrating patient experience, particularly those related to the clinical benefit-risk evaluation, into the clinical development of drugs for rare diseases. It also seeks to strengthen communication and collaboration among regulatory authorities, drug development entities, and patients involved in drug development for rare diseases, ultimately facilitating the marketing authorization of these drugs to address clinical needs.

II. Main Contents

The "CARE Plan" is a pilot work designed to implement the concept of patient-centered drug development in the field of drug development for rare diseases.

The Implementation Framework for the Patient-Centered Action for Rare Diseases Encouragement ("CARE Plan") (hereinafter referred to as the Implementation Framework) serves as a supporting document for the "CARE Plan". The Implementation Framework divides the drug development process into five stages, covering the entire product life cycle. These stages include:

Stage A: Project initiation; Stage B: Pre-clinical trial phase (Pre-IND); Stage C: Pre-pivotal study phase; Stage D: Pre-marketing application/marketing application phase (Pre-NDA/NDA); Stage E: Post-marketing phase. The Implementation Framework provides detailed descriptions of the activities and specific implementation approaches that reflect the concept of "patient-centered" development at different stages of drug development. These primarily include how to collect patient experience data relevant to each development stage, how to apply such data in the development process, and how to design and conduct clinical trials that incorporate and reflect patient experience considerations.

III. Organization and Implementation

The Guidance on Application for the Patient-Centered Action for Rare Diseases Encouragement ("CARE Plan") (hereinafter referred to as the Application Guidelines) and the Implementation Framework provide detailed information on the procedures and specific requirements for applicants intending to participate in the "CARE Plan", and may serve as references for voluntary application.

Applicants may voluntarily apply to participate in the corresponding stage of the "CARE Plan" based on the current stage of drug development, through established communication channels. The CDE will select one to two representative drug candidates at each of the stages A, B, C, D, and E for inclusion in the "CARE Plan". Drugs included in the pilot plan are required to conduct and complete the relevant work corresponding to their development stage in accordance with the requirements established in the Implementation Framework. While conducting routine drug application and registration work in accordance with current regulations and procedures, applicants are also required to report the progress or outcomes of the relevant activities specified in the Implementation Framework to the CDE.

Applicants may submit an application to join the "CARE Plan" to the CDE through communication channels. The CDE will evaluate whether to approve the inclusion of a drug in the CARE Plan based on the characteristics of the product, the indication being developed or applied for, and the implementation plan submitted by the applicant. The evaluation will be conducted by the CDE's Rare Disease Working Group in collaboration with the relevant indication-specific review team. When necessary, a communication meeting may be convened to discuss the details of the implementation plan.

Upon preliminary agreement to include a drug in the "CARE Plan", the CDE will publish a public notice for five working days. Drugs for which no objections are received during the publicity period will be officially included in the "CARE Plan".