Q&A

FAQ on Rare Disease Drugs (Orphan Drugs) in China

by Grace Wang
Apr 07, 2022

In recent years, rare disease drugs have been receiving more attention from Chinese authorities. The government has enacted a series of policies for improving the availability of rare disease drugs, including releasing the Catalog of Rare Diseases, expediting the review of rare disease drugs, and adding more rare disease drugs to the National Reimbursement Drug List (NRDL).

This article answers questions about China's policies on rare disease drugs, mainly regarding clinical data, marketing authorization, and reimbursement.

    1. How Does China Define a Rare Disease Drug?

    There is no official definition of "rare disease drug" in China. Rare disease drugs, also known as orphan drugs, generally refer to drugs with significant clinical value for treating rare diseases that affect a small proportion of the population.

    Although without a clear definition, China has a list on which the diseases are officially recognized as rare diseases. In 2018, the National Health Commission and another four authorities issued the first Catalog of Rare Diseases, covering 121 rare diseases, such as congenital scoliosis, Fabry disease, and multiple sclerosis.

    More rare diseases are likely to be added to the catalog if they meet all four conditions1 below:

    • Domestic and international evidences prove that the disease has a relatively low incidence or prevalence;

    • The disease poses immense harm to the patient and his or her family.

    • There is a clear diagnostic method for the disease;

    • There is an economically feasible treatment/intervention for the disease, or the disease has no effective treatment/intervention but has been included in a National Science and Technology Major Project.

    2. Does China Accept Foreign Clinical Trial Data?

    Whether the Center for Drug Evaluation (CDE) accepts foreign clinical data depends on the data's quality.

    CDE Acceptance

    Foreign Clinical Data

    Fully accepted

    • The data is authentic and reliable, and complies with ICH GLP and pre-approval inspection requirements;

    • The data can support the efficacy and safety evaluations;

    • The data is free from racial factors that may affect the drug's efficacy and safety.

    Partially accepted

    • The data is authentic and reliable, and complies with ICH GLP and pre-approval inspection requirements;

    • The data can support the efficacy and safety evaluations but has racial factors that make it uncertain whether the data is applicable to the Chinese population.

    • The drug applicant should analyze the influencing factors, consult with CDE, and conduct required clinical trials later.

    *The data of rare disease drugs can be conditionally accepted, but the applicant should collect the efficacy and safety data for post-approval evaluation.

    Not accepted

    • The data is severely problematic in authenticity, completeness, accuracy, or traceability;

    • The data cannot support the efficacy and safety evaluations;

    • The drug applicant should conduct clinical trials in China.

    Source: Technical Guidelines for Accepting Foreign Clinical Trial Data of Drugs2

    Therefore, if a rare disease drug's overseas clinical data is acceptable to NMPA, the applicant has no need to conduct clinical trials in China. What the applicant needs to do is having a consultative meeting with CDE. After reaching a consensus with CDE, the applicant can directly submit the new drug application (NDA) to CDE.

    3. Are There Any Preferential Policies for Expediting the Approval of Rare Disease Drugs?

    Yes. China has Priority Review as one of the four Expedited Programs for Drug Registration. Priority Review can shorten the time of the technical review from 200 workdays to 70 workdays for urgently-needed rare disease drugs approved overseas but not in China. Such urgently-needed foreign drugs are collected in the List of Overseas New Drugs Urgently Needed in China's Clinical Settings. The list currently covers 74 drugs, among which 34 are for rare diseases.

    To obtain a Priority Review designation, the applicant should submit the Priority Review application with NDA to CDE. Detailed procedures are shown in the diagram below.

    baipharm-priority-review-of-drugs-in-china.jpgPriority Review of Drugs in China

    4. What Are the Procedures for a Rare Disease Drug to be Approved in China?

    Generally, drug applicants should first submit the investigational new drug (IND) application and then the new drug application (NDA) to CDE. But rare disease drug applicants can apply for the exemption of clinical trials so they may be allowed to skip the IND application.

    In addition, if it is a foreign orphan drug that already has clinical data, the applicant should have a meeting with CDE to see if the data will be accepted for applying for marketing approval in China. If accepted, the applicant can directly submit the NDA application to CDE.

    baipharm-new-drug-registration-procedures-in-china.jpgNew Drug Registration Procedures in China

    5. How Many Rare Disease Drugs Have Been Approved in China?

    According to Illness Challenge Foundation, China has approved 87 drugs for 46 rare diseases by the end of 2021.

    6. What Rare Diseases Still Have No Effective Drugs in China?

    Among the 121 rare diseases officially recognized in China, 75 diseases, such as Leber Hereditary Optic Neuropathy and Laron Syndrome, have no approved orphan drugs for effective treatment. To encourage the development and import of rare disease medicines, China has released a series of preferential policies, including waiving the tariffs for importing four active pharmaceutical ingredients (API) of orphan drugs.

    7. Does China Have Any Pricing & Reimbursement Policies for Rare Disease Drugs?

    China doesn't have mandatory prices or reimbursements for rare disease drugs but have National Reimbursement Drug List (NRDL), which currently includes 58 drugs for 28 rare diseases. The NRDL-listed orphan drugs have lowered their prices and are covered or partially reimbursed by social medical insurance. 

    For example, Biogen cut the price of Spinraza (Nusinersen Sodium Injection) for spinal muscular atrophy (SMA) from 700,000 yuan per dose to 33,000 yuan per dose to get into NRDL. With different reimbursement proportions in different provinces, each Spinraza injection costs around 10,000-17,000 yuan.

    Appendix: Rare Disease Drugs Included in NRDL After the Release (June 8, 2018) of the First Catalog of Rare Diseases in China


    Rare Disease

    Orphan Drug

    Approval   Year

    Company

    1

    Amyotrophic Lateral Sclerosis   (ALS)

    Edaravone and Sodium Chloride

    2019

    Mitsubishi Tanabe Pharma

    2

    Hemolytic Uremic Syndrome (HUS)

    Eculizumab

    2018

    Alexion

    3

    Castleman Disease

    Siltuximab

    2021

    BeiGene

    4

    Fabry Disease

    Agalsidase Alfa

    2020

    Takeda

    5

    Fabry Disease

    Agalsidase Beta

    2019

    Sanofi

    6

    Gaucher Disease

    Velaglucerase Alfa

    2021

    Takeda

    7

    Hemophilia

    Human Coagulation Factor Ⅸ

    2020

    China Biologic Products

    8

    Hemophilia

    Emicizumab

    2021

    Roche

    9

    Hemophilia

    Eftrenonacog Alfa

    2021

    Sanofi

    10

    Hereditary Angioedema

    Lanadelumab

    2020

    Takeda

    11

    Hereditary Angioedema

    Icatibant

    2021

    Takeda

    12

    Homozygous Hypercholesterolemia

    Evolocumab

    2018

    Amgen

    13

    Huntington Disease

    Deutetrabenazine

    2020

    Teva Pharmaceuticals

    14

    Hypophosphatemic Rickets

    Burosumab

    2021

    Kyowa Kirin

    15

    Idiopathic Cardiomyopathy

    Tafamidis Meglumine

    2020

    Pfizer

    16

    Pulmonary Arterial Hypertension

    Sildenafil Citrate

    2020

    Pfizer

    17

    Pulmonary Arterial Hypertension

    Treprostinil

    2020

    Zhaoke Pharmaceutical

    18

    Mucopolysaccharidosis (Type I)

    Laronidase

    2020

    Sanofi

    19

    Mucopolysaccharidosis (Type II)

    Idursulfase Beta

    2020

    CANbridge

    20

    Mucopolysaccharidosis (Type IVa)

    Elosulfase Alfa

    2019

    BioMarin

    21

    Multiple Sclerosis

    Teriflunomide

    2018

    Sanofi

    22

    Multiple Sclerosis

    Fingolimod

    2019

    Novartis

    23

    Multiple Sclerosis

    Fampridine

    2021

    Biogen

    24

    Multiple Sclerosis

    Dimethyl Fumarate

    2021

    Biogen

    25

    Multiple Sclerosis

    Ofatumumab

    2021

    Novartis

    26

    Neuromyelitis Optica

    Satralizumab

    2021

    Roche

    27

    Paroxysmal Nocturnal   Hemoglobinuria

    Eculizumab

    2018

    Alexion

    28

    Spinal Muscular Atrophy

    Nusinersen

    2019

    Biogen

    29

    Spinal Muscular Atrophy

    Risdiplam

    2021

    Roche

    30

    Systemic Sclerosis

    Nintedanib

    2020

    Boehringer Ingelheim

    31

    Tyrosinemia

    Nitisinone

    2021

    Chinese Medicines

    Related:

    Grace Wang
    ChemLinked Regulatory Analyst / Editor
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