The 15th Rare Disease Day: China's Policy & Market Overview

by Grace Wang Mar 04, 2022

February 28 marks this year's International Rare Disease Day, a day for raising people's awareness of rare diseases and improving the availability of their treatments. Rare diseases refer to diseases that generally have a low incidence. One rare disease affects a small percentage of the population, but a huge number of over 300 million1 people are suffering from about 7,000 types2 of rare diseases worldwide.

In China, the number of rare disease patients has exceeded 20 million, with more than 200,000 new patients annually. There are some common predicaments among patients: misdiagnosis or missed diagnosis; no treatments are available for their diseases; and some available therapies are too expensive.

To solve the problems, China has been making efforts on multiple aspects—improving hospitals' diagnosis and treatment capabilities, encouraging drug R&D, expediting marketing approval, and reimbursing part of the medical expenses.

1. China Improves Diagnosis and Treatment of Rare Diseases

  • In 2018, National Health Commission (NHC) and other four authorities issued China's First Catalog of Rare Diseases, including 121 rare diseases like congenital scoliosis and spinal muscular atrophy (SMA).3

  • In February 2019, NHC released the Guidance for Diagnosis and Treatment of Rare Diseases (2019), introducing the aforementioned 121 rare diseases in terms of cause, epidemiology, symptom, auxiliary examination, identification, diagnosis, treatment, and monitoring.4

  • In February 2019, NHC selected 324 hospitals to establish a network for coordinated diagnosis and treatment of rare diseases.5 Hospitals in the network receive rare disease patients collectively and make referrals to give corresponding care to patients according to their disease stages. In the network, national-level hospitals and provincial-level leading hospitals receive rare diseases patients with difficult and precarious situations from member hospitals. After cooperative diagnosis, patients with clear diagnosis and in recovery or stable stage are referred back to member hospitals.

  • In November 2019, China Rare Disease Diagnosis and Treatment Information System launched by NHC came into use at the 324 hospitals mentioned above. The system has collected information of about 500,000 rare disease cases6, which helps the authority know the disease distribution to allocate medical resources accordingly.

2. China Encourages Development and Import of Rare Disease Drugs and Expedites Marketing Authorization


Regulation/Policy/Measure/Guideline

Effective Date

R&D

Foreign clinical data can be accepted (if the data is free from ethnic differences) for supporting marketing approval of rare disease drugs that have been approved overseas.7

May 23, 2018

Technical Guidelines for Clinical Research of Rare Disease Drugs says real-world study can support the approved drug to add a new indication of rare disease.8

Jan. 6, 2022

Review

Technical review's time limit is reduced to 3 months9 for rare disease drugs in the List of Overseas New Drugs Urgently Needed in China's Clinical Settings.

Oct. 30, 2018

Priority Review Program

technical review's time limit is reduced to 70 workdays10 for rare disease drugs that have been approved overseas.

July 1, 2020

Tax

Value-added tax (VAT) is reduced to 3% for 35 rare disease drug products (21 in the 1st batch; 14 in the 2nd   batch) and 4 active pharmaceutical ingredients.11 *The drugs are listed in the appendix at the end of this article.

1st batch: March 1, 2019

2nd batch: Oct. 1, 2020

Tariffs are reduced to zero for 4 foreign APIs (penicillamine, pirfenidone, riluzole, and bosentan) of rare disease drugs.12

Jan. 1, 2021

3. China Promotes Availability of Rare Disease Drugs Via Reimbursement and Volume-based Procurement

So far over 40 approved drugs have been included into the National reimbursement Drug List (NRDL), a list of drugs reimbursed by the national healthcare insurance. Thanks to the price cut to get into the NRDL, some rare disease drugs have become affordable to patients. For example, Biogen's Spinraza for treating spinal muscular atrophy cut its price from 700,000 yuan per dose to 33,000 yuan per dose, relieving the economic burden for SMA patients. [Read More].

China's volume-based procurement (VBP) program also makes rare disease drugs available to patients at lower prices. Ambrisentan for treating pulmonary hypertension entered the VBP program after its price was slashed from 115.97 yuan/tablet to 20 yuan/tablet in 2020, contributing to its higher availability among patients.

4. Rare Disease Drug Companies Tap into Market Potential in China

China has approved more than 60 rare disease drugs, but they are far from meeting all the clinical needs. Moreover, the clinical needs are expected to grow as the cases increase. According to Frost & Sullivan, China's rare disease drug market will expand from $1.3 billion in 2020 to $25.9 billion in 2030, with a compound annual growth rate of 34.5%.13

Seeing the market potential, some Chinese pharmaceutical companies have set up pipelines to make rare disease drugs or made licensing deals to introduce foreign drugs into China.

For instance, Canbridge Pharma is a China-based company focusing on developing rare disease therapies. It has established pipelines for seven biologics, small molecule drugs, and candidate drugs to treat rare diseases such as Hunter Syndrome, lysosomal storage diseases (LSD), and Fabry Disease.14 The company has three rare disease drugs approved by China NMPA, namely Hunterase, Nerlynx, and Caphosol, all introduced from foreign countries.

More pharmaceutical companies can be on the way to developing rare disease therapies as China may offer more incentive policies in the future. Ding Lieming, deputy of the National People's Congress and CEO at Betta Pharmaceuticals, says at the NPC's 2022 meeting, he will propose waiving registration fees and setting marketing exclusivity for rare disease drugs.15 With more regulatory support in the future, more drugs for rare diseases may emerge in China.

Related Article:

Appendix: 35 Rare Disease Drugs and 4 Active Pharmaceutical Ingredients (API) with the Preferential Value-added Tax of 3%

1st Batch: 21 Rare Disease Drugs with VAT of 3% since March 1, 2019

No.

API

Drug Product

Approved Dosage Form

Tax Number

1

bosentan

Bosentan Tablets

Tablet

30049010

2

Ambrisentan

Ambrisentan Tablets

Tablet

30049090

3

Riociguat

Riociguat Tablets

Tablet

30049090

4

Macitentan

Macitentan Tablets

Tablet

30049090

5

Iloprost

Iloprost Solution for Inhalation

Solution

30043900

6

Treprostinil

Treprostinil Injection

Injection

30043900

7

Pirfenidone

Pirfenidone Capsules

Capsule

30049090

8

Nintedanib

Nintedanib Esilate Soft Capsules

Capsule

30049090

9

/

Imiglucerase for Injection

Injection

30049090

10

/

Alglucosidase Alfa for Injection

Injection

30049090

11

Miglustat

Miglustat Capsules

Capsule

30049090

12

/

Recombinant Human Growth Hormone Injection

Injection

30043900

13

Sapropterin

Sapropterin Dihydrochloride Tablets

Tablet

30049090

14

/

Recombinant Human Interferon Beta 1a Solution for Injection

Injection

30021500

15

Penicillamine

Penicillamine Tablets

Tablet

30049090

16

Riluzole

Riluzole Tablets

Tablet

30049090

17

/

Human Coagulation Factor Ⅷ

Injection; lyophilized   powder for injection or infusion

30021200

18

/

Recombinant Human Coagulation Factor Ⅷ For Injection

Injection

30021200

19

/

Recombinant Human Coagulation Factor IX For Injection

Injection

30021200

20

/

Human Prothrombin Complex

Injection; lyophilized powder for injection or infusion

30021200

21

/

Recombinant Human Coagulation Factor VIIa for Injection (Eptacog alfa, activated)

Injection

30021200

 

1st Batch: 4 APIs with VAT of 3% since March, 2019

No.

API

Drug Product

Approved Dosage Form

Tax Number

1

Bosentan

/

/

29359000

2

Pirfenidone

/

/

29337900

3

Penicillamine

/

/

29309090

4

Riluzole

/

/

29342000

 

2nd Batch: 14 Rare Disease Drugs with VAT of 3% since Oct 1, 2020

No.

API

Drug Product

Approved Dosage Form

Tax Number

1

Recombinant Human Interferon   Beta-1b

Recombinant Human Interferon Beta-1b for Injection

Injection

30021500

2

Teriflunomide

Teriflunomide Tablets

Tablet

30049090

3

Nusinersen Sodium

Nusinersen Sodium Injection

Injection

30049090

4

Fingolimod

Fingolimod Hydrochloride Capsules

Capsule

30049090

5

Selexipag

Selexipag Tablets

Tablet

30049010

6

Eculizumab

Eculizumab Injection

Injection

30021500

7

Emicizumab

Emicizumab Injection

Injection

30021500

8

Bosentan

Bosentan Dispersible Tablets

Tablet

30049010

9

Deutetrabenazine

Deutetrabenazine Tablet

Tablet

30049090

10

Siponimod

Siponimod Tablets

Tablet

30049090

11

Agalsidase Beta

Agalsidase Beta for Injection

Injection

30049090

12

Elosulfase Alfa

Elosulfase Alfa Injection

Injection

30049090

13

Laronidase

Laronidase Concentrated Solution for Infusion

Injection

30049090

14

Human Coagulation Factor Ⅸ

Human Coagulation Factor Ⅸ

Injection

30021200


Grace Wang
ChemLinked Regulatory Analyst
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