This year’s Feb. 28 marks the 16th International Rare Disease Day. In China, there are around 20 million1 rare disease patients, with more than 200,000 additional patients each year.
Drugs for treating rare diseases are also called orphan drugs. They are difficult to develop due to several reasons:
- the number of subjects of clinical trials is relatively small;
- the R&D work is lengthy and costly;
- the rare disease market is not so lucrative as the clinical need is not huge, etc.
However, fortunately, China has shown a supportive attitude towards rare disease drug development in a series of official documents. Among the encouraging announcements to improve the availability and affordability of rare disease drugs, the regulations and policies introduced in this article are significant in terms of the drugs’ marketing authorization review timeline and pricing.
1. China’s Rare Disease Catalog
China has not given an official definition to rare diseases, but it released the first Catalog of Rare Diseases in 2018. The catalog, including 121 rare diseases such as congenital scoliosis, Fabry disease, and spinal muscular atrophy (SMA), is meant to encourage the industry to develop therapeutic drugs for these diseases.
One shortcoming of the catalog is that it has not covered all rare diseases in China. Nevertheless, it will be appropriately updated, according to the National Healthcare Commission (NHC)’s reply2 in last December to a proposal for more efforts into rare disease diagnosis and treatment.
China does not stipulate that only drugs for diseases in the catalog are recognized as rare disease therapies. In reality, drugs outside the catalog can be recognized as long as its indication is a rare disease.
2. Clinical Research and Marketing Authorization Regulations & Policies
2.1 Foreign clinical data can be accepted
China National Medical Products Administration (NMPA) announced in 2018 that if foreign clinical data is free from ethnic differences, the data can be accepted as evidence supporting the marketing authorization of rare disease drugs.3
2.2 RWD can be accepted
According to Technical Guidelines for Clinical Research of Rare Disease Drugs effective since 2022, China Center for Drug Evaluation (CDE) can accept the real-world data to support a rare disease to be the new indication of an approved drug.4
2.3 Technical review period is shortened for rare disease drugs approved overseas
China has not designated a special approval pathway for rare disease drugs, yet it has been trying to shorten their review time. The Expedited Program of Priority Review accelerates the technical review from 200 workdays to 70 workdays for the rare disease drugs that have been approved overseas.5
2.4 Pilot use is permitted in Hainan
Even without China NMPA’s national marketing authorization, rare disease drugs & medical devices can apply for pilot use at Bo’ao Winhealth Rare Disease Medical Center in southern China’s Hainan province. The pilot use program can be a springboard into the Chinese nationwide market.
The below is a list of rare disease drugs approved by China NMPA in 2022:
17 Rare Disease Drug Approved by China NMPA in 2022 | ||||
No. | Drug Product | Approved Indication | Marketing Authorization Holder | Whether the Indication is in the Catalog |
1 | Edaravone Sublingual Tablet | Amyotrophic lateral sclerosis (ALS) | Nanjing Biosnwill Medicine | Yes |
2 | Riluzole Oral Suspension | ALS | ITALFARMACO, S.A. | Yes |
3 | Eliglustat Tartrate Capsules | Gaucher Disease (GD) | Beijing Kailai Tiancheng Medical Technology | Yes |
4 | Icatibant Acetate Injection | Hereditary angioedema (HAE) | Jiangsu Hansoh Pharmaceutical | Yes |
5 | Nitric Oxide for Inhalation | Pulmonary hypertension (PH) | INO Therapeutics LLC | Yes |
6 | Inebilizumab Injection (UPLIZNA) | Neuromyelitis optica spectrum disorders (NMOSD) | Horizon Therapeutics Ireland DAC | Yes |
7 | Naxitamab Injection | Neuroblastoma | Y-mAbs Therapeutics, Inc. | Yes |
8 | Melphalan Hydrochloride for Injection | Neuroblastoma | Xi'an Libang Pharmaceutical | Yes |
9 | Vigabatrin Powder for Oral Solution | Infantile spasms | Easton Biopharmaceuticals | Yes |
10 | Vigabatrin Powder for Oral Solution | Infantile spasms | Dr. Reddy's Laboratories | Yes |
11 | Clobazam Tablets | Lennox-Gastaut syndrome (LGS) | Yichang Humanwell | Yes |
12 | Romiplostim for Injection (ROMIPLATE) | Primary immune thrombocytopenia (ITP) | Kyowa Kirin | No |
13 | Luspatercept for Injection (REBLOZYL) | β-Thalassemia | Celgene | No |
14 | Emapalumab Injection (Gamifant) | Hemophagocytic lymphohistiocytosis (HLH) | Swedish Orphan Biovitrum | No |
15 | Human Interferon γ for Injection | Chronic granulomatous disease (CGD) (newly approved indication) | Shanghai Kaimao Biopharmaceutical | No |
16 | Mogamulizumab Injection (Poteligeo) | Mycosis fungoides and Sézary syndrome | Kyowa Kirin | No |
17 | Spesolimab Injection (Spevigo) | Pustular psoriasis | Boehringer Ingelheim | No |
Source: China NMPA Database | ||||
3. Pricing & Reimbursement
Rare disease drugs are generally expensive with their original prices. To make the drugs more affordable, China has been trying to include more rare disease drugs into the National Reimbursement Drug List (NRDL), a list of drugs fully or partially reimbursed by China’s social health insurance. To get products into the NRDL, companies usually need to negotiate with the National Healthcare Security Administration (NHSA). The negotiations generally involve price cuts.
In the latest round of NRDL adjustment, seven rare disease drugs entered the list.
Seven Rare Disease Drugs Added to 2022 NRDL | |||
No. | Drug | Company | Rare Disease |
1 | Lanadelumab Injection | Takeda | Hereditary angioedema (HAE) |
2 | Risdiplam Powder for Oral Solution | Roche | Spinal muscular atrophy (SMA) |
3 | Dimethyl Fumarate Enteric Capsules | Biogen | Multiple sclerosis (MS) |
4 | Ofatumumab Injection | Novartis | MS |
5 | Inebilizumab Injection | Horizon Therapeutics | Neuromyelitis optica spectrum disorders (NMOSD) |
6 | Riluzole Oral Suspension | Zhaoke Pharma | Amyotrophic lateral sclerosis (ALS) |
7 | Treprostinil Injection | Zhaoke Pharma | Pulmonary hypertension (PH) |
Source: China 2022 NRDL | |||
The rare disease drugs’ prices are lowered remarkably after NRDL admission. For example, Risdiplam’s price dropped from 63,800 yuan to 3,780 yuan per 60 mg.6
Another price-influencing policy is tax reduction. According to China’s 2023 Tariff Adjustment Plan, interim import tariffs are exempted for four active pharmaceutical ingredients (APIs) of rare disease drugs.
Four APIs with Provisional Zero Tariffs | |||
Tax Code | Product | 2023 Most-favored-nation (MFN) tariff rate % | 2023 Interim Tariff Rate % |
29309090 | Penicillamine | 6.5 | 0 |
29337900 | Pirfenidone | 9 | 0 |
29342000 | Riluzole | 6.5 | 0 |
29359000 | Bosentan | 6.5 | 0 |
Source: China 2023 Tariff Adjustment Plan | |||
BaiPharm’s Comment
China has made efforts and progress in approving and reimbursing rare disease drugs. But it will be better if more improvements are made in encouraging the development of new rare disease drugs, introducing overseas drugs into China, etc. to benefit both patients and pharma companies.
BaiPharm is a consulting company offering regulatory compliance and market access services for pharmaceuticals. Welcome to contact BaiPharm if you would like to know more about China’s drug regulations.
Related:
FAQ on Rare Disease Drugs (Orphan Drugs) in China
The 15th Rare Disease Day: China's Policy & Market Overview
In Light of the 14th Rare Disease Day, How Is China Doing on Rare Disease Drugs?
The First Catalog of Rare Diseases in China
List of Overseas New Drugs Urgently Needed in China’s Clinical Settings
China to Cut VAT on the 3rd List of Anti-cancer and Rare Disease Drugs
