Biogen's Drug for Rare Disease SMA Gets Access to More Patients in China Due to NRDL

by Grace Wang Feb 14, 2022

In December 2021, Biogen cut the price of Spinraza (Nusinersen Sodium Injection) from 700,000 yuan per dose to 33,000 yuan per dose. The move was for getting a place in China's National Reimbursement Drug List (NRDL).

Thanks to the price cut, nearly 30 patients1 across China got administered with Spinraza on Jan. 1, the first day of the new NRDL took effect.

Spinraza Entered the Chinese SMA Treatment Market Via Priority Review

SMA, a rare disease characterized by muscle atrophy or weakness, currently affects 30,000-50,000 patients in China.2 The disease's incidence is 1/10,000-1/6,000 among infants. According to estimation, about 1,023 to 1,705 among the average 10,232,667 newborn babies4 in China during the past three years suffer from SMA.

Biogen became the first SMA therapy approved by China National Medical Administration on Feb. 25, 2019, about two years after the approval by the U.S. Food and Drug Administration on Dec. 23, 2016.5.

Given Spinraza's significance in treating SMA patients, NMPA granted the priority review designation to the drug and completed the review within only 173 days.

Thus, Spinraza established its advantage by becoming the first drug in China's SMA treatment market.

However, the drug was too expensive for patients from average families until it got included in the NRDL.

Being listed in NRDL: Pathway to More Patients

The inclusion into the NRDL enhances Spinraza's market position with a lowered price. Each injection costs less than 33,000 yuan thanks to social healthcare insurance.

Based on different reimbursement rates in different provinces, each Spinraza injection will cost around 10,000-17,000 yuan6. The initial course of treatment needs four doses of Spinraza within 63 days. After that, a maintenance dose should be administered every four months. So the first year's medical expense is estimated to be about 100,000 yuan, and each following year around 50,000 yuan.

Besides, the NRDL is connected with the drug procurement by public hospitals. After being included into the NRDL, Spinraza has been listed on 34 provincial drug procurement platforms by Jan. 4, so the drug expects a higher profile across the Chinese market.

China's Policy for Rare Diseases Drugs

From marketing approval to inclusion into the NRDL, Spinraza marks an example of successfully utilizing the China's preferential regulations and policies for rare disease drugs.

China's Regulations and Policies for Rare Disease Drugs

Clinical Data

For importing overseas drugs into China:

Foreign clinical data is permitted for supporting marketing approval if there's no ethnic difference in the data.7

Review and Approval

For overseas new rare disease drugs with urgent clinical needs but have not been approved in China:

- Priority review program: technical review will be completed within 70 workdays.8

- Dedicated review channel: technical review will be completed within three months from the acceptance of the application.9

*List of Overseas New Drugs Urgently Needed in China's Clinical Settings

Tax

For Chinese domestic and imported drugs:

Value-added tax (VAT) for the first batch of 21 rare disease drugs and four active pharmaceutical ingredients has been reduced to 3% since Mar. 31, 2019.10

Apart from the measures mentioned above, China issued the First Catalog of Rare Diseases to encourage pharmaceutical companies to develop therapies for the listed diseases. 

China also tries to extend the NRDL to cover more drugs, so that they can be reimbursed and affordable to more patients.

Rare Disease Drugs Added to NRDL in 2021

No.

Drug

Disease

Category

1

Human Coagulation Factor Ⅸ

Hemophilia

Category II

2

Icatibant Acetate Injection

Hereditary angioedema

Category II

3

Nusinersen Sodium Injection

Spinal muscular atrophy (SMA)

Category II

4

Fampridine Sustained-release Tablets

Multiple sclerosis (MS)

Category II

5

Agalsidase Alfa Concentrated Solution for Infusion

Fabry disease

Category II

6

Evolocumab Injection

Heterozygous familial hypercholesterolemia (HeFH) and cardiovascular disease.

Category II

7

Tafamidis Soft Capsules

Transthyretin amyloid cardiomyopathy (ATTR-CM)

Category II

Notes:

Ÿ   Category I drugs are 100% covered by the reimbursement of the basic medical care insurance system; category II drugs are partially reimbursed with varied rates in different regions.

Ÿ   MAH refers to marketing authorization holder.

China currently has more than 20 million rare disease patients.11 In such an emerging market, BaiPharm recommends drug makers leverage the government policies to establish advantages. Contact BaiPharm if you need help with pharmaceutical market entry projects.

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Grace Wang
ChemLinked Regulatory Analyst
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