Accessing Innovative Orphan Therapies in China: Local Advancements

One of the most promising aspects of China's orphan drug access landscape is found at the local level. Provinces are pioneering innovative methods to fund and expedite access to innovative therapies for patients with rare diseases.

Improving Affordability

Local funding mechanisms for rare disease therapies are gradually evolving into several models in China, such as the "dedicated rare disease fund" model in Qingdao, the "critical illness insurance" model in Zhejiang and Jiangsu, the "medical assistance" model in Foshan, and the "commercial health insurance" model in Beijing and Shanghai.

These pilot programs primarily aim to establish independent funding pools outside the basic medical insurance catalog, providing coverage for high-priced orphan therapies not included in the catalog.

Qingdao relies on fiscal allocations, contributions from insured individuals (20 yuan per person per year), BMI personal accounts, and special reserve funds to establish a dedicated rare disease fund.

Zhejiang and Jiangsu require provincial critical illness insurance participants to pay 2 yuan per person per year to create a separate funding pool for rare diseases.

Foshan utilizes government aid, lottery funds, and charity as a rare disease funding model.

In terms of commercial health insurance, Beijing and Shanghai leverage City Commercial Health Insurance ("Huiminbao") to fund orphan drug access.

Beijing has connected Boao Lecheng Medical Pilot Zone and Beijing “Puhuibao.” Insured individuals who receive treatment with drugs listed in the Boao Lecheng formulary can be reimbursed through Beijing City Commercial Health Insurance (CHI). Meanwhile, Shanghai's "Huhuibao" includes some ultra-rare diseases in the specific high-cost drug protection catalog, allowing reimbursement rates for these drugs to reach 70%.

Despite these encouraging developments, currently, the absence of a dedicated rare disease fund is the biggest 'shortcoming' in China's diversified security system for rare diseases.

Accelerating Access

Multinational companies are leveraging local initiatives that prioritize and trial innovative drugs to expedite the introduction of new orphan therapies in China. These policies provide a unique opportunity to treat patients with drugs and devices approved overseas but not yet in China.

The Beijing Tianzhu Comprehensive Bonded Zone has established a pilot area for rare disease drug coverage, implementing a unique 'white list' system. This system, which includes a white list for rare disease drugs, pilot medical institutions, and import drug trading enterprises, is designed to expedite the approval and import process for rare disease drugs. It allows for single approval, multiple imports, and use by multiple medical institutions, with the ambitious goal of fully implementing ten types of RD drugs by 2024.

Currently, VOXZOGO® for treating achondroplasia in pediatric patients, Deflazacort for treating Duchenne muscular dystrophy (DMD), and Cablivi® for treating acquired thrombotic thrombocytopenic purpura (aTTP) have all been introduced into the Beijing Tianzhu rare disease drug pilot zone, providing new treatment options for patients.

The Hainan BoAo Lecheng International Medical Tourism Pilot Zone, also known as Hope City, is another leading destination for many innovative therapies entering China. Recently, multinational pharmaceutical companies have begun exploring breakthrough enzyme replacement therapy for patients with acid sphingomyelinase deficiency (ASMD) in this zone.

Advancing Rare Disease Diagnosis and Treatment

Since the introduction of China's First List of Rare Diseases, the National Network to Collaborate on Diagnosis and Treatment of Rare Diseases (NCDTRD) was established in February 2019, encompassing 324 hospitals to enhance rare disease patient care.

More recently, the China Alliance for Rare Diseases' STEP Project has released guidelines, formed collaboration groups, and established diagnosis and treatment centers for Lysosomal Storage Disorders (LSDs).

In July 2023, multinational companies supported the China Primary Health Care Foundation in launching a nationwide high-risk screening project for Lysosomal Storage Disorders (LSDs). Over four years, this initiative involved 425 hospitals, conducted 37,900 free tests, and diagnosed approximately 1,231 patients, significantly enhancing early and accurate diagnoses for rare diseases such as Gaucher disease, Pompe disease, Fabry disease, and mucopolysaccharidosis type I (MPS I).

References:

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2. Understanding China’s Access Environment for Orphan Therapies: Advancing Policies and Diagnosis & Treatment Capacities, NRDL+ Newsletter, 2023.6.7., https://www.nrdlplus.com/understanding-chinas-access-environment-for-orphan-therapies-advancing-policies-and-diagnosis-treatment-capacities/
3. Navigating China's Access Landscape for Orphan Therapies: Insights into Funding Pathways, NRDL+ Newsletter, 2023.6.21, https://www.nrdlplus.com/navigating-chinas-access-landscape-for-orphan-therapies-insights-into-funding-pathways/
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