Zhejiang Province Announces Results of Rare Disease Drug Price Negotiations: Sanofi's Myozyme and Fabrazyme Included-VCBeat

On June 2, the Zhejiang Provincial Healthcare Security Administration publicly announced the negotiation results for two rare-disease specialty drugs on its official website. Sanofi’s alglucosidase alfa (brand name: Myozyme) and agalsidase beta (brand name: Fabrazyme) were included. The negotiation process commenced with a notice issued on April 30, 2020, followed by negotiations on May 21, and the results were released on June 2, spanning slightly over one month. The public comment period for the negotiation results ran from June 2, 2020, to June 10, 2020.

Negotiation Results for Special Drugs for Rare Diseases in Zhejiang Province

Zhejiang's Medical Insurance Policy for Special Drugs for Rare Diseases

At the end of last year, Zhejiang Province issued the “Notice on Establishing a Guarantee Mechanism for Rare Disease Medications in Zhejiang Province,” which specified that the Provincial Healthcare Security Administration would, in accordance with“Expert Review, Price Negotiation, and Dynamic Adjustment”principles, based on the rare disease drugs recommended by the Provincial Expert Committee on Rare Disease Medications, and taking into account factors such as the province’s level of economic and social development and the balance of the Rare Disease Drug Security Fund, organize and conduct negotiations on rare disease medications.

Covered individuals are: (1) insured persons under the Basic Medical Insurance of Zhejiang Province who have held Zhejiang household registration (hukou) for at least five years at the time of initial diagnosis; and (2) insured persons under the Basic Medical Insurance of Zhejiang Province who are under five years of age, hold Zhejiang household registration (hukou), and whose biological father or mother has held Zhejiang household registration (hukou) for at least five years.

Regarding cost coverage, when patients with rare diseases receive treatment at designated hospitals, they are responsible for paying the out-of-pocket portion, while the remaining costs are settled directly between the medical insurance agency and the designated hospital. Medications for rare diseases are excluded from the total medical insurance budget management and the drug proportion assessment metrics of the designated treatment hospitals.

Introduction to Two Selected Medicines and Rare Disease Treatments

The two aforementioned negotiated drugs are specialty medications belonging to the therapeutic areas of Pompe disease and Fabry disease, bringing hope to patients in China.

Meierzhan

Myozyme is a specialty drug developed by Sanofi for the treatment of Pompe disease. It was first launched in the United States in 2007 and received approval from the China Food and Drug Administration (CFDA) in late 2016 for the treatment of patients with Pompe disease.

Pompe disease is an autosomal recessive disorder caused by a deficiency of acid alpha-glucosidase. Based on the timing of symptom and sign onset, it is generally classified into the infantile form and the juvenile/adult-onset form. The incidence in newborns is only 1 in 40,000. Due to the high technical requirements for diagnosis and the low incidence rate, clinical diagnosis is challenging. The average diagnostic delay worldwide is as long as 7–10 years. For patients with infantile Pompe disease, most die within one to two years after diagnosis.

Pompe disease causes severe and irreversible damage to patients' muscles, including cardiac muscle, skeletal muscle, and respiratory muscles. While children of the same age are already able to run and jump, patients with Pompe disease are confined to wheelchairs, rely on ventilators for survival, and inevitably progress toward death, causing immense suffering for both patients and their families.

Fabrazyme

Fabrazyme is a specific therapeutic agent for the treatment of Fabry disease. In late December 2019, it was approved by the National Medical Products Administration for long-term enzyme replacement therapy in patients diagnosed with Fabry disease, including adults, children, and adolescents aged 8 years and older. This marks the first approved specific therapy for Fabry disease in China. Currently, Fabrazyme is marketed in multiple countries and regions, including Europe and the United States.

Fabry disease (also known as Fabry's disease) is a rare X-linked lysosomal storage disorder. Patients often experience burning pain in their hands and feet, which can last from a few minutes to several days, sometimes recurring. In severe cases, it can significantly impair daily life and cause serious damage to organs such as the kidneys, heart, brain, and nerves. Without effective treatment, it poses a significant threat to life. Symptoms of this disease typically appear during childhood or adolescence. Additionally, male patients tend to have more severe clinical symptoms than female patients. The average lifespan of male patients is shortened by 20 years compared to the general population, while for female patients, it is reduced by approximately 10 years. According to statistics, there are over 300 diagnosed Fabry disease patients in China.