Evrysdi® (Risdiplam) Included in China's National Reimbursement Drug List to Enhance Access for SMA Patients

ShanghaiJanuary 19, 2023PR Newswire -- On January 18, 2023, the National Healthcare Security Administration and the Ministry of Human Resources and Social Security issued the latest announcement regarding Roche's drug Evrysdi.^®(Evrysdi Oral Solution for Suspension) has been officially included in the "National Basic Medical Insurance, Work-related Injury Insurance, and Maternity Insurance Drug Catalog (2022 Edition)" (hereinafter referred to as the "National Medical Insurance Catalog"). This move reflects the country's high attention to ensuring medication access and health rights for patients with rare diseases, marking another significant milestone in the joint efforts of all parties to alleviate the burden on patients and families affected by Spinal Muscular Atrophy (hereinafter referred to as "SMA").

Evrysdi^®In June 2021, it was officially approved by the National Medical Products Administration for the treatment of SMA patients aged 2 months and above, becoming the first orally administered disease-modifying treatment approved in China for the treatment of SMA.

Focus on Long-term Diagnosis and Treatment Dilemmas,Unmet needs in SMA remain

SMA is a rare hereditary neuromuscular disease, with an incidence rate of 1 in 10,000 live newborns.^[1], with approximately 1,000 new SMA patients added in China each year.^[2]; About 80% of patients will develop the disease within 18 months after birth.^[3], the progression of the disease can lead to abnormalities in multiple systems throughout the body. Without effective treatment, most infants with severe SMA will pass away before the age of 2.^[4]。

Due to the lack of overall awareness of diseases in society, SMA patients and their families have long faced challenges in obtaining a confirmed diagnosis and prolonged diagnostic times. Additionally, the limited number of users for rare disease medications, coupled with high research and development costs and significant difficulties, has led to extremely restricted treatment options and low affordability. Therefore, it is an urgent wish for many SMA patients and their families that more drugs with proven efficacy and convenient usage be included in medical insurance.

Moreover, like most rare disease groups, many SMA families are also plagued by numerous practical challenges such as heavy caregiving burdens and difficulties in integrating normally into society, urgently needing the support of a more comprehensive social security system.

Oral Medications Show Advantages, Medical Insurance Boosts Innovation Accessibility

Since gaining U.S. FDA approval in August 2020, Evrysdi^®It has been approved in more than 80 countries worldwide, with thousands of patients in the real world having received Evrysdi.^®The treatment has been widely proven for its efficacy and safety. As the first small-molecule disease-modifying therapeutic drug targeting mRNA, it can penetrate the blood-brain barrier, distribute in central and peripheral tissues, and systemically increase SMN protein levels. Research results show that Evrysdi^®Type 1 SMA patients treated have shown significantly improved survival rates compared to natural history, achieving motor milestones and improvements in respiratory and swallowing functions.^[5]; Patients with Type 2 and Type 3 SMA showed improvement in motor function and life independence after medication.

Its breakthrough oral administration method allows patients to undergo home treatment without hospitalization, invasive surgery, or the concurrent use of other medications. This not only improves patient compliance with medication but also alleviates the pressure on public healthcare resources in hospitals. Meanwhile, Evrysdi^®The use of stepwise dosing further reduces the medication burden for younger patients, facilitating earlier treatment.

"Risdiplam has brought China's SMA treatment into the 'oral era.' Its inclusion in the national medical insurance catalog will significantly improve the accessibility of innovative therapies, reduce the burden on patients and their families, and provide an important supplement to existing clinical treatment options, addressing the limitations of drugs currently in the catalog. For many SMA families who have long faced the dilemma of 'lack of medical resources and medicines,' the availability of innovative drugs also means the accessibility of 'hope for life,'" said Professor Wang Yi from Fudan University Affiliated Children’s Hospital. "In the long term, this will also drive the development and improvement of China's overall SMA diagnostic and treatment standards, as well as comprehensive disease management."

"SMA is a disease that requires lifelong treatment, and its long-term treatment burden has always been a focal point of attention for all parties. As a patient care organization, we have accompanied patient families through many twists and turns and moments of helplessness, which is why we are particularly excited about the inclusion of innovative oral medications in medical insurance. The continuous improvement in treatment accessibility will more effectively support us in fulfilling our mission to 'help patient families face the disease with more hope and dignity,'" said Xing Huanping, Executive Director of the Meier SMA Care Center.

Policy Efforts in Rare Disease Prevention and Treatment, Government-Enterprise Collaboration Accelerates the Improvement of Guarantees

In 2018, the National Health Commission and five other departments jointly formulated the "First List of Rare Diseases," which included SMA. The "Rare Disease Diagnosis and Treatment Guidelines (2019 Edition)" were also released the following year; the National Medical Products Administration successively issued three batches of the "List of Overseas New Drugs Urgently Needed Clinically," along with the priority review and approval policy, accelerating the market entry of rare disease drugs in China. This series of measures demonstrates the country's attention to the rare disease population and its determination to advance the cause of rare disease prevention and treatment as well as implement the national strategic goal of universal health. Evrysdi^®The accelerated approval and market launch in China through the priority review and approval channel, as well as its inclusion in the medical insurance this time, are vivid proof of this determination.

In response to the national call, Roche has been actively contributing to the construction of a multi-level medical security system for rare diseases by launching charitable assistance programs, participating in national medical insurance negotiations, and engaging in city-level惠民insurance expansion. At the same time, Roche collaborates with various sectors to carry out series of disease education and humanistic care projects such as the "Empowering the Future – SMA Super Mom Comprehensive Support Initiative," aiming to gather broader forces to build an integrated, comprehensive support system encompassing "screening, diagnosis, treatment, and assurance" for patient families.

The "successive good news" in the SMA field is a microcosm of Roche's deep cultivation in the neuroscience field. Facing one of the most difficult disease areas to conquer and cure, Roche is stepping up efforts to advance and expand its related product pipeline, moving towards the goal of "freeing humanity from the burden of neurological diseases and creating a tomorrow where the nervous system no longer limits human potential."

^[1] Wang Wenqiao, Mao Shanshan, Ma Ming. "Research Progress on Nutritional Management in Children with Spinal Muscular Atrophy." Journal of Clinical Pediatrics 40.3 (2022): 235-240.

^[2] National Bureau of Statistics. Statistical Communiqué of the People's Republic of China on the 2021 National Economic and Social Development [EB/OL]. http://www.stats.gov.cn/tjsj/zxfb/202202/t20220227_1827960.html, 2022-10-25.

^[3] Expert Consensus on Multidisciplinary Management of Spinal Muscular Atrophy. Chinese Medical Journal. 2019. 99(19): 1460-1467.

^[4] Office of the National Health Commission. Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition) [Z]. 2019-02-27

^[5] Baranello G, et al. N Engl J Med. 2021 Mar 11;384(10):915-923.