Koselugo® (Selumetinib) for Neurofibromatosis Type 1 Included in China's 2023 National Reimbursement Drug List

Improve the accessibility of medicines for pediatric patients and reduce the economic burden on their families.

ShanghaiDecember 13, 2023PR Newswire -- Today, in the "National Basic Medical Insurance, Work-related Injury Insurance and Maternity Insurance Drug Catalog (2023)" (hereinafter referred to as the "Medical Insurance Catalog") published by the National Healthcare Security Administration and the Ministry of Human Resources and Social Security, AstraZeneca's rare disease drug Cosentyx^®(General Name: Koselugo, Selumetinib Sulfate Capsules) is listed therein. The "National Reimbursement Drug List (NRDL)" will officially take effect on January 1, 2024. Selumetinib was approved in China on April 28, 2023, for the treatment of pediatric patients aged 3 years and older with symptomatic, inoperable plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1). The inclusion of selumetinib in the NRDL will significantly improve the accessibility of this innovative drug for children with NF1, reduce the financial burden of treatment on their families, and thus bring positive changes to the growth and lives of these young patients.

Zhang Shuyang, President of Peking Union Medical College Hospital"China sees over 200,000 new cases of children with rare diseases each year. The impact of these children with rare diseases extends to at least 6-7 people, including the child's parents, their future offspring when they grow up, etc., affecting one-tenth of China's population."^[1]"We are delighted to see that, through this year's National Reimbursement Drug List (NRDL) negotiations, additional medications for rare pediatric diseases have been included. This marks another step forward in the collaboration between the government and enterprises to make rare disease drugs more 'affordable' for patients. However, despite the price reductions achieved through NRDL negotiations and their inclusion in the NRDL, which have improved patient accessibility, challenges remain in actual implementation. These include slow progress in equipping medical institutions, low regional coverage, and limited affordability. The level of implementation varies significantly across regions. Addressing these issues requires efforts and attention from multiple sectors of society to tackle the problem of medication access for rare disease populations. Continued efforts should be made to expedite the process for pediatric drugs to be covered under outpatient special disease and chronic disease benefits, ensuring that children with rare diseases can truly 'access' the medications they need."

NF1 is a rare, progressive genetic disorder, with 30%-50% of NF1 patients developing plexiform neurofibromas.^[2],[3]``, resulting in symptoms such as disfigurement, pain, impaired motor and airway function, vision damage, and bladder or bowel dysfunction.^{[1],[4],[5],[6],[7]}Plexiform neurofibromas often occur at birth, with varying degrees of severity, and may reduce life expectancy by up to 20 years compared to the general population.^{[1],[6],[8],[9],[10]}。

Ni Xin, President of Beijing Children's Hospital, Capital Medical UniversityNF1 patients face numerous clinical issues that require prolonged and frequent hospitalizations, severely impacting the lives and health of children. Currently, the main clinical treatments for NF1 include surgical resection and plastic surgery, but most NF1 cases are inoperable, and surgeries carry high risks and recurrence rates. Therefore, there is an urgent need for effective drug therapies for NF1. Selumetinib, as the only NF1-related plexiform neurofibroma treatment approved in China, has been included in the National Reimbursement Drug List (NRDL), demonstrating the nation's attention and care for children with NF1. This greatly improves the accessibility of the drug, which is particularly significant for children and families who previously could not afford the treatment costs, offering hope for a better life for more affected children.

Professor Li Qingfeng, Vice President of the Ninth People's Hospital affiliated to Shanghai Jiao Tong University School of Medicine, and Director of the Department of Plastic and Reconstructive Surgery"I-type neurofibromatosis (NF1) may present with plexiform neurofibromas in about half of the patients, which can cause pain, neurological dysfunction, and skeletal deformities, and can be life-threatening in severe cases; patients often develop symptoms during childhood. Moreover, due to the progressive rapid growth of plexiform neurofibromas during childhood and adolescence, it can easily lead to lifelong cognitive impairment, learning difficulties, and impaired social functioning in patients. The disease not only affects the physical health of children but also significantly hinders their daily lives."

Zou Yang from the Bubble Home Neurofibromatosis Care Center"I am deeply moved to tears as a staff member of the Neurofibromatosis Patient Organization and also a family member of a child patient upon the announcement of this medical insurance catalog. This has profoundly demonstrated the determination and acceleration of the country and government in ensuring that patients with rare diseases have access to and can afford medications. This is of great significance for further filling the gaps in patient accessibility. However, we still see challenges in the 'last mile' regarding the long-term availability and convenience of medication for rare disease patients—such as inconsistent regional coverage limits and, in some areas, medical insurance caps that fail to meet the ongoing medication needs of rare disease patients. Nevertheless, I believe that with continuous efforts from all parties and the research and implementation of solutions, every patient and family affected by rare diseases will truly gain access to necessary drugs and treatments, and welcome the dawn of overcoming these conditions."

Leo Wang, Executive Vice President of AstraZeneca Global, President of International Business and China"Children with NF1 and their families often bear a heavy disease burden, facing enormous challenges both physically and mentally. AstraZeneca always puts patients at the center, not only continuously providing high-quality innovative products for rare disease patients but also committing to promoting the accessibility and affordability of rare disease drugs in multiple ways. Besides cooperating with the government to include rare disease drugs in the medical insurance catalog through medical insurance negotiations, we have also been exploring multi-level medical security models with various partners and actively participating in building a sound rare disease diagnosis and treatment ecosystem, enabling rare disease patients to have access to, afford, and benefit from good medicines."