Childhood Cancer: What New Drugs Are Available Abroad and How to Use Them Domestically?

Childhood Cancer: What New Drugs Are Available Abroad and How to Use Them Domestically?

Source: Sunflower Children's Hospital  

Author: Professional Team  

Editor: Chen Li  

Date: December 28, 2024  

"Shortage of Medical Resources for Children’s Cancer"

Little Leo was diagnosed with neuroblastoma, and by the time it was discovered, the cancer had already spread throughout his body: tumors were found in his head, left jaw, spinal canal, and bone marrow. Children like him come from all corners of the country: Sichuan, Tianjin, Hebei, Heilongjiang... Regardless of where they are from, their experiences are strikingly similar: sudden onset of illness, multiple misdiagnoses, being shuttled between various hospitals until a malignant tumor is confirmed.

They all have "little bald heads." After losing their eyebrows and hair, they seem to have lost the vibrant colors typical of their age, creating a stark contrast with the brightly decorated cartoon figures in the hospital corridors and rooms. Even so, one must cautiously inquire with the parents standing nearby to discern the child's gender.

According to the World Health Organization (WHO), childhood malignant tumors have become the second leading cause of death among children, following accidental injuries. Over 1,000 children are diagnosed with cancer every day worldwide.

Childhood cancer is not a death sentence; in high-income countries, the survival rate for children with cancer is very high, with more than 80% of diagnosed children able to survive. However, in some low- and middle-income countries, only about 20% of children diagnosed with cancer are able to survive.

One reason for this disparity in survival rates is the difference in effective treatment options available.

The phrase "shortage of medical resources" perfectly summarizes the current state of childhood cancer treatment. Pediatric tumors are not merely smaller versions of adult tumors; they each have their own characteristics.

Although relevant departments in Europe and the United States are actively working to promote the development of pediatric drugs, there remains a significant gap in the number of approved pediatric cancer drugs as compared to those for adults. The population of pediatric cancer patients is considerably smaller than that of adult cancer patients. Thus, for economic reasons, very few companies are willing to develop drugs specifically for pediatric tumors and rare diseases.

Moreover, the timeline for expanding adult drugs to include pediatric indications is generally quite lengthy. In January 2021, the FDA approved a supplemental new drug application (sNDA) for the targeted cancer drug Xalkori (crizotinib), to treat children and young adults with anaplastic lymphoma kinase (ALK)-positive, relapsed or refractory systemic anaplastic large cell lymphoma (ALCL). ALCL is a rare form of non-Hodgkin lymphoma (NHL), accounting for about 30% of NHL cases in young people. Approximately 90% of ALCL cases in youth are ALK-positive. This ALK inhibitor has actually been used for lung cancer treatment since 2011.

Even with FDA approval, pediatric cancer drugs still require further review and approval by the National Medical Products Administration (NMPA) in China. Of the 44 pediatric cancer drugs approved by the U.S. National Cancer Institute, 30 have been approved by the NMPA, and among these, 18 drugs have been approved for pediatric indications.

"More than 25 Innovative Drugs Approved Abroad"  

"Policy Support May Bring New Hope"

Given the real challenges of clinical trials, policy support will be a crucial element in the development of pediatric drugs. To allow patients to access new drugs more quickly, China has approved the use of drugs that are available abroad but not yet on the domestic market in the Boao Lecheng Pilot Zone in Hainan. This initiative allows for real-world data collection to support future domestic approvals. Such an approach will play a significant role in the field of pediatric drugs.

For example, regarding little Leo's neuroblastoma, children in China have been able to access targeted therapies that are not yet available domestically since 2022. Between January 2022 and March 2024, the NMPA approved 10 pediatric oncology drugs, including daratumumab, which was approved for sale in 2021, and selumetinib, which is used for neurofibromatosis type 1. The drug naxitamab was approved at the end of 2022 for treating relapsed or refractory high-risk neuroblastoma in children aged one year and older.

NMPA Approved Pediatric Oncology Drugs Overview

According to the "Handbook of Innovative Drugs for Childhood Cancer" compiled by the Hainan Provincial Women and Children’s Medical Center and the Clinton Health Access Initiative (CHAI), as of March 2024, more than 25 innovative drugs for childhood cancer have been launched abroad but have not yet received domestic registration or pediatric indications. It is hoped that this handbook will provide more information for families of affected children in China, igniting further hope.

"Some Approved Pediatric Oncology Innovative Drugs Not Yet Launched Domestically"

Of course, not all news surrounding children with cancer is bad.

One year and seven months old, little Mia also suffered from neuroblastoma. After 13 months and traveling 15,000 kilometers, undergoing chemotherapy, surgery, and GD2-targeted therapy, five-year-old Mia has made a complete recovery and can even participate in mini marathons and long-distance hiking activities!

Hope springs eternal that, in the near future, through the further efforts of the government, enterprises, doctors, and social welfare advocates, more children will conquer cancer and regain their health!

Editor: Huang Shi  

Typesetting: Xia Yu  

Proofreader: He Fei