2024 National Health Insurance Negotiation: Which Pediatric Cancer and Rare Disease Medications Are Included?

2024 National Health Insurance Negotiation: Which Pediatric Cancer and Rare Disease Medications Are Included?

Source: Sunflower Children  

Author: Huang Shi  

Editor: Zhengty  

Date: December 24, 2024  

On the morning of November 28, the National Healthcare Security Administration held a press conference to introduce the adjustments to the national health insurance drug catalog for 2024 and announced the new drug list. This adjustment added a total of 91 new medications, including 26 oncology drugs (4 of which are for rare diseases), 15 medications for chronic diseases such as diabetes (including 2 for rare diseases), 13 rare disease medications, 7 anti-infection drugs, 11 traditional Chinese medicines, 4 psychiatric medications, and 21 drugs from other fields.

Let’s take a look at which pediatric oncology and rare disease medications have been included in this year's national health insurance negotiations and which drugs are worth looking forward to.

Larotrectinib Sulfate

Larotrectinib sulfate capsules/oral solution may be one of the highlights among pediatric oncology drugs in this health insurance negotiation. It is the world's first targeted drug that does not differentiate based on tumor origin. This drug is a tropomyosin receptor kinase (TRK) inhibitor, which works by blocking certain cellular signals that may help prevent cancer cell growth. Clinical results from six independent expansion analyses related to larotrectinib were presented at the 2022 ASCO conference, involving patients with 25 different types of tumors. Common cancer types include soft tissue sarcoma (46%; infantile fibrosarcoma: 19%, other sarcomas: 27%), thyroid cancer (12%), lung cancer (9%), salivary gland cancer (10%), and colorectal cancer (6%). There have already been reports of effective treatment data for larotrectinib in various pediatric and adult patients. It appears to be more effective in pediatric patients than in adults. Larotrectinib acts quickly, with cases of rapid tumor shrinkage observed about a week after medication initiation. Most children achieve partial response (PR) or complete response (CR) within 1 to 2 treatment cycles.

Polatuzumab Vedotin

Another standout drug is Polatuzumab vedotin, an injectable treatment. It is a new hope for pediatric lymphoma patients and elderly lymphoma patients alike. This drug belongs to a class known as antibody-drug conjugates and is used to treat a type of malignant lymphoma called diffuse large B-cell lymphoma (DLBCL). A major characteristic of lymphoma is that it becomes harder to treat with each recurrence. The risk of relapse in diffuse large B-cell lymphoma is very high. Even patients receiving initial treatment with R-CHOP therapy have a 40% chance of progressing to relapse or refractory status. However, existing second-line and subsequent treatment options for relapsed/refractory DLBCL show that the 5-year and 10-year overall survival rates for patients are only 25.0% and 8.6%, respectively. These patients often have poorer prognoses and shorter survival times. The advent of Polatuzumab vedotin has broken the treatment dilemma for relapsed/refractory DLBCL. A global multicenter randomized controlled trial (GO29365) showed that Polatuzumab vedotin combined with bendamustine and rituximab for the treatment of relapsed/refractory diffuse large B-cell lymphoma extended the median overall survival of patients compared to those receiving bendamustine and rituximab alone by nearly three times, reducing the risk of death by 58%.

Waiting for CAR-T Products: Uncertain Future

In the field of pediatric cancer treatment, no one is unaware of Emily's story. She is the first person in the world to be "cured" by CAR-T therapy and has been cancer-free for over 12 years. In May 2010, just 5 years old, Emily was diagnosed with acute lymphoblastic leukemia (ALL). In April 2012, she enrolled in a CAR-T clinical trial, becoming the first child in the world to receive CAR-T cell therapy. By 2024, Emily is now 18 years old and has been "cured" of cancer for over 12 years. She has been accepted into the University of Pennsylvania, where she received her treatment, and is starting a new chapter in her life.

Currently, CAR-T cell therapies in China primarily target diffuse large B-cell lymphoma, refractory or relapsed multiple myeloma, and refractory follicular lymphoma. However, due to their high costs, these therapies were not included in this year's health insurance negotiations. "No negotiations for drugs over 500,000 yuan; drugs over 300,000 yuan won't be included even if negotiated." This is an unwritten rule from the National Healthcare Security Administration regarding "sky-high prices." Currently, six CAR-T drugs have been approved in mainland China, four of which applied to participate in this year's health insurance negotiations. However, as a new type of precision targeted therapy for cancer, CAR-T drugs generally cost over one million yuan. Without effective cost reductions, it is unlikely that we will see CAR-T drugs included in the health insurance list in the foreseeable future.

Medications for Pediatric Rare Diseases

In addition to oncology drugs, there are also medications for pediatric rare diseases included in the health insurance plan. Eculizumab (Eculizumab hydrochloride capsules) has been successfully included; it is the world’s first and only approved specific complement bypass factor B inhibitor and the oral monotherapy for adult paroxysmal nocturnal hemoglobinuria (PNH). This offers PNH patients the possibility for a better quality of life and brings hope to more families affected by rare diseases. According to the China Rare Disease Alliance, a total of 249 out-of-catalog drugs passed formal review during this health insurance catalog negotiation, including 38 rare disease medications. Ultimately, 13 rare disease drugs succeeded in negotiation or bidding, bringing a glimmer of hope to affected patient groups.

Transplant Complications Medications

Additionally, the world's first and only approved ROCK2 inhibitor for chronic graft-versus-host disease (cGVHD) in patients aged 12 and older who have inadequate response to corticosteroid therapy—Belumosudil (Breztri) has also successfully entered the health insurance list. In recent years, allogeneic hematopoietic stem cell transplantation has rapidly developed in China, with numbers ranking first globally in 2023. However, chronic graft-versus-host disease (cGVHD) has become a significant clinical challenge post-transplant. The standard first-line treatment for cGVHD is corticosteroid therapy (steroid pulse), but this treatment has considerable side effects. With the emergence of innovative drugs like ROCK2 inhibitors, the challenges of treating fibrosis in refractory cGVHD may be effectively reversed. The inclusion of these drugs in the health insurance catalog will help improve the survival and quality of life for more cGVHD patients.

Rare Tumor Medications

Compared to previous years, this year has also seen several rare tumor medications included in the health insurance catalog. This is a new change following last year's rare disease catalog adjustments, as some rare tumors have been included in the second batch of rare disease listings. These drugs include Berotralstat capsules for glioblastoma, Narsofermin injection for giant cell tumors of bone, Trametinib capsules (indicated for NRAS mutation advanced melanoma patients failing anti-PD-1/PD-L1 treatment), Daratumumab injection for multiple myeloma, and Octreotide acetate microspheres for gastrointestinal and pancreatic neuroendocrine tumors. However, these rare tumor medications have not yet received approval for pediatric indications.

Overall, the inclusion of pediatric medications in this year's national negotiations appears to be "low-key." In the national negotiations of 2021 and 2022, the number of newly added pediatric medications surged from single digits to 34 and 22, marking "big years" for pediatric medications entering health insurance. After that, in the 2023 negotiations, only 9 new pediatric medications were added, and this year's new additions have not been clearly announced. The Sunflower Children’s Association will continue to monitor this topic, hoping for more new medications for pediatric cancers next year and for more existing drugs to be included for pediatric indications, enabling more families of children with tumors to access and afford their needed medications.

Igniting Hope with Expertise!

References:  

1. Xinhua Health | "Medicine Box" Upgraded Again: Health Insurance Drug Catalog Continues to "Refresh" for Seven Years  

2. The Seventh Health Insurance Negotiation Begins: Multiple Star Innovative Drugs Compete  

3. The 2024 Health Insurance Negotiation Concludes: A Big Year for Innovative Drugs, the Era of Bispecific Antibodies and ADCs Begins  

4. Breaking News | 13 New Rare Disease Medications Added! 2024 National Health Insurance Drug Catalog Updated  

5. Chinese Expert Consensus on the Diagnosis and Treatment of Chronic Graft-Versus-Host Disease (cGVHD) (2021 Edition)  

6. Expert Consensus on Larotrectinib Treatment for TRK Fusion Pediatric Tumors in China (2023)  

Author: Huang Shi  

Cover Source: Visual China  

Layout: Xia Yu  

Proofreading: Xin Yue