Childhood cancer drug current landscape and pipeline characteristics

Published: October 2023

WHO analysed the current landscape and pipeline of childhood cancer drugs up through July 2022. Products are reported by drug category, type, target, phase of development, regulatory approvals, route of administration, oral formulation, storage temperature, light protection and malignancy type. Trials are reported by countries where trials are conducted, WHO Region, income group, and sponsor. See below for details on the scope, analysis, and limitations.

See also:

What you see | Scope, analysis and limitations | Data sources 

What you see

The data visualization shows the:

• Tab 1: Number of childhood cancer drugs by:
  
  • Drug category (chart A.1)
  • Drug type (chart A.2)
  • Target (chart A.3)
  • Phase (chart B.1)
  • Route of administration (chart B.2)
  • Oral formulation (charts B.3 and B.4)
  • Storage temperature (chart C.1)
  • Light protection (chart C.2)
  • Malignancy type (chart D.1 and chart D.2)
• List of childhood cancer drugs by phase, category, type, and malignancy type, with further information on each product (hover over the malignancy types in chart E to open a pop-up window for additional information, including a list of all registered clinical trials for drugs in either phase I or phase II ).

• Tab 2: Number of clinical trials on childhood cancer drugs by:
  
  • Location where trials are conducted (chart F.1, chart F.2 and chart F.3)
  • Sponsor (chart G)
  • Year of trial registration (chart H)

Products can also be viewed by grouped phases (select the phases of interest using top left tick boxes).

Note: A drug can have multiple targets, routes of administration, and formulations. They can also be studied for multiple diseases and covered by multiple trials present in multiple locations-- the numbers displayed in these charts may total to more than the total number of drugs or trials.

Points to note:

Tab 1: Overview of the childhood cancer drugs

• Out of the 440 drugs identified:
  
  • Drugs were divided into 9 general drug categories. The three most common drug categories  were molecular targeted therapies (135; 31%), followed by immunotherapy (108; 25%) and then cytotoxic chemotherapy (93; 21%). Together, the top two categories, molecular targeted and immunotherapy made up 56% of the drugs currently in use (chart A.1).
  • Drugs were divided into more specific drug types, based upon physical characteristics and/or mechanism of action. Of those, the three most common were small molecule  (139; 32%) followed by monoclonal antibody (55; 13%), and then vaccine (49; 11%) (chart A.2).
  • 132 specific drug targets of molecular targeted or immunotherapy were identified. By clicking on a specific target (chart A.3), the drugs with that specific target will be listed in chart E and characteristics of those specific drugs in the remainder of the visualization charts. Certain drugs may have more than one target
  • Approximately two thirds of all drugs are in phase I (98; 22%) or in phase II (176; 40%) of development.
  • The most common routes of administration are intravenous (196; 45%), and oral (170; 39%) (chart B.3).
  • Only 33% of the oral drugs studied were available in paediatric friendly formulations (57 out of 170 drugs) (chart B.3). Criteria for paediatric-friendly included at least one of the following: commercial oral liquid, data available regarding compounding into liquid, available crushable formulation.
  • 18% (78) of the drugs require refrigeration and 25% (112)  require light protection. This information was not available for approximately 50% of all the drugs (charts C).
  • Gliomas, neuroblastoma, and osteosarcoma were the top three malignancies with the highest number of drugs in clinical trials. Note that one drug can be studied for multiple malignancies (charts D).

Tab 2: Overview of the clinical trials on childhood cancer drugs

• A total of 2,159  childhood cancer clinical trials have been registered in the ICTRP database between 2007-2022 of which:
  
  • 47% (1,006 trials) are conducted in the region of the Americas, followed by the Western Pacific region (843; 39%) and the European Region (588; 27%) (chart E.1).
  • 74% (1,601 trials) are located in high income countries (chart E.2).

To explore the data further

• Select the grouped phases (top left tick boxes) to see the drugs and trials characteristics by these groups.
• Select a specific malignancy type (chart D.2) or any other specific element or combination of elements to display the corresponding data in the other charts. For example, by selecting Gliomas in chart D.2, we can see that 156 drugs are studied for this malignancy type, of which 62 (40%) are at phase II (chart B.1). 70 are available in oral formulation (chart B.2) of which 24 are paediatric friendly (chart B.3). 
  
• Hold the ‘Ctrl’ key on your keyboard to select more than one option. For example, in addition to the selection above, by selecting phase II in chart B.1, we can see that 10% of the 62 corresponding drugs are known to require light protection (chart C.2).
• Hover the cursor on a bar or a cell in a table to see more information in a pop-up window. For example, hover over the malignancy type in chart E to see the list of corresponding clinical trials (the list of clinical trials is only available for drugs in phase I and phase II)
• Undo a selection by clicking ‘undo’ or ‘reset’ near the bottom of the page or by clicking the same element again.

Scope, analysis and limitations of the data

Scope

• This current landscape and pipeline analysis focuses on drugs in use in paediatric cancer clinical trials registered over the past 15 years (2007-July 2022) with information on the specific drugs included in each of the trials (categories, targets, types, phases, storage needs, etc.).
• Trials were restricted to cancer treatment studies (registries, biology studies, supportive care studies, psychosocial studies, etc. were excluded). Minimum age of study participants had to be less than 16 years old.
• Data regarding Chimeric antigen receptor (CAR) T-cell therapy were also collected, analysed separately and displayed on a different dashboard.

Analysis

• Data collection involved the following sources:
  
  • Drug lists:
    • Drugs were extracted by reviewing the full entries of all relevant trials for childhood cancers registered in the International Clinical Trials Registry Platform (ICTRP)
  • Drug details information were collected from the following sources:
    • International Clinical Trials Registry Platform (ICTRP) trial entry
    • Clinical information platform and literature
    • Drug information embedded in individual protocols
    • Direct communication by email or phone with principal investigator or drug company, where applicable.
  • Trial detailed information was collected from:
    • International Clinical Trials Registry Platform (ICTRP)
• Drug category consists of a general grouping by drug mechanism of action.
• Drug type is a more specific clinically relevant characterization of the agent based upon physical characteristics and/or mechanism of action.
• A target was listed if the drug, primarily molecularly targeted agents and immunotherapies, had a specific target.
 

Limitations of the data

• This analysis relies on data available in the public domain or from contacted trial leads or pharmaceutical companies.
• Up-to-date data on some drugs was not available (e.g., for the storage temperature or the light protection status), particulary for those drugs earlier in the development
• Paediatric formulation analysis covers only oral drugs.
  
• This analysis does not cover primarily adult cancers that are occasionally seen in children.
• Specific malignancies included for a drug were based upon study inclusion criteria which were sometimes general  (e.g. solid tumours) or included a long list of malignancies that qualified and may not reflect actual trial enrollment.