WHO review of the drugs pipeline for neglected tropical diseases

Published: March 2024

WHO analysed the state of paediatric drugs research and development targeting neglected tropical diseases (NTDs), comparing these efforts to those for adult drugs and considering the overall burden of these diseases. 
The analysis spans trials from 1999 through 2022, and provides insights into the trials ‘recruitment status, the number and location of trials, the diseases being targeted, the age group of participants enrolled, and the organizations sponsoring these trials.
Additionally, it details the progress of drug development, noting the most advanced phase reached for drugs targeting various NTDs.

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:

  • Number of trials and burden of diseases by: 
    • Recruitment status (chart A.1)
    • Age group (chart A.2)
    • Disease (chart A.3)
    • Income group (chart A.4)
    • WHO region (chart A.5)
    • Country (charts A.6)
    • Type of sponsor (chart A.7)
    • Sponsor (chart A.8)
  • Drugs for NTDs, characterized by:
    • Highest phase of development (chart B.1)
    • Disease (chart B.2)
  • List detailing the drugs included in the analysis (chart B.3)

 

Note: A drug can be studied for multiple diseases and covered by multiple trials being conducted in multiple locations; therefore, the numbers displayed in these charts may total to more than the total number of drugs or trials.

 

Points to note
  • Overview of the NTD trials:
    • Between 1999 and 2022, 669 clinical trials targeting NTDs were conducted, of which 21.4% included children aged 0-4 years (143 trials), 49.5% (331) covered ages 5-14, and nearly 80% included the 15-59 age group (527) (chart A.2).
    • The majority of recruiting trials focus on three primary diseases, accounting for over 50% of the total: leishmaniasis (40; 32.8%), dengue (19; 15.6%), and leprosy (13; 10.7%). (select “recruiting” status in chart A.1, and see chart A.3).
    • Despite carrying the highest disease burden, rabies clinical trials are significantly underrepresented, with only 1.3% of identified trials (unselect "recruiting" in chart A.1 and see chart A.3).
    • Lower middle-income countries are at the forefront of hosting clinical trials for NTDs, with 385 trials (57.5%). Among these, India stands out with the greatest number of trials (125; 32.5%). This significant activity in India aligns with its status as the country facing the most substantial NTD burden within this income group. (select “Lower middle income” group in A.4, and see A.6).
    • Africa, bearing the highest disease burden among WHO regions, ranks second in trial numbers (159; 23.8%), with countries with trials representing less than a quarter of the total DALYs in this region (1,472 of 6,115 thousand DALYs) (chart A.5).
    • The majority of trials are sponsored by academic and research institutions (393; 58.7%), followed by pharmaceutical and biotechnology companies (96; 14.3%) (chart A.7).
    • Looking at the individual sponsor, Drugs for Neglected Diseases Initiative (DNDi) sponsored the highest numbers of trials with 39 trials, followed by the Swiss Tropical and Public Health Institute (18) and Mashhad University of Medical Sciences (16) (chart A.8).
  • Overview of the NTD drugs:
    • Among all identified drugs targeting NTDs, the majority are in phase 3 of development (120 drugs; 33.1%) (chart B.1).
    • The diseases receiving the most focus are leishmaniasis, with 110 drugs tested in clinical trials; dengue (47); and leprosy (41) (chart B.2).

To explore the data further

  • Select a specific age group (chart A.2) or any other specific element or combination of elements to display the corresponding data in the other charts. 
  • Hold the ‘Ctrl’ key on your keyboard to select more than one option. 
  • Hover the cursor on a bar or a cell in a table to see more information in a pop-up window.
  • 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

  • Our analysis covers research on neglected tropical diseases from 1999 to 2022, focusing on clinical trials where the drugs used in the intervention groups were clearly listed on the WHO ICTRP platform.
  • Neglected tropical diseases not listed under chart A.3 are not included in this analysis.

Analysis

  • The analysis leverages data from the WHO's International Clinical Trials Registry Platform (WHO ICTRP) and AdisInsight by Springer Nature for trial information and drug identification. The WHO Global Health Estimates (GHE-2019) were also used to evaluate the overall burden of the neglected tropical diseases through Disability-Adjusted Life Years (DALYs).
  • The term "Trial countries’ DALYs" refers to the Disability-Adjusted Life Years (DALYs) for countries that have hosted clinical trials.
  • "Global DALYs" denotes the DALYs across all countries, irrespective of whether they have conducted trials.
  • Drugs or combinations of drugs that were known by various names have been consolidated under a single unique identifier.

Limitations of the data

  • Automated data mining techniques were used to extract information on the primary disease targeted in each trial, using text-based data fields. It's important to note that if a NTD is not included in our analysis, this does not necessarily imply there are no trials concerning that disease. Rather, it may reflect limitations in our data mining process, which could have missed capturing trials related to the specific NTD.
  • For some of the diseases included in our analysis, specific corresponding DALYs data were not available.
  • Our analysis is restricted to trials that have been registered on the ICTRP platform and further narrowed to those trials where the intervention drugs could be precisely identified.

 

Publication

Paediatric research and development landscape for neglected tropical diseases: Report on 1999–2022 period

Neglected tropical diseases (NTDs) are a diverse set of 21 diseases and disease groups that mainly affect resource-poor populations in tropical and subtropical...