1.3 Improved access to essential medicines, vaccines, diagnostics and devices for primary health care
1.3.4 Research and development agenda defined and research coordinated in line with public health priorities
Scorecard
The scorecard shows the strength of the output: establishing priorities for research and development (R&D) throughout WHO, making the global accelerator for paediatric formulations a formal WHO network and advancing the agenda for new antibacterial products to counter the threat of antimicrobial resistance. As the work towards this output is largely led and coordinated at headquarters, it involves less technical support to countries, which does not reflect a deficiency but the lack of relevance of these indicators for the scope of the output.
Achievements and challenges
WHO plays a catalytic role in R&D of innovative products, particularly in areas in which the market is not providing the necessary products or technologies to support vaccine delivery.
To ensure reliable access to resources in the R&D pipeline and clinical studies, WHO has continued to promote the Global R&D Observatory and the International clinical trials registry platform, which has extended its user database and centralized all COVID-19-related clinical studies. A standardized process for developing target product profiles has been developed and the role of these documents clarified.
WHO also convened initial consultations to develop joint scientific advice, whereby product developers can approach WHO for advice on whether a product meets the criteria for having likely public health value. The aim of the procedure is to ensure that submissions of products for prequalification or a policy recommendation meet WHO requirements, thus accelerating approval and uptake. Joint scientific advice will be pilot-tested in 2021.
The pre-clinical development and the clinical antibiotic pipelines have been assessed in relation to coordinating the global One Health response of the Tripartite as well as with other agencies and the private sector. A concept for reviewing priority bacterial pathogens has been developed and collection of data has started. The Antibacterial agents in preclinical development report and Antibacterial agents in clinical development review were downloaded more than 12 000 and 22 000 times, respectively, from the WHO website. The concept of a project for identifying priority fungi and the respective antifungal agents that are in clinical development was developed. An expert group was established to draw up the first list of priority fungal pathogens, and the first meeting was held in April 2020 to start the list and identify gaps in R&D.
To support establishment of a Global Antibiotic Research and Development Partnership to mobilize resources and partners to develop life-saving treatments for drug-resistant infections, a consultation was organized within the WHO Secretariat to identify research priorities.
With the European Investment Bank and other partners, WHO developed a concept and a financial model for an impact investment fund for R&D on antibiotics. WHO mobilized the support of the pharmaceutical industry to build the AMR Action Fund involving the pharmaceutical industry, the European Investment Bank and the Wellcome Trust, which was launched in July 2020. The Fund will invest around US$ 1 billion in antibiotic development. Progress was also made in normative work with a number of articles published in peer-reviewed journals.
The Global Accelerator for Paediatric Formulations Network (GAP-f) was hosted, which provides a sustainable mechanism to ensure that the most urgent, optimal paediatric formulations are developed to the highest standards of safety and efficacy and made available for use in children in a timely manner. The GAP-f network, formally launched in October 2020, comprises 30 organizations which contribute to one or more of GAP-f’s four working groups. A donor forum and an ad hoc external advisory committee were also created, in addition to the routine meetings of the steering group and working groups.
PAediatric Drug Optimization (PADO) guidance for prioritizing paediatric drugs is being prepared. Formal PADO processes for antibiotics and medicines for childhood cancer are anticipated in 2021, to be added to processes for HIV, tuberculosis and hepatitis.
At a leading conference on retroviruses, CROI 2021, GAP-f presented an analysis of population pharmacokinetics to determine the lowest body weight at which children could be treated with available daclatasvir formulations. Possible use of adult formulations could accelerate extension of hepatitis C treatment for children globally.
GAP-f has contributed to the development and launch of the new Technology Landscape, released by Unitaid, and is collaborating with the Bill & Melinda Gates Foundation to identify and prioritize paediatric formulations for inclusion on the WHO Model List of Essential Medicines for Children. GAP-f also launched a new quarterly webinar series, #BetterDrugs4Kids, the first of which was a webinar entitled “Best practices in clinical research for the development of paediatric formulations”.
Of the challenges faced, the most significant were the diversion of human resources to the COVID-19 pandemic and resource constraints, which led to major changes to workplans and a switch to virtual meetings. Both resource constraints and competing priorities of partners have resulted in some delays for GAP-f.