This page highlights the overall pipeline of corporate processes relating to health products. It contains summaries of the steps, links to guidance, and examples of the end product for each process. The pipeline can be started at any point.
In depth knowledge of the health R&D pipeline including resource allocation, the technology landscape and the geographical distribution of R&D capacity and infrastructure available allows funders, governments, and researchers to better understand the current situation and more importantly, where the greatest needs exist. The aim is to identify funding, technological and geographical gaps which can ultimately assist in better policy development and better-informed decision-making processes potentially leading to better targeted and increased investments in appropriate health technologies.
WHO staff develop in depth landscape analyses drawing on resources and networks. Resources include data published in the Global Observatory on Health Research and Development, a comprehensive source of data and analyses on global health R&D for human diseases, and the International Clinical Trials Registry Platform which allows researchers and patients to have a global transparent view of the registered clinical trials and to find their results.
Timely knowledge of the pipeline, including estimated trial end-dates, allows proactive preparations for WHO prequalification assessment and policy recommendation processes to avoid delays to market introduction and wide-scale implementation of innovative health products. The information contained in both platforms allow for deeper analysis of specific therapeutic areas to provide both an overall picture of the landscape and information on the status of the products in trials. They directly inform the development of Target Product Profiles (TPPs) to address unmet needs and to take account of novel technological developments underway. Sometimes this may be linked with future horizon scanning work to identify potential future scenarios and how to shape towards the most desired future. Guidance is available for this type of activity.
Setting research agendas and research priorities identifies the most critical research required to improve public health. A systematic approach results in a quality research agenda that can align stakeholders to achieve public health improvements. This includes aligning researchers and funders with global or national research needs.
A research agenda outlines research needs in a particular subject to improve public health. A research prioritization exercise then aims to rank these topics. The topics may be grouped depending on the health area. Priorities may be made according to different values such as cost, feasibility or potential for impact. The research priorities may be different in different contexts.
WHO published a systematic approach to research priority setting which can be adapted to different contexts. The guidance gives examples of several different methodologies. Staff undertaking a research prioritisation exercise can also approach researchsupport@who.int for further support.
The Global Accelerator for Paediatric Formulations (GAPf) provide an in-depth paediatric drug optimization (PADO) process to analyse paediatric drug formulation needs.
Clear guidance from WHO on required product characteristics can encourage R&D that will be of most benefit by informing product developers, regulatory agencies, procurement agencies and funders on R&D and public health priorities.
Target product profiles (TPPs) or preferred product characteristics (PPCs) documents are planning tools for the development of health products. TPPs and PPCs specify the intended use, target populations and desired attributes of products and guide research and development (R&D) outputs for funders and developers. WHO TPPs and PPCs are intended to support the development of missing health products on specific areas of public health needs. WHO TPPs have a specific focus on access, equity and affordability which are considered integral parts of the innovation process. The availability of TPPs/PPCs de-risks the work of product developers by expressing data required for regulatory, WHO Prequalification and policy decisions.
WHO has developed guidance to harmonize practices used to develop TPPs/PPCs to ensure full transparency in the processes used to express interest in particular health products and their respective attributes. Staff developing a target product profile can also approach researchsupport@who.int for further support.
The TPP Directory is an online searchable database of characteristics used to describe desired health products, including medicines, vaccines, diagnostics and medical equipment. It focuses on health product profiles for which there is a limited market or incentive for research and development.
The Directory includes TPPs developed by external sources such as product development partnerships, commercial entities and other organizations as well as profiles for products prioritized for global action by WHO and authored by WHO.
While TPPs provide high level guidance on desirable attributes for health products and guidance on target populations, these are not sufficiently granular to enable appropriate design of pivotal trials or clinical studies. Well designed, comprehensive studies are necessary to support manufacturer’s claims and regulatory submissions as well as the development of WHO recommendations and other normative and standard-setting products. Faulty design resulting in incomplete data sets can lead to delays in regulatory approvals, WHO prequalification listing and policy recommendations and therefore wide-scale implementation of urgently needed health products and interventions. Therefore WHO can supply guidance to developers and other researchers to align on the evidence required to inform policy and prequalification decisions. This aims to result in evidence available to make the highest quality recommendations.
WHO has produced a starter document for staff of the items needed to be included. WHO develops this methodological guidance as part of a consultative process involving scientific, regulatory, public health, end user and implementation expertise that yields possible approaches to create appropriate trial and study designs. Staff developing evidence generation guidance can also approach researchsupport@who.int for further support.
The generation of policy recommendations for health interventions and the WHO prequalification (PQ) assessment of health products are key elements in ensuring access to quality, safe, efficacious and appropriate products. Facilitating the production of necessary evidence to satisfy the data needs of these two processes can be difficult, especially for product developers with limited regulatory and health technology assessment experience. Incomplete product dossiers and gaps in evidence can lead to longer timelines to issuance of guidelines and prequalification listings. Furthermore, obtaining the right information from the relevant teams at WHO to ensure quality submissions for either processes can prove difficult.
The Coordinated Scientific Advice procedure represents a novel, standardized WHO corporate platform for interaction between product developers, the relevant technical department(s) and the Prequalification team. The procedure allows product developers to request scientific advice directly from the teams involved in the development of relevant guidelines and the WHO PQ team through a single-entry point at WHO. The advice provided to the product developers is coordinated internally to ensure alignment in positions and requirements from all relevant WHO staff. The process is aimed at innovative products of potential high public health value and commercially available products under development for a different indication/intended use.
Product developers can submit specific questions to WHO regarding the development of their product and obtain scientific advice around the design of pivotal phase III trials and/or clinical studies required to support any claims for prequalification and policy development purposes. The advice is coordinated internally between the WHO relevant technical department(s) and the Prequalification Team and presented to the product developer in a face-to-face meeting and in a final, written advice report. The process is voluntary, free of charge and non-binding for WHO and the product developer and in no way pre-empts a positive outcome for a future Prequalification or guideline development submission.
The formulation of policy recommendations, guidelines and other normative-setting products is a key pillar in ensuring access to health products and interventions to reach WHO’s triple billion targets and universal health coverage. Ensuring sufficient evidence is available to support any WHO recommendation is crucial.
WHO issues guidelines, recommendations and other normative setting products through a thorough, unbiased evidence retrieval and evaluation process in collaboration with all relevant stakeholders including target populations, intended users, national programmes, funders and methodologists. The process is transparent and well documented and follows clear guidelines.
Development of public health recommendations/guidelines at WHO relies on a clear set of procedures describing steps and criteria allowing optimal assessment of available evidence, as outlined in the WHO’s Handbook for Guideline Development.1 In order for WHO to assess evidence for public health recommendations, the following elements are key to consider: quality of evidence, patients’ values and preferences, resource implications, balance of benefits and harms, acceptability, feasibility, human rights, health equity, equality and non-discrimination, societal implications and health systems considerations.
Lack of regulatory capacity or inconsistency in the implementation of regulatory requirements globally can lead to the supply of health products of uncertain quality, safety and/or efficacy resulting in significant waste of public funds and potentially leading to serious global public health incidents.
WHO Prequalification aims to ensure access to quality, safe, efficacious/well performing, appropriate in vitro diagnostics, medicines, vaccines and vector control products for high burden/priority diseases. The prequalification process consists of a transparent, scientifically sound assessment of health products with a potential public health impact. This information, in conjunction with other procurement criteria, is used by UN and other procurement agencies to make purchasing decisions regarding IVDs, medicines, vector control products and/or vaccines.
The prequalification assessment varies slightly for each of the product streams but generally includes a product dossier review, product testing or performance evaluation, and inspection of the manufacturing sites. In addition, post-market surveillance activities contribute to ensuring the level of quality of the products after their listing is maintained.
Effective and efficient regulatory systems protect the public and enable timely access to quality medical products.
WHO plays a crucial role by building capacity in Member States, encouraging international regulatory cooperation, including convergence, harmonization, information- and work-sharing, reliance and recognition.
Different approaches, including WHO collaborative registration procedures, support a timely access to health products of assured quality, safety, efficacy and performance.
Using the principles of reliance and good regulatory practices, collaborative registration procedures enable National Regulatory Authorities to use the outputs from work already carried out to accelerate their decision-making, in line with international best practices.
More information is available: Regulation and Prequalification (who.int)
The WHO Emergency Use Listing Procedure (EUL) is a risk-based procedure for assessing and listing unlicensed vaccines, therapeutics and in vitro diagnostics with the ultimate aim of expediting the availability of these products to people affected by a public health emergency. Regulation and Prequalification (who.int)
The expert review panel (ERP) and the expert review panel for diagnostics (ERPD) was created by WHO and the Global Fund to Fight AIDS, Tuberculosis and Malaria (GFATM) to allow time-limited procurement of FPPs and diagnostics that are urgently needed and are yet to be prequalified and have not undergone stringent regulatory assessment. ERP relies on a group of independent technical experts to conduct a risk-based assessment of the products. The principal users of the ERP mechanism are the Global Drug Facility, GFATM, UNFPA, UNICEF and WHO's procurement unit. But any procurer can request an ERP/ERPD evaluation.