WHO target product profiles to shape global research and development.

Research and development leading to new and improved health products is essential for achieving healthier lives for populations worldwide. However, new products in development do not always match the global need for products for neglected diseases and populations. To promote research, provide an incentive for investment and align products with the needs of end-users, research needs to be better coordinated and prioritized. The World Health Organization (WHO) has developed target product profiles that define the characteristics required in new health products to address the greatest public health needs. A WHO target product profile document presents a need and provides guidance on what to include to consider access and equity as part of the research and development plan from the outset. WHO has also set up the Target Product Profile Directory, a free-to-use online database of characteristics used to describe desired health products, including medicines, vaccines, diagnostic tools and medical equipment. Here we describe the process of developing a WHO target product profile, and the benefits of this type of guidance. We urge product developers to share product profiles addressing unmet needs in public health, to help in progress towards global targets for better health and well-being.

Promouvoir la santé des populations à travers le monde va de pair avec la recherche-développement responsable de la conception et de l'optimisation de produits sanitaires. Pourtant, les nouveaux produits à l'étude ne répondent pas toujours aux exigences mondiales des populations et maladies négligées. En vue de promouvoir la recherche, de favoriser les investissements et d'aligner les produits sur les besoins des utilisateurs finaux, les travaux doivent être mieux coordonnés et leurs priorités, mieux définies. L'Organisation mondiale de la Santé (OMS) a donc élaboré des profils de produits cibles qui déterminent les caractéristiques requises pour les nouveaux produits sanitaires, afin qu'ils correspondent davantage aux besoins les plus criants en matière de santé publique. Un profil de produit cible établi par l'OMS est un document qui met en évidence un besoin et fournit des indications sur les aspects à prendre en compte pour garantir l'accès et l'équité dès le départ dans le plan de recherche-développement. L'OMS a également publié un Répertoire des profils de produits cibles, une base de données en ligne consultable gratuitement qui reprend les caractéristiques employées pour décrire les produits sanitaires souhaités (médicaments, vaccins, outils diagnostiques et équipements médicaux). Dans le présent document, nous détaillons le processus de développement d'un profil de produit cible par l'OMS, mais aussi les avantages que comportent de telles indications. Nous encourageons vivement les laboratoires à partager les profils de produits qui répondent à des besoins non satisfaits en matière de santé publique, afin de contribuer à avancer vers les objectifs mondiaux de santé et de bien-être.

La investigación y el desarrollo de productos sanitarios nuevos y mejorados son esenciales para que las poblaciones de todo el mundo vivan más sanas. Sin embargo, los productos nuevos en desarrollo no siempre se ajustan a las necesidades mundiales de productos para enfermedades y poblaciones desatendidas. Para promover la investigación, incentivar la inversión y adaptar los productos a las necesidades de los usuarios finales, es necesario coordinar mejor la investigación y establecer prioridades. La Organización Mundial de la Salud (OMS) ha elaborado perfiles de productos específicos que definen las características que deben reunir los productos sanitarios nuevos para satisfacer las principales necesidades de salud pública. Un documento de perfil de producto específico de la OMS presenta una necesidad y ofrece orientación sobre lo que debe incluirse para tener en cuenta el acceso y la equidad como parte del plan de investigación y desarrollo desde el principio. La OMS también ha creado el Directorio de Perfiles de Productos Específicos, una base de datos en línea de uso gratuito con las características utilizadas para describir los productos sanitarios deseados, incluidos medicamentos, vacunas, herramientas de diagnóstico y equipos médicos. En el presente documento, describimos el proceso de elaboración de un perfil de producto específico de la OMS y las ventajas de este tipo de orientación. Instamos a los desarrolladores de productos a compartir perfiles de productos que aborden necesidades no cubiertas en salud pública para contribuir al avance hacia los objetivos mundiales de mejora de la salud y el bienestar.

Creating equity in health research to drive more and better evidence.

Health research is rapidly changing with evidence being gathered through new agile methods. This evolution is critical but must be globally equitable so the poorest nations do not lose out. We must harness this change to better tackle the daily burden of diseases that affect the most impoverished populations and bring research capabilities to every corner of the world so that rapid and fair responses to new pathogen are possible; anywhere they appear. We must seize this opportunity to make research easier, better and more equitable. Currently too many nations are unable to generate the evidence or translate it to directly change health outcomes in their own communities. It is essential to act and harness this emerging change in how research data can be generated and shared, so that all nations sustainably gain from this development. There are positive examples to draw on from COVID-19, but we now need to act. Here we present an initiative to develop a new framework that can guide researchers in the design and execution of their studies. This highly agile system will work by adapting to risk and complexity in any given study, whilst generating quality, safe and ethical data.

WHO International Standard for evaluation of the antibody response to COVID-19 vaccines: call for urgent action by the scientific community.

The first WHO International Standard and International Reference Panel for anti-SARS-CoV-2 immunoglobulin were established by the WHO Expert Committee on Biological Standardization in December, 2020. The WHO International Antibody Standards are intended to serve as global reference reagents, against which national reference preparations or secondary standards can be calibrated. Calibration will facilitate comparison of results of assays (eg, of the neutralising antibody response to candidate COVID-19 vaccines) conducted in different countries. Use of these standards is expected to contribute to better understanding of the immune response, and particularly of the correlates of protection. This Personal View provides some technical details of the WHO Antibody Standards for SARS-CoV-2, focusing specifically on the use of these standards for the evaluation of the immune response to COVID-19 vaccines, rather than other applications (eg, diagnostic or therapeutic). The explanation with regard to why rapid adoption of the standards is crucial is also included, as well as how funders, journals, regulators, and ethics committees could drive adoption in the interest of public health.

Meeting report: WHO consultation on accelerating Lassa fever vaccine development in endemic countries, Dakar, 10-11 September 2019.

At the time of writing in 2019, there have been 754 confirmed cases of Lassa fever in Nigeria, 21% of whom have died. Lassa is on the priority pathogen list for WHO's R&D Blueprint for Action to Prevent Epidemics. In September 2019, WHO convened 67 scientists, regulators, ethicists, public health officials, funders and vaccine developers to discuss the end-to-end clinical development plan for Lassa fever vaccines. The substantial increases in vaccine trial capacity in Africa were reviewed, together with lessons learned from the evaluation of vaccines against HIV, TB, malaria, and Ebola in Africa. Participants agreed on a pathway for Lassa vaccine trial progression, as outlined in WHO's Lassa fever R&D roadmap and the WHO Lassa fever Target Product Profile. Two Phase 1 trials of Lassa vaccines have already started, and it was agreed that continuing interactions between high income and African regulatory and ethics authorities and WHO will be important in progression towards Phase 2b/3 efficacy trials in Lassa fever endemic areas. There was agreement that, for diseases whose burden is mainly in Africa, it should be the norm that African regulatory authorities are consulted on trial design/progression before first-in-human Phase 1 trials. Phase 2b-3 vaccine trial capacity needs to be in place in high Lassa fever burden areas where efficacy trials will take place. Licensure of one or more Lassa fever vaccines suitable for West African populations is a realistic goal in the next 5 years, with CEPI and WHO aligned on the pathway forward for vaccine development.

Respiratory syncytial virus vaccine research and development: World Health Organization technological roadmap and preferred product characteristics.

The respiratory syncytial virus causes a considerable respiratory disease burden globally, most markedly in young infants, in low and middle income countries. A diverse product pipeline illustrates the recent intensification of research and development activities for vaccines and monoclonal antibodies against RSV. With the aim to ensure that product development activities are directed to address the public health needs, the World Health Organization has developed a research and development technical roadmap and articulated product characteristics preferences.

Maternal immunization against Group B streptococcus: World Health Organization research and development technological roadmap and preferred product characteristics.

Group B streptococcus, found in the vagina or lower gastrointestinal tract of about 10-40% of women of reproductive age, is a leading cause of early life invasive bacterial disease, potentially amenable to prevention through maternal immunization during pregnancy. Following a consultation process with global stakeholders, the World Health Organization is herein proposing priority research and development pathways and preferred product characteristics for GBS vaccines, with the aim to facilitate and accelerate vaccine licensure, policy recommendation for wide scale use and implementation.

WHO consultation on group B Streptococcus vaccine development: Report from a meeting held on 27-28 April 2016.

Globally, group B Streptococcus (GBS) remains a leading cause of sepsis and meningitis in infants in the first 90days of life. Intrapartum antibiotic prophylaxis (IAP) for women at increased risk of transmitting GBS to their newborns has been effective in reducing part, but not all, of the GBS disease burden in many high income countries (HICs). In low- and middle-income countries (LMICs), IAP use is low. Immunization of pregnant women with a GBS vaccine represents an alternative strategy to protecting newborns and young infants, through transplacental antibody transfer and potentially by reducing new vaginal colonization. This vaccination strategy was first suggested in the 1970s and several potential GBS vaccines have completed phase I/II clinical trials. During the 2015 WHO Product Development for Vaccines Advisory Committee meeting, GBS was identified as a high priority for the development of a vaccine for maternal immunization because of the major public health burden posed by GBS in LMICs, and the high technical feasibility for successful development. Following this meeting, the first WHO technical consultation on GBS vaccines was held on the 27th and 28th of April 2016, to consider development pathways for such vaccines, focused on their potential role in reducing newborn and young infant deaths and possibly stillbirths in LMICs. Discussion topics included: (1) pathophysiology of disease; (2) current gaps in the knowledge of global disease burden and serotype distribution; (3) vaccine candidates under development; (4) design considerations for phase III trials; and (5) pathways to licensure, policy recommendations and use. Efforts to address gaps identified in each of these areas are needed to establish the public health need for, the development and deployment of, efficacious GBS vaccines. In particular, more work is required to understand the global disease burden of GBS-associated stillbirths, and to develop quality-assured standardized antibody assays to identify correlates of protection.

Report from the World Health Organization's third Product Development for Vaccines Advisory Committee (PDVAC) meeting, Geneva, 8-10th June 2016.

The third meeting of WHO's Product Development for Vaccines Advisory Committee (PDVAC) was held in June 2016, with a remit to revisit the pathogen areas for which significant progress has occurred since recommendations from the 2015 meeting, as well as to consider new advances in the development of vaccines against other pathogens. Since the previous meeting, significant progress has been made with regulatory approvals of the first malaria and dengue vaccines, and the first phase III trials of a respiratory syncytial virus (RSV) vaccine candidate has started in the elderly and pregnant women. In addition, PDVAC has also supported vaccine development efforts against important emerging pathogens, including Middle Eastern Coronavirus (MERS CoV) and Zika virus. Trials of HIV and tuberculosis vaccine candidates are steadily progressing towards pivotal data points, and the leading norovirus vaccine candidate has entered a phase IIb efficacy study. WHO's Immunization, Vaccine and Biologicals (IVB) department is actively working in several pathogen areas on the recommendation of PDVAC, as well as continuing horizon scanning for advances in the development of vaccines that may benefit low and middle income countries (LMICs), such as the recent licensure of the enterovirus 71 (EV71) vaccine in China. Following on from discussions with WHO's Strategic Advisory Group of Experts (SAGE) on Immunization, PDVAC will also look beyond licensure and consider data needs for vaccine recommendation and implementation to reduce the delay between vaccine approval and vaccine impact.

Meeting report: WHO consultation on Respiratory Syncytial Virus (RSV) vaccine development, Geneva, 25-26 April 2016.

Respiratory syncytial virus (RSV) is a leading viral cause of respiratory morbidity and mortality in infants and young children worldwide. Low and middle income countries (LMICs) account for approximately 99% of the global mortality estimates in this population, with up to 200,000 RSV deaths per year. The vaccine product development pipeline is diverse with the most advanced clinical candidate currently in phase III efficacy testing in pregnant women. In addition, a long-acting RSV-neutralizing monoclonal antibody (mAb) to be administered at birth to prevent serious RSV-related respiratory disease is in late stage clinical development, as are additional conventional mAb for use in high-risk infants. Thus, there is a realistic possibility that an effective new intervention to prevent RSV disease will be available in the next 5-10year horizon. In anticipation of this outcome, the Strategic Advisory Group of Experts for Immunization (SAGE), WHO's vaccine policy recommendation body, reviewed the status of RSV vaccine and monoclonal antibody development in April 2016. Although substantial progress towards licensure has broadened the research agenda to consider intervention impact and cost effectiveness, significant gaps remain in the data that will be needed to inform and support a policy recommendation for implementation. These aspects were the focus of WHO's second consultation on RSV vaccines and single dosage extended half-life mAb for prophylaxis.