About neglected diseases

Neglected diseases disproportionately affect developing countries, causing significant morbidity and mortality in already disadvantaged populations – more than six million people die each year due to neglected diseases such as tuberculosis (TB), malaria and sleeping sickness. Because these diseases are overwhelmingly restricted to neglected populations, there is insufficient commercial market to attract the R&D investment by private industry that is necessary to develop new health technologies to bridge existing product gaps.

 

However, with the support of public and private funding the product pipeline for neglected diseases has more than doubled in size over the last decade. Over three-quarters of these product candidates are being developed by innovative collaborations between the public and private sector – such as product development partnerships (PDPs) – meaning that products are being designed specifically to be affordable and appropriate for developing countries. With sustained, appropriate support, we will see the delivery of many exciting, much needed products and technologies over the next 15 years.

 

About the R&D pipeline tracker

The pipeline analysis presented here looks at 35 neglected diseases within the scope of the G-FINDER survey, covering all product categories including drugs, vaccines, diagnostics and vector control products, and all stages of research from early stage R&D through to product registration. You can find a detailed explanation of the methodology behind this review and notes on the limitations of the data below.

 

Using the R&D pipeline tracker

The site is designed to be interactive and provides both visualisations and tables which can be filtered to give more granularity:

• The first visualisation displays the number of candidates in the pipeline by disease, product type, archetype, complexity and R&D stage

• The second data visualisation lists each of the candidates, showing their developers. It can be filtered by disease, product and R&D stage

• The third and fourth visualisations provide the same information, but for the diseases within each disease category.

 

Methodology

We undertook a global review of candidate products for neglected disease in order to capture snapshot of the R&D pipeline as of August 31, 2019; this followed a previously published review of the R&D pipeline as of August 31, 2017.

 

Scope

The scope of candidate inclusion aligned with the G-FINDER survey on global funding for R&D for neglected diseases conducted by Policy Cures Research. The G-FINDER scope is based on three key principles: that the disease or health issue disproportionately affects low- and middle-income countries; that there is a need for new products (i.e. there is no existing product, or improved or additional products are needed); and that there is a market failure (i.e. there is insufficient commercial market to attract private R&D investment).

 

The scope of the G-FINDER project is reviewed annually in consultation with an expert Advisory Committee. As a result of these consultations, the disease scope used for the pipeline review in 2019 was wider than in 2017, with the addition of the following diseases: hepatitis B and mycetoma. Snakebite envenoming was added to the G-FINDER scope after the conclusion of the 2019 pipeline review and was therefore not included in the scope of this analysis.

Accordingly, the R&D pipeline review presented here comprises 35 individual disease and six multiple-disease groups.

The G-FINDER scope includes drugs, microbicides, biologics, vaccines, diagnostics and vector control products. Not all product types are included for every disease or health condition: the product category is excluded if a viable commercial market is thought to exist, and additional restrictions are applied to some disease and product categories if there is potential commercial (high-income country market) overlap. For example, the G-FINDER scope excludes dengue vaccines, and HIV/AIDS drugs are included only if they are label-extensions or reformulations specifically intended for developing country use (e.g., pediatric or slow-release formulations; fixed dose combinations; low-dose formulations for prophylaxis; long-acting injectables for treatment or prophylaxis). Medical devices (except for diagnostics), and general or supportive therapies (e.g., oral rehydration or nutritional supplements) are not included in the scope of G-FINDER and were therefore not included in the pipeline reviews. Dues to changes in the G-FINDER scope between the 2017 and 2019 as described above, two disease-specific product areas were included for the first time in the 2019: vaccines for leprosy, and biologics for HIV.

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The pipeline review included candidates at all stages of development, from discovery through to product registration. Drugs, vaccines, and reproductive health candidate products were classified in the following five development stages: discovery; pre-clinical studies; and clinical development (Phase I, Phase II and Phase III). Diagnostics and vector control products follow different product development and regulatory pathways, so these candidate products were classified under the following stages: concept and research; feasibility and planning; design and development; and clinical validation and launch readiness. Candidates were no longer considered to be part of the R&D pipeline – and therefore were excluded from this analysis – once granted regulatory approval by a stringent regulatory authority, or if their development had been placed on hold indefinitely.

Further details on the diseases and product areas within the scope of the pipeline analysis are provided in the G-FINDER R&D matrices and scope documents.

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Data source and validation

We reviewed and cross-referenced all major sources of available data on the R&D pipeline within the scope outlined above. Sources included: the G-FINDER R&D funding database; the World Health Organization “rainbow tables”; publications of the WHO Initiative for Vaccine Research (IVR) which included background documents and meeting reports and presentations prepared for the WHO Product Development for Vaccines Advisory Committee (PDVAC) and Strategic Advisory Group of Experts (SAGE) on immunizations; reports of the WHO Vector Control Advisory Group (VCAG) reports; WHO R&D roadmaps; the WHO vaccine pipeline tracker; WHO prequalification lists; multiple Unitaid landscape and technical reports; multiple clinical trial portals including ClinicalTrials.gov, the AIDSinfo clinical trial database, the Chinese Clinical Trial Registry, the Clinical Trials Registry – India and the WHO International Clinical Trials Registry Platform; disease-specific pipeline updates by the HIV Vaccines Trials Network and the Treatment Action Group; publicly available company and product development partnership R&D portfolios; journal publications; conference and meeting proceedings; and university, government, and non-profit organization websites.

Candidates were only included if an authoritative source could confirm they were in active development. The following sources were considered to be authoritative:

• The website of the candidate developer, if recently updated

• Recent reports or other materials from international organizations such as WHO and Unitaid

• Clinical trial portals

• Correspondence with product developers

• Correspondence with experts in the field, including FIND; the Innovative Vector Control Consortium (IVCC); the International AIDS Vaccine Initiative (IAVI); Netherlands Leprosy Relief; Program for Appropriate Technology in Health (PATH); the Sabin Vaccine Institute; and the US National Institute of Allergy and Infectious Diseases (NIAID).

 

New feature

The current update includes additional classification of product type by archetypes and complexity based on the adapted version of Portfolio-To-Impact Model (P2I model). 

 

Disclaimer

It is inevitable that some candidates might have been missed, while others will have been included as active when in fact they are no longer so. We welcome additions, removals, questions and feedback on our methodology. It is important to note that although the disease and product scope of the pipeline is aligned with that of the G-FINDER survey, the candidate- and organisation-level information provided here should not be combined with G-FINDER financial data for direct analysis. Attempting to do so would result in a distorted picture for a number of reasons, including that much investment information reported by participants in the G-FINDER survey is for a portfolio of products rather than specific candidates (or indeed is for multiple diseases, rather than just one); that in order to prevent double-counting of financial flows G-FINDER does not report onwards funding provided by PDPs and other intermediaries, and thus does not show the full amount of funding received by individual product developers; and that G-FINDER only uses primary data, whereas the pipeline data came from a variety of sources as described above. The time required for the collection and publication of financial data means that the most recently-published G-FINDER data is for FY2018, while this pipeline was current as at August 2019.

 

References

1. Policy Cures Research. G-FINDER neglected diseases R&D matrix [Internet]. Available from: https://gfinder.policycuresresearch.org/staticContent/pdf/Y10-2017_R&D_Matrix_Neglected_Disease_PCR.pdf

2. Policy Cures Research. G-FINDER neglected diseases scope [Internet]. Available from: http://www.policycuresresearch.org/downloads/Y10_G-FINDER%20ND%20R&D%20scope.pdf

3. Policy Cures. The Unrecognised Revolution in Global Health [Internet]. Available from: http://pipeline.policycures.org/

4. Policy Cures, GHTC. Saving lives and creating impact [Internet]. Available from: http://www.policycures.org/downloads/Saving%20lives%20and%20creating%20impact.pdf

5. BVGH. Global Health Primer - a review of drug, diagnostic, and vaccine pipelines [Internet]. Available from: https://bvgh.org/global-health-primer/

6. World Health Organisation (WHO). Tables of malaria vaccine projects globally [Internet]. WHO. Available from: http://www.who.int/immunization/research/development/Rainbow_tables/en/

7. World Health Organisation (WHO). Research and development [Internet]. World Health Organisation. Available from: http://www.who.int/immunization/research/en/

8. World Health Organisation (WHO). Vector Control Advisory Group (VCAG) [Internet]. WHO. Available from: http://www.who.int/neglected_diseases/vector_ecology/VCAG/en/

9. World Health Organisation (WHO). WHO Pesticide Evaluation Scheme: ‘WHOPES’ [Internet]. Available from: http://www.who.int/whopes/en/

10. Unitaid. Publications [Internet]. Unitaid. Available from: https://unitaid.eu/publications/

11. Treatment Action Group. Pipeline Report [Internet]. Available from: http://www.treatmentactiongroup.org/pipeline-report