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Neglected disease R&D product pipeline.

 
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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 pipeline

The pipeline analysis presented here looks at 33 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 pipeline

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

The global R&D pipeline for neglected diseases is the result of a comprehensive review conducted by Policy Cures Research in 2017, and represents a snapshot of the pipeline as at August 2017.

 

Scope

The scope of the R&D pipeline presented in this analysis was developed based on the G-FINDER landscape reports on global funding of R&D for neglected diseases.

 

The G-FINDER scope is based on three key principles: the disease or health issue disproportionately affects the developing world; there is a need for new products (i.e. there is no existing product, or improved or additional products are needed); and there is market failure (i.e. there is insufficient commercial market to attract R&D investment by private industry). Accordingly, the R&D pipeline presented here encompasses the 33 neglected diseases included in the G-FINDER survey.

 

Product types included were drugs, vaccines, diagnostics and vector control products; however, in line with the G-FINDER scope, not all product types were included for all areas. Microbicide candidates for HIV have been included under the drug category. Medical devices and general or supportive therapies (e.g. oral hydration or nutritional supplements) were excluded.

 

The pipeline includes candidates at all stages of development - from discovery through to registration. For drugs and vaccines, development stage was broken down into discovery; pre-clinical studies; and clinical trials (further divided into Phase I, Phase II and Phase III trials). Diagnostics and vector control products have different regulatory pathways, and the development stage for these products was broken down into concept and research; feasibility and planning; design and development; and clinical validation and launch readiness. Screening programmes were excluded as these were not considered to represent discrete pipeline candidates. Candidates were no longer considered to be part of the R&D pipeline – and therefore excluded from this analysis – once granted regulatory approval by a National Regulatory Authority, or if their development had been placed on hold indefinitely.

 

In line with the scope of the G-FINDER report, additional restrictions were applied to selected disease and product categories with potential commercial (high-income country market) overlap; for example, drug candidates for HIV/AIDS were only included if they were label-extensions or reformulations specifically intended for developing country use (e.g. pediatric or slow-release formulations; fixed dose combinations; or low-dose formulations for prophylaxis).

 

For further details on the specific diseases and related product areas within the scope of the pipeline analysis, please see the Policy Cures Research R&D matrix[1] and R&D scope document.[2]

 

Data source and validation

Data on the candidates in the global R&D pipeline for poverty-related and neglected diseases was collected by Policy Cures Research, building on previous pipeline analyses including the 2015 neglected diseases product pipeline developed by Policy Cures,[3] pipeline data collected for the 2012 Policy Cures/Global Health Technologies (GHTC) report ‘Saving lives and creating impact: Why investing in global health R&D works’,[4] and the BIO Ventures for Global Health (BVGH) Global Health Primer.[5]

 

To bring the R&D pipeline candidate data up to date as at August 2017, and to expand on the scope of previous efforts, Policy Cures Research reviewed and cross-referenced all major sources of available data on the R&D pipeline for poverty-related and neglected diseases. Sources included the G-FINDER R&D funding database; the World Health Organization ‘rainbow tables’[6]; background documents prepared for the WHO Product Development for Vaccines Advisory Committee[7]; WHO Vector Control Advisory Group (VCAG) reports[8]; WHO Pesticide Evaluation Scheme (WHOPES) reports[9]; Unitaid landscape and technical reports[10]; disease-specific pipeline updates prepared by BVGH and the Treatment Action Group[11]; publicly available company and product development partnership R&D portfolios; journal publications; clinical trial registration portals; and university, government, and non-profit organisation websites.

 

Candidates were only included if an authoritative source could confirm they were still 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 FY2016, while this pipeline was current as at August 2017.

 

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