Global Health R&D: Evidence, Priorities, Coordination

World Health Assembly Agenda Item 13.5 is descriptively-yet-uninformatively labelled “Follow-up of the report of the Consultative Expert Working Group on Research and Development: Financing and Coordination (CEWG).” But that anodyne title actually masks an important milestone in the World Health Organization’s long-running efforts to increase R&D around neglected diseases and diseases of poverty.

The annual World Health Assembly is taking place from 22-31 May.

Last year, in Resolution WHA69.23, Member States directed the Secretariat to implement several of the recommendations in the CEWG Report. This year, the Secretariat is back with the results (WHA70/22): a Global Observatory on Health R&D, a Health Product Profile Directory, a planned voluntary pooled funding mechanism, and an Expert Committee on Health Research and Development.

To draw attention to and support for these initiatives, Switzerland, South Africa, and the European Commission co-hosted at a side event on 25 May at the Graduate Institute, with panellists from EuropeAid, WHO, and TDR (the Special Program for Research and Training in Tropical Disease, which is housed within WHO and co-sponsored by UNICEF, UNDP and the World Bank). The panellists provided an overview of the CEWG and gave a sneak peek at the new tools and their findings.

How Did We Get Here?

Martin Friede (Coordinator, Initiative for Vaccine Research, WHO) spoke first, bringing the audience up to speed on the CEWG’s history and process. In 1990, the Commission on Health Research and Development published a startling finding: worldwide, less than 10% of R&D spending was going towards the diseases responsible for over 90% of preventable deaths. Advocates for equitable access to medicines cited this “10/90 gap” as a telltale sign of market failure—proof dispositive that the market is not an effective or ethical arbiter of health R&D priorities.

But if not the market, then what? Member States needed an alternative priority-setting mechanism that could guide R&D investment in place of, or alongside, market forces. So in 2010, they created the CEWG to come up with one (Resolution WHA63.28; the CEWG replaced a previous Expert Working Group.)

The CEWG’s first task was to articulate a set of principles to guide priority-setting. This was ultimately settled at last year’s WHA, where Member States agreed “that health research and development should be needs-driven and evidence-based and be guided by the following core principles: affordability, effectiveness, efficiency, and equity; and that it should be considered a shared responsibility.” (WHA69.23).

But operationalizing these principles requires a vast amount of data; for example, on burden of disease, funding flows, drugs in development, existing patents and licensing agreements, ongoing studies & clinical trial, previously-tried-and-failed approaches, etc. Some of this information was already being collected, but was scattered and not easily accessible. Other types of data weren’t even gathered. So the CEWG’s second task was to compile all the evidence needed to support priority-setting in a usable form. And then its third task was figuring out how to finance it all.

WHA70: Where Are We Now?

With context in place, Vasee Moorthy (Coordinator for Research, Ethics & Knowledge Uptake, Health Systems and Innovation Cluster, WHO) and Robert Terry (Knowledge Manager, TDR) unveiled two tools that WHO has developed to help manage this massive evidence base.

The first is the Global Observatory on Health R&D, a “centralized and comprehensive source of information and analyses on global R&D activities for all human diseases.” The Observatory is the most complete source of R&D information ever, compiling existing databases (e.g. G-Finder, Pubmed.gov) and gathering new data where necessary and feasible. WHO is also working with major public and private R&D funders (e.g. US National Institutes of Health, Gates Foundation, Wellcome Trust) to develop classifications and standards around the data and metadata they collect, which will enable expanded interoperability in the future.

The Observatory lays the foundation for R&D priority-setting by allowing policymakers, funders, and researchers (and anyone else) to get an overview of existing research; identify gaps & opportunities; monitor trends (including funding trends); and compare activities across countries and diseases. Moorthy showed a few sample queries and visualizations, then invited the audience to go online and explore further.

The second tool is a Health Product Profile Directory developed by TDR. Product profiles are essentially detailed “wish lists” to guide R&D. They identify the target drugs/vaccines/diagnostics needed and describe their minimum or ideal characteristics (e.g. target population, route of administration, clinical & microbiological characteristics, stability, drug interactions). Currently, most of the product profiles developed are not made public and there is no standard format or content.

The Directory is a centralized database of current profiles, allowing researchers and funders to identify existing needs and providing templates for future profiles. Inclusion in the Directory does not imply WHO endorsement of a given profile. Nor is the Directory complete. Terry noted that while the pharmaceutical industry often cites the public health community’s “failure to describing priorities” as the reason there isn’t more R&D on neglected diseases (as opposed to market failure), companies have waxed “hot and cold” about sharing profiles for fear of revealing propriety information. TDR is currently working with the Gates Foundation and others to expand the Directory.

On the governance front, there are two notable developments. First, Member States have approved the terms of reference for the Expert Committee on Health R&D, which will be responsible for curating the list of recommended R&D priorities. The Committee is expected to begin work by the end of the year. Second, TDR has presented an operational plan for what Terry calls a “credible and effective” voluntary pooled funding mechanism to support R&D on the priorities identified by the Expert Committee.

Where to Next?

Unsurprisingly, the biggest challenge as things move forward will be mobilizing donor resources for the pooled fund. According to Observatory data, investments in R&D for neglected diseases peaked in 2009 and then declined 7% between 2012-2015, But even in this challenging environment, Kevin McCarthy (Senior Health Advisor, European Commission-EuropeAid) expressed guarded optimism. He explained that health R&D would normally fall outside EuropeAid’s remit. But the fact that the European Parliament has identified access to medicines (especially in developing countries) as a priority area created space for EuropeAid to get involved and work with WHO on these projects.

Likewise, Tania Dussey-Cavassini (the outgoing Swiss Ambassador for Global Health) called attention to the “unusual” combination of hosts for the event, suggesting that it indicates broad-based support for the R&D agenda among Member States. The take-home message here is that if this level of political will continues, progress is possible.

Furthermore, Robert Terry pointed out that one of the most valuable aspects of the Observatory and the Product Profile Database is that they can be used by anyone. Even if political will lapses, private investors can use these tools to direct R&D funds towards the areas of greatest need. He also noted that “if we can agree on priorities, it is possible that we can move closer to coordinating R&D activity. [However] the problem is that everyone likes coordination, but no one wants to be coordinated.”

Mara Pillinger is a PhD Candidate at George Washington University. Follow her on Twitter @mplngr.