A GLOBAL HEALTH R&D OBSERVATORY DEVELOPING A CASE FOR ITS DEVELOPMENT

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1 DRAFT WORKING PAPER 1 1 May 2013 A GLOBAL HEALTH R&D OBSERVATORY DEVELOPING A CASE FOR ITS DEVELOPMENT Executive Summary In order to ensure alignment of the proposed Global Observatory for Health R&D with expectations of Member States and effective use of resources, the project will need to be planned and developed in a phased approach. Linkages between the Observatory, the priority-setting and coordination mechanisms and modes of funding will need to be defined and established. The Observatory will build on existing sources as much as possible, and provide technical support to Member States to establish a system to monitor national R&D investments and pipelines, recognising the considerable gaps that exist in the capacity of many countries to produce this data. Success will rely on establishing effective networks and encouraging Member States to both contribute to and gain benefit from an Observatory. A sustainable business model will be developed in consultation with key stakeholders, but an initial assessment based on the WHO Global Health Observatory is that $11,500 million will be needed to support initial development of the Observatory and capacity building in Member States for the first 5 years. 1 In order to deepen the analyses presented in the Background document prepared for the open-ended meeting of Member States in November where the report and the feasibility of the recommendations proposed by the Consultative Expert Review Group s report were discussed - the WHO Secretariat has developed draft Working Papers focusing on four main elements: the global observatory for health research and development (R&D); R&D coordination and prioritization; R&D financing; and options for demonstration projects. The Working Papers are drafts and will be revised based on feedback received. 1

2 Goal There is an overarching consensus among research and development (R&D) stakeholders that a global mechanism - working title Global Observatory for Health R&D - would be a valuable asset in order to monitor and analyse relevant information on health R&D, building on national and regional observatories (or equivalent functions) and existing global data collection mechanisms with a view to contributing to the identification of gaps and opportunities for health R&D. In order to realise these functions, the Global Observatory for Health R&D would provide an online platform that would: Monitor and report on financial flows for global health R&D; Integrate information on R&D financial flows with the product pipeline and other resources that support innovation and access to medical technologies; Provide information, reports and analysis to inform policy makers, donors and researchers with special focus on developing countries and global health; and Support capacity building in the governance of R&D and innovation for improved access. The information and data on the Observatory would enable users to: Analyse data on financing and policy for global health R&D; Produce analysis to inform National R&D portfolios management; Guide R&D priority setting at national, regional and global levels; Benchmark activities e.g. between countries; and Monitor and Evaluate trends against national, regional and global strategies. Background Today there is an incomplete picture of how much the world is spending on health and diseaserelated research and development (R&D). As such it is difficult to align, or even begin to coordinate, health R&D investments with international public health priorities. 2,3 Efforts to track and map global 2 Public health, innovation and intellectual property: global strategy and plan of action (2008). World Health Assembly resolution WHA Health Research Classification Systems Current Approaches and Future Recommendations European Science Foundation Science Policy Briefing

3 health research investments are complex, incomplete, resource-intensive, and caveat-laden. 1 While existing initiatives, for example the G-FINDER survey, HRweb and the LATTES network, are demonstrating alternative approaches to the collection of data and resource flows, none are comprehensive and all come with certain limitations in the diseases they cover, the degree to which public and private funding can be assessed and the absence of a standard system for reporting R&D data. However, the numerous data sources that are now available online provides a new opportunity, not available to previous initiatives, to bring this data together (see Table 1). Learning and expertise is available in other sectors, such as meteorology, environment and physics, which are experienced in bringing together large and complex data. With the current interest in establishing a Global Observatory for Health R&D, there is a real opportunity to create a valuable new resource. Project outline developing a Global Observatory for Health R&D There is no off-the-shelf solution to create an R&D Observatory. The technical challenges to its establishment are significant and, to add real value, a global platform of this type requires a longterm commitment and sustainable sources of support. Therefore, the development of the Observatory requires a phased approach: 1) A research phase to map current initiatives and identify user requirements. 2) A planning phase to develop a sustainable business case. 3) A pilot testing phase to create the necessary technology and design of the platform. Each phase will need to be accompanied by communication, advocacy and resource mobilization activities to ensure the final Observatory is fit for purpose. Phase 1: Research - mapping current initiatives and user requirements The first phase would be a six-month study to cover three aspects: 1) A comprehensive mapping of existing initiatives, identification of possible synergies and knowledge/technology gaps. 2) A technical review inviting input from many varied providers both inside and outside health as to the various options that could be used to access, collate and analyse R&D data. 3) A comprehensive needs assessment of users to identify what they would want and use from an R&D Observatory and what they would support in order to make the platform sustainable. In Phase 1, the initial work is a mapping of existing initiatives. Therefore the preliminary outputs will be a map of what already exists what it measures and what challenges it faces for scale up. The table at the end gives a first impression of what this looks like. Specifically, this would include: 3

4 a global analysis of registered clinical trials a global analysis of published output a global analysis of funding using existing data A number of initiatives already provide some of this data (see Table 1), but large gaps exist particularly in low income countries where the need is greatest. An informal workshop has already been held on 14 February 2013 hosted by the Wellcome Trust at its HQ. The participants were drawn from: UKMRC (led the delivery of the latest HRCS report for UK), Policy Cures (G-Finder Report), Bill & Melinda Gates Foundation, NIH including Fogarty, European Commission, OECD, UNESCO, RICYT, Eurostat, Research Africa, COHRED, Wellcome Trust and WHO including TDR. A report of this meeting is available. 4 Other consultations will need to be held at national, regional and global levels in order to make the most of what is already available and to build capacity for those Member States that need support in collating this type of data. There is a need for harmonization with the other health Observatories that exist or are being planned, including: the Regional Health Observatories (for example in AFRO, EURO and EMRO), the Global Health Observatory, and the APO Asia Pacific Observatory for Health Systems and Policies and the Global Observatory for ehealth. Input will also be sought from many different types of data providers to learn from other sectors e.g. meteorology, physics, and the big internet companies to ensure that many sources of innovation can be tapped into to design the most efficient process for this type of Observatory. The output from Phase 1 will be a more comprehensive understanding of what challenges exist and potential approaches to meeting them. Phase 2: Planning a sustainable business case This phase will involve working with a select group of stakeholders to establish a Steering Group to turn the information from phase 1 into a technical specification and a static model for dissemination and feedback from the potential users. It could potentially last 6 12 months. A sustainable business case for the platform will need to be developed including the appropriate governance arrangements and realistic funding models. The final R&D Observatory could take many forms but it is most likely to be a distributed system with regional and/or national systems feeding into a mechanism. Input could be from aggregated providers such as Ministries or companies or from individual researchers using their own research profiles on social network sites e.g. the LATTES 4 WHO informal workshop on monitoring R&D resource flows. Report of a workshop hosted by Wellcome Trust 14 February

5 system of Brazil. An important function of the R&D Observatory will be to provide support and training to build capacity in the collection and analysis of R&D flows and how to link them to the product pipeline. The link between the R&D Observatory and any mechanism for the coordination or harmonization of global R&D for health will need to be mapped out as it develops. See Draft working paper 2 - Coordination and priority setting in R&D to meet health needs in developing countries. The analysis and reporting functions could be undertaken by many different users with the Observatory acting as a repository for best practices in managing R&D portfolios, setting priorities and linking the R&D through product pipelines to impact public health through improved access. The output from Phase 2 will be a comprehensive business plan that has identified the needs of users and the resources required to support the development of the Observatory in the long-term. Phase 3: Pilot testing and platform design One key aspect of this new development will be to embed the R&D Observatory within the proposed Global Innovation and Access Platform under the GSPA-PHI. There is a regional version already launched by PAHO. Some of the technological and user interface issues are already being explored in the Regional Innovation Platform. 5 WHO has within house, the technology and licences to design and launch a platform and is near to launching a similar platform in another technical area to provide real time analysis on chemicals and food safety. 6 This phase will require the identification of service providers to build and road test the final design. The final governance arrangements will need to be put in place and the Observatory will need to begin operations. On-going costs will be a small Secretariat at WHO linked to a series of regional/national providers. Translation to other UN languages will be a consideration. On-going support and capacity building in WHO Member States for the management of research systems will need to be a function of the R&D Observatory. The Observatory will also need to be maintained and the technology developed must ensure that the most efficient systems are used. This will be based on the underlying principle of always seeking 5 Global Forum for Health Research: Monitoring financial flows for health research series

6 to harvest data wherever possible, being linked with existing initiatives and building regional and national capacity to enable improved governance of R&D and innovation for improved access. An estimated budget for the first five years of operation which is subject to change in light of the planning phase is USD $ 11,500 million. 6

7 Draft Budget: USD over the first 5 years $11,500,000 These figures are based on initial discussions within WHO in running the Global Health Observatory, with PAHO as it developed its Regional Platform on Access and Innovation for Health Technologies. As such they are indicative at this stage. All figures USD $ Year 1 Year 2 Year 3 Year 4 Year 5 Phase 1 Research Mapping of initiatives 50,000 Technical review, interviews 30,000 User needs assessment 50,000 Phase 2 Planning Stakeholder meetings 100, , , , ,000 Business case development 50,000 Technical specification for tender 50,000 Phase 3 Development First static Build Web-based (English) 180,000 Pilot Beta version (English) 200,000 Final version post user testing 200,000 Steering Group 50,000 50,000 Going live ongoing cost* IT Maintenance 80,000 80,000 80,000 Translation to UN languages 250,000 Innovation Technological /Apps development Assisting data collection / capacity building 200, , , , ,000 1,000,000 1,000,000 1,000,000 Governance Group 50,000 50,000 50,000 Staff (WHO secretariat) 1 x Professional staff P5 (FTE) 327, , , , ,000 1 x Professional Staff P3 (FTE) 223, , , , ,000 1 x Professional Staff P3 (FTE) 223, , ,000 1 x Administration (50%) 103, , , , ,000 Overheads 13% 185, , , , ,790 Year Totals 1,615,900 2,127,790 2,888,280 2,605,780 2,353,790 7

8 Table 1: Current Sources of data to monitor resources in support of health R&D 7 Type of indicator Source Description Limitations Inputs UNESCO Institute for Statistics Biennial survey, re-launched in 2006, tracking overall R&D expenditures, R&D personnel and number of researchers reported by non-eu, non-oecd and non-ricyt countries. Limited scope only certain geographies, conditions, types of research Organization for Economic Cooperation and Development (OECD) Eurostat Utilizes data from OECD and RICYT, in addition to the country reporting. Main output is Gross Domestic Expenditure on R&D (GERD) but also includes expenditures by field of science (medical and health sciences is one) Annual survey tracking overall R&D expenditures & R&D personnel reported by 42 countries of which 34 are member countries. Main output is Gross Domestic Expenditure on R&D (GERD), but also reports expenditures by field of science (medical and health sciences) and socio-economic objectives (health) by sector of performance. Annual survey of EU-27 Member State expenditures on overall R&D, of which 6 are not OECD members. Limited granularity & level of detail only up to Health & Medical R&D Limited comparability different methodologies including different scope and inclusion criteria The joint questionnaire does not specifically deal with healthrelated R&D, and relevant figures have to be picked out from various sections of the databases and built up into an estimated total. Methodology as per UNESCO and OECD (joint questionnaire). RICYT G-Finder Reports totals and health related R&D. Annual survey, started in 1990, tracking principally overall R&D expenditures, R&D personnel, publications and patents reported by Portugal, Spain and 29 countries in the Americas, Reports totals and health related R&D. Annual survey of R&D disbursements for neglected disease R&D started in OECD and Eurostat do not publish exactly the same selection of data from the responses. Countries do not reply to all sections of the questionnaire. Challenging to ensure and incentivize individual country reporting. ASEAN Asia S&T Portal: ST indicators Restricted to 31 neglected diseases and the pharmaceutical tools used to prevent, control and treat them. Data collected from 240 public, private and philanthropic organizations from 34 countries. Indicators on science, technology and innovation in ASEAN countries, Australia, China, India, Japan, Republic of Korea Data thereby limits the effects that monitoring/tracking of R&D can have in terms of offering a policy support to governments and institutions. 7 Adapted from: J.A. Røttingen, S. Regmi, M. Eide, A.J. Young, R.F. Viergever, C. Årdal, J. Guzman, D. Edwards, S.A. Matlin, R.F. Terry. Mapping available health R&D data: what there, what s missing and what role for a Global Observatory. Lancet, 2013, in the press. 8

9 for Asian countries. and New Zealand. Number of indicators varies depending on country. Organization of Islamic States: R&D GFHR: Monitoring Financial Flows: Global studies published An overview of the current developments in the 57 OIC member countries measuring Human Resources in R&D, R&D Expenditures, High Technology Exports, Scientific Publications, and Patent Applications. Reports collating and analyzing expenditure data on health R&D drawing on OECD, UNESCO, RICYT and other sources; alternating annually between global surveys and studies focused on specific diseases or public investments by individual countries. African Union/NEPAD: ASTI & AOSTI Treatment Action Group (TAG) reports HIV Vaccines and Microbicides Resource Tracking Working Group reports UNESCO Institute for Statistics The African Science, Technology and Innovation Indicators (ASTII) Initiative is a program within the African Science and Technology Consolidated Plan of Action being coordinated by NEPAD Agency. Participating countries conduct Research and Development (R&D) and Innovation surveys, the outcomes of which are captured in the African Innovation Outlook (AIO). First round of R&D and Innovation surveys with 19 countries have been finalized. Annual survey of Tuberculosis R&D investments started in Annual survey of HIV&AIDS R&D investments started in See above OECD/Eurostat See above RICYT See above Processes National databases Some databases created and used by funders or research institutions as a mechanism to manage their research portfolio. Limited comparability Limited scope No global or international databases. Although global database is not likely to be feasible, agreement on classification may allow for harvesting of data. 9

10 WHO International Clinical Trials Registry Platform (ICTRP) The ICTRP provides a single point of access to information on more than 200,000 clinical trials registered at 15 different national or regional registries around the world. For the purposes of registration, a clinical trial is any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcomes. Not comprehensive - compliance with registration needs to be improved. Stronger enforcement needed. Registration data uploaded by trial managers leading to variable data quality. Data quality and adherence to standards can be improved. Outputs Thomson Reuters and others Medline and Pubmed Thomson Reuters Web of Science covers research published in more than 12,000 scientific journals and conference proceedings and is presently one of the most extensive sources of R&D outputs. There are also other sources. MEDLINE is the U.S. National Library of Medicine's (NLM) premier bibliographic database that contains over 19 million references to journal articles in life sciences with a concentration on biomedicine. PubMed comprises more than 22 million citations for biomedical literature from MEDLINE, life science journals, and online books. PubMed provides free access to MEDLINE and links to full text articles when possible. Broad but not fully comprehensive coverage of the literature. Predominance of public funded research. Bias towards reporting of positive results. Lag period between active research and publication. No clear link between publication and inputs (financing). Virtual Health Library (BIREME) Open access online source of extensive health related literature for the Latin American and Caribbean region supported by PAHO WHO. Integrates a range of bibliographic and other data sources for the health sciences. WIPO (PATENTSCOPE) Searchable database administered by the World Intellectual Property Organization (WIPO) where over 14 million patent documents including over 2 million published international patent applications (PCT) can be searched. Not easy to use or interpret the results of searches. Requires specialized training. Re:Search (WIPO) WIPO Re:Search provides public access to intellectual property for pharmaceutical compounds, technologies, know-how and data available for research and development for neglected tropical diseases, tuberculosis, and malaria. WIPO Gold WIPO GOLD is a free public resource which provides a onestop gateway to WIPO s global collections of searchable IP data. It aims to facilitate universal access to IP information. National and regional databases Databases created and used by national and regional patent offices National or regional databases Some databases created and used by national regulatory authorities including: No global or international database Drugs@FDA: official information about FDA approved innovator and generic drugs and therapeutic biological products currently approved for sale in the United States. Limited scope only certain conditions, products The European Medicines Agency (EMA) : decentralized agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of 10

11 medicines developed by pharmaceutical companies for use in the European Union. WHO prequalification system database Products prequalified by the WHO de-facto regulatory process. Outcome/ Impact Effectiveness of technologies/interventions in reducing disease burden. Real world health benefits achieved which will depend on sales/uptake and adherence. May be captured by the effective coverage measure. No global or international database. Considerable work needed to link research contribution to expected and real health impact. 11