1. R&D Blueprint : platform technologies to improve control of severe infectious diseases R&D Blueprint : platform technologies to improve control of severe infectious diseases David Wood WHO/HSI/EMP/TSN Briefing to IPC Dec 2015 1

2. 2 | Production platforms for the R&D Blueprint |  Context – lessons learned from Ebola R&D  The R&D Blueprint  Request for ideas on production platform technologies Outline of presentation

3. 3 | Production platforms for the R&D Blueprint | Lessons learned from Ebola R&D

4. 4 | Production platforms for the R&D Blueprint | To compress the usual timeline for unproven interventions from years to months, by working in parallel on: Development Testing Licensure Use Initial challenges (Ebola R&D) Ebola R&D Landscape of clinical candidates and trials http://www.who.int/medicines/ebola- treatment/rd_clinical_candidates_trials_ report/en/

5. 5 | Production platforms for the R&D Blueprint | 1) Clinical trials underway by December 2014 in the affected countries, each of which were "clinical trial naïve" prior to the epidemic 2) Efficacy results available or expected on at least: (a) 1 vaccine (b) 6 diagnostics (c) 5 therapeutics Achievements

6. Pending questions for Ebola R&D Survivors – future support of survivors; study and treatment of sequelae Future public health use of investigational products – Vaccines; e.g contacts of survivors; front line staff; disease flare-ups – CP; frozen CP banked for any near future recrudescence Future research studies – For example in non human primates Health systems capacity strengthening – Ebola has illustrated need across West Africa; how to build R&D capacity 6 6 | Production platforms for the R&D Blueprint |

7. 7 | Production platforms for the R&D Blueprint |  Need for new R&D funding models to support the development of products where the market is non- existent, unknown, or unreliable  Future R&D must encompass far more than vaccines, diagnostics and drugs e.g personal protective equipment; social science research  Collaborative fora or systems already in existence at the time of the 2014-2015 EVD crisis saved time  Countries want their own health and academic personnel to work, and learn, in collaboration with external experts Lessons learned WHO Ebola R&D Summit (11-12 May 2015) http://www.who.int/medicines/ebola-treatment/WHO_EVD_Meet_HIS_15.3_report/en/

8. 8 | Production platforms for the R&D Blueprint |  Need to establish target product profiles (TPPs), and global R&D roadmaps, for priority novel interventions  Arrangements for carrying out clinical trials should be agreed in advance e.g protocols, review mechanisms, reference preparations for assays, approved standards of care  Data generated during public-health emergencies, including negative results, should be openly shared Lessons learned (continued)

9. 9 | Production platforms for the R&D Blueprint |

10. The broader R&D challenges "The job now is to harness the lessons from Ebola to create a new R&D framework that can be used for any epidemic-prone disease, in any infectious disease emergency. What is needed is an R&D preparedness plan with clear rules, well-defined platforms for information sharing, and agreed procedures to expedite development and clinical trials". WHO Director General, Dr M Chan, 11 May 2015 10 10 | Production platforms for the R&D Blueprint |

11. 11 | Production platforms for the R&D Blueprint | Two Key and complementary objectives 1) to develop and implement a roadmap for R&D preparedness for priority pathogens 2)to enable roll-out of an emergency R&D response as early and as efficiently as possible 11

12. Five work-streams designed to identify key actions required to achieve the objectives 12 12 | Production platforms for the R&D Blueprint |

13. 13 | Production platforms for the R&D Blueprint | 13 Work-stream 2 activities underway R&D Readiness – An infographic summarizing current timelines to initiate efficacy trials for priority products – Action plans for phase 1/2 clinical trial material for each priority pathogen – A biobanking strategy – Horizon-scanning for useful technologies to nurture and adapt – Action plan to improve scientific knowledge for prioritizing and evaluating health interventions MERS CoV R&D Roadmap – A full MERS-CoV roadmap, from basic research through to commercialization of products, that can be used as an exemplar to develop a generic template for other priority pathogens – A full set of MERS Target Product Profiles (vaccine, medicines, blood products, Dx)

14. 14 | Production platforms for the R&D Blueprint | Work-stream 2 Activities underway Regulatory preparedness – Improved preclinical models – Improved clinical trial designs for efficacy – Globally agreed quality, safety and efficacy specifications for products for priority pathogens – Improved safety evaluation methodologies for health technologies used in public health emergencies – Improved regulatory readiness for emergency approval of products Platform production plan – An request for ideas on production platforms - see http://www.who.int/medicines/ ebola- treatment/public_consultplatfor ms23112015.pdf http://www.who.int/medicines/ ebola- treatment/public_consultplatfor ms23112015.pdf 14

15. 15 | Production platforms for the R&D Blueprint | Production platform plans

16. 16 | Production platforms for the R&D Blueprint | Objectives of production platform plan Ideas are requested for flexible development and production platform technologies to manufacture candidate products for evaluation in Phase 1 clinical trials before any confirmed epidemic threat Also to cover manufacture of candidate products for Phase 2 and 3 clinical evaluations during a potential epidemic 16

17. 17 | Production platforms for the R&D Blueprint | Scope Vaccines, therapeutics (drugs and blood products), and diagnostics Targeted against 5 to 10 top priority diseases, to be defined through the Blueprint process Only proposals that can address more than three priority pathogens will be considered. 17

18. 18 | Production platforms for the R&D Blueprint | Access Candidate products developed through this process should be affordable for use in populations in which they are tested and/or needed The priority pathogens may affect any country but options to address affordability in low and middle income countries (LMICs) need to be included in each proposal The submissions should explain how IP issues will be managed to ensure fair and equitable access, especially for LMICs, to any product(s) developed through the proposed platform(s). 18

19. 19 | Production platforms for the R&D Blueprint | Public health use Candidate products developed through this mechanism and that are found to have a favorable benefit-risk profile should be available in sufficient quantity to enable potential use in disease control efforts Therefore submissions should include strategies to assure readiness for production at an appropriate scale to contribute to epidemic control 19

20. 20 | Production platforms for the R&D Blueprint | Key dates Submission of ideas by Friday 15 January 2016 Workshop to present the ideas, by 28 February 2016 Notification of invitation to submit a phase 2 proposal by 15 March 2016 Phase 2 proposal deadline (invited proposals only) by Friday 29 April 2016 Second workshop, where proponents will be invited to present the shortlisted platform ideas, convened by WHO with Member States and other organizations which fund Research and Development by 31 July 2016 20

21. 21 | Production platforms for the R&D Blueprint | World Economic Forum initiative Vaccine innovation session, Davos 21 January, "to support R&D Blueprint" 90 minute discussion with the CEOs of GSK, J&J, Merck and Sanofi. SII invited Gates and Wellcome Trust have both indicated interest to have high-level representation WEF following-up with World Bank, IAVI, GAVI and PM of Norway Aim of session (preliminary thinking) - to discuss (a) key principles and (b) governance options for a vaccine innovation network 21

22. 22 | Production platforms for the R&D Blueprint | WHO has organized a series of consultation to address some of the already identified challenges 22

23. 23 | Production platforms for the R&D Blueprint | 23

24. 24 | Production platforms for the R&D Blueprint |  Ebola R&D Landscape of clinical candidates and trials –http://www.who.int/medicines/ebola- treatment/rd_clinical_candidates_trials_report/en/http://www.who.int/medicines/ebola- treatment/rd_clinical_candidates_trials_report/en/  Request for ideas on production platform technologies –http://www.who.int/medicines/ebola- treatment/public_consultplatforms23112015.pdfhttp://www.who.int/medicines/ebola- treatment/public_consultplatforms23112015.pdf  Proposed data sharing framework –http://www.who.int/medicines/ebola- treatment/blueprint_phe_data-share-results/en/http://www.who.int/medicines/ebola- treatment/blueprint_phe_data-share-results/en/ Selected links to resources