1. Gaps from the Perspective of NGOs/ Foundations/PDPs IoM Workshop: International Regulatory Harmonization Amid Globalization of Biomedical Research & Medical Product Development Washington, DC February 13-14, 2013 Vincent Ahonkhai, MD, FAAP

2. © 2013 Bill & Melinda Gates Foundation | 1 Overview of topics for this presentation Top priority areas for harmonization Key gaps in the current systems Barriers to closing those gaps Recent approaches to overcoming the barriers and addressing the gaps Thoughts on the path forward

3. © 2013 Bill & Melinda Gates Foundation | 2 The perspective of creating access and ensuring quality for the public market Product developmentRegistrationPost-registration  Initial ("home-country") registration  WHO prequalification  Country registrations

4. © 2013 Bill & Melinda Gates Foundation | 3 Diagnostics and medical devices is the top-priority product class for better harmonization Priority ranking for product classes from a global health perspective: 1.Diagnostics and medical devices No international standards 2.Vector control products WHO Pesticide Evaluation Scheme has become central process 3.Drugs and vaccines More work to do, but area where there has been the most effort 1 2 3

5. © 2013 Bill & Melinda Gates Foundation | 4 Key gaps in the global health regulatory landscape Product developmentRegistrationPost-registration  Timely and effective ethical and regulatory approval for trials  Infrastructure and expertise to support and enforce GLP and GCP  3-step process of initial registration, WHO prequalification, and country registrations  Consistent and appropriate standards for registration submission  Communication and transparency to reduce duplication of efforts  Infrastructure to detect and report safety and effectiveness data  Technical expertise to interpret and act on safety and effectiveness data

6. © 2013 Bill & Melinda Gates Foundation | 5 Barriers to closing the key gaps Diversity of countries  The communicable disease burden and family health needs of low- and middle-income countries (LMIC) are distributed across 100+ countries around the world  Wide range of standards, activities, capabilities, and aspirations across those countries Regulator resources  Lack of sufficient and sustained financing for critical activities  Difficulty attracting, developing, and retaining staff with technical expertise in a competitive market for limited talent Trust and transparency  Limited mechanisms for LMIC regulators to confidently rely on activities and assessments (like GMP inspections) of other regulators Sponsor resources  Lack of regulatory experience and capacity at global health PDPs compared to commercial developers  Prioritization of commercial interests for product development and launch over public market access

7. © 2013 Bill & Melinda Gates Foundation | 6 Recent approaches to overcoming the barriers and addressing the gaps Product developmentRegistrationPost-registration  African Vaccine Regulatory Forum (AVAREF)  European & Developing Countries Clinical Trials Partnership (EDCTP)  Critical Path to TB Drug Regimens (CPTR)  WHO Prequalification programs (PQ)  African Medicines Regulatory Harmonization (AMRH)  WHO Global Vaccine Safety Blueprint  Safety Surveillance Working Group (SSWG)  WHO Pesticide Evaluation Scheme (WHOPES)

8. © 2013 Bill & Melinda Gates Foundation | 7 Thoughts on the path forward We need a better understanding of the landscape of issues  The global regulatory system is too complex to make decisions based only on intuition or anecdote Comprehensive stakeholder engagement is critical for informed decision- making and for successful implementation of change There are many issues we could address: we must deliberately assess where we can have the most impact, and deprioritize other areas We have had successes, and we need to continue to support and expand those going forward  E.g., the development, introduction, and safety surveillance of MenAfriVac (meningitis A vaccine for the African meningitis belt)

9. © 2013 Bill & Melinda Gates Foundation | 8 Thank you