1. CPTR Key Accomplishments: Research Resources Group October 3, 2012

2. CPTR Structure Regulatory Science Consortium Research Resources Group Drug Development Coalition ADVISORY PANEL

3. CPTR Structure Regulatory Science Consortium Research Resources Group Drug Development Coalition

4. Ensuring an Ethical Process Clinical Trial Capacity Resources Needs & Funding Stakeholder Engagement Access & Appropriate Use Of New Drugs Value of Consortium Approach Global Regulatory Pathways Research Resources

5. Increase the likelihood of successful TB drug development by developing resources and creating the infrastructure necessary to support the development of novel TB regimens The goals of the CPTR Research Resources Group are to work collaboratively with CPTR partners to identify and address challenges in regimen development, including: Supporting capacity-building activities for TB clinical trial sites Soliciting global participation in addressing regulatory challenges Promoting engagement all stakeholders involved in a TB trial Fostering access to new TB therapies and ensuring appropriate use 5 Research Resources Group: Mission

6. Research Resources Group: Workgroups 6 Access & Appropriate Use Workgroup Increase access to TB therapy by those in greatest need both before and after formal approval Shorten normative, regulatory, and policy processes to accelerate access to life-saving TB therapies Clinical Trials Infrastructure Workgroup Increase clinical trial and laboratory site capacity and quality Global Regulatory Pathways Workgroup Identify and address challenges in the current regulatory process Identify and promote new and existing regulatory pathways to facilitate efficient review processes Stakeholder & Community Engagement Workgroup Identify and develop resources to facilitate participatory approaches within TB drug trials Ensure effective and appropriate stakeholder and community engagement in TB drug trials

7. Access & Appropriate Use Key Accomplishments – Paper on Compassionate Use and Expanded Access accepted and awaiting publication in the International Journal of TB and Lung Diseases Next Steps – Expanding the understanding of challenges related to pre-approval and post-approval access to TB drugs/drug regimens to determine appropriate next steps – Working to facilitate the uptake of WHO recommendations on compassionate use 7

8. Clinical Trials Infrastructure Key Accomplishments – Providing a forum for clinical trial networks, donors, and researchers to discuss collaborations at annual TB Union meetings – Developing the TB Clinical Trial Online Database which links experienced TB sites with groups looking for this capacity Next Steps – Developing commonly-agreed standards for assessing and ensuring quality of mycobacterial laboratories 8

9. Clinical Trials Database Example 9

10. Global Regulatory Pathways Key Accomplishments – Laying the groundwork for joint regional review for TB clinical trials and marketing authorizations which aim to increase the efficiency of review processes Next Steps – Present concepts for joint review and other modified regulatory pathways to global audience to get feedback and support 10

11. Requirements for Joint Review Key Required Elements Manufacturer/Sponsor Agreement to participate, as owners of the data NRAs Consensus from all countries involved on the joint process or ‘accepted’ review Designated person from each country involved to participate in review process Experts Conduct review and/or support NRA staff working through review and questions Neutral Support Partner Facilitate the review procedure and information dissemination Provide venue for discussion Funding Source Cover associated expenses (e.g. information dissemination, meetings, travel, etc.) All Agreement on format of submission Agreement on timeframe of specific activities

12. Stakeholder & Community Engagement Key Accomplishments – Released the Good Participatory Practice Guidelines for TB Drug Trials (GPP-TB) which p rovides a principle-based framework for effective engagement of all stakeholders and works towards establishing a common language for incorporating a participatory approach to TB drug research Next Steps – Developing tools and materials to facilitate the implementation of the GPP-TB – Identifying clinical trial sites to ‘pilot’ the GPP-TB 12

13. Objective of the GPP-TB Guidelines Encourage greater attention to community interests Help establish shared standards, expectations, and accountability for effective and outcome- driven engagement throughout all phases of TB drug trials. 13

14. Thank you 14