The Reagan-Udall Foundation (RUF) for the FDA is a partner in the CPTR Initiative, a global initiative launched by the Bill and Melinda Gates Foundation, the Critical Path Institute, and the TB Alliance to accelerate the development of new tuberculosis (TB) multi-drug regimens. In conjunction with the CPTR team, RUF is proactively bringing together a broad range of international TB stakeholders with diverse perspectives, including: regulators, drug developers, academic scientists, and advocates to identify, prioritize, and work toward resolving regulatory science issues and challenges of drug development and treatments for people with TB.
Though sometimes considered a disease of the past, TB remains a global health priority. The 2014 World Health Organization’s Global Tuberculosis Report reports that in 2013 alone, 9 million people fell ill with TB and 1.5 million died from the disease. This burden of disease is not shared equally—22 high-TB burden countries account for 80% of all TB cases. Confounding the problem further, is the threat of multi-drug resistance–480,000 cases of MDR-TB were reported in 2014. Further, the number of TB and MDR-TB cases is likely a low estimate, as roughly 3 million cases go undiagnosed or unreported each year. An estimated US $8 billion annually is needed to enact a full response to TB; however, currently there is an annual shortfall of US $2 billion. The CPTR partnership exemplifies the type of multi-stakeholder collaboration that will be necessary to address the global challenge that TB presents.
The current first-line regimen used to treat active, drug-sensitive TB contains 4 drugs that are nearly 50 years old. This treatment takes six to nine months to complete and can have severe side effects. While there has been some recent progress with the approvals of bedaquiline (by FDA in 2012; by EMA in 2014) and delamanid (by EMA in 2014), there is still an urgent need for new treatments that are shorter, simpler, more effective, less toxic, and less expensive in order to defeat TB.
CPTR is championing a new paradigm to speed promising TB treatments to patients. It focuses on shifting the unit of development from an individual drug to combinations of drugs, which can be tested together and developed as a regimen from early clinical testing. Advances in regulatory science will help clearly evaluate experimental TB drugs both on their own and within the context of a regimen. This new approach has the potential to shorten the time needed to develop new TB treatment regimens by decades, as well as significantly reduce development costs.
Why the Reagan-Udall Foundation?
As part of the CPTR, RUF’s focus is on the regulatory scientific process. While the goal of the CPTR initiative is to accelerate the development of new TB drug regimens, RUF is focused on incorporating and addressing concerns from the broad range of international TB stakeholders involved: regulators, drug developers, academic scientists, and advocates. The ultimate goal is to improve overall public health.
RUF serves as a neutral convener in organizing meetings with and between these stakeholders to facilitate an open dialogue. These discussions serve as a platform for identifying critical needs within TB drug development. RUF is supporting regulatory science research activities, working to improve current pathways for TB drug development and advancing the science behind innovative pathways for the development of novel combination drug regimens.
RUF’s areas of work within CPTR:
- Working to ensure early, effective, and appropriate collaboration with key stakeholders and communities involved in TB clinical research.
- Identifying and developing tools and methods to facilitate participatory approaches in TB research.
2. Global Regulatory Pathways
- Working to foster dialogue along key regulatory agencies, WHO, and National TB Control Programs (NTPs) to develop strategies to mitigate regulatory science challenges.
- Identifying more efficient regulatory pathways to facilitate TB drug/regimen development
Project Funding – This project is supported by the following grants:
• Bill & Melinda Gates Foundation – Grant (2011-2014) – $977,165
• Bill & Melinda Gates Foundation – Grant (2014-2017) – $1,229,613
Project Status: Ongoing