Developing an aggregator mechanism for late-stage clinical trials of neglected disease product candidates

There have been significant improvements in recent years in the early stage development of products for poverty-related and neglected diseases (PRNDs). However, there are still major challenges in the funding of late-stage clinical trials of candidate products for these diseases. For vaccine development specifically, Rappuoli and colleagues recently concluded that “these improvements in the early development process have revealed a new, and possibly more perilous, Valley of Death in the late vaccine development phase.”

There are three major challenges in conducting phase III trials for PRND product development:

1. Such trials are expensive and companies often shy away from investing in them because there is no commercial market for most PRNDs.
2. There is poor coordination on late-stage trials across R&D initiatives. At present, there is no overarching global mechanism that is “steering the ship”—there is no universally agreed upon process for prioritizing R&D investments for PRNDs, for selecting the most promising candidates, or for coordinating the multiple, overlapping research programs worldwide. The result is duplication, waste, and ultimately delays in the development of products.
3. Current R&D efforts for PRNDs are “top-down”—they are controlled by high-income countries (HICs) and have generally done poorly at including decision-makers from high-burden countries. It is policymakers in low-and middle-income countries (LMICs) who are in the trenches when it comes to controlling PRNDs—yet they are often not at the table when it comes to deciding on what gets funded, where research is conducted, who gets access to intellectual property, and where and how the technologies end up being manufactured. All these steps need to be “globalized” if we are to develop and deploy new control tools.

This study examines whether and how these challenges could potentially be addressed through a new kind of global funding platform for late-stage clinical trials (an “R&D aggregator”). Two key aims of this study are

• To assess the demand for and design of an R&D aggregator
• To assess the health and economic benefits of a global aggregator (i.e., to assess the investment case).

Sabine Campe, Open Consultants
Wenhui Mao, Center for Policy Impact in Global Health
Kaci Kennedy McDade, Center for Policy Impact in Global Health
Mohamed Mustafa Diab, Duke Center for Policy Impact in Global Health
Osondu Ogbuoji, Center for Policy Impact in Global Health
Marco Schäferhoff, Open Consultants
Shantih van Hoog, Open Consultants
Gavin Yamey, Center for Policy Impact in Global Health

Robert Karanja, Villgo Kenya
Dr. Mziwandile Madikizela, SAMRC
Vipul Chowdhary, Technical Consultant
Dr. Yangmu Huang, Technical Consultant

Working paper: Yamey G, Schäferhoff M, Moran M, Diab MM, McDade KK, Mao W, Chodavadia P, Zimmerman A, Huang Y, Chowdhary V, Karanja R, Madikizela M, Ogbuoji O. Developing an aggregator mechanism for late-stage clinical trials of neglected disease product candidates. The Center for Policy Impact in Global Health. Duke Global Working Paper Series: number 23, October 2020. Available at: https://centerforpolicyimpact.org/wp-content/uploads/sites/18/2020/10/Aggregator-for-Late-Stage-Trials_Working-Paper-FINAL.pdf

This project is funded by the Bill & Melinda Gates Foundation.