Clinical Trials and Regulatory Pathways for Neglected Diseases

Defining the Issue

Neglected diseases -- which include malaria, tuberculosis, and a dozen other parasitic, soil transmitted, bacterial, and tropical infections – affect an estimated one billion people, including 400 million children, in Africa, Asia, tropical regions of Latin America, and parts of the Middle East. Malaria and tuberculosis alone kill an estimated 2.6 million people each year. Less deadly neglected diseases disable, deform, and increase their sufferers' vulnerability to other infectious diseases like HIV/AIDS.

A recent surge in investments from the Bill & Melinda Gates Foundation, the U.S. National Institutes of Health, and other donors have supported product development partnerships (PDPs) that have helped spur progress in developing new health products for neglected diseases. But two substantial bottlenecks prevent these products from reaching those in need. First, the research and regulatory capacity in many neglected disease-endemic settings is not adequate to support the clinical trials that need to occur there in order to complete the development of these products. Second, current levels of financing are insufficient to support the clinical development of these products.

Addressing these twin challenges requires increased funding for late-stage clinical trials and capacity building, and greater attention to how these trials and their regulatory pathways can be improved to reduce unnecessary costs, delays, and risks to trial subjects. The working group developed practical, scalable, and feasible strategies for meeting those needs and providing evidence to support their implementation.

Improving the Clinical Development and Regulatory Pathway

The working group’s final report identifies practical and feasible actions to address the persistent challenges of late stage clinical development for neglected disease products, and recommends a two pronged solution:

(1) the development of regional pathways for the review of clinical trials in disease-endemic settings, and;

(2) better, faster, and cheaper clinical trials by focusing on key parameters and objectives of trials, evidence-driven approaches, and early engagement among trial sponsors.

The report outlines the responsibilities different global actors should bear, while pointing out opportunities and needs at the local, national, and intra-institutional levels. Finally, the report recommends approaches against existing clinical trial data to demonstrate the feasibility and potential time, money, and lives saved.

Working Group Participants

The Working Group brought together a group of high-level stakeholders and experts from industry, PDPs, regulatory authorities, investigators, donors, technical agencies, and the global health policy community. Members served in a personal capacity, independent of their institutional affiliation.

Members:

• Vincent Ahonkhai, Senior Regulatory Officer, Global Health Delivery, Bill & Melinda Gates Foundation
• Ernst Berndt, Professor of Applied Economics, MIT Sloan School of Management
• Fred Binka, Project Manager, INDEPTH Malaria Clinical Trials Alliance (MCTA)
• Tom Bollyky, Chair, Visiting Fellow, Center for Global Development
• Mike Brennan, Senior Advisor, Global Affairs, AERAS Global TB Vaccine Foundation
• Richard Chin, Chief Executive Officer, Institute for OneWorld Health
• Liliana Chocarro, Former Scientist, Regulatory Pathways, World Health Organization
• Ralf Clemens, Head, Global Development of Vaccines, Novartis
• Iain Cockburn, Professor of Finance and Economics, Boston University School of Management
• David Dilts, Director of Clinical Research, Knight Cancer Institute; Professor, Division of Management, Oregon Health & Science University; and Co-Director, Center for Management Research in Healthcare
• Paul Huckle, Senior Vice President, Global Regulatory Affairs, GlaxoSmithKline
• John Hurvitz, Partner, Covington & Burling LLP
• Richard Kingham, Partner, Covington & Burling LLP
• Judith Kramer, Executive Director, Clinical Trials Transformation Initiative and Associate Professor of Medicine, Duke University School of Medicine
• Marc LaForce, Global Program Leader, Meningitis Vaccine Project, PATH
• Orin Levine, Executive Director, PneumoADIP and Associate Professor, Johns Hopkins Bloomberg School of Public Health
• Melinda Moree, Chief Executive Officer, BIO Ventures for Global Health
• John Purves, Former Head, Quality of Medicines Sector, Pre-authorization Evaluation of Medicines for Human Use Unit, European Medicines Evaluation Agency
• Vijaya Ramachandran, Senior Fellow, Center for Global Development
• David Shoultz, Business Officer, Global Health, Infectious Disease Development, Bill & Melinda Gates Foundation

Observers:

• Yuppadee Javroongrit, Assistant Director & Head of International Affairs & Investigational Drug Group, Drug Control Division, Food and Drug Administration, Ministry of Public Health, Thailand and Co-Chair, ASEAN Consultative Committee on Standards and Quality/Pharmaceuticals Product Working Group
• Margareth Ndomondo-Sigondo, Pharmaceutical Coordinator, New Partnership For Africa's Development (NEPAD)
• Wendy Taylor, Senior Advisor, Innovative Finance and Public Private Partnerships, USAID