Global Vaccines 202X: Access, Equity, Ethics 2-4 May 2011 The Franklin Institute Science Museum Philadelphia, USA Sustainability: The R&D Pipeline for Neglected Disease Vaccines Elizabeth L. Ponder, PhD May 4, 2011
BIO Ventures for Global Health (BVGH) Mission: BIO Ventures for Global Health is an independent, non-profit organization whose mission is to save lives by accelerating the development of novel, biotechnology-based drugs, vaccines, and diagnostics that address the unmet medical needs of the developing world. We have a unique perspective. We look at health problems facing poor countries through a dual lens: Expertise in Industry Expertise in Global Health
The Global Health Primer provides a landscape analysis for neglected disease products • Updated online database for 17 neglected diseases: www.bvgh.org/globalhealthprimer.aspx • Covers drugs, vaccines, and diagnostics • Highlights product needs and opportunities
There are 12 approved and 160 vaccines in development for neglected diseases 106 Products in Clinical Development
Preventive vaccines receive the largest portion of neglected disease R&D funding G-FINDER ANALYSIS: Mary Moran & Javier Guzman, Policy Cures
Neglected disease vaccines are in development by 169 organizations representing 35 countries Number of Developers per Country 6 10 12 12 80 55
Looking across the vaccine landscape, several trends emerge: • Newer vaccines in development for neglected diseases are increasingly scientifically complex due to new technologies and targeting more challenging organisms • Proof of concept trials for new vaccines are progressing, but scientific learning beyond efficacy from these efforts has been minimal • A lack of financing options, clear policy statements, or WHO prequalification of an approved vaccine severely limits access Early Innovation Clinical Proof of Concept Approval and Access
Early-stage neglected disease vaccines focus on newer technologies In use in approved vaccines No approved vaccines in humans
Parasitic diseases represent the majority of neglected diseases but the minority of products Unicellular parasitic diseases: Multicellular parasitic diseases: • Schistosomiasis • Soil transmitted helminthiasis • Onchocerciasis • Lymphatic filariasis • Malaria • Sleeping sickness • Chagas disease • Leishmaniasis
Proof of concept studies are advancing but still have a long way to go PATH and BVGH (2011) The Case for Investment in Enterotoxigenic Escheriscia coli Vaccines. Clemens JD et al. (1988) J Infect Dis 158: 372-377. Peltola H et al. (1991) Lancet 338: 1285-1289. Rerks-Ngarm S et al. (2009) NEJM361: 2209-20. Casares S et al. (2010) Vaccine28: 4880-94. PAHO et al. (2004) “Progress in Shigella vaccine development.” in Vaccines: preventing disease & protecting health. Colditz GA et al. (1994) JAMA271: 698-702 WHO (2004) Weekly Epidemiological Record 79: 27-38 Moran M et al. (2011) Neglected Disease Research and Development: Is the Global Financial Crisis Changing R&D? Policy Cures.
Neglected disease vaccines challenge the traditional industry model for clinical evaluation • Traditional Industry Model: How can we help vaccines that will fail, fail faster? • For Neglected Diseases: How can we help vaccines that will fail, not just fail faster, but also fail smarter? How can we maximize learning from vaccine trials with partial efficacy? As products fail, or show only partial efficacy, how do we decide what to do next?
Key needs for future neglected disease vaccine development include: • A focus on parasitic disease vaccine development to understand the technical feasibility and potential health impact • Increased investment in the integration of scientific research questions with efficacy studies for vaccines to maximize return on public investment • More operational and clinical research on existing vaccines to support the development of clear policy statements, accelerate WHO prequalification, and inform decisions on new product needs Early Innovation Clinical Proof of Concept Approval and Access