Volume 5, Number 6—December 1999
Development of Orphan Vaccines: An Industry Perspective
|1. Provide information, prioritize, and secure demand •Increase awareness of disease: set-up of special interest groups (patients, parents, professionals), expert groups, and national forums. •Acquire epidemiologic data on selected infectious diseases to guide decision-making: obtain access to data registries with comparable case-definitions across countries, and obtain information from specialized units and experts, scientific literature, patient organizations,and pharmaceutical manufacturers associations. •Establish the suitability of vaccine prevention vs. other options: realistic comparisons of vaccination with patterns and costs of other alternatives, such as treatment or vector control. •Ensure political support for orphan vaccine initiatives and organize tripartite partnerships between public, private, and nongovernmental sectors.|
|2. Facilitate vaccine research and development and national/regional approvals •Promote innovative research and development technologies that could be applied to blockbuster vaccines or, alternatively, promote low-cost traditional vaccine technologies. •Encourage public/private sector links: academic/industrial research groups. •Set international standards of quality, safety, and efficacy and define minimum amount of data required for licensure. •Make recommendations on appropriate schedules, target ages. •Promote national and regional ex-U.S. and European Community incentives on Orphan Drug Policies (Latin America, Asia). •Expand and harmonize orphan drug policies as part of the ICH process (decrease time to regulatory approval).|
|3. Ensure market/funding visibility, production and distribution •Reduce investment risks for manufacturers by providing realistic demand estimates. •Fund development of orphan vaccines for developing countries through various institutional bodies, such as CVI, WHO, UNICEF, PAHO, WB, USAID, NIH, CDC, PATH, other donor bodies, and nongovernmental organizations and foundations (e.g., Gates Foundation) on the basis of target assistance for the neediest countries based on total gross national product. •Strengthen political and public health collaboration between orphan programs (European Community, United States) and other countries to create a supranational office dedicated to orphan vaccines (World Office of Orphan Vaccine Development or CVI) that could harmonize and coordinate funding (from research to manufacturing) from various sources. •Identify and expand the pool of the committed purchasers based on expected coverage criteria. •Promote and support protection of intellectual property. •Clarify compensation programs that may assume responsibility for liability. •Evaluate tiered pricing (high/low) feasibility at two levels: •Multinational: traveler or military vaccines in industrialized countries, endemic community vaccines in developing countries. •National: a private market for the high GNP per capita subgroup, a public market for the low GNP per capita subgroup. •Establish manufacturing strategies, such as campaigning to subsidize orphan vaccine cost investments by large volume sales of EPI vaccines. •Strengthen the vaccine distribution network for the targeted population.|
ICH, International Conference on Harmonization; CVI, Children's Vaccine Initiative; WHO, World Health Organization; UNICEF, United Nation's Children's Fund; PAHO, PanAmerican Health Organization; WB, World Bank; USAID, U.S. Agency for International Development; NIH, National Institutes of Health; CDC, Centers for Disease Control and Prevention; PATH, Program for Appropriate Technology in Health; EPI, Expanded Program of Immunization.