Developing Biodefense Countermeasures: Lessons from the Orphan Drug Act and Project BioShield Anthrax Contracts

US preparedness for a potential bioterrorist attack must include a comprehensivestockpile of countermeasures against biological agents. The Project BioShield Act wasenacted in 2004 to promote the development of biodefense countermeasures. Under theact, however, only a limited number of such products have successfully been developedand none have yet received regulatory approval. This dissertation seeks to understand thebarriers and ineffective incentives that challenge the development of suchcountermeasures by analyzing the following: 1) Evaluation of factors (technical,regulatory, economic, legal, political and military) surrounding the development ofbiodefense products; 2) Comparison of the impact of two acts with the aim to incentivizethe development of products with little market appeal: Project BioShield and the OrphanDrug Act; and 3) Comparison of four companies with a BioShield anthrax contract in amultiple-case study. The purpose of these analyses is to identify opportunities to removebarriers that challenge the development of biodefense countermeasures.This dissertation finds that the most critical barriers to the successful development andapproval of biodefense countermeasures are regulatory and political. The regulatorypathway is still unproven, and the political barrier is apparent because the government isnot only the sole customer, but also the regulator, legislator, and collaborator of thebiodefense industry. Radical strategies to lower the regulatory and manufacturingbarriers have been proposed but still need to be implemented by the Department ofHealth and Human Services. This initiative suggests, however, that without significantfederal funding and keen involvement, the development of biodefense countermeasurescould be stumped. This dissertation proposes an alternative model where the resourcesand burdens are shared between the private industry, the government, and a thirdcontractor party on standby. Additional opportunities to remove barriers include the needfor federal commitment to maintain biodefense and BioShield funding, continuousimprovement of federal coordination and communication, using a simplified acquisitionprocedure as opposed to the current approach favoring full and open competition,investment in biodefense product(s) and associated technological platforms, and thepromotion of a government-industry-academia-military partnership. The alternativemodel enhancement to the biodefense drug model suggested in this dissertation alongwith BioShield can create the necessary foundation to stimulate the development ofbiodefense countermeasures for preparedness against potential bioterrorist attacks.