Reading Room Production — page 452

An issue that was cited as an example for how markets might be improved for these products was the use of the Priority Review Voucher System applied to biodefense products as is now afforded to industry for orphan products under the Orphan Drug Act. If a system could be developed wherein the voucher was transferable or saleable to another industry partner, it would represent a value proposition that could be understood by the investor community as a potential opportunity for return on investment. There was discussion regarding the theoretical vs. practical aspect of this advantage for priority review, but agreement that another feature regarding 3 months of granted exclusivity on review would be of value. Regulatory Approval Pathway On the issue of better defining the pathway to regulatory approval of the product, a general description of the preferred process is that the FDA should be very specific in the requirements that a company is expected to demonstrate for a candidate study, rather than the current system where a company provides data for a study, and is then told whether it meets the reviewers' approval. Dr. Zeke Emanuel pressed for more specificity on the steps where this pathway could be made more transparent. This is an action item for the industry to be further defined with more dialogue. One concrete example of how better understanding of a path to approval would affected a company's effectiveness in producing countermeasures was given by Novartis regarding use of an adjuvant to increase immunogenicity of a vaccine. Adjuvants stimulate an immune response at a lower antigen concentration, and potentially produce a broadened response profile to similar antigens, thus increasing the manufacturing yield per unit of production. "In general, you can have more product produced if the material is more potent" was the phrase used to define this issue. In Europe, this pathway to use of adjuvant through their regulatory agency permitted the use of adjuvants in influenza vaccine, so what is the reason that the U.S. cannot seem to provide a reasonable pathway to accomplish a similar allowance ? There is a need for harmonization of the regulatory pathway with the EMEA (the European Medicines Agency) on issues such as this. It would also permit global companies more opportunity for accessing larger markets. Zeke Emanuel pointed out that the issue of adjuvants had to also account for the perceptions of safety by the U.S. population and must be factored into the market issues concerning this approach. Merck added an example regarding lack of a clear understanding of the regulatory path to have an anthrax lethal factor inhibitor approved. • Action item: identify what steps or processes are needed for international harmonization in regulatory pathways, with adjuvants as an exemplar • Action item: industry needs to be more specific in what steps should be taken to improve the FDA's study review process (prospective vs. retrospective feedback). Liability