The Public Health Emergency Medical Countermeasures Enterprise Innovative Strategies to Enhance Products from Discovery Through Approval: Workshop Summary (2010) / Chapter Skim
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Appendix D: Case Studies of HHS Chemical, Biological, Radiological, and Nuclear Medical Countermeasure Development Programs, Executive Summary
Appendix D: Case Studies of HHS Chemical, Biological, Radiological, and Nuclear Medical Countermeasure Development Programs, Executive Summary
Pages 91-112
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From page 91... ...
By Robert Kadlec Vice President Global Public Sector PRTM The US government has a long and complex history of medical countermeasure development programs for chemical, biological, radiological and nuclear threats. The goal of this document is to revisit those experiences in order to glean insights into overarching challenges, successful strategies, and areas for improvement across the mission space.
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Medical Countermeasures for Hematopoietic Acute Radiation Syn-drome (hARS)
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APPENDIX D 93 Case Study Summary: Right Side/Response Activities Preparedness Assessment Framework Mission Components
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94 EMERGENCY MEDICAL COUNTERMEASURES ENTERPRISE The Elements of Risk Scientific/Technical Risks Assessing the risks of commercial drug development is complicated by the source of the candidate product, therapeutic class, and product type. There are generally higher clinical approval success rates for in-licensed candidates as compared to candidates that are self-originated.
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The findings also suggest that product development programs and associated pipeline strategies must be tailored for the desired product category. While the anti-infective statistics above provide some optimism for medical countermeasures for biodefense indications, the many challenges of working with these threat agents limit any real ability to compare across categories of threat agents.
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Companies prefer when a candidate fails early in its development, as it limits actual and opportunity costs. For the development of CBRN medical countermeasures, failure must be viewed similarly: inevitable, acceptable, and ideally occurring at an early stage in development.
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BARDA has not yet utilized the Other Transaction Authority that has been granted to it, but NIAID has shown some success in using MTAs, CDAs, and modified clinical trial agreements to effectively collaborate with private sector partners. The liability protections enacted in the PREP Act 8 has assuaged the concerns of many firms that develop medical countermeasures.
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Therefore, the opportunity costs alone limit the number of industry partners interested in initiating CBRN medical countermeasure programs. Companies involved in the CBRN medical countermeasure market may also encounter unexpected financial challenges that require additional assistance from the USG.
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Perceptions of the CBRN medical countermeasures market, and hence profit potentials, have their own set of influences as to the kinds of companies and investors attracted to the CBRN medical countermeasure enterprise. To date, with few exceptions, large, experienced pharmaceutical companies have not entered the CBRN medical countermeasure market because of lack of sufficient profit or other incentives.
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There is an association between the risks of medical countermeasure development and perceived national security risk. High risks associated with CBRN medical countermeasure development intuitively translate to greater risk from a national security perspective.
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US GOVERNMENT RESPONSES TO RISK The USG has conducted research and development of CBRN medical countermeasures since World War II. The Department of Defense (DOD)
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Congress affirmed the priority of CBRN medical countermeasures by directing the Secretary of HHS to • ensure development and acquisition of smallpox vaccine; • accelerate the approval of priority medical countermeasures as a fast-track product through the FDA; • direct the FDA to develop the Animal Rule; and • accelerate research and development on medical countermeasures against top-priority threat agents. Congress also played a significant role in incrementally lowering the perceived barriers to medical countermeasure development.
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President Bush proposed the Project BioShield Act in 2003, which attempted to address the need for a guaranteed USG market for CBRN medical countermeasures. The original proposal would have permitted payment for acquisition directly from the US Treasury, which would have circumvented the annual Congressional authorization and appropriation process.
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under the Assistant Secretary of Preparedness and Response (ASPR) to support, coordinate, and provide oversight of advanced development of pandemic vaccines and CBRN medical countermeasures.
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There have been successes in the HHS medical countermeasure enterprise, but also ample opportunity to make improvements in its efficiency and effectiveness. The definition of success for the development of CBRN medical countermeasures can be broadly characterized as having a licensed, safe and efficacious medical countermeasure that can be rapidly distributed and administered to mitigate sickness, injury, or death resulting from exposure to CBRN agents.
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2. If considered solely on the basis of financial risk, most CBRN medical countermeasures would never be developed.
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The 2007 HHS PHEMCE Strategy and Implementation Plan for CBRN Threats represented the first effort by the US government to project future CBRN medical countermeasure needs. However, this guidance was only at the level of product category, and provided no additional detail as to the desired characteristics or quantities of each class of product.
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8. With few exceptions, experienced pharmaceutical companies have not entered the CBRN medical countermeasure market.
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HHS can improve the likelihood of success for current and future programs by exploring some of the following approaches. First, the perceived USG priority and need for CBRN medical countermeasures is inconsistent and unclear.
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HHS has not budgeted or appropriated sufficient advanced development funding for any one medical countermeasure program, much less for all medical countermeasures for all 13 identified material threats. There is a significant level of concern regarding the long-term outlook for the existing USG funding for CBRN medical countermeasures.
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The regulatory process supporting the CBRN medical countermeasure development enterprise demands improvement. Incomplete and sometimes conflicting guidance to participating companies from HHS agencies has created confusion and may have increased costs.
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It requires managing the scientific, technical, and regulatory risks to produce a profitable outcome. The US government's effort to successfully develop, procure, stockpile, and effectively use CBRN medical countermeasures is even more challenging.
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