Mar 26, 2010 (CIDRAP News) – The National Biodefense Science Board today capped a hurried review by recommending that the federal government launch a major effort to better focus its activities to develop medical countermeasures (MCMs) for chemical, biological, radiological, and nuclear (CBRN) threats, starting by choosing at least three new countermeasures for priority efforts.
The recommendation was one of 23 in a 103-page report that the NBSB approved in a meeting this morning. The report is part of a comprehensive review of the national MCM efforts, ordered by Health and Human Services (HHS) Secretary Kathleen Sebelius last December. The action was prompted in part by the initially slow deliveries of vaccine for pandemic H1N1 influenza.
As part of the overall review, Dr. Nicole Lurie, HHS assistant secretary for preparedness and response (ASPR), asked the NBSB in late January to produce a written report by today on the issues and challenges facing what is known as the Public Health Emergency Medical Countermeasure Enterprise, or PHEMCE. The review included a workshop to review the strategic management, leadership, and accountability structure of the program.
Besides recommending prioritizing new countermeasures, the NBSB report calls on HHS to, among other things:
- Coordinate with the White House on a "unifying end-to-end national strategy" to address CBRN threats
- Empower Lurie to be the operational leader of MCM development
- Change the guidance for the Food and Drug Administration's "animal rule," which allows the FDA to provide emergency authorization of a drug on the basis of evidence from animal experiments, if human trials are too dangerous
- Give the public more information about CBRN threats, as part of a sustained communication plan
At the meeting, Lurie noted that the broad MCM program review also included a workshop convened by the Institute of Medicine and discussions by the President's Council on Science and Technology.
Earlier indications were that ASPR's goal was to complete the overall review by Mar 31, but it was clear at the meeting that the timetable has slipped a bit. "We are looking at April—we are coming down to the wire," ASPR spokeswoman Gretchen Michael told CIDRAP News today.
John G. Grabenstein, RPh, PhD, a vaccine expert at Merck, was one of three NBSB members who led the fast-tracked report-writing effort. In presenting it today, he commented, "The effort has been enormous, especially when you consider that we all have day jobs."
The report says the NBSB identified three themes for the additional effort to develop MCMs: "prioritization" of the threats and the needed countermeasures, "synchronization" or coordination of efforts across government agencies, and "anticipation," referring to the need to foresee logistical problems in the distribution and dispensing of countermeasures.
"To do all of that, you need leadership," Grabenstein said. "If there is not a concerted effort, if there's not a voice reminding people to keep the emphasis on these weapons countermeasures during periods of calm, we will be taken by surprise."
Grabenstein and John S. Parker, MD, who served as another co-chair of the report development, both emphasized that the report was not requested "because something was going wrong," in Parker's words, but because the administration wants to improve the MCM effort. Parker works for Scientific Applications International Corp.
"Our most important conclusion is that leadership, discipline, and synchronized effort are not lacking but are unfocused," the report states. "This problem can be overcome by the HHS secretary assembling the agency leaders, designating the ASPR as the coordinating authority, and directing a synchronized, prioritized, common effort toward the nation's goals."
The board recommends that the HHS chief confer with the white House on a national strategy for countering CBRN threats, including the possibility of establishing a position on the National Security Council to lead the strategy.
The third recommendation is that the HHS secretary "promptly identifies at least three high-priority new MCMs the department will develop to counter CBRN threats, with target timelines. At least one of these should address radiation exposure." That step, which will "crystallize" the PHEMCE effort, should be coupled with the development of broader prioritized lists of threats to guide further efforts, the report says.
The panel doesn't specify which three MCMs should top the list. "We have all the efforts since 2001—surely the top three or top few should be apparent," said Grabenstein.
Under the Pandemic and All-Hazards Preparedness Act, the ASPR has the lead role in managing MCM activities, and "this office must be fully empowered to undertake this role," the report says. In line with this, the ASPR should be assigned to "refine the HHS acquisition structure and metrics, to provide accountability for the MCM program."
Parker commented that it has long been difficult to adapt federal acquisition regulations to medical and biological products. "We hope HHS takes the opportunity and importance of this mission to work with the Congress . . . that they can actually come up with the structure to make it reasonable and productive to produce a biological," he said.
Another recommendation says the HHS secretary should designate the Biomedical Advanced Research and Development Authority (BARDA) as "the MCM Portfolio Director, to coordinate technical aspects of balancing the countermeasures menu."
The HHS secretary is also urged to work with the Department of Homeland Security to overcome obstacles to the timely distribution and administration of MCMs to people in need, including children and those with limited functional ability.
Lurie asked the board if it was their intent that all MCMs end up in the Strategic National Stockpile, or if some products might be stored by the manufacturers.
Parker replied that there may be cases in which manufacturers could store a product in bulk and distribute it in an emergency. "I don't think it's the intent of the paper to say that the only distribution system is the national stockpile," he said.
In connection with MCM distribution, Grabenstein said the question of home stockpiling of antibiotics remains unresolved. "Can the states and feds get products out in time?" he asked. "It's a big piece that needs to get resolved."
Another section of the report calls for "consistent, adequate, and balanced funding" for countermeasures development. It recommends that the HHS secretary determine the CBRN-related budget needs of all the relevant agencies for fiscal year 2011 and request an appropriate revision of the administration's proposed budget.
Further, the panel says HHS should develop a coordinated budget request for 2012 and beyond, plus a legislative plan to seek multi-year funding authority. At the same time, the agency will need to seek reauthorization of the Project Bioshield fund, which expires in 2013.
The fourth section of the report, "Function and Activity," includes a lengthy set of eight recommendations. It suggests that the government take a "decentralized" approach to the discovery and early development of MCMs, "harnessing the creativity and innovation of the nation's biotechnology companies." For advanced development, a more centralized approach involving public-private partnerships or federal research and development centers is recommended.
The panel also recommends that the FDA come up with a plan for designating candidate MCMs for high-priority review and a plan to revise the "animal rule."
Testimony from many countermeasure developers convinced the NBSB that the FDA's January 2009 draft guidance for industry on the animal rule "contains unrealistic expectations" for CBRN countermeasures, the report says. The board recommends a rewrite of the guidance within 6 months.
Some other recommendations under the "function and activity" heading call on the ASPR to:
- Identify needs for more pediatric products for the national stockpile
- Come up with a plan for creating and maintaining "dossiers" on the top 20 MCMs to facilitate the granting of emergency use authorizations for them in an emergency
- Create MCM distribution plans for children and for people with functional limitations
In addition, the heads of the National Institutes of Health and National Institute for Allergy and Infectious Diseases (NIAID) are urged to devise a plan for "how to align resources for MCMs to the national prioritized lists of research goals and product requirements." The NIAID, along with BARDA, is also advised to develop a plan to address the need for screening and diagnostic tests that can be used in clinical settings.
On the communication side, the board says the ASPR should make plans to give the public more information about CBRN consequences and to provide information about MCMs before and during emergencies. It says the government has failed to explain to the public the urgent need for countermeasures.
"PHEMCE and ASPR leaders need to think of themselves as leading a very specific type of research and development organization with a distinct primary leader," the report states. "The primary leader needs to develop a strategy that brands the PHEMCE in such a way that the American public understands the important roles of HHS and ASPR in preparedness and response."
The report won warm praise from Lurie, who said at the meeting, "You've done an incredibly meaningful piece of work." She called the document "very, very thoughtful, meaningful, and provocative."
"This is really a report that will help promote lasting and systemic change in a system that needs to be transformed," she commented.
Mar 26 NBSB meeting agenda
Feb 10 CIDRAP News story "HHS advisors face short deadline for analyzing biodefense"