HHS is hoping to redefine its relationship with biotech and pharmaceutical firms that are developing Rx countermeasures though a series of "pivotal studies" under which government works more closely with industry at the beginning stages of development.

Nicole Lurie, HHS assistant secretary for preparedness and response, told the Pharmaceutical Research and Manufacturers of America's recent annual meeting, "Shortly, we'll begin what I will say tongue in cheek are some pivotal studies to test ways that we change the way we work with some of our industry partners."

The pilot programs "will involve getting together our top scientists at NIH and CDC and FDA and BARDA [Biomedical Advance Research and Development Authority] to work together with companies upfront [and] all the way through the development process, so that we can anticipate problems and challenges together ... rather than to confront unanticipated surprises and slow things down at the back end."

The programs will involve up to six products and up to six companies, and Lurie joked that there's been so much interest in participating "we're probably going to have to have a lottery."

Selection will, of course, be non-random, and one issue that HHS may be considering is the size of the firm that the department could be working with.

"It's clear that much of the innovation right now in this industry - not all of it, but a lot of it - comes from small biotech companies and small innovator companies," Lurie said. "And then, as things stand now, many of them don't necessarily have all of that scale-out manufacturing capacity and surge capacity that large, experienced companies do."

Struggles With Manufacturing

Established firms also have production challenges, Lurie noted. "We hear as we talk to large, experienced companies that sometimes their ranks are also thin. I was talking to a company just last week that said, 'You don't understand. We have six people in our whole company that know how to do a given set of processes in vaccine manufacturing.'"

While vaccines are only one element of medical countermeasures, the delays in the production of the H1N1 vaccine last fall highlighted concerns that the production technologies that "we depend on, tried and true over the past 50 years, need to be supplemented with newer, faster and more flexible technologies," Lurie said.

Many are pointing to cell-based vaccine production as the answer to these production concerns, but while industry and government are actively engaged in the issue, the technology may not yet have firms' full devotion. FDA recently finalized a guidance on production issues for cell-based vaccines, and the event was touted in an HHS press release, but during drafting the document failed to generate a single comment from industry.

How To Engage - And Who To Engage

HHS' challenges for medical countermeasure development go beyond optimizing a single manufacturing technology. The government wants to create an environment that will encourage rapid development and production of countermeasures.

To decide how best to do that, the government has solicited advice from the Institute of Medicine, the National Biodefense Science Board, the President's Council of Advisors on Science and Technology, and PRTM Management Consultants.

The aim of the review was to "really improve this enterprise, from identifying which countermeasures are really needed to additional research to advanced R&D and ultimately to distribution and administration of the final product," Lurie said.

"What we see already in our review - and it's not quite done - [is] that we across government have to be more strategically aligned in pursuing medical countermeasures in a much more integrated way, working toward a common goal that meets a well-defined and really clearly articulated set of threats, including those from Mother Nature," Lurie said.

Who will do the creating and producing is perhaps as big a question as how it will be done. Some smaller biotech firms see the potential for a huge payday, but lack the regulatory and manufacturing experience to deliver on their ideas smoothly. Large manufacturers, on the other hand, may be better equipped to move forward on projects, but could become wary after the high-profile contract revisions related to anthrax antibiotics and H1N1 flu vaccines.

At a recent IoM meeting to discuss development of bioterror products, Chuck Ludlam, a former top staffer for BIO and Sen. Joe Lieberman, I-Conn., said that working with Big Pharma would be the key to success, but those firms would need "goal line" incentives such as "cash, tax benefits, patent benefits and liability protections" (¹ Also see "Revive BioShield II: Bring Big Pharma Back Into Medical Countermeasures " - Pink Sheet, 1 February, 2010.).

- M. Nielsen Hobbs ( ² n.hobbs@elsevier.com )

Rx Countermeasures: HHS Launching "Pivotal Studies" To Engage Industry

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