President Obama is expected soon to sign into law a five-year reauthorization of the array of federal biodefense programs created after 9/11, following overwhelming passage of the final version of the measure earlier this week by the U.S. House of Representatives.
The House’s 370–28 vote on Monday came five days after the U.S. Senate passed by unanimous consent an identical version of the Pandemic and All-Hazards Preparedness Reauthorization Act (PAHPRA). The bill (HR 307) now goes to Obama, who is expected to sign it. How closely future funding meets PAHPRA objectives, however, won’t begin to emerge until Obama submits a budget for FY 2014.
The reauthorization extends much of the original Pandemic and All-Hazards Preparedness Act from federal fiscal years 2014 through 2018, with some significant changes.
For example, PAHPRA reauthorizes the federal Special Reserve Fund (SRF) to purchase medical countermeasures against such diseases as anthrax, botulism, and smallpox for the Strategic National Stockpile. SRF will be funded at $2.8 billion over the five-year period—half the $5.6 billion over 10 years set aside when the fund was created a decade ago. If the SRF dips below $1.5 billion, HHS must notify Congress, and explain the potential impact on priorities for medical countermeasures (MCMs) to address chemical, biological, radiological, and nuclear threats.
SRF was created through the Project BioShield Act of 2004, one of two bills being renewed through the new legislation. The other is the Pandemic and All-Hazards Preparedness Act of 2006, which established the Biodefense Advanced Research and Development Authority (BARDA) within the U.S. Department of Health and Human Services (HHS).
The measure authorizes HHS Secretary Kathleen Sebelius to support innovation under BARDA by promoting “vaccine-manufacturing technologies, dose-sparing technologies, efficacy-increasing technologies, and platform technologies.”
Perhaps the most important provision of the reauthorization for drug developers is a new requirement that Sebelius facilitate written management plans governing development and regulatory review of MCMs.
Drug sponsors would launch the process through a written request to Sebelius’ office that would include a proposed “regulatory management plan.” FDA would work with sponsors to hammer out such a plan within 90 days. Regulatory management plans would include developmental milestones and performance targets and goals for MCM candidates, with BARDA director Robin Robinson, Ph.D., prioritizing which MCMs may receive plans if resources aren’t available for all candidates.
"The regulatory management plans help solidify the process for figuring out how to get new medical countermeasures through the FDA and into the stockpile. Working with sponsors to develop these unique clinical studies and identify appropriate animal models will better enable the FDA and sponsors to make scientific decisions and hopefully expedite the regulatory process," Phyllis Arthur, senior director, vaccines, immunotherapeutics, and diagnostics policy for the Biotechnology Industry Organization (BIO), told GEN.
"The cornerstone issue with MCMs is that the clinical process is not very clear because they are not basic drugs. Sponsors have the opportunity to work with BARDA and FDA to develop clinical studies, identify the right animal models, and determine milestones in the absence of human efficacy trials, so that when an emergency comes, there are assurances that these products are going to work," Arthur said.
Another change, intended to speed up MCM R&D, will extend from eight years to 10 years the time during which a security countermeasure should qualify for approval or licensing for inclusion in Project BioShield.
PAHPRA formalizes Sebelius’ authority over MCMs by authorizing her to extend the expiration date of MCMs during emergencies if the extension is intended to support the U.S.’ ability to protect public health or military preparedness and effectiveness, and the extension is supported by “an appropriate scientific evaluation.” Sebelius is also authorized to allow MCM makers to deviate from good manufacturing practice when warranted by “the circumstances of a domestic, military, or public health emergency or material threat.”
PAHPRA also requires Sebelius to solicit input from Nicole Lurie, M.D., assistant secretary for preparedness and response, and Dr. Robinson on pediatric studies for MCMs. Sebelius must notify both about all pediatric studies for which FDA commissioner Margaret Hamburg, M.D., issues written requests. Pediatric issues will also be addressed through a new National Advisory Committee on Children and Disasters to be created under the reauthorization. The panel’s work would end September 30, 2018.