Bill Tasks FDA with Setting up New Regulatory Pathways

| 28 February 2013

The US Senate has approved a piece of legislation passed earlier this month by the House that would generate new incentives for industry and government officials to prepare and respond to threats and diseases, including a new regulatory pathway of sorts that would facilitate the development and approval of medical countermeasures.


The bill, known as the Pandemic and All-Hazards Preparedness Reauthorization Act (PAHPRA) of 2012 was passed by the House of Representatives on 22 January 2013, just four days after it was introduced into the House at the start of the 2013 session.

The bill received overwhelming support-a relative rarity in the overly-partisan climate that made its mark on the 112th Congress-passing in a 395-29 vote.

The senate took up the bill shortly thereafter, and it passed the Health, Education, Labor and Pensions (HELP) Committee on 14 February 2013. Congress's website notes that the bill was passed with a series of minor amendments, which would require it to be resubmitted to the House to be voted on once again.

Assuming passage by the House, the bill could be on the president's desk for his signature sometime in March 2013.

Similar Bill, Same Major-and New-Provisions

The bulk of the bill, however, remains the same, and several sections have the potential to affect regulated industry. One area of particular note: new regulatory management plans (RMPs), which are intended to allow companies to "obtain scientific feedback and interactions regarding the development and regulatory review of eligible medical countermeasures by facilitating the development of written regulatory management plans."

FDA would be tasked with working with sponsors within 90 days of the submission of an RMP request. If one is agreed to, it would set "targets and goals for timely and appropriate responses by [FDA} to the triggers described," including meetings, written feedback, decisions and other activities.

These provisions could constitute a new sort of medical countermeasure pathway, offering guidance and triggers not necessarily available to other products.

New Incentives

Other sections are also tooled toward generating incentives through regulatory processes.

Title II, "Enhancing Medial Countermeasure Review," is tooled to provide FDA with the ability to authorize the temporary approval of medical products in medical emergencies. In some cases, this would allow FDA to use a Special Protocol Assessment (SPA), which is a statement from FDA indicating that uncompleted clinical or non-clinical trials form a sufficient basis for approval. This can sometimes be synonymous with the so-called "Animal Rule," which allows FDA to approve some products based on efficacy studies conducted in animals if it is intended to treat a rare and dangerous pathogen.

However, the law would also expand FDA's authority relative to the prior authorization of the act. Currently, FDA has the authority to approve products only for "emergency" situation. Under the new legislation, it would also have the authority to approve products in the case of a "threat justifying emergency authorized use." Additionally, under current law, FDA could only declare such a threat if it was already present; under the new language, it could declare that "the circumstances exist" that could facilitate such an emergency. The agency could also recognize such threats for "US citizens living abroad."

Such approvals would be temporary, existing for a period of not more than one year. After that time period, FDA would need to either extend the approval or facilitate the regulatory review of the product.

CGMPs and Biodefense

FDA would, however, be authorized to approve on an emergency basis products that do not meet current good manufacturing practices (CGMPs).

"With respect to the emergency use of a product for which an authorization under this section is issued (whether an unapproved product or an approved use of an approved product), the Secretary may waive or limit, to the extent appropriate given the applicable circumstances described in subsection (b)(1)," the legislation states.

The section could be seen by some as a response to recent drug shortages caused by CGMPs at a number of US and foreign facilities, which received a flurry of congressional attention earlier in 2012.


Another section, "Emergency Use of Medical Products," gives government agencies broader regulatory authority, most of which is aimed at being able to approve, stockpile and maintain medical products. For instance, the Department of Health and Human Services-in practice, FDA-has the authority to extend the expiration dating for products introduced into interstate commerce. This could become crucial in a case where a pandemic requires widespread use of a product such that supplies of non-expired product run out. Under ordinary circumstances, the company would expose itself to legal liability and potential enforcement action if it shipped products that had exceeded their approved shelf life. Section 564(A)(b) of the PEPRA reauthorization would allow FDA to extend that date if it is supported by scientific analysis. Similar authority is given to DHHS to determine if products manufactured under deficient CGMPs would be suitable for public consumption.

PEPRA would continue to allow federal authorities, including DHHS, to stockpile unapproved drug products so long as they are not used without first obtaining FDA approval. The intent of such stockpiles is to obtain products that are likely to be effective for a condition that has no known treatment, and to have them ready in case such a threat does emerge.

REMS and Interagency Activities

Risk Evaluation and Mitigation Strategies (REMS)-the way in which FDA places postmarketing controls on potentially dangerous products-might also be waived during emergencies to facilitate the more rapid adoption and release of a product. Some REMS plans involve elements to assure safe use, which can involve time delays for training or additional testing.

The legislation also directs FDA to be directly involved in inter-agency coordinating activities, such as those intended to "enhance medical countermeasure activities." The agency has long been involved in coordinating such activities, such as a relatively recent meeting intended to develop countermeasures for mass casualty scenarios involving burn victims.

Some of those therapies might also be reviewed through the Animal Rule models, and PEPRA calls on FDA to release an Animal Rule guidance document no later than 18 months after the passage of the law.


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy