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Fostering innovation to protect national health and security

By Mary Falvey, Office of the Chief Scientist, FDA

Regulatory Science SymposiumThe Food and Drug Administration (FDA) is known for the approval of safe and effective medical products; less well known is FDA’s pivotal role in building our nation’s counterterrorism capacity. A critical responsibility is fostering the development and approval of medical countermeasures (MCMs), including drugs, vaccines, diagnostic devices, and other equipment, so that the nation is prepared to respond to a naturally occurring or intentional public health threat. Like many other collaborative efforts, advances resulting from FDA’s Medical Countermeasures initiative (MCMi) can lead to improvements in how medical products for routine diseases are developed.

At the end of May, FDA’s Office of Counterterrorism and Emerging Threats convened its second annual Regulatory Science Symposium.  Attendees got a first-hand look at some of the complex regulatory challenges of working within the MCM space and how FDA, its federal partners, industry, and academia are working together to tackle those challenges.  

The Symposium highlighted recent successes in MCM development—the cutting-edge scientific approaches and mechanisms being brought to bear to develop a variety of treatments and ensure that safe and effective medical products are available when they are needed. (See program highlights). 

More than 300 people were in real or virtual attendance during this three-day forum.  In addition to more than 40 speakers from industry, academia, FDA, and other governmental agencies, 54 posters showcased the breadth and depth of MCM research underway as part of the MCMi. As the pre-eminent leader in regulatory science, FDA planned the Symposium to offer stakeholders the opportunity to learn firsthand what’s going on in MCM regulatory science, understand how collaborations come about, and network with MCM experts and researchers from around the world. In a unique event, on day three of the Symposium, attendees heard about a pilot training program to ensure collection of high-quality data during research in maximum biosecurity labs, where researchers work with highly toxic biologic threats under very complex conditions. (See Pilot training)

As part of its commitment to protect the United States from public health threats, including biological and emerging infectious disease threats, FDA is eager to engage with medical product sponsors and we encourage sponsors to reach out to FDA early and often!  For more information, visit MCMi’s Symposium Web page

Regulatory Challenges Unique to Medical Countermeasures

As a regulatory agency, FDA’s role is to promote the most efficient regulatory pathway for product approval and market release, while ensuring that new treatments and cures are safe and effective. In 2011, FDA released a Strategic Plan for Advancing Regulatory Science and recently renewed a solicitation for research opportunities, including for MCM development.  

Collaboration is the cornerstone of many of FDA’s regulatory science efforts.  Collaboration is crucial to successfully preparing for and responding to public health emergencies, which present their own unique challenges.  Because we cannot be prepared for every conceivable threat, FDA's goal in advancing MCM regulatory science research is to work with stakeholders to develop a more sophisticated and nimble science and manufacturing base so that problems can be detected early and a process will be in place that can respond swiftly and effectively to multiple types of threats.

To augment efforts already underway in its medical product centers, in August 2010, FDA launched the Medical Countermeasures initiative (MCMi) with the goal of fostering collaborations with other governmental agencies, state and local governments, industry, and academia to ensure that medical products, testing devices, and other equipment are available to respond to chemical, biological, radiological, and nuclear threats and emerging infectious diseases. 

MCMi Program Highlights

One important goal of MCMi is establishing feasible and clear regulatory pathways for MCM product innovators — based on the most Regulatory Science Symposiumadvanced science — to accelerate development.  Although the regulatory pathway for biodefense medical products is generally the same as for any nonbiodefense medical product, there is one major difference.  In many cases, it would be neither ethical nor feasible to conduct efficacy studies in humans, so the Animal Rule may be the only pathway to approval.  Under the Animal Rule, FDA may grant marketing approval for drugs and biologics based on adequate and well-controlled animal efficacy studies.

The use of the Animal Rule is not confined to the development of medical countermeasures. It can also be used to develop drugs intended to ameliorate or prevent other serious or life-threatening conditions (e.g., some emerging diseases or infections, injury due to toxic industrial chemicals, snake venom) when it would be unethical to conduct efficacy studies in. Using the Animal Rule is clearly a scientific challenge, but a number of products have been approved using this pathway.

On day two of the Symposium, FDA’s Dr. Yulia Yasinskaya from the Center for Drug Evaluation and Research (CDER) presented a detailed case study on raxibacumab, the first monoclonal antibody to treat inhalational anthrax and the first new molecular entity approved using the Animal Rule.  Inhalational anthrax is a rapidly progressive disease with high mortality. Naturally occurring inhalational anthrax in humans is sporadic and rare, and thus clinical trials are not feasible.  Dr. Yasinskaya reviewed the animal models for inhalational anthrax, the animal efficacy study designs and their associated endpoints, and all the data supporting raxibacumab’s approval. 

A high-priority MCM and a key topic at the Symposium was development of MCMs to treat acute radiation syndrome.  MCMi has established an Action Team on acute radiation syndrome to explore treatments that can be used during a nuclear event (e.g., an accident).  Successful outcomes in addressing acute radiation syndrome will produce dividends in other medical areas too, including preventing and treating radiation injury in the fields of cancer chemotherapy and bone marrow transplantation.

Development of MCMs to treat traumatic brain injury was another topic of interest at the Symposium. MCMi’s Warfighter Action Team engages regularly with the Department of Defense (DoD) to help identify regulatory science gaps and policy challenges affecting the development and availability of critical MCMs for the warfighter. In addition to traumatic brain injury, research is underway to develop biodosimetry devices for determining radiation exposure levels and mobile medical devices that can be used in a combat zone. 
Turning to a somewhat different dimension of emergency preparedness, Dr. George Lunn of FDA’s Center for drug Evaluation and Research described the large quantities of medical products being stockpiled for quick release and distribution, if required during an emergency. But because emergencies rarely occur, this means that these products may sit in stockpiles for years, and for many, the shelf life will have expired.  Dr. Lunn discussed the Shelf Life Extension Program (SLEP), managed by the DoD, whereby FDA tests stockpiled medical products near or past expiration.  If the data permit, the expiration dates can be extended in the event of an emergency. Tamiflu is such a product, and a number of batches have already had their expiration dates extended.

Pilot Training

FDA and colleagues from the University of Texas Medical Branch, Galveston National Laboratory (UTMB — GNL) shared their experiences in designing a robust, collaborative, and educational pilot program to ensure the collection of the highest quality data during research in maximum biosafety level labs.  The pilot course was held in April 2013.  Biological threats that cause life-threatening diseases for which there are no MCMs, like ebola virus, can only be tested in such labs, and these environments can present formidable challenges, especially with regard to data recording, record keeping, inspections, and equipment validation.  The pilot course brought researchers and regulators together to educate one another on the challenges both face when working with data gathered in these maximum biosafety environments.  Key goals included identifying approaches that will ensure the high-quality data and the most efficient review of data coming from studies performed in these environments.