WASHINGTON — Since taking office, FDA Commissioner Margaret Hamburg, MD, has pushed for science to play a stronger role in the agency, both in the rationale for its decision-making and in meeting its basic purpose.
FDA, she has said, should play a strong role in developing new tools, standards and approaches to assess the safety and efficacy of FDA-regulated products.
With that goal in mind, FDA released its Regulatory Science Strategic Plan last month. The plan identifies priority areas of regulatory science where new or enhanced engagement by FDA is essential to the agency’s success in preserving the public health. In doing so, it steers the agency into areas usually considered the sole domain of basic researchers or product manufacturers.
“As new discoveries yield increasingly complex products, this strategic plan ensures that our experts are equipped to make science-based decisions resulting in sound regulatory policy,” said Hamburg. “It positions us to foster innovation through better science without compromising our high safety standard.”
Much of the plan deals with FDA’s ability to exploit new technology and keep on top of emerging sciences. One priority concerns clinical studies. The plan notes that clinical development programs are “lengthy and expensive with uncertain outcomes,” which conflicts with the need to get new medicines to patients in a timely manner.
FDA should take a hand in developing better science for clinical studies, the plan states. The agency will collaborate with other federal departments and private industry to develop and refine new trial designs, endpoints, and analysis methods. By utilizing large, pooled clinical trial data sets, FDA can help identify potential trial endpoints, as well as explore differences in specific populations and different subsets of disease — essentially leveraging FDA’s access to data to help ongoing trials. FDA also will help identify and qualify new and improved biomarkers for safety and efficacy, the plan states.
The plan also calls on the agency to take an active role in improving manufacturing practices. New technologies applied to the manufacturing process are often complex, and it is not always well understood how they impact product safety, efficacy or quality. Many questions remain unanswered and need to be addressed by FDA, in collaboration with academia and industry, the plan states.
For example, does a continuous manufacturing process create poorer product quality compared with a batch approach? Also, what role do excipient ingredients (inactive ingredients used to deliver the active ones) and complex dosage forms have on product safety?
The agency also plans to work with industry to create new, more sensitive methods to detect microbial contamination in products and find better ways to decontaminate pharmaceutical products that are not amenable to conventional sterilization methods.
While this might seem to be a departure from FDA’s traditional role as only a regulator, agency officials said it remains within FDA’s mandate. According to FDA, expanding understanding of the basic science, by both by FDA and product developers, will allow for a more informed review process.
“I think it’s important to note that this is an area we identified as a gap that exists between basic research and discovery, and actual delivery of products to the market and to the patients who need them,” explained Vicki Seyfert-Margolis, PhD, FDA senior advisor for science innovation and policy, in an FDA podcast last month. “What this will change is an awareness, not only within the agency in terms of really beefing up the scientific knowledge base, but also how we’re going to be engaging the external world, including other government agencies, academia and industry partners, to try to accomplish some of the tasks that have been laid out in this plan.”
“Clearly, these are things that we need to do to help ensure better, safer products,” she said. “I think it’s something that the entire scientific and medical product enterprise needs to be investing in.”
Regulatory Science Priority Areas
1. Modernize Toxicology to Enhance Product Safety
2. Stimulate Innovation in Clinical Evaluations and Personalized Medicine to Improve Product Development and Patient Outcomes
3. Support New Approaches to Improve Product Manufacturing and Quality
4. Ensure FDA Readiness to Evaluate Innovative Emerging Technologies
5. Harness Diverse Data through Information Sciences to Improve Health Outcomes
6. Implement a New Prevention-Focused Food Safety System to Protect Public Health
7. Facilitate Development of Medical Countermeasures to Protect Against Threats
to U.S. and Global Health and Security
8. Strengthen Social and Behavioral Science to Help Consumers and Professionals Make Informed Decisions about Regulated Products
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