While the law does contain specific directions for stakeholders and policymakers to implement certain new programs and guidelines, perhaps its biggest influence will be on the rules that are rethought or relaxed in an effort to streamline innovation.
Since the FDA plays such a pivotal role in regulating new medical devices, therapies, and medication, the agency will be taking a second look at some of its processes in order to prevent barriers to innovation or unnecessarily high costs in light of the new law, said Commissioner Scott Gottlieb, MD, in a recent blog post.
“We’re at a point in science where new medical technologies hold out the promise of better treatments for a widening number of vexing conditions,” he wrote. “To build upon such opportunities, FDA will soon unveil a comprehensive Innovation Initiative.”
“It will be aimed at making sure our regulatory processes are modern and efficient, so that safe and effective new technologies can reach patients in a timely fashion.”
The interests of public health demand that regulatory agencies leverage modern tools, techniques, and advances in science to create cost-effective and speedy pathways for safe and effective therapies, Gottlieb asserted.
Big data analytics and computer simulations can now provide valuable insight into the clinical trials process.
Known as “in silico” testing, the use of computer modeling to supplement drug and device development is a promising avenue for evaluating safety and moving new therapeutics through the approvals process.
“FDA’s Center for Drug Evaluation and Research (CDER) is currently using modeling and simulation to predict clinical outcomes, inform clinical trial designs, support evidence of effectiveness, optimize dosing, predict product safety, and evaluate potential adverse event mechanisms,” Gottlieb explained.
“To take just one example of the benefits of these approaches, as we enter an era of drug individualization, modeling and simulation that incorporates aspects of individual physiology and genetics in drug metabolizing enzymes is being used to identify patient subgroups that need dose adjustments.”
These methodologies are being used to assess the effect of drug interactions with kidney and liver problems and to identify clinical management strategies that can be included in drug labels where necessary.
Computer models are also being used to create behavior models for the placebo arms of trials related to Parkinson’s disease, Huntington’s disease, Alzheimer’s, and muscular dystrophy.
Because some of these diseases are relatively rare, smaller patient sample sizes often limit the design and execution of drug trials. Analytics models can help scientists to predict behavior patterns and extrapolate results.
“FDA is working hard to maximize the authorities and resources Congress granted us to advance medical innovation for patients,” said Gottlieb. “To ensure smooth coordination and communication across the agency, we established an intra-agency Cures Steering Committee.”
The team has combed through the legislation and conducted a detailed analysis of all its provisions, he added, helping to develop a checklist of FDA deliverables. Gottlieb anticipates that the online list will become a valuable tracking tool to keep the public up to date on the agency’s progress.
The FDA has already developed a work plan to divvy up the $500 million in funding provided by the 21st Century Cures Act.
The money will be disbursed over the next nine years, subject to annual appropriations, in order to help cover the costs of implementing the law’s provisions.
“We’re at the beginning of a transformative era in science and medical technology,” he concluded. “Through our implementation of Cures, and our efforts to build on its provisions through a new Innovation Initiative, we hope that our collective efforts will help consumers benefit from this new progress.
“FDA’s headway in pursuing the opportunities enabled by Cures illustrates the agency’s enthusiasm and commitment to the law—both its letter and its spirit.”