FDA Commissioner Scott Gottlieb, M.D. said in a statement that the proposed modernization of the Food and Drug Administration‘s Drug Review Office will give their clinicians and scientists more time, better tools and greater support to advance the clinical and regulatory principles that the FDA uses to evaluate new drugs for safety and efficacy.
“One principal aim of these proposed changes is to elevate the role of our scientists and medical officers to take on even more thought leadership in their fields. Our professional staff members are our house officers. They are the backbone of our process and the heart of our public health mission,” Dr. Gotlieg said. “We want to give our clinicians and scientists more time, better tools and greater support to advance the clinical and regulatory principles that the FDA uses to evaluate new drugs for safety and efficacy. One goal of this modernization of our process, for example, will be the ability to issue much more product-specific guidance documents. We’ll develop hundreds of new clinical guidance documents and make sure they stay up-to-date to reflect the latest science.”
Scientific and medical advances are giving the FDA more opportunities to more fully address diseases and illness. Whereas one time all that patients may have hoped for was the ability to control a chronic disease, and perhaps slow its advance or lessen its insidious effects, more patients today can expect the ability to arrest the march of illness or achieve an outright cure.
However, with this progress comes more complexity. Not only challenges related to the science of how drugs are discovered, but also the manner in which they’re developed. For very novel drugs targeting unmet needs, this often doesn’t follow the traditional three phases of clinical trials.
To promote the most promising opportunities and address the corresponding intricacy of these new endeavors, the FDA has introduced many fundamental advances in how it evaluates drugs for safety and effectiveness, as well as the manner in which clinical trials are guided. These include adaptive approaches to clinical development such as the introduction of seamless trial designs or master protocols or tissue agnostic product approvals. Each allows the agency to better marry the scientific prospect more closely to the approaches that can best unlock these opportunities.
So do the introduction of new scientific domains into the development and review process. This includes the more widespread use of modeling and simulation, the greater use of real-world evidence in the pre- and post-market setting, and the adoption of better tools for collecting and evaluating more real-time safety information after products are approved.
Some advances in their processes are going to be achieved through more modern and scientifically rigorous approaches to how they collect and evaluate clinical data as part of the drug development process. But other features of the modernization will require a change in the way that we configure the structure and function of the process for reviewing this information.
To advance these public health goals, the director of our Center for Drug Evaluation and Research (CDER), Dr. Janet Woodcock, has proposed to her center an important series of new steps to modernize the organization and functions of CDER’s Office of New Drugs.
We believe that some of the new organizational structures we’re considering could also allow our review staff to have more time for reviewing and providing feedback to sponsors on clinical protocols. One goal is to engage sponsors earlier in the development process to ensure that trial designs are efficient and structured in the most effective way to identify risks and measure benefit. Equally important, there will also be an increased ability to engage external stakeholders, such as disease specialists, academic researchers and regulatory partners at other agencies. And with patient-focused drug development becoming a reality, ongoing relationships and interactions with patient groups are becoming an important part of our regulatory practices.
We believe that part of these modernization efforts will be enabled through increased organizational efficiency and possible structural changes that would ultimately flatten the overall matrix of our review process. As part of this effort, for example, we’re considering creating many new therapeutic-specific divisions that’ll have more ability to engage in discrete areas of medicine.
The goal is to make sure that the drug review divisions are therapeutically focused to promote efficient review and provide greater scientific leadership to academic, industry and patient groups. We believe this will deepen internal collaboration and enhance external scientific exchange.
In turn, outside stakeholders and patients, who are focused on specific therapeutic areas, will have more points of contact to the therapeutic areas they’re most focused on. In the past, the structure was organized in a way to match our workload to different divisions and offices. The goal was to make sure that there was balance across the different offices and divisions. So the organizational structure doesn’t always follow the subdivisions of medicine. That’s why we think these proposals may create a superior structure – one that’s based on the optimal clinical and public health configuration. In turn, the administration will then staff these different divisions in a way that matches our manpower to the flow of applications and critical tasks like post-market safety.
The proposed structural changes under consideration are one key element of this modernization effort, but the heart of our proposal is the reforms aimed at improving how the FDA does its work.
Our goal is also to make the scientific disciplines that conduct review more tightly integrated around a common review process, and a common review template that’s more easily collaborated around. Dr. Gotlieb hopes to make the entire review process more integrated across the discrete areas of science and regulatory expertise that are critical components of informing our overall mission.
They also want to make the entire review process and the development of the key review memos better organized, so that our medical staff can document their findings more efficiently and spend more of their time on advancing scientific work in their fields. According to our own assessment, we believe the new alignment and processes will improve efficiency by 20 percent at a minimum overall. This is, in part, the result of better workflow and workforce management and greater internal collaboration across the different review functions. The additional efficiency Gotlieb expects to achieve will be channeled toward the development of more product guidance and thought leadership. They will engage the external community more closely in their work, especially patients who inform our patient-focused drug development, with the aim of advancing more modern regulatory principles.
“This proposed modernization of the organization and function of our Office of New Drugs is a major undertaking,” Gotlieb said. “It’s the culmination of the work and leadership of Dr. Woodcock and her talented team. It reflects her longstanding commitment to advancing the public health by making sure our science and organization match the complexity and opportunity of the novel products that we’re tasked with evaluating. As in all our work, the goal is to make sure that we continue to fulfill our mission and meet the aspirations of patients, and protect and promote the public health.”