FDA Commissioner: Agency Focused on Reducing Inefficiencies and Costs of New Drug Development

During the keynote speech at the RAPS 2017 Regulatory Convergence, Scott Gottlieb, MD, commissioner of the FDA, explained that the agency is focused on making drug development more efficient and cheaper in order to expedite approvals and reduce downstream price. 

In order to contextualize the need for reducing the time between clinical inception and market approval, Dr Gottlieb pointed to the initial discovery of germ science and the gap of time between its discovery and clinical acceptance.

“The germ theory was first proposed… in 1762,” Dr Gottlieb explained. “But these ideas were initially met with skepticism. Humanity struggled to understand how germs caused disease. This disbelief had predictable consequences. Epidemics would continue to ravage society for centuries to come. It took 300 years from the first discovery of the germ theory of disease to see these principles established as a part of the practice of medicine.”

He noted that while this gap is shrinking, the FDA is focused on accelerating the approval process and diminishing the lag time between inception and approval.

“It’s taken just two decades from the first mapping of the human genome, and its translation into technologies that allow us to edit those gene sequences as a way to target deadly diseases that afflict children,” he said. “This gap in time between the discovery of a basic scientific principle, and the adoption of new treatments based on the original insight, has been shrinking as science evolves.”

However, he stressed that numerous inefficiencies and difficulties are making the clinical development gap longer than it should be.

“But at the same moment that we’re shrinking the sequence of time between a basic science discovery and the creation of practical solutions for patients that are harnessed from these breakthroughs, the process for developing those opportunities is also getting more costly and uncertain,” Dr Gottlieb said. “In many cases, it’s also getting more prolonged. This is especially true when it comes to new drug development.”

Dr Gottlieb explained that the new drug development process is becoming so costly for both pharmaceutical manufacturers and consumers that scientific breakthroughs are at risk of going undiscovered.

“We’re on an unsustainable path, where the cost of drug development is growing enormously, as well as the costs of the new medicines,” he said during his speech. “We need to do something now, to make the entire process less costly and more efficient. Otherwise, we won’t continue to realize the practical benefits of advances in science, in the form of new and better medicines.”

He emphasized that drug developers are not the only ones who suffer as a result of clinical inefficiencies. 

“Even when we can afford to develop breakthroughs, more people will have a hard time paying for them if we can’t reduce the cost of drug development, and find better ways to capture those savings.”

Dr Gottlieb also pointed out that this process reduces competition in specialty areas where the drug development burden is high, further increasing drug prices. 

“The high cost of development also reduces competition,” he said. “With the high costs, it can be less viable to develop some drugs, especially if you’re second or third to market. Yet, we know this kind of competition lowers prices.”

Dr Gottlieb said that many of the policies he has enacted at the FDA during his brief tenure have been aimed at reducing the costs of drugs, such as boosting generic competition, closing gamed loopholes, and ending monopolies. However, he explained that in order to truly impact
prices that the agency must focus more on reducing the burden at beginning of the drug development process. 

“We also need to address the other side of the continuum, where the costs start: With the high and rising expense of developing a novel drug,” he said.

Dr Gottlieb suggested that the current oncology drug space could act as a model for how all drug development should be. He called oncology development focused, efficient, and fast. 

“These are cases where the biological rationale for a treatment is firmly established, where proof of concept can be more easily obtained, and where the drugs are typically targeting significant unmet medical needs… The risks and benefits can be made obvious through more focused studies… The time it takes for FDA to review the results, can unfold efficiently. This would suggest that drug development overall is getting more efficient. But this experience is generally the exception.”

Dr Gottlieb emphasized the need for adaptive clinical trial designs in order to quickly match clinicians with targeted study participant groups.

“We’re seeing wider use of adaptive approaches, which allow scientists to enrich trials for patient characteristics that correlate with benefits, or that help predict which patients are least likely to suffer a certain side effect,” he explained. “This predictive information is valuable. It can be incorporated in a new drug’s label and help inform more careful prescribing.”

He also pointed to better communication between the FDA and the various stakeholder involved in the drug development process as a way to reduce inefficiencies. 

“The Agency also needs to engage in more communication between sponsors, investigators,
[institutional review boards], and other stakeholders involved in the development program,” he said. “This is not a ‘business as usual’ approach. It may require a much more iterative process, with greater communication between all of the stakeholders involved in the clinical trial processes.”

Dr Gottlieb closed by warning that unless stakeholders come together to solve the problem burdening drug development and access, scientific advances will go unachieved. 

“Unless we find ways to modernize how we approach our work, and make more efficient use of our resources, then we’re going to get fewer medicines, and higher costs,” he concluded. “We’re not going to realize the benefits of the scientific advances we’re seeing as quickly, if we see them at all.”

—David Costill