Life Science Compliance Update

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September 28, 2017

Gottlieb Speaks to RAPS Regulatory Conference

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United States Food and Drug Administration (FDA) Commissioner Scott Gottlieb recently spoke with attendees of the Regulatory Affairs Professionals Society (RAPS) Regulatory Convergence conference about the steps the FDA is taking to make the clinical end of drug development more efficient and effective.

Gottlieb started by speaking and providing an overview of the history of medicine, all the way back to the “germ theory,” first proposed by Girolamo Fracastoro in 1546 through the late 1850s when Louis Pasteur and Robert Koch offered convincing evidence that germs caused disease and the 1870s, when sanitation was finally used in the medical field.

Gottlieb goes on to note that there is always a period between when the discovery of “a central principle of biological science” is discovered, to its eventual translation into the tools of clinical medicine. He notes that as science continues to evolve, that time period in shrinking and that primary ideas are more quickly reshaping the way we approach illnesses.

Gottlieb stated that the cost of drug development is on “an unsustainable path, where the cost of drug development is growing enormously, as well as the costs of the new medicines.” He also noted his belief that something needs to be done now, to make the whole process less costly – and more efficient – recognizing that if nothing is done, “we won’t continue to realize the practical benefits of advances in science, and in the form of new and better medicines.”

He noted that:

At FDA, we’ll continue to take steps to bring more competition to the drug market, as one way to reduce costs and improve access. All of the efforts I’m going to discuss today are not just about advancing science that can treat illness. That’s a key part of our mission. But it’s also about making sure patients can have access to these opportunities.

He noted some of the actions that the FDA has already announced that are aimed at addressing the cost of medicine and access to drugs, including: taking steps to boost generic competition and target firms that “game [FDA] rules” to extend drug monopolies past the point Congress intended for.

He focused on the idea that the FDA needs to focus on holding down high costs where they start: “with the high and rising expense of developing a novel drug.”

He did not place the blame at the feet of the pharmaceutical industry, instead noting:

Even while some drug development programs move quickly, most take many years. The costs are also high, and growing. There’s been criticism of the various estimates of how much it costs to develop a new drug. But we know some drug programs can easily top $1 billion, just in direct outlays.

We also know that the average cost of developing a single new drug continues to increase at a pace that often dwarfs even the rate of increases in other healthcare costs. Take just one time period, between 2003 and 2013, and one estimate of these costs. Over that time period, the cost of developing a drug rose by 145 percent after correcting for inflation, according to the Tufts Center for the Study of Drug Development.

Gottlieb stated that the FDA has taken steps to modernize its approach to the way it collects clinical information needed to make decisions about the safety and effectiveness of new drugs, including seeing wider use of adaptive approaches, which allow scientists to enrich trials for patient characteristics that correlate with benefits, or that help to predict which patients are less likely to suffer a particular side effect.

Other concepts the FDA is seeking to better adapt to clinical development include common control studies and the wider use of large simple trials. FDA is also advancing the use of ‘Master Protocols’ to enable more coordinated ways to use the same trial structure to evaluate treatments in more than one subtype of a disease or type of patient.

The Agency also needs to engage in more communication between sponsors, investigators, IRBs, and other stakeholders involved in the development program. 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.

To better delineate how the FDA approaches the overall development and evaluation of drugs targeted to certain unmet medical needs, it plans to begin work on at least ten new disease-specific guidance documents over the next year. Some of these documents are already underway. Among the diseases targeted are areas of significant unmet need like Amyotrophic Lateral Sclerosis (ALS).

Interestingly, in response to a question on commercial speech and FDA regulations, he made clear that while the FDA has lost recent court cases related to the First Amendment, "Our regulations cannot be in conflict with the courts," noting that the agency is currently "in a period of ambiguity."

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