Former FDA Commissioner Discusses Future of Drug Approvals

Dr Robert Califf cites trends to monitor in FDA drug approvals and safety evaluations.

Former FDA Commissioner Dr Robert Califf expressed concerns over President Donald Trump’s stated plans to speed up drug approvals and significantly reduce FDA regulations.

In an interview with Kaiser Health News, Dr Califf—–who stepped down from the agency just last month––shared his takeaways about ensuring that safety is maintained and that the drugs are actually doing what they are designed to do.

The battle over costly drugs is ongoing, with many stakeholders looking for new ways to decrease prices.

In a White House statement, President Trump said, “We’re also going to be streamlining the process so that from your standpoint when you have a drug you can actually get it approved, if it works instead of waiting for many, many years. The US drug companies have produced extraordinary results for our country but the pricing has been astronomical for our country.”

Dr Califf countered that faster drug approvals do not necessarily mean less expensive drugs.

“What I’m concerned about it that when people hear ‘faster approval,’ you get an image of the FDA sitting on this application and, you know, twiddling thumbs,” Dr Califf told KHN. “That’s not what happens.”

Instead, the slow areas of the approval process occurs before drug manufacturers submit an application to the FDA, with a trial and error process that can take many years, according to Califf.

He acknowledges that faster drug development could bring development costs down. However, he said that there is not a direct relationship between the cost of development and the price of devices and drugs.

In regards to the 21st Century Cures Act—–which was signed late last year by former President Barack Obama––Dr Califf applauded the piece of legislation, saying the bill is well-rounded and will speed up product development.

According to KHN, the Cures Act encourages the FDA to consider pieces of information during the approval process, as well as clinical trials that include biomarkers and real world evidence.

“Using real world evidence in clinical trials is one that I’m particularly excited about as a way to get better answers at a lower cost and faster at the same time,” Dr Califf told KHN.

If the FDA approval process is accelerated, Dr Califf is concerned about maintaining drug safety standards and ensuring thorough clinical trials, KHN reported.

“The concept of safety is much more complex than most people think about until they look into it deeply,” Dr Califf told KHN. “All drugs have risk. None of them are absolutely safe. And the actual safety risks are only revealed through clinical trials with the same quality and number of patients involved as it takes to look at efficacy.”

An estimated 92% of drugs do not reach market because they failed to show any benefit or had unexpected toxicity during human clinical trials, according to KHN. Califf expressed concerns that drugs could be deemed safe based on limited evidence as a result of the streamlined development track.

In a White House press release, President Trump said, “We’re going to streamline the FDA, we have a fantastic person that I think I we’ll be naming fairly soon, he's going to streamline the FDA and you're going to get your products either approved or not approved but it's going to be a quick process. It’s not going to take 15 years. And we're going to do I think a tremendous—–I think we're going to make a tremendous difference to you. I read where it costs sometimes $2.5 billion on average, actually, to come up with a new product. Is that correct? 15 years, $2.5 billion to come up with a product where there's not even a safety problem. So it's crazy. I’m surprised you can't get them to move faster than that.”

Understaffing within the FDA can have a negative impact on the approval process, Dr Califf revealed. The FDA has steadily been hiring staff to keep up with the growing industry, but there are still more than 700 unfilled positions within the agency, according to KHN.

In an effort to reduce the size of government, President Trump imposed a 90-day hiring freeze on the agency. Dr Califf called the potential freeze unfortunate, because new staff is needed to meet the faster approval timelines, and to provide timely feedback and advice that aids manufacturers in choosing the right studies required for approval, while effectively speeding up development.

“A hiring freeze at this time when we are just revving up with the 21st Century Cures is unfortunate,” Dr Califf told KHN. “I hope that the dust will settle soon and the FDA can get back to its hiring.”

Lastly, Dr Califf said that the FDA currently has an established adverse event database, but after Vioxx was pulled from the shelves in 2004, the agency started developing the Sentinel system.

Sentinel uses claims data to examine the safety of a drug, according to KHN. The system includes 180 million individual electronic records and tracks each dispensed prescription, hospitalization, and serious outcomes. According to Dr Califf, the system is being used every day by the FDA and is continuing to improve. Furthermore, Sentinel can now be accessed by academia and industry.