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FDA’s No. 2 Official Sounds Off on Right to Try, Opioid Crisis

February 14, 2018
During his first State of the Union address two weeks ago, President Donald Trump expressed his belief that patients with terminal conditions should have access to experimental drugs that could potentially save their lives, saying, “It is time to give these wonderful Americans the right to try.”

During a fireside chat yesterday at the BIO CEO & Investor Conference in New York, FDA Principal Deputy Commissioner Rachel Sherman shared her thoughts on the Right to Try debate and discussed the agency’s commitment to meaningfully address the nation’s opioid crisis.

Sherman noted that patients with terminal illnesses have had a right to try experimental drugs through the FDA’s expanded access program for decades.

“Expanded access is something the agency has done since 1975, and we almost never deny a single patient’s request,” she said. “But there is a safety check on the disease, the diagnostics and who’s administering it. There’s also informed consent and a requirement that someone besides the person who might be selling the product hears about the bad things.”

She continued, “In the initial iterations of [the Right to Try] bill, there was no stipulation about manufacturer notification. So if the first patients who took an experimental drug dropped dead, they were under no obligation to tell anyone. We share BIO’s sentiment that no responsible company would be terribly interested in this approach.”

Sherman said FDA Commissioner Scott Gottlieb has been meeting with congressional leaders to ensure Right to Try legislation has appropriate checks so desperate patients and their families can make smart decisions about trying drugs in early stages of the FDA’s review process.

“Our primary and only concern in this bill is that patients be protected,” she said. “So we want to make sure that the patient is aware of the medication’s history and that there is some type of informed process that ensures someone besides the seller is aware of what’s happening.”

BIO CEO Jim Greenwood, who moderated the panel, asked Sherman about arguments made by some Right to Try advocates that FDA involvement in the process was denying life-saving drugs to terminal patients. He asked whether the FDA was hearing from biopharmaceutical companies concerned about legal liability or worried that negative outcomes could be held against them in a drug’s application before the agency.

“Let me clear that up, because that’s an urban myth,” Sherman said. “Over 100,000 patients have received some form of access [to experimental drugs] over the last 20 to 25 years. … We don’t hold data against anyone. If something untoward happens to someone, we look and see why. But I challenge anyone to find an example of a drug development program that has been in any way harmed by expanded access.”

On the opioid crisis, Greenwood pointed out that Purdue Pharma – the maker of Oxycontin – announced over the weekend its plans to stop marketing opioid products to doctors and to cut its sales force by more than a half.  He said BIO believes the only long-term answer to the opioid crisis is for the U.S. biopharmaceutical industry to innovate our way out of the crisis.

BIO released a report yesterday on the state of innovation in the pain and addiction space. Currently, there are 125 novel drugs to treat pain before the FDA – 87 percent of which use non-opioid receptors. However, the FDA has approved only two novel medications to treat pain in the last decade.

Greenwood asked Sherman whether FDA’s Division of Anesthesia, Analgesia and Addiction Products had the capacity to speed non-addictive painkillers to market, noting that the odds of pain medications in Phase 1 being commercialized are just 2 percent.

“We handle crisis well,” Sherman responded. “Hurricanes, shutdowns, HIV – we’re good when dealing with serious problems like this.”

Sherman referenced a recent BIO survey of its membership on their experiences with the FDA that reported concerns with the FDA’s analgesia and addiction division. Sherman called those results “fascinating” and said the biopharmaceutical industry’s level of satisfaction with different divisions “doesn’t necessarily correspond to the internal impression at FDA…. But can that division handle the workload? The answer is yes, we’ll figure it out.”

Greenwood concluded by asking Sherman if the FDA believed that relaxing research restrictions on controlled substances like marijuana could help researchers find new pathways to understand and treat pain.

“When you were in Congress, how would you answer that question?” Sherman replied.

“I would’ve referred it to the FDA for comment,” Greenwood retorted.

“FDA has no comment,” Sherman chuckled, “but you could speak to the DEA [Drug Enforcement Agency] about not taking the physician out of their workflow.”