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Healio – Gottlieb sees ‘watershed opportunity’ to shape future of FDA’s regulatory process

Healio – Gottlieb sees ‘watershed opportunity’ to shape future of FDA’s regulatory process

When diagnosed with Hodgkin lymphoma at the age of 33 years, FDA Commissioner Scott Gottlieb, MD, was told he had a 90%
chance of survival.

“Ninety percent doesn’t sound so good if it’s you,” Gottlieb, now 44, told HemOnc Today following a keynote presentation at the Friends of Cancer Research Annual Meeting. “So, to me, that 10% sounded pretty grim.”

Gottlieb — the FDA’s 23rd commissioner — pored over “stacks of studies” in an effort to find available therapy that might improve his chances of survival. He is approaching his new role in a similar way in an attempt to loosen FDA regulations that get in the way of patients benefiting from the promise of cutting-edge therapies.

“I had a cancer that was imminently curable,” Gottlieb said. “The situation I faced is very different than when somebody is told they have metastatic pancreatic cancer and their chance of surviving 5 years is 30% or 20%, or even more grim than that.

“That person isn’t looking for a study to try to get from 30% to 40%,” Gottlieb added. “For them, available medical therapy is most likely to fail. They’re looking for something brand new, a magic bullet, something transformative that can dramatically improve their lives. The challenge for us is to think about how we design a regulatory process that serves all patients.”

‘The stars are aligned’

Since being named commissioner of the FDA, Gottlieb has spearheaded attempts to address the opioid epidemic, crusaded for the reduction of nicotine in cigarettes, orchestrated a crackdown on what he called “deceitful” stem cell clinics that claim to have cures for disease, and has witnessed the first approval of chimeric antigen receptor T-cell gene therapy in the United States.

“He’s the right person at the right time for this job,” said Ellen V. Sigal, PhD, chairperson and founder of Friends of Cancer Research, an advocacy group that supports collaboration among health care partners to promote advances in science, policy and regulation with the goal of quickening patients’ access to life-saving treatments, according to the organization’s website.

“He comes in knowing the agency and the government well, and at a time when [research] science is extraordinary and we have an opportunity to get it to patients,” Sigal added. “He’s a physician, so he knows the needs of patients. He’s a survivor, so he knows what it feels like to be a patient, and he’s driven to really make a difference. Right now, the stars are aligned with science and with the regulatory environment.”

Gottlieb, who served under former FDA Commissioners Lester Mills Crawford, DVM, PhD, and Andrew C. von Eschenbach, MD, during the George W. Bush administration, said he’s been given a “watershed opportunity” to shape the country’s regulatory process for years to come.

“This is an inflection point in science when you think about what’s happening with cellbased therapies, targeted therapies and gene therapy,” Gottlieb said during his keynote address. “Things we couldn’t really conceive of 20 years ago or even 10 years ago — it seemed like it would be a lifetime before they came to fruition — are now showing practical results because we’re thinking differently about the regulatory process. That’s what we’re trying to do at FDA right now. There are product areas and new technologies that require us to think completely differently about how we apply our regulatory principles to new kinds of products.”

Opioids, tobacco

Gottlieb has been particularly vocal about the opioid epidemic that claimed the lives of an estimated 59,000 Americans in 2016.

The FDA has advocated the use of medication-assisted treatments such as methadone, buprenorphine and naltrexone for those addicted to opioids, such as heroin.

On Tuesday, the FDA issued a warning against the herbal supplement kratom — which is used for chronic pain — linking its use to 36 deaths.

“We’re looking at ways to change provider training and try to reduce or rationalize dispensing and prescribing,” Gottlieb said. “We need to address the new addiction crisis, but we also need to make sure we’re very cognizant about how we address the needs of patients who are currently addicted and want to transition to a life of sobriety and who need medical therapy to do that.”

In July, the FDA announced plans to regulate the tobacco industry by reducing nicotine levels to nonaddictive or minimally addictive levels. Gottlieb said he hopes some of those restrictions are in place before or shortly after the new year. “I think we’re still going to be within that time frame,” Gottlieb said. “If it slips, it won’t slip by much. We’re moving forward with that policy-making.”

New discovery

A former advisor for several pharmaceutical companies, including Bristol-Myers Squibb, Gottlieb said one of the priorities of the FDA is to find ways to bring more competition into the drug industry to bring down rising costs.

The FDA’s Oncology Center of Excellence — which leverages the combined skills of regulatory scientists and reviewers with experts in the fields of drug development, biologics and medical devices — serves as a model for other branches of the FDA, mostly due to the dedicated efforts of its director, Richard Pazdur, MD, Gottlieb said. “We need to make sure the Oncology Center of Excellence is securely funded because that really is the model for how we’re going to make this work,” Gottlieb said.

Gottlieb said he would like to see the FDA take more of a “lifecycle” approach to the drug and medical device review process, ensuring that researchers with domain expertise who were accustomed to working in individual silos be included as integral parts of review teams.

“I think we sometimes separate people into such discreet silos that sometimes it feels like you’re working for a ‘to-do-for-review timeline,’ as opposed to working for a public health goal and to achieve a certain outcome by making a certain product available to patients who need it,” Gottlieb said. “We need to make sure the structural line of the review process is aligned to the ultimate goal.

“We know timelines are exceedingly important and a way to make sure we’re being accountable, but our goal is not to review a product on a 6-month timeline,” Gottlieb added. “Our goal is that a safe and effective product comes to market in a timely fashion and impacts people’s lives in a positive way.”

In August, the FDA reported enforcement actions against two large stem-cell clinics in Sunrise, Florida, and Beverly Hills, California, and a biotech company in San Diego that were “creating harm” for patients by administering cell-based therapies for regenerative medicine. The agency is constructing framework that will better regulate stem-cell therapy and reward academic institutions with compelling research data, Gottlieb said.

“Regenerative medicine is a very rapidly evolving field and the traditional model for how we regulate medical products might not apply or be absolutely suited to how regenerative products are being developed and promulgated and used,” Gottlieb said.

“Trying to ask an academic physician to come in and file a license with the FDA is burdensome, expensive and they’re probably not going to have sufficient data to do it. One of the things we’re going to do is allow academic researchers who follow a common protocol for having manufactured cells to band together and pool their data and follow a common license with FDA. We’re trying to find a way to adapt our regulatory touch.”

With ample funding, the FDA is in a position to move the science of medicine into a time of new discovery, Gottlieb said.

“We can always do more with more,” he said. “A properly resourced agency is in everyone’s broad interest, certainly from a public health standpoint, even from the standpoint of regulated industry.

“The agency is at a point where it’s better resourced than it’s ever been historically, but the resources are not equally distributed across the agency,” he added. “There are pockets where I do think we struggle more, not necessarily on the medical products side, but on the food side. … But, I do feel we’re operating from a much stronger platform than at any time I’ve ever known the agency and I’ve been here through multiple cycles. I feel fortunate to be running it at this point in time.”…