Gottlieb leaves mixed legacy on drug prices, opioids, tobacco use
As a former adviser to several federal entities, FDA Commissioner Scott Gottlieb, MD was familiar with public service before he assumed his current position in early 2017.
But he also had ties to the pharmaceutical industry and, thus, not all were convinced he would make good on his promises to change the status quo on drug prices, opioids and tobacco.
“I was concerned that he would be overly deferential to industry interests,” C. Michael White, PharmD, FCP, FCCP, of the department of pharmacy practice at the University of Connecticut, told Healio Primary Care Today.
Arthur Caplan, PhD, founding head of medical ethics at the New York University School of Medicine, said he too was not convinced Gottlieb would make an impact.
“When he was first announced I thought, ‘Well, he’s got ties to the pharmaceutical industry, he’s this entrepreneur, what is he really going to do?’” Caplan said in an interview.
Both White and Caplan said Gottlieb’s actions during the past 24 months ran counter to what was expected.
“I was becoming a fan of Dr. Gottlieb,” White said. “He was willing to speak directly to the public and health care providers about issues impacting the FDA or drug safety and tried to tie in the experts from his agency so it wasn’t all about him. His explanations were well thought-out and showed the organization was trying to do the right thing for the American people.”
“Gottlieb was given space to operate, people were willing to defer to him because he was not as tied to other objectives such as trade, immigration that the administration is trying to accomplish,” Caplan said. “For an administration that often I criticize as not taking science seriously, they opened up enough room to let Gottlieb shine.
“The momentum he was building toward vaping, opioids and would have started to extend into the pharmaceutical industry of study design and research ethics is going to be lost,” Caplan said.
Clinicians, policy experts, medical societies and special interest groups offered a deeper look at how well Gottlieb followed through on plans to make an impact on drug prices, opioids and tobacco.
In a speech to FDA employees shortly after being appointed commissioner nearly 2 years ago, Gottlieb said the agency would take “meaningful steps to get more low-cost alternatives to the market, to increase competition, and to give consumers more options ... [especially] when it comes to complex drugs and biosimilars.”
In some regards, the FDA did meet this objective. The agency approved a record-tying 46 drugs in 2017. Cancer medications led the way, with 16 oncology and 11 hematology drugs approved, followed by medications to help treat infectious diseases, CVD, rheumatoid arthritis, Parkinson’s disease and constipation.
“Gottlieb improved the process by which biosimilar drugs are approved; which is always a positive,” Robert A. Freeman, PhD, professor in the department of pharmacy practice and administration at the School of Pharmacy and Health Professions at the University of Maryland Eastern Shore, told Healio Primary Care Today.
The FDA also broke its record with 763 generics approved in 2017, then surpassed that total with 781 approvals in 2018.
The FDA has also taken limited steps towards the Trump administration’s proposal to lower drug prices by announcing plans to promote competition and access to drugs while keeping the agency’s Risk Evaluation and Mitigation Strategy, or REMS, as intact as possible.
Caplan said Gottlieb’s steps toward the FDA approvals were unexpected but critically important.
“He took on some of the toughest emerging opponents. ... These are some of the biggest, most powerful players and he was willing to take them on and hold them accountable,” he said.
Others agreed Gottlieb had put the pharmaceutical industry on notice.
Speaking on behalf of the Campaign for Sustainable Drug Pricing, health policy expert Lauren Aronson in a statement called Gottlieb an “invaluable voice in Washington in the fight to lower the price of prescription drugs.”
She cited several areas where Gottlieb "demonstrated important leadership" in the area of drug pricing: cutting down on abuses to REMS, encouraging the development of biosimilars, raising awareness of brand manufacturer ‘gaming’ of FDA rules to deny would-be generic competitors purchasing the samples of brand-name drugs necessary to perform mandated FDA testing, and his work on the CREATES Act, that according to Aronson, “would crack down on anticompetitive tactics employed by brand name drugmakers to keep generic competitors out of the market.”
Generic, tainted supplements
Other areas under the realm of the FDA were not completely changed during Gottlieb’s tenure and left blemishes on his legacy, White said in the interview.
“The FDA has a horrible mess on its hands with the generic drug and supplement industry. The FDA of yesteryear was overly lax in inspecting foreign manufacturers and it put Americans at risk,” White said.
The FDA has started the process of changing this. Several weeks ago, Gottlieb proposed several initiatives, including creating a tool that quickly notifies the public and appropriate manufacturer when a product with an illegal or possibly dangerous ingredient is discovered, revisiting current FDA regulations regarding dietary supplements, and ensuring the FDA regulatory framework is fluid enough to adequately assess product safety while encouraging innovation.
White said patients will still be at risk while this initiative goes through regulatory channels.
“The FDA has been trying to catch up, but there are so many manufacturers. And without adequate oversight, people are still at risk of getting substandard products. Having dietary supplements with little to no active ingredients in them or being tainted with prescription or banned drugs is a real problem. The issue here is not his alone but part of his legacy,” he said.
White also alluded to the impurity found in several generic versions of angiotensin II receptor antagonists for the treatment of patients with hypertension and heart failure. The FDA has since stated that patients who have the maximum possible exposure to the impurity experience a small risk, but White still found the revelation troubling.
“The debacle with the angiotensin receptor blockers having potential carcinogens in them is troublesome. That this issue has lingered for so long and is not resolved is only the latest but a huge black mark on the industry. This is not his fault, but it was not fully addressed during his tenure,” White said.
In that first speech to FDA employees, Gottlieb also promised “forceful steps” that would provide health care professionals with tools that would allow opioids to be used only by those who need them.
To that end, Gottlieb and the FDA announced several initiatives to curb the opioid epidemic, most recently proactively developing and testing a product label to encourage development of an over-the-counter version of the opioid overdose antidote naloxone and approving a mobile medical app that could increase the time a patient participates in an outpatient treatment program for opioid use disorder.
Lawrence Greenblatt, MD, an internal medicine doctor at Duke Health, noted some of Gottlieb’s other accomplishments in this area.
“Gottlieb showed more flexibility and innovation than one typically expects to see in a large federal bureaucracy. Under his lead, the FDA began to look at a number of new strategies: broadly offering training under the REMS program to health professionals involved with pain management, packaging short-acting opioids in limited quantities for easier prescribing, and seizing of mail from countries known to be exporting illicit fentanyl to the United States,” Greenblatt said in an interview.
He added that opioid prescriptions have dropped nearly 25% in the past 3 to 4 years, but other statistics indicate the opioid epidemic may still not have reached its tipping point under Gottlieb.
“Opioid deaths continue to climb steeply, mainly due to heroin, illicit fentanyl and fentanyl analogues. Most individuals with opioid use disorder have difficulty accessing affordable evidence-based treatment. Rather than focusing on improving safety, many health care providers have altogether abandoned prescribing chronic opioid therapy leading to additional suffering and possibly fanning the flames of the illicit opioid crisis. We will be left wondering if bolder strokes and earlier actions might have saved many lives,” Greenblatt said.
Caplan noted other than the FDA approval of Dsuvia (sufentanil, AcelRx Pharmaceuticals) — which the organization Public Citizen’s Health Research Group said is five to 10 times more potent than fentanyl — the agency was taking the right steps toward curbing the opioid epidemic.
“He made sure doctors knew what they were prescribing and counterbalancing the misinformation from the companies,” Caplan said.
Another priority Gottlieb stated was to “protect kids from the dangers of tobacco use.”
He took several steps in this area, including targeting retailers in both physical and online shops that sell Juul-related products to minors, revisiting significant parts of two policies that restrict access to newly regulated noncombustible tobacco products, electronic nicotine delivery systems and e-cigarettes.
The American Heart Association issued a statement lauding Gottlieb’s accomplishments in this area upon hearing of his resignation.
“Commissioner Gottlieb departs the FDA having established himself as a tireless champion of tobacco control,” said CEO Nancy Brown. “He elevated the war on tobacco use — and particularly the epidemic of electronic cigarette use among youth — to unprecedented levels. Because of his efforts, millions more people nationwide are aware of the grave threats posed by e-cigarettes and other tobacco products that are addicting a new generation of youth.”
Some have expressed concern his departure will stall this process.
“Gottlieb significantly moved the needle forward on sound tobacco regulation to protect the public, including our nation’s youth. His departure raises serious questions about the extent to which the current administration will move forward with plans to ban menthol in cigarettes as well as other flavors in cigars,” Cristine Delnevo, PhD, MPH, director of the Center for Tobacco Studies at Rutgers School of Public Health in New Jersey, said in an interview.
Another organization lauded Gottlieb’s role in the fight against youth tobacco but lamented the lack of progress in bringing his ideas to fruition.
“We applaud FDA Commissioner Scott Gottlieb for his strong leadership in making the fight against tobacco use a centerpiece of his tenure at the FDA,” Matthew L. Myers, president of the Campaign for Tobacco-Free Kids, said in a statement.
“However, to date, none of his proposals have been adopted, and there are no rules in place to prohibit the sale of the flavored, high-nicotine products that have led to the skyrocketing use of e-cigarettes by our kids. Commissioner Gottlieb’s legacy will depend on whether his many proposals are implemented and, in the case of the youth e-cigarette epidemic, strengthened going forward,” Myers said.
The change in leadership in any organization usually causes upheaval and uncertainty, and Gottlieb’s departure will be no exception.
“Whether the next commissioner will have the political capital to move [Gottlieb’s initiatives] forward remains uncertain,” Caplan said. "I would advise Gottlieb’s successor to keep the momentum going and focus where he was on tobacco use, opioids and vaping, since they are big health crises we face."
The Campaign for Tobacco-Free Kids and the American Heart Association also encouraged the FDA to continue to pursue Gottlieb’s agenda.
“We urge the FDA and the administration to quickly implement Commissioner Gottlieb’s proposals to accelerate progress in reducing smoking by issuing rules to limit nicotine levels in cigarettes (and other combustible tobacco products) and to ban menthol cigarettes and flavored cigars,” Meyers said.
“We urge the FDA in the strongest possible terms to move forward with effective regulation of an industry that continues to prioritize profits over the lives of consumers,” Brown said in the statement. “The work he began in tobacco control and other important areas must continue. His efforts to accelerate generic approvals and competition, and to educate consumers about the link between nutrition and chronic, preventable disease must move forward. The FDA has a critical role to play in empowering consumers with nutrition information and encouraging the food industry to make healthier food options available.”
Many of Brown's and Caplan's thoughts were echoed by Jack Resneck Jr., MD, AMA board chair.
“[Gottlieb] has taken decisive action to confront the opioid epidemic, taken on pharmaceutical companies charging high drug prices, and is working to address nicotine addiction and limit youth use of e-cigarettes. As a physician and in public service, Dr. Gottlieb has devoted his career to improving public health and tackling the challenges facing the health system. We hope his successor picks up where he left off,” Resneck said in a statement. – by Janel Miller
Disclosures: The sources for this article report no relevant financial disclosures.