There’s no shortage of areas to debate when it comes to FDA policies, the need for reform and the argument against the erosion of safety standards.
In fact, there were so many drug and device-related fields to cover, President Trump’s nominee for the top job at the Food and Drug Administration Scott Gottlieb didn’t get a chance to comment on food safety, supplements, and his other potential jurisdictions.
In Tuesday’s nomination hearing, broadcast live, the Senate Health, Education, Labor and Pensions Committee pushed other major areas to the fore.
They included Gottlieb’s “unprecedented” industry ties, drug and device approval speeds, science versus ideology, biosimilars and generics, and Gottlieb’s biggest concern of all: the opioid epidemic.
Handling the boss
Both sides of the aisle seemed concerned about President Trump’s hiring freeze in government departments, including FDA. Committee chair Sen. Lamar Alexander (R-TN) addressed it directly in his opening remarks, asking how Gottlieb would lead a strong agency incapable of hiring new talent.
According to Alexander, former commissioner Robert Califf’s top priority with the passing of the 21st Century Cures Act was to be able to hire and pay the necessary talent to review investigational drugs and devices.
Gottlieb reiterated the importance of FDA possessing a world-class workforce, armed with the resources and tools they need to do their job.
The most direct sentence he could summon, however, was to note that he has been vocal about the need to maintain a strong FDA workforce in the past.
“I will continue to make my opinions known on that issue,” he said.
Others questioned how he could maintain a science-first, objective approach while working beneath an ideological president.
“For those who have worked with me, I haven’t been shy about offering my unvarnished advice,” Gottlieb replied. “We mentioned 866 articles I’ve written where I’ve offered very clear thoughts. I’m going to continue to offer people my very clear thoughts on whatever issues I’m asked to opine on, including my boss’s.”
Unsurprisingly, the political parties returned to their respective corners when Gottlieb’s extensive industry ties were flagged. Democrat senators were concerned about his ability to remain impartial, while Republican’s viewed it as valuable experience in the field.
Sen. Patty Murray (D-WA) began her address by expressing disappointment in the limited amount of time the committee had to review Gottlieb’s application papers, which chronicle his many connections. The full package was received on Friday, Murray said.
“We’ve had just a handful of days to fully understand the extent of Dr. Gottlieb’s unprecedented financial entanglements with the industries he would regulate as FDA commissioner.”
Murray repeated the term “unprecedented” several times, as Senator Alexander reminded the audience that the most recent FDA commissioner, Robert Califf, had extensive industry ties too.
“I’m glad to know that you’ve got a background and experience in the issues before you,” Alexander told Gottlieb.
For his part, the nominee promised to work with ethical experts throughout his tenure. He has already withdrawn from multiple compromising positions and has met the minimum impartiality requirements for the position.
Part of the damage can’t be undone, however, in the eyes of the Democrats. It was pointed out that, if approved, he would likely oversee regulatory filings from some of the many companies that he has consulted for or financially invested in, including Cerecor and CRISPR Therapeutics.
As a prolific writer and speaker, Gottlieb has an established track record indicating he would like to streamline the approval process. Some committee members, particularly Sen. Elizabeth Warren (D-MA) were particularly concerned about this pro-industry approach.
Gottlieb’s biggest message here was that it doesn’t need to be a trade-off.
“We should reject the false dichotomy that it all boils down to a choice between speed and safety,” he said.
The approval process can be improved from many angles, he argued, such as through adapted clinical trials that don’t infringe on the scientific rigor applied.
“We should be reminded always that we save lives by allowing good things to happen,” Gottlieb told the committee. “But we also save lives when we keep bad things from happening.”
Biosimilars and generics
Sen. Mike Enzi (R-WY) floated the issue of biosimilars in question session. Since 2012, four biologics have been approved, he said, but only two made it to the market. By 2021, some 70 more biologics patents will expire, creating a major opportunity for healthcare savings.
Since 2012, four biologics have been approved, he said, but only two made it to the market. By 2021, some 70 more biologic patents will expire, creating a major opportunity for healthcare savings.
“I think many of us have been disappointed by the economic savings we’ve seen from biosimilars so far,” Gottlieb said.
His answer was concise but touched on one of the major issues; FDA giving guidelines to biologics manufacturers in a timely fashion. Uncertainty has clouded the field until now.
He also stated that the FDA needs to answer specific questions, such as whether or not biosimilars can be used interchangeably.
The discussion moved on, returning later to generic drugs with a question by Sen. Todd Young (R-IN) that played right into one of Gottlieb’s top priorities.
“There are literally billions of dollars worth of drugs each year that are sold as branded drugs at high prices but should be subject to generic competition,” the nominee said in agreement.
A huge part of the problem, Gottlieb said, was the inability of current FDA guidelines to handle new complex drugs. The standard measures for bioequivalence and bioavailability no longer suffice. That confusion prolongs the exclusivity period of the drug, as generics struggle to break into the market.
“This is an area I want to work on,” he said.
The committee and Gottlieb all agreed on the urgency of the opioid epidemic.
“The opioid epidemic in this country is having staggering human consequences,” Gottlieb said in response to a question by Alexander. “I think this is the biggest crisis facing the agency.”
While he said an “all of the above” approach is needed, he singled out opioid alternatives as one of the greatest opportunities to turn the tide on the crisis.
That could include new pain relieving drugs and devices that can administer them in a more localized way. He also mentioned the need for medical options that help patients maintain sobriety.
Photo: VladimirSorokin, Getty Images
Drug developers, economists, and politicians are smart people. But maybe a four-year-old could have predicted this one: What goes up, must come down.
Are drug prices finally on the way down?
Looking at some of the major 2017 approvals thus far, there is some evidence to suggest the culture is slowly shifting.
Even Teva Pharmaceuticals appears to have turned a new leaf. On Tuesday, the Israeli company announced that its Huntington’s disease therapy Austedo (deutetrabenazine) had received an FDA nod. Rather than altering the course of the disease, it helps treat patients’ involuntary movements.
Leerink analyst Jason Gerberry reported that Austedo will be priced at around $60,000 a year. That compares to an older version of the drug called Xenazine (tetrabenazine), which sells for $152,000 per year. A generic version goes for $96,000.
Austedo caps off a progressive few weeks.
In late March, Roche subsidiary Genentech got the green light to market Ocrevus (ocrelizumab) for relapsing-remitting and primary-progressive multiple sclerosis (the latter being an industry first).
Genentech announced an intended list price of $65,000 — pretty reasonable given the drug delivered a 47 percent reduction in annualized relapse rates compared to Rebif, a first-generation MS therapy that sells for around $86,000.
On the same day, Regeneron Pharmaceuticals’ Dupixent (dupilumab) was approved for moderate-to-severe eczema. It got stamped with a list price of $37,000 a year, a significant discount on older drugs that sell for around $50,000 per year.
Based on EvaluatePharma’s predictions, Ocrevus and Dupixent will be the highest grossing drugs to enter the market in 2017. The pricing on those drugs matters.
Also in March, Newron Pharmaceuticals finally earned U.S. marketing authorization for Xadago (safinamide), an iterative MAO-B inhibitor for the treatment of Parkinson’s disease. The drug was already approved for sale in Europe.
Xadago doesn’t represent a huge therapeutic gain and Newron – to its credit –priced it that way. Without insurance, a 30-day supply of the drug will cost $670, to be marketed in the United States by US WorldMeds.
This all comes in stark contrast to Marathon Pharmaceuticals, the outlier in the conservative pricing trend. But the public, political and industry reaction to its proposed list price speak volumes too.
After receiving marketing approval for its Duchenne muscular dystrophy drug, Marathon slapped an $89,000 price tag on what is essentially a decades-old corticosteroid, available overseas for around $0.60 per dose.
Marathon eventually backed down after much outcry from the media and a harshly worded letter courtesy of Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings, (D-Md.). Marathon has since sold the franchise to PTC Therapeutics.
It seems there’s no longer any tolerance for the ‘how far can we push this’ approach. The smart players are instead asking ‘what is reasonable and sustainable in our current healthcare climate?’
But it’s early days. A handful of good examples doesn’t signal an industry shift. That will take many more years and wider changes to the healthcare system.
Photo: TAW4, Getty Images
Former Vice President Joe Biden took the stage at the American Association for Cancer Research (AACR) annual meeting on Monday, with a speech titled ‘The Beau Biden Cancer Moonshot: Progress and Promise.‘
“What a difference a year makes,” he said in his opening remarks, streamed live by AACR.
Some 15 months after the Cancer Moonshot’s launch and one year after his first AACR keynote speech, there was a lot to report back to the Washington, D.C., crowd. What a difference a year makes, indeed.
There’s a movement.
Biden is not the leader of the cancer moonshot. He’s the inspiration.
“Look, I don’t have the answers,” he said. “But you all possess the potential to generate these answers.”
Trained as a lawyer, Biden educated himself about cancer after his son, Beau, was diagnosed with a brain tumor. He later passed away. That experience didn’t give him the expertise to navigate medicine’s way to a cure. It made him passionate about it, in a way that he can serve as a focal point for the necessary people to come together.
Silicon Valley showed up, politicians on both sides of the aisle, and Nobel laureates.
“I got a call from the chairman of the board of IBM,” Biden said. “Did I want Watson, the supercomputer, to partner with the department of defense and the VA?”
With unprecedented data sharing and collaboration, the National Cancer Institutes launched the Genomic Data Commons to pool the information garnered through The Cancer Genome Atlas (TCGA), a database of 14,000 individuals’ genomic and health records. It has now expanded to 30,000 genomes.
Amazon called, Biden said, and agreed to open its cloud-computing platform for scientists using these massive databases. Since June, the data has been accessed 80 million times by researchers around the world.
Public support has been overwhelming. There is hope once again.
“You’ve lighted a fire under the public,” he said. “They’re beginning to believe again.”
There’s a cultural shift.
“For decades, we thought we could tackle cancer one discipline at a time,” Biden told the audience of cancer experts.
It’s not enough. Cancer uses every tool, system, and pathway at its disposal. The science community needs to meet each of those mechanisms head on, by uniting immunologists, virologists, geneticists, data scientists, chemical, biological and computer engineers and more. That’s happening, Biden said. The age of individual achievements in science is over.
Since its launch, the Cancer Moonshot has seeded at least 80 new collaborations. Many government-related projects have begun, he said, bringing together unlikely partnerships between the likes of NASA and the Department of Veterans Affairs.
There’s success in Washington.
In December, Congress passed the 21st Century Cures Act, which authorized an additional $6.3 billion in funding over seven years for health-related research, including $1.8 billion earmarked for cancer specifically.
Biden had the privilege to preside over the Cures Act, he said, which achieved remarkable bipartisan support.
With the passing of the Act, Republican Senator Mitch McConnell stood up to propose that the cancer initiative takes the name of Biden’s late son. The Beau Biden Cancer Moonshot.
“Those things don’t happen very much these days,” he recalled with a lot of emotion in his voice. “There is genuine, genuine bipartisan support.”
By this stage, the speech was starting to speak to something much more than the Cancer Moonshot. Late in the Obama administration, both sides had come together to pass something worthwhile.
“This is what [the American people] expect their government to do,” Biden said.
Oh, what a difference a year makes.
One year on, President Trump has taken the White House and is outlining major cuts to the NIH, the EPA — to the entire scientific field.
“The message sent out a few weeks ago in the President’s budget is counter to this hope and the progress we’ve made,” the 47th vice president of the United States told the audience.
He didn’t hold back.
“On the cusp of saving and extending lives for Americans, the President of the United States is not only not doubling-down on our investment, he’s proposing Draconian cuts.”
Funding would be set back 15 years, Biden said. By one estimate, new grant funding would be cut by 90 percent, given the multi-year commitments that the NIH has already made.
The ex-VP doesn’t believe the budget blueprint will pass Congress. However, the message it sends has already done a world of harm, communicating that science is not valued or worthy in the United States.
What a difference a year makes.
For Biden, the Cancer Moonshot was always about two things. It needed to inject urgency into the biomedical march towards a cure while also shifting the culture towards more collaboration, passion, and hope.
“You can not turn back the clock,” he said.
Not on his watch anyway.
Photo: MANDEL NGAN, AFP/Getty Images
Chatter at an early morning session on the third day of the 43rd annual meeting in Washington, D.C., was largely focused on the state of healthcare — and the survival, or repeal, of the Affordable Care Act — under the new President Trump administration.
A conversation about the state of healthcare at the Association of Community Cancer Centers’ annual meeting mostly focused on speculation over the Affordable Care Act’s future under the Trump administration. But the discussion shifted to the standing of the Cancer Moonshot Task Force, put in place during the final year of President Obama’s tenure.
Dr. Kavita Patel of the D.C.-based think tank The Brookings Institution and Dan Todd of Todd Strategy, both former Capitol Hill staffers, said any push for increasing the funding for cancer research will most likely come from agency heads, at the Food and Drug Administration, the Centers for Disease Control and Prevention, and the National Institutes of Health.
“There’s a commitment on Capitol Hill,” Todd said. “But the moonshot folks all went to work with former Vice President Biden. If there’s nobody [in the White House], it’s going to atrophy.”
The task force, helmed by former Vice President Joe Biden whose son, Beau, died of brain cancer in 2015, left D.C. the same day as the rest of the White House staff when President Obama left on Jan. 20. Today, it lives on as the nonprofit Biden Cancer Initiative, and the former vice president said the nonprofit’s work will focus on bringing down the cost of cancer treatments, enabling wider access to clinical trials, and supporting community oncology efforts.
Even before Obama and Biden left the White House, national cancer research received a boost. In December, the 21st Century Cures bill was passed into law. Through the law — the Capitol Hill commitment to which Todd referred — Congress appropriated $1.8 billion in new funding for cancer research.
Whether President Trump’s White House will take up the mantle of moonshot cancer research is an uncertainty. Although former members of the Cancer Moonshot Task Force had spoken with the incoming administration about the research — which includes a conversation between Biden and his successor, Vice President Mike Pence, about continuing the work — the new White House’s proposed budget removes $6 billion in funding from the National Institutes of Health.
“Trump’s [administration] agreed to help continue some of those efforts,” Patel said. “Doing that but releasing a budget where you’re cutting the NIH by billions of dollars does not make sense to me.”
Photo: azerberber, Getty Images
A Department of Health and Human Services directory listing Dr. Donald Rucker as the new national coordinator for health IT seems to suggest the Trump Administration has filled the role. The news was first reported by Politico.
— David Pittman (@David_Pittman) March 31, 2017
An email from a spokesperson for the Department of Health and Human Services in response to MedCity News said, “We are not commenting on personnel at this time.”
The move would mark Rucker’s first foray into the government. He was the chief medical officer at Siemens Healthcare USA for 13 years, according to his LinkedIn profile. For the past four years, he has worked as an adjunct professor at Ohio State University Wexner Medical Center for clinical emergency medicine and biomedical informatics.
He also served as a clinical assistant professor of emergency medicine at University of Pennsylvania Medical School.
Dr. Karen DeSalvo had served as head of the ONC for roughly two years until she shifted over to the role of Assistant Secretary of Health in 2016. Dr. Vindell Washington took over the role until he stepped down in January this year. Since then, Dr. Jon White has served as the acting national coordinator.
Correction: We incorrectly reported that Fierce Healthcare was the first to report the story, but have since discovered that Politico initially reported the story, which is available only to paid subscribers. We regret the error.
Mix concern for the future well-being of the insurance exchange market along with a healthy dose of ¯_(”/)_/¯ and you’ll have a decent analysis of healthcare in the U.S. circa 2017.
At the Association of Community Cancer Center’s 43rd annual meeting this week in Washington, D.C., there was a discussion about the state of healthcare under President Trump’s administration — a timely subject, given the recent failure of House Republicans to pass the American Health Care Act (AHCA), their repeal and replace version of the Affordable Care Act, or what’s commonly known as Obamacare.
Moderated by the ACCC’s director of health policy Leah Ralph, the discussion included Dr. Kavita Patel of the Washington, D.C.-based think tank The Brookings Institution and Dan Todd of Todd Strategy. As former Capitol Hill staffers, both offered instructive comments on congressional Republicans’ failure to pass the AHCA.
“The House is always this kind of chaotic, welcome-to-the-jungle kind of mess,” Patel said. “Bottom line: They didn’t have the votes.”
Todd echoed the sentiment, also noting that a dose of political miscalculation led House Republicans to believe all their members would vote for an Obamacare repeal bill. In recent days, members of the GOP have blamed the Freedom Caucus, the more conservative wing of the Republican Party in the House, for scuttling the AHCA. (Notably, President Trump tweeted this admonishment: “The Freedom Caucus will hurt the entire Republican agenda if they don’t get on the team, & fast.”). This happened about eight minutes before the discussion on Thursday.
Political dynamics aside, what does any of this mean for cancer treatment in the U.S., and the general state of healthcare? That was the question both Patel and Todd tried to make sense of for conference attendees.
The insurance exchange marketplace is where about 10 million people currently get their insurance, and how it fares in the year ahead is what Todd tackled head-on. Since passage of the ACA seven years ago, it’s now apparent that the small group market doesn’t look like the large group market — it looks much like the Medicaid market, made up of people who are sicker and older, and is highly cost-sensitive. The fix employed by President Obama’s administration, which wasn’t contemplated in the original healthcare law, was a risk adjustment payment paid to plans.
“Will the Trump administration make those payments like the Obama administration did? My gut tells me no,” Todd said. “If no, you don’t have a healthy market.”
Patel responded in kind, noting that if the Trump administration does away with cost-sharing subsidies, the result will be “people who have cancer who can’t afford insurance.” The reason? The financial stability of the federal healthcare marketplace will begin to falter. So far, the Trump Administration hasn’t said one way or the other whether it will continue providing those subsidies for insurers who participate in the federal marketplace, although House Speaker Paul Ryan said that the Trump administration should keep making those payments to insurers “to avoid destabilizing the market,” the Wall Street Journal reported Thursday.
Patel also highlighted the essential benefits component of the ACA, which required all plans sold in the marketplace to cover cancer screening, treatment, and follow-up care. The ACA tied out-of-pocket maximums paid by patients to essential benefits.
Health and Human Services Secretary Tom Price has signaled that Republicans will dismantle elements of Obamacare even without the votes in Congress. As the Chicago Tribune reported, Price described in testimony this week how “his department could make insurance plans cheaper by scaling back several federal mandates, including what the ACA currently defines as ‘essential benefits’ in coverage.”
“You get rid of [essential benefits], you actually get rid of those out-of-pocket annual maximums, which are crucial for cancer patients,” Patel said.
Although both the White House and Ryan vowed they would renew their efforts to repeal the ACA, it’s too soon to tell what will happen with healthcare this year.
Put another way: ¯_(”/)_/¯.
Featured photo: Justin Sullivan, Getty Images
On January 31, seven leaders of the biopharma world flew to Washington to discuss industry politics face-to-face with President Trump.
All seven were men.
It was almost inevitable that no woman would be granted a seat at the table. Since the rise of Big Pharma, all the major players around the world have had a male at the helm.
That’s about to change. On March 31, GlaxoSmithKline’s CEO of eight years, Andrew Witty, will formally retire and take a sliver of the proverbial glass ceiling with him. His replacement, Emma Walmsley, will become the most powerful businesswoman in the United Kingdom, overseeing its third-largest company.
British-born Walmsley comes from a consumer – not science – background. She currently serves as CEO of GSK Consumer Healthcare, a 21,000-employee division that markets many over-the-counter products such as Panadol (acetaminophen) and Sensodyne toothpaste. Her education; a master’s degree in classics and modern languages from Oxford University.
Deborah Dunsire, a former CEO of Millenium Pharmaceuticals who now heads Waltham-startup XTuit Pharmaceuticals, commended the GSK board.
“It is terrific to see a woman heading GSK! Given her excellent experience, performance and skills it is gratifying to see the board of GSK not allowing her or any other candidate’s gender to influence the decision,” Dunsire said. “Not surprising to find that the GSK board has over 25% women. With more diverse boards we will see more diversity in the C-Suite.”
Lynn Seely, president and CEO of Myovant Sciences shared a similar sentiment.
“It is a major step forward for the Life Sciences industry that GSK has recognized leadership, performance, experience and not gender as key criteria for selection of a CEO. GSK’s decision is inspiring for those of us working to change the face of board rooms.”
Walmsley’s move to GSK came after a long tenure at cosmetics giant L’Oreal. In a post on Sheryl Sandberg’s Lean In website, she recalled when and how her career path changed.
I had been to a networking lunch with Andrew Witty the Chief Executive of GSK—someone I had long admired for his pioneering approach to the healthcare industry and reputation for values-based leadership. An inspiring conversation ended up spiralling into a job offer alarmingly fast. To be the President of GSK’s global consumer healthcare business, which operates in over 100 countries with £5 billion in sales and has thousands of employees.
Seven years later, Walmsley will succeed Witty as CEO — though the move comes with a touch of controversy. It was reported several weeks ago that GSK had approved a 25 percent cut on her paycheck.
GSK’s remuneration committee explained its rationale.
“Taking into account the fact that this is Emma’s first CEO role, reductions have been made to all elements of her remuneration package in comparison to Sir Andrew’s current arrangements. Her overall package for 2017 will be approximately 25% less than that received by Sir Andrew.”
The investor reaction to Walmsley’s hiring has also been mixed. GSK shares slid marginally when her appointment was first announced. It was viewed by some as a prioritization of its consumer healthcare division at a time when some analysts are calling for the company to be split.
That’s one of many challenges the incoming CEO will face in the years ahead. Another is the patent expiration date looming over GSK’s biggest breadwinner, Advair, a treatment for asthma and COPD. The FDA rejected Mylan’s generic version of Advair on Thursday, buying GSK a little more time.
Coincidentally, it’s Mylan trying to take a bite out of Advair sales. It’s perhaps the second largest biopharma company run by a female, Heather Bresch.
That’s two women at the top. We’re not doing too badly, right?
Lest we give up on the glass ceiling conversation, here’s timely proof from the Daily Mail that it remains incredibly relevant:
Walmsley’s credentials in this order are; mother of four, has a husband, soon-to-be-CEO of the U.K.’s third largest company. Also relevant to her new position: She “lives with her family” in a £3.7 million house in South-West London.
There’s a long way to go, but women finally have at least one seat at the table when it comes to Big Pharma.
Correction: a previous version of this article misspelled Walmsley’s name in the headline.
In a role that is normally reserved for regulators like the Federal Trade Commission or the U.S. Food and Drug Administration, the New York Attorney General’s office has announced a settlement with three health app developers amounting to $30,000.
The settlement resolved allegedly misleading claims ranging from accuracy to “irresponsible” privacy practices, according to a statement from the office of Attorney General Eric Schneiderman.
It’s interesting because it could mean other state attorney generals will step up and take similar action against digital health companies as the public and private sectors attempt to rein in a sector of digital health likened to snake oil salesmen by the American Medical Association.
The three companies singled out in the settlement had apps that had been downloaded several thousand to 1 million times. The companies and apps include:
Cardiio, which produces an app of the same name to measure heart rate. The company’s touchless heart rate monitor had received certification from Happtique as part of a program that was later called into question and dismantled. The company also took part in Rock Health’s accelerator in 2012. The company was formed by MIT researchers but had not been endorsed by the institution.
Runtastic, an Austria-based company that produces an app with the same name to measure heart rate and cardiovascular performance under stress. The AG’s office also took issue with Cardiio and Runtastic for failing to test the app’s accuracy with users who used it in “vigorous exercise,” despite marketing the app for that purpose.
MobiHealthNews noted that Adidas acquired Runtastic in 2015.
Matis, an Israeli company, sells My Baby’s Beat, has claimed that the app could transform any smartphone into a fetal heart monitor. The AG’s office directed the company to post “prominent” disclaimers so that consumers understand that their apps are not approved by the FDA. The AG office also took issue with Matis for failing to be more scientifically rigorous, such as evaluating its own product against a fetal heart monitor or Doppler. For its part, Matis said in a statement posted on its website that the company had never presented its device as a medical tool to measure baby health indicators. But why else would you use it? As an ice-breaker at cocktail parties?
Privacy was also an issue with these apps. Developers have to improve consumer privacy protections to get affirmative consent, according to the settlement. The companies also have to disclose that they collect user data and share it. That’s an important point because this data includes GPS location, unique device identifier, and “deidentified” data that third parties may be able to use to re-identify specific user, the statement said.
Privacy with connected devices and apps underscore a longstanding source of concern from the FTC about how open companies are with customers about how personal health data and other personal information is used.
Photo: Getty Images
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