Tag: FDA

Why Former FDA Commissioner, Scott Gottlieb, Joined the Aetion Board | Carolyn Magil, CEO Aetion

Nov 13, 2019

By JESSICA DAMASSA, WTF HEALTH

Big news from Aetion CEO, Carolyn Magil, as she talks about the addition of former FDA Commissioner, Scott Gottlieb, to her Board. WHOA. What a HUGE endorsement of support for what Aetion is building…which is what, exactly? Carolyn explains how the company is using real world data (any data outside of clinical trial data) to figure out how different people will react to the same drug. That means they’re using data from health insurance claims, EMRs, wearables, pharma registries, etc. to ultimately save the time, money, and headache of finding out which medicines will work best for which patients. What’s more? A priority for Aetion is helping bring to light how populations usually under-represented in clinical trials (women, seniors, kids) will react to certain treatments. Backed by $77M in funding, and now the former FDA head, tune in to find out what’s next for Aetion.

Filmed at the HIMSS Health 2.0 Conference in Santa Clara, CA in September 2019.

The Untold Reality of Medical Device Shortages in the U.S.

Nov 5, 2019• 1

By CHAUN POWELL, MBA and SOUMI SAHA, PharmD, JD

Say the word “shortage” to a healthcare professional and chances are the first thing that will come to mind is drug shortages. With good reason, too – there are more than 100 drugs currently at risk or not readily available for U.S. hospitals, according to the Food and Drug Administration’s (FDA) drug shortage list.

Shortages don’t just apply to drugs, however, and as 2019 has shown, healthcare providers must become more focused on shortages of the medical device variety. The shutdown of multiple medical device sterilization facilities in 2019 is poised to jeopardize the availability of devices that are critical to routine patient care. On Nov. 6, the FDA is hosting a panel to hear from stakeholders, including hospital epidemiologists and healthcare supply chain experts, on the risks associated with facility shutdowns and potential action steps.

The industry as a whole is in need of meaningful solutions. As taxpayers, patients and key stakeholders in healthcare, we must collaborate to eliminate interruptions to our healthcare supply chain. For those invested in improving healthcare from the inside, this means working across competitive boundaries and borrowing best practices from sister industries as we work to identify the root cause of these issues and provide meaningful and preventative solutions.

A Bizarre Claim of Right to Try

Jan 18, 2019• 4

By ARTHUR CAPLAN, KELLY MCBRIDE FOLKERS, and ANDREW MCFADYEN

A patient with glioblastoma recently received an experimental cancer vaccine at the University of California, Irvine. Notably, this is being hailed as the first case of someone utilizing the Right to Try Act of 2017. ERC-USA, a U.S. subsidiary of the Brussels-based pharmaceutical company Epitopoietic Research Corporation, says it provided its product, Gliovac, to the patient at no cost. The vaccine is currently undergoing Phase II clinical trials. A handful of people in Europe have received access to it through “compassionate use.” This patient did not qualify for ongoing clinical trials in the U.S. The patient, who remains anonymous, is the first known individual to receive an experimental medicine that has not been approved by the FDA, as permitted under the federal right to try law.

Glioblastoma is a nasty cancer – John McCain and Ted Kennedy passed away after battling the disease for just over a year. We believe that patients with terminal illnesses, like those with glioblastoma, should have every reasonable tool at their disposal to treat their disease.

That being said, we’ve argued before that right to try laws are not the best way to help desperate patients. They still aren’t. The number of cases claimed to date is exactly one. And, further examination of what we know about this case does not make a strong argument for the widespread usage of the right to try pathway.

Health in 2 Point 00, Episode 63 Walgreens & Fedex partnership, Verily’s adherence program, & more!

Dec 21, 2018• 1

Today on Health in 2 Point 00, Jess and I get festive for the holidays. In this episode, Jess asks me about Walgreens and its new partnership with FedEx for next day prescription delivery and with Verily to help patients with prescription adherence. She also asks me about blockchain startup PokitDok getting its assets acquired by Change Healthcare. Lots of job changes are happening as well. Amy Abernethy, the chief medical officer at Flatiron Health, was named Deputy Commissioner of the FDA. Rasu Shrestha, who was previously at the University of Pittsburgh Medical Center, is the new chief strategy officer of Atrium Health. Finally, Zane Burke, who recently stepped down as president of Cerner, was just hired as Livongo’s new CEO, while Glen Tullman remains executive chairman of the company. Dr. Jennifer Schneider was also promoted from the company’s chief medical officer to president. We have one more episode of Health in 2 Point 00 for 2018, so be on the lookout for our year-end wrap-up. —Matthew Holt

Last Month in Oncology with Dr. Bishal Gyawali: November 2018

Dec 17, 2018• 2

By BISHAL GYAWALI MD

Keynote speech

There was a very sobering piece in NEJM by the FDA last month in which the authors try to explore what went wrong with the Keynote-183, Keynote-185 and checkmate 602 trials testing PD-1 inhibitors combinations with pomalidomide or lenalidomide and dexamethasone in multiple myeloma. Interim analysis of Keynote 183 and 185 revealed detrimental effects on overall survival (OS) with hazard ratios of 1.61 and 2.06, not explained by differences in toxicities alone. The checkmate 602 trial was also halted in light of these findings and also showed higher mortality in the nivolumab combination arm.

In the thoughtful NEJM piece, the authors make at least three important points. First, they question why these PD-1 inhibitors were tested in combination despite their having limited single-agent activity. In fact, a couple of years ago, Vinay Prasad and I asked the same question: why are novel cancer drugs being tested in combination despite having limited activity as a single agent? We found that these drugs, even when ultimately approved, provide relatively low value and recommended that drugs with poor single agent activity not be tested in combinations unless there are specific reasons to expect synergy.

The second important point in the article is that many cancer drug approvals are lately based on durable response rates in single arm trials without a control group, a situation in which it is difficult to evaluate the safety and efficacy of drug combinations. Indeed, without an RCT, the oncology community would never have known these signals of detrimental effect. If the FDA had approved these PD-1 inhibitors in multiple myeloma on the basis of non-randomized trials, which it often does in other oncology contexts, who knows how long it would have taken to recognize the increased mortality in patients—and at what cost. This is another reason why we need RCTs more now than ever. Finally, the authors point out that these PD-1 inhibitors in multiple myeloma were directly advanced to phase 3 trials after phase 1 trials were completed, without phase 2 information. Indeed, in a recent paper, Alfredo Addeo and I showed that a substantial percentage of drugs that fail in phase 3 trials do not have supporting phase 2 data.
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GAO: FDA Can’t Monitor Device Recalls

Dec 1, 2018

By MERRILL GOOZNER

The Government Accountability Office has weighed in on the failure of the Food and Drug Administration to properly monitor medical device recalls. Its review of the 3,510 recalls between 2005 and 2009 — 40 percent of which were cardiovascular radiological or orthopedic devices — found:

Several gaps in the medical device recall process limited firms’ and FDA’s abilities to ensure that the highest-risk recalls were implemented in an effective and timely manner. For many high-risk recalls, firms faced challenges, such as locating specific devices or device users, and thus could not correct or remove all devices.

“The gist of this report is that the FDA can’t tell if recalls of high-risk devices were carried out successfully because it lacks criteria for assessing device recalls and doesn’t routinely review recall data,” said Sen. Charles Grassley, R-Ia., who along with Sen. Herb Kohl, D-Wis., called for the report. “Recalls are typically voluntary, and patients would be better served if the FDA took a thorough approach to post-market surveillance of medical devices.”

FDA Mobile Medical Applications NPRM

Dec 1, 2018

By JOHN HALAMKA, MD

Many have asked me for an analysis of the new FDA Mobile Medical Applications NPRM.

The FDA will not seek to regulate mobile medical apps that perform the functionality of an electronic health record system or personal health record system. However, the FDA defined a small subset of mobile medical apps that may impact the functionality of currently regulated medical devices that will require oversight. Here’s a thoughtful analysis by Bradley Merrill Thompson of Epstein Becker Green, which he has given me permission to post:

“Today, FDA published the long-anticipated draft guidance on the regulation of mobile apps—more specifically, what the agency calls “mobile medical apps”. This draft reflects significant efforts by FDA in a fairly short amount of time, and we applaud that work. Much of the framework of the FDA guidance is consistent with the work the mHealth Regulatory Coalition (MRC) published on its website earlier this year (www.mhealthregulatorycoalition.org). While FDA has done a good job getting the ball rolling, there are a number of areas that require further work. We all (including FDA) recognize that this draft guidance is certainly not the end of the story.

The regulatory oversight recommended in today’s draft guidance applies only to a small subset of mobile apps, which FDA defines as any software application that runs on an off-the-shelf, handheld computing platform as well as web-based software designed for mobile platforms. To be regulated, as a first step the app would have to first meet the definition of a medical device and then as a second step either (1) be used as an accessory to another regulated device or (2) “transform” the handheld platform into a device, such as by using the platform’s display screens or built-in sensors.

Is There an Independent Unbiased Expert in the House?

Dec 1, 2018

By SHANNON BROWNLEE

Last week, U.S. Food and Drug Administration Commissioner Margaret Hamburg told the advocacy group Public Citizen that the FDA may loosen conflict-of-interest rules for experts who serve on the agency’s advisory panels. These panels wield considerable power when it comes to FDA decisions about approving drugs and medical devices, and for pulling them off the market when evidence surfaces that they may cause patients harm.

Why loosen the rules? Commissioner Hamburg said the agency is having trouble finding experts to fill its advisory panel slots. In other words, anybody expert enough to be on an FDA panel undoubtedly has a conflict.

Or maybe the FDA just isn’t looking very hard. In 2008, Jeanne Lenzer — an independent journalist — and I created a list of more than 100 experts in fields ranging from epidemiology to neurology to emergency medicine, every one of them independent from industry conflicts of interest. We made the list available to our fellow journalists at the website, Healthnewsreview.org, a site that grades health stories. Dozens of journalists from top news outlets, including the New York Times,Bloomberg, and the Wall Street Journal, have requested the list, and used it to find sources for their stories — or at least we hope they have.

Pfizer Pioners New Ways to Frustrate Generics

Dec 1, 2018

By Merrill Goozner

The best-selling drug in pharmaceutical industry history, Pfizer’s cholesterol-lowering Lipitor, lost its patent protection Thursday. But the huge savings that consumers, insurance companies and the government usually realize when generic versions of a best-selling pill hit the market are still six months away, and, consumer advocates fear, may never come to pass.

The reason is the unprecedented series of side deals that Pfizer has signed in recent months with some insurers and pharmacy benefit managers to offer lower-priced versions of Lipitor, known generically as atorvastatin. They also are offering consumers $4 co-pays – comparable to prices paid at discount outlets like Walmart and Costco – so they’ll continue buying the brand name version of the drug.

Government officials fear the full cost of the drugs might then be passed along to insurers and Medicare, although the companies involved say that won’t happen.

The goal of the maneuvers is to keep as many of the estimated 8 to 10 million Americans who take Lipitor ($7.2 billion in U.S. sales in 2010; $10.7 billion worldwide) on either the branded product or on an “official” generic, which in Lipitor’s case will be marketed by Watson Pharmaceuticals. They will sell for about half the price of the branded product for about six months, when a number of generic makers are expected to hit the market. Their versions of atorvastatin could sell for as low as $50 a month, which is less than a tenth its current price and comparable to other generic statin drugs already on the market.

Food Safety: It’s Déjà Vu All Over Again

Dec 1, 2018

By Marion Nestle

In preparation for the holiday season, Secretary of Health and Human Services Kathleen Sebelius and Agriculture Secretary Thomas Vilsack held a press conference to promote the departments’ efforts on food safety.

They announced release of the administration’s progress report from its Food Safety Working Group.

They also highlighted additional places to get government information about food safety at home:

I didn’t pay much attention to these announcements until I read the slightly snarky account in Food Chemical News (December 22).

The Obama administration patted itself on the back today with a new report that both lists the accomplishments over the past three years of its Food Safety Working Group (FSWG) and identifies the group’s top priorities for the coming year.

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