Categories

Tag: FDA

Pandemic Fears: What the AIDS Battle Should Teach Us About COVID-19

By ANISH KOKA, MD

As the globe faces a novel, highly transmissible, lethal virus, I am most struck by a medicine cabinet that is embarrassingly empty for doctors in this battle.  This means much of the debate centers on mitigation of spread of the virus.  Tempers flare over discussions on travel bans, social distancing, and self quarantines, yet the inescapable fact remains that the medical community can do little more than support the varying fractions of patients who progress from mild to severe and life threatening disease.  This isn’t meant to minimize the massive efforts brought to bear to keep patients alive by health care workers but those massive efforts to support failing organs in the severely ill are in large part because we lack any effective therapy to combat the virus.  It is akin to taking care of patients with bacterial infections in an era before antibiotics, or HIV/AIDS in an era before anti-retroviral therapy.  

It should be a familiar feeling for at least one of the leading physicians charged with managing the current crisis – Dr. Anthony Fauci.  Dr. Fauci started as an immunologist at the NIH in the 1960s and quickly made breakthroughs in previously fatal diseases marked by an overactive immune response.  Strange reports of a new disease that was sweeping through the gay community in the early 1980’s caused him to shift focus to join the great battle against the AIDS epidemic. 

Continue reading…

The FDA Needs to Set Standards for Using Artificial Intelligence in Drug Development

By CHARLES K. FISHER, PhD

Artificial intelligence has become a crucial part of our technological infrastructure and the brain underlying many consumer devices. In less than a decade, machine learning algorithms based on deep neural networks evolved from recognizing cats in videos to enabling your smartphone to perform real-time translation between 27 different languages. This progress has sparked the use of AI in drug discovery and development.

Artificial intelligence can improve efficiency and outcomes in drug development across therapeutic areas. For example, companies are developing AI technologies that hold the promise of preventing serious adverse events in clinical trials by identifying high-risk individuals before they enroll. Clinical trials could be made more efficient by using artificial intelligence to incorporate other data sources, such as historical control arms or real-world data. AI technologies could also be used to magnify therapeutic responses by identifying biomarkers that enable precise targeting of patient subpopulations in complex indications.

Innovation in each of these areas would provide substantial benefits to those who volunteer to take part in trials, not to mention downstream benefits to the ultimate users of new medicines.

Misapplication of these technologies, however, can have unintended harmful consequences. To see how a good idea can turn bad, just look at what’s happened with social media since the rise of algorithms. Misinformation spreads faster than the truth, and our leaders are scrambling to protect our political systems.

Continue reading…

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

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.

Chaun Powell
Soumi Saha

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.

Continue reading…

A Bizarre Claim of Right to Try

Kelly McBride Folkers
Andrew McFayden
Arthur Caplan

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.

Continue reading…

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

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

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.
Continue reading…

Surrogate End Points Ain’t all that Bad

By CHADI NABHAN MD MBA

Dr. Chadi Nabhan, MD, MBA

Life is busy, yet we somehow find time to stay engaged on social media, remain engrossed in the 24/7 news cycle, and continue our futile efforts to resist clickbait. While social media can allow us to mindlessly scroll through feeds, it also provides an avenue to provoke vigorous dialogue, however diverse, controversial, or even rooted in unfettered biases. These exchanges have served as the primordial soup for a virtual friend or foe-ships. Tense and argumentative Twitter exchanges are especially entertaining given the challenges in justifying a position in fewer than 280 characters. Thus, tweetorials have emerged to explain a point of view via a thread of comments since it is not always easy to do so in 1 or 2 tweets. The longer the tweetorial, the more heated the debate. What I am trying to get at here, somewhat obtusely, is the concept of surrogates.

I have already suggested a surrogate. Length of a tweetorial is a surrogate for degree of controversy of the topic. Meaning, length is a surrogate, a proxy. We are surrounded by surrogates. Longer wait lines at restaurants and bars imply a hipper joint or tastier menu. My child being extra nice to me is a surrogate for him wanting more time on electronics. Not a day goes by without folks arguing about surrogate endpoints. I wanted to dig deeper into surrogates and since I am a physician, I’m focusing on surrogates in medicine. Apologies to those who thought I would be discussing restaurants or exotic trips.

I want to make sure my definition of surrogates is accurate: Merriam-Webster dictionary for enlightenment. The first use of the word “surrogate” was in 1533, B.T. (“Before Twitter”). A surrogate is defined as “one appointed to act in place of another” or “one that serves as a substitute”. We use surrogate endpoints in clinical trials as a substitute for other end points.

Continue reading…

Our Guide to Pre-Approval Access to Drugs For Both Doctors & Patients

By ALISON-BATEMAN HOUSE

In April 2016, I published guidance, in the form of a mock case study, on how to access a drug before it has been approved by the FDA—what’s known as pre-approval (or expanded or compassionate) access. This is an updated version of that guidance, reflecting multiple important changes in the pre-approval landscape over the past year. In particular, the FDA rolled out a new, streamlined form for single-patient requests, and Congress passed the 21st Century Cures Act, which, among many other things, mandated that certain pharmaceutical companies provide public information about their pre-approval access policies.

Patients (and physicians) trying to access an unapproved drug outside of a clinical trial can feel as though they’re navigating uncharted waters. Many physicians don’t know that the FDA permits the use of unapproved drugs outside of clinical trials; those who do know often have no idea how to access such drugs for their patients. Those physicians who know about pre-approval access are largely specialists in certain areas—often, oncology or rare diseases—and they are generally self-taught: they didn’t learn about pre-approval access in medical school or in their residencies. Thus, while some physicians have become very accustomed to requesting pre-approval access to drugs, the majority lacks this knowledge. In this essay, I use a fictional case to trace the process for requesting access to an unapproved drug. I hope to explode several myths about the process, especially the beliefs that the FDA is the primary decision-maker in granting access to unapproved drugs and that physicians must spend 100 hours or more completing pre-approval access paperwork.

Imagine you are a physician, and you have a pregnant patient who has tested positive for the Zika virus. She is only mildly ill, but she’s terrified that the virus, which has been linked with microcephaly and other abnormalities, will harm her unborn child. She’s so concerned that she is contemplating an abortion, even though she and her husband have been trying to have a child and were overjoyed to learn she was pregnant.

Continue reading…

The “21st Century Cures” Draft Bill: A Step Away From Transparency

Screen Shot 2015-05-18 at 10.25.01 AM

There is optimism that Congress will soon pass the 21st Century Cures bill. The draft bill proposed by the House Energy and Commerce Committee aims to foster medical innovation by streamlining the FDA regulatory process and increasing NIH research funding by $10 billion. The draft bill has overwhelming bipartisan support and will benefit patients, medical researchers and pharmaceutical companies. However, it also includes a passage, which aims to amend the Sunshine Act and exempt pharmaceutical companies from reporting the payments they make to physicians for continued medical education (CME) programs. The supporters of this change argue that physicians learn about the latest developments in medical science through CME programs and that requiring the disclosure of these payments would discourage pharmaceutical companies from financially supporting educational programs. Ultimately they believe it could inhibit the diffusion of medical innovation among doctors.

I took a look at the data released by CMS on the financial transactions between the pharmaceutical companies and individual physicians. In the last five months of 2013, more than $120 million were paid to physicians who participated (as faculty or speakers) in CME programs. The payments constitute 26 percent of the total financial transactions between pharma and individual physicians. The proposed change essentially allows pharmaceutical companies to hide more than a quarter of their payments to physicians. Exempting the pharmaceutical companies from reporting the largest part of their financial relationship with doctors will not help to foster medical education, rather it will add to current suspicions about the unjustified impact of such payments on the drugs that physicians prescribe to their patients.

If CME programs legitimately increase the awareness of physicians about the latest medical innovations and provide them with unbiased information about new drugs, then both pharmaceutical companies and those physicians who serve as speakers and faculties of such programs should be extremely proud of their role as champions of innovation and envoys of the latest knowledge in the medical community. If that is the case, one would wonder why they wouldn’t embrace and support the efforts that shed light on their noble role.

Patients heavily rely on the recommendations of their doctors to make any kind of decision regarding their health and thus have the right to be informed about the possibility that their doctors have a conflict of interest. Congress should refrain from amending the Sunshine Act and avoid jeopardizing the patients’ right to have access to information.

Niam Yaraghi is a fellow at the Brookings Institute Center For Technology Innovation. His posts appear regularly on THCB and on the Brookings Institute Tech Talk blog, where this post first appeared. This post also appeared as an opinion column on the US News and World Report site.

Registration

Forgotten Password?