regulation


A Republican Staffer on the Gottlieb Hearing

This week at a Senate hearing Scott Gottlieb defended his nomination to be FDA commisioner.  Last night at happy hour we caught up with a Republican staffer who was willing to be candid in exchange for remaining unnamed.

 

Staffer:  Oh, yeah, the Gottlieb nomination.  Sweet little thing.  The nomination, I mean.  Seems like the hearing was weeks ago with this whole “nuclear” warfare going on in the Senate over Gorsuch.  

Mendelspod:  The Republicans looked pretty pleased with Gottlieb, as did some of the Democrats.

How to Improve Lab Tests in the Absence of FDA Regulation?

Rubbing shoulders at molecular medicine conferences these days one senses a sigh of relief when you talk about laboratory developed tests (LDTs). With the FDA’s decision to put regulation on hold coupled with the expected confirmation of Scott Gottlieb as FDA commissioner, those in the lab testing business seem to be confidently settling back to the status quo. And those who were arguing that all we need is a “beefed up” CLIA to hold labs to better testing standards don’t appear to be motivated to do so anymore.

Several questions arise when it comes to LDTs. First of all, if regulation was truly important for enabling this revolution we call precision medicine, then why couldn’t the Obama administration get it issued? In other words, is the status quo so bad? Secondly, without the FDA even threatening to regulate, will we see the “beefed up” CLIA that many labs argued is the best way forward? Without the stick of the FDA, is the carrot gone too?

Russell Garlick is the CSO of SeraCare, a private company that has worked to improve clinical laboratory standards for over thirty years. The company recently added a new business unit for precision medicine diagnostics, and Russell was brave enough to come on today and address these questions.

As for the status quo being good enough, Russell isn't happy.

“Many of the organizations undertaking clinical trials to recruit oncology patients have lost confidence because LDTs in one geography of the United States don’t perform the same as in other parts of the United States,” he says.

Russell has worked many years with labs on IVDs--the already regulated group of diagnostic tests. He sounds disappointed that the FDA has dropped their focus on LDTs, but is hopeful that existing organizations, such as the College of American Pathologists, or even private companies such as SeraCare might step in and seize an opportunity to improve things.

“There’s a lot of status quo. And frankly it’s a little bit disappointing,” he says, “because the laboratories can benefit from [improved standards]. It’s that inertia to do something new and different."

Flint Whistleblower Says Today's Science Is to Blame for Its Own Lack of Public Trust

Marc Edwards is telling a different story than the one most of us have been reading and hearing lately. But then he’s used to it.

Marc was the engineer from Virginia Tech who was called one day in September, 2015, by a resident of Flint, Michigan. A Ms Lee Ann Walters wanted Marc to check out her water. When Marc and his team got to Flint they uncovered super high levels of lead in the potable water, with over 100,000 people exposed to high lead levels and 12,000 people with lead poisoning. You know the rest.

But you may not know that a very similar story to Flint played out in the nation’s capital in 2003. A Washington DC water crisis led to a hearing in which Congress found that the CDC had released “scientifically indefensible” reports on the water.

Marc Edwards exposed those reports and lost a contract with the EPA over it.

Aghast at the world of academic science which he says is "gamed by a system of quantitative incentives" and at government agencies who often overlook the truth, Marc now takes aim at the whole system of science. Last year he co-authored a report Academic Research in the 21st Century: Maintaining Scientific Integrity in a Climate of Perverse Incentives and Hypercompetition. The report warns of a tipping point where science “itself becomes inherently corrupt and public trust is lost, risking a new dark age."

The integrity of science has been a major theme here on the program, so while Marc is not a biomedical researcher, his experience in exposing bad science resonates within our own life science community.

In line after quotable line (“The idea of science as a public good is getting lost. In science our product is truth, and our brand is trust. The greatest proportion of truth seekers are not going into science as opposed to other human endeavors."), Marc fillets today’s scientists in government and academia, arguing that the system of science is skewed towards quantitative markers rather than quality: the pressure to publish more papers each year, citations, how much funding, etc.

Marc thinks things have gone so wrong that the war on science today (and yes, he does think there is a war on science), is more the fault of the scientists than any political movement. Somewhat with irony, but more with sadness, he says:

“The Flint water crisis was so bad it restored my faith in politicians. I mean that’s how screwed up it was. The politicians behaved themselves really well. The people who have been indicted are the scientists and engineers."

In-Situ Sequencing, CRISPR Patents, and Racist Milk Drinkers: February 2017 with Nathan and Laura

Commentators Nathan Pearson and Laura Hercher join us to look back on February’s genomics headlines.

Beginning this time with science, Nathan says we should be expecting great things from new in-situ sequencing. Laura found it encouraging that the National Academy of Sciences shifted to be more in support of genome editing. Theral asks what life forms are left to sequence for the Earth BioGenome Project?

Then it’s back to politics. Are the departure of Liz Mansfield from the FDA and Matt Might from the White House the beginning of a brain drain from government agencies in the new administration? We finish with some stories about racism that might fit under the heading “family genomics and black history month."

Hank Greely on “The End of Sex" and Other Stuff

Each year at this time we bring on a guest who is somewhat out of the way of our normal lineup, for example, a science fiction writer or a philosopher. Today Theral interviews a law professor who loves to philosophize and write about the impact of biotechnology on our lives now and in the near future. His newest book out this year, “The End of Sex and the Future of Human Reproduction,” is another comprehensive and provocative example of what has made Stanford’s Hank Greely such an in-demand speaker both to scientist and non-scientist audiences alike.

“My prediction in the book is that in twenty to forty years, most people with good healthcare will conceive their children in a lab using stem cell derived eggs--and sometimes sperm—and then do whole genome sequencing preimplantation genetic diagnosis and pick the embryo they want,” says Hank at the outset of today’s extended interview.

Whereas sci-fi writers and the mainstream press often play into what Hank calls "our need for scary bedtime stories," he seeks to understand and elucidate the actual--and less dramatic--"muddling through" of new technologies into our lives.

In addition to discussing the book, we talk with Hank about his relationship to his colleague scientists at Stanford, what he thinks is the breakthrough technology of 2016, and the future of the FDA in the era of Trump.

How to Scale Cancer Genomics, with Marco Marra, UBC

Back in 2009 at the annual AGBT meeting for sequencing, Marco Marra presented one of the first cases of cancer treatment using whole genome sequencing.

We caught up with Marco at his office at the University of British Columbia where he heads the Department of Medical Genetics. Marco also directs the Genome Sciences Center which is part of a very special organization called the BC Cancer Agency.

In 2012 Marco and his team began a pilot project at the agency to scale up their work from just a one off case to more routine treatment. While doing whole genome and whole transcriptome testing is not yet “standard of care” for cancer patients, the scientists and researchers at the agency have the opportunity to sit down with oncologists on a weekly basis and explore its use with several patients at a time.

What are the major questions and challenges Marco has encountered in scaling? How is the regulatory environment for genomic testing in Canada? And which camp does Marco adhere to when it comes to whole genome sequencing: quantity or quality?

Join us as we talk to the number two cited scientist in all of Canada.

What Does the Election Mean for Genomics? November 2016 with Nathan and Laura

While everyone is asking what will become of Obamacare, we ask our regular commentators, Nathan Pearson and Laura Hercher, specifically about genomics and medicine.

Nathan begins by saying that data scientists everywhere should be humbled. Does the failure to predict the election send out warnings about big data predictions in genomics?

Laura points out that Obamacare covers many of the new genetic tests which have become available in the past decade, such as screening tests for hereditary breast and ovarian cancer syndrome and lynch syndrome. Coverage for these tests is now up in the air.

"It is cruelly absurd to talk about the value to the human race of identifying the people with these syndromes if we don't then give them the ability to act on the information," she says.

No matter what happens to Obamacare, isn't there bipartisan support for genetic testing and for research funding? (See the passage yesterday of the 21st Century Cures Act.)

Both Nathan and Laura agree that genomic medicine will continue apace. However, they worry that under a Trump administration the less fortunate will become even more vulnerable and have less access to improvements in healthcare. They point to an area of testing that is already highly politicized: prenatal screening. Will women lose access to testing in an era that reverses gains made in women's reproductive rights?

We finish with a local election in the Florida Keyes where residents approved the use of Oxitec's genetically modified mosquitos. Fear, Laura points out, can quickly change suspicion into acceptance.

With FDA Guidance on LDTs Still Not Out, What Are Labs Doing?

As we get closer to the election and the end of 2016, the debate over LDT regulation has gone quiet. At this time last year, there was one hearing after another, first in the Senate, then in the House. The FDA’s Jeffrey Shuren was called before congress and drilled over the nuances of the guidance as well as asked when it would be released. He said, in the first half of 2016.

Though there has been no guidance released, the FDA has continued sending letters out to individual labs, requesting certain LDTs be approved before the labs market them. In March of this year, the FDA put a couple labs and two Texas hospitals on notice that were marketing “high risk” unregulated diagnostics. This surprised many in the laboratory community. These tests were diagnostics to detect the Zika virus, and any delay could negatively impact public health. The FDA told the labs they expected them to submit a request for emergency authorization (EUA).

So what are labs across the country doing? What are they supposed to be doing? Are they shying away from developing new LDTs? Are they proactively working to develop 'clinical validity’ for their tests, something they haven’t had to do under CLIA (the current regulatory statue for labs), but would be required to pursue by the FDA?

Some lab directors, such as today’s guest, say they haven’t changed a thing and are in “wait and see” mode. John Longshore is the Director of Molecular Pathology for the Carolinas Pathology Group and Carolinas HeathCare System, an integrated health network with more than 40 hospitals. He’s optimistic that laboratories are being heard on Capitol Hill and that it won't come down to FDA guidance. Referring to a recent Senate HELP meeting in September 2016 on the topic of LDTs, he says he's confident "that we will have regulation through congressional legislation rather than FDA guidance.”

The debate continues . .

September 2016 with Nathan and Laura

There were many headlines this past week heralding the first three parent baby to be born. But in fact, as our commentators point out in today’s look back on last month’s genomics news, three parent babies have been around for some time. So why are couples going to Mexico for mitochondrial transfer today? Why is it not legal in the U.S.?

Nathan points out that every one of our ancestors back ten generations ago gave us a hundred times more DNA than the mitochondrial donor might give to a three parent baby. Yes, the donor is a parent, he says, but she’s also just a distant cousin. "This shouldn’t freak anybody out.”

Laura doesn’t like the term “three parent babies” at all:

"It’s like in the early days when we went around, ‘oh is that a test tube baby?’ This is a human being, a kid on this planet—you can’t call this boy a ‘three parent baby’. He has two parents. They are the people raising him.”

Our second story involves regulation as well. This month the FDA approved Sarepta’s drug to treat Duchenne muscular dystrophy. Here’s a new drug that could help a patient population desperately in need, and yet, most of the key opinion leaders in our industry are very disappointed with the FDA. Why? The answer comes down to whether the FDA should include more than "just the science” in their decisions.

The All American EpiPen Timeline

Millions of years ago - Bees sting humans.  Certain humans eat nuts.  Anaphylactic shock happens.

1973 - Some All American humans are increasingly afraid that other humans will attack on them with chemicals.  The Pentagon asks scientists at a company called Survival Technology, Inc. to develop a quick treatment for when humans are fried by nerve gas.

1977 - All American Shel Kaplan invents the CombiPen.



New to Mendelspod?

We advance life science research, connecting people and ideas.
Register here to receive our newsletter.

or skip signup