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.

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.

10 Genomics Questions for the Presidential Candidates

ScienceDebate.org has just released 20 science questions for the presidential contenders.  We thought we'd send in our own list of 10 genomics related questions.  Here they are:

 

1.  Will you get your genome sequenced, and 

   A.  Donald, will you show us what percentage of Neanderthal you have?

   B.  Hillary, will you show us the variants you keep on your private home server?

 

2.  If Obama could be cloned, should his clone be able to run for another term?

 

3.  Which of the following would make the best Moonshot:

Yes to FDA Regulation of LDTs, But We Need a New Framework, Says David Spetzler

We set up an interview with David Spetzler, the CSO of Caris Life Sciences, to hear about some promising new liquid biopsy tests they are developing. And we do that in today’s show. But first, the interview takes a turn toward the regulation of molecular tests. Spetzler says that Caris is already doing as many quality certifications as possible short of FDA oversight.

“We are actually quite favorable to increases in regulation,” he says, “We think there’s a lot of bad science being done, quite honestly, because it’s expensive to take the additional steps to insure that high quality work is being performed. And we’re doing those steps, so we might as well get credit for it.”

At the same time, Spetzler says that an appropriate framework to evaluate the utility of molecular profiling is missing. Traditional blind trials don’t work for molecular tests, he warns. The studies must be more longitudinal and be focused on more than just one biomarker at a time. Caris is currently running a study that “backs away from individual biomarker validation and focuses on process validation to identify underlying treatment opportunities for patients with particular molecular profiles.”

We do get to a discussion of Caris’ upcoming liquid biopsy tests, such as one that screens for breast cancer. This past year we’ve seen an explosion in the industry of these new non-invasive, blood-based tests. But whereas most companies are looking at cell free DNA (cfDNA) or circulating tumor cells (CTCs), Caris has come up with a novel platform using exosomes. These are small vesicles that handle intracellular communication and are not only present in all biological fluids but much more ubiquitous than cfDNA or CTCs.

Do Alternate Proposals to Regulate LDTs Stand a Chance?

As the FDA works away on final guidance for regulating LDTs, various professional groups unhappy with the course of the FDA have put together and hurried their own proposals up to Captiol Hill. The Diagnostic Testing Working Group (DTWG) has had their proposal drafted into legislation which has already been revised once in the House Energy and Commerce Committeee, while proposals from the College of American Pathologists (CAP) and the Association of Molecular Pathology (AMP) have been presented to both the Senate and the House. Various other groups, including the American Medical Association (AMA) and the American College of Medical Genetics (ACMG), have either signed on in support of one of these proposals or are independently lobbying Congress as well.

What chance do these organizations have at persuading Congress to step in and change the course of LDT regulation?

Here to help us parse through these options and get a sense of congressional reception is Scott McGoohan, the new Director of Science and Regulatory Affairs at BIO. Scott says that all of the proposals fit into “three buckets:”

First, there’s the guidance that the FDA is working on which will bring regulatory oversight to thousands of tests which heretofore have not been regulated.

Second, there’s the CAP and AMP proposals which call for the modernization of CLIA which will include increased oversight of laboratory developed tests. These proposals reserve a small set of high risk tests for FDA oversight.

And third, there’s the DTWG who are hoping to get Congress to establish a new center at the FDA for all in vitro diagnostics.

One thing is for sure, says Scott. Whichever proposal wins out, “the general sense is that there will be a change coming with increased regulatory standards for laboratory developed tests.”

Scott joins BIO at a very busy time in Washington for biotech policy. The House has passed the 21st Century Cures Initiative and the Senate Help Committee is doing active work on their health innovation agenda.

“It is an incredible time [in Washington] and an incredible opportunity for transformative regulatory change,” says Scott.

Certainly not all regulatory change is transformative, as Scott reminds at the conclusion of today's show with a story about a crazy bill in Wisconsin.

The World of DIY Genomics with K T Pickard

K Thomas Pickard is not at all into sports cars. So when he hit midlife crisis, it wasn’t a Porsche or a golf club membership that would reenergize his quiet moments. Nope. K T got his genome sequenced.

Introduced to genomics through a super computing company he worked at twenty-five years ago, K T went on to make a career for himself in medical imaging. Yet always in the background lurked a curiosity to know more about genomics. K T's inner geneticist found satisfaction in the past couple years. First he got his 23andMe data and then participated in Illumina’s Know Your Genome program, eventually doing trio sequencing on his wife, his daughter and himself. K T has written peer reviewed articles on his findings.

What was the process like for K T? For example, how did he get his genome analyzed once it was sequenced and what did he learn? How does K T compare genomics to the more established field of radiology where he had his day job?

K T’s genomics hobby has led to founding a non-profit to advocate for “neurodiversity” and the San Francisco Bay Area chapter of Genomics Coffee.

Creating the Foundation of Genomics: Marc Salit, NIST

What is a human genome? Well it’s the three billion letters of our DNA. But how is it measured? How do we know when we have it accurately represented?

These are questions that will have to be answered as precision medicine takes hold; for we must have defined standards that will be the basis for regulatory policy, commerce, and better research. These are also the questions that are foremost on the mind of today’s guest.

Marc Salit is the leader of the Genome Scale Measurement Group at the National Institute of Standards and Technology or NIST. In today’s show, he explains how NIST played a pivotal, foundational role in enabling the ‘Century of Physics.' Now Marc and NIST are looking for the right set of standards to enable the already-upon-us “Century of Biology.”

The human reference genome is an example of a standard that Marc and his team are developing. Currently they are piloting what they call “Genome in a Bottle,” a physical reference standard to which all other human genomes can be measured. How far is the team to having a complete reference genome, and what is an example of the way they are working with the FDA to ensure safe and meaningful genomic tests? Join us as we peer in at the foundation of genomics.

Still Unhappy with FDA’s Plan to Regulate LDTs, Professional Lab Groups Go Direct to the Senate

It's no secret that America's molecular testing laboratories by and large are worried that the FDA's plan to regulate laboratory developed tests, or LDTs, will severely harm patients. Now they have a new proposal which they are taking directly to Capitol Hill.

Roger Klein is the Medical Director of Molecular Oncology at the Cleveland Clinic. He’s also serving as the spokesperson for the Association for Molecular Pathology (AMP) on the controversial topic of regulating LDTs.

Several weeks ago Roger was in Washington arguing before the Senate’s HELP Committee presenting them a newly drafted proposal that he, AMP and other professional groups feel to be a better solution to regulating LDTs. Currently the FDA is on a path to issue their final guidance for LDTs in a way that treats these lab tests as medical devices.

Klein and the professional lab groups are highly concerned that the FDA’s proposed regulation will be too restrictive and will curtail an industry just as it is blooming. Their proposal: to beef up the existing CLIA regulation so that it includes determining a test’s clinical validity. So far, CLIA has been concerned only with the analytical validity of a test.

But wait a minute, haven't we heard these arguments before? Roger explains in today’s interview that his proposal before the Senate is much more complete than what they had in January.

Brian Kennedy and Aubrey de Grey on their Converging Approaches to Aging Research

Last week we attended the 2015 Rejuvenation Biotechnology Conference where we heard about the latest developments in aging research.

We were fortunate enough to sit down with two of the major figures in the field of aging research, Aubrey de Grey, CSO of the SENS Research Foundation and Brian Kennedy, CEO of the Buck Institute for Research on Aging. Brian and Aubrey have gone about their work in different ways but say that their approaches are now converging as the momentum behind aging research increases.

How do the two see the field since Calico and Human Longevity emerged? What developments in the past year stand out to them? Join us for an exclusive interview with two of the aging field’s visionary leaders.

The Sports Genes with Jeremy Koenig, Athletigen

Jeremy Koenig is a molecular biologist and an athlete. His interest in both led him to found a new direct-to-consumer genetic testing company called Athletigen.

Still it its early days, the company has curated several genetic markers which tell about a person’s athleticism. With their first report now available, the company makes it super easy to get started if you have already used 23andMe’s service. The athletigen.com portal will link in to 23andMe and retrieve your raw data. Within minutes you can be looking into whether you’re more of a power or endurance athlete, or whether your body will respond well to the paleo diet, among other predispositions. The report is free.

So how will the company make money, we ask Jeremy, who is also CEO. And, being a Canadian company, what regulatory hurdles does the company face? The DTC genetic testing space has been a treacherous one. The winners have been those companies which offer non health related products, such as genetic ancestry or paternity, or those which have sold large data sets to big pharma.

In today's show, Jeremy points out that the human body is the ultimate technology, and that athletes push this technology to its limits. Athletes can learn from their genes. And the rest of us can learn from studies of athletes and their genes. Will the field of sports genetics take off and open up new possibilities for research and precision medicine?

“One of the things my coach said to me as an athlete in college - I was a hundred meter sprinter - he’d say, 'Jeremy, we are all wearing different bodies, and you need to embrace yours. Don’t think that you need to do what the best sprinters in the world are doing right now. You need to do what’s good for Jeremy.' I think everyone needs to take stock of their own DNA,” says Jeremy.

 



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