regulation


Historic Consensus Reached on Biospecimen Standards: Carolyn Compton, NBDA

Guest:

Carolyn Compton, Professor of Pathology, ASU Bio and Contact Info

Listen (4:54) A historic new consensus

Listen (6:33) CAP committed to enforcement

Listen (2:50) Five core variables

Listen (5:16) Non-governmental leadership

Listen (4:20) Problem unique to biomedicine

Listen (3:25) Ramifications for our audience

Listen (4:48) Does this mean more work and higher prices?

A unique event happened at ASU last week that has the potential to positively impact all of biomedical research and therefore patients.

Carolyn Compton has been a tireless crusader for higher standards in biospecimen collection, handling, and storage. (See our interview earlier this year with Carolyn here where she argues that uneven sample quality is a major reason that biomedical research has such poor reproducibility rates.) Beginning as a pathologist herself, Carolyn has served at various organizations, including a stint at the National Cancer Insitute, which have prepared her for a special leadership role on the topic of biosamples. Last week she saw her long time dream come true.

“I’m delighted to report that we did in fact come to consensus after four days of discussion on what standards would be needed to raise the quality of ALL biospecimens from ALL patients to a standard that would be acceptable—perhaps not optimized—transparent and of known quality level for all patient samples. This is huge!” Carolyn exclaims in today’s show.

What does Carolyn mean by consensus? As we’ve been uncovering in our recent series on the topic, there have been no uniform standards in the collection, handling, and storage of biospecimens. It’s all been done with “poetic license” Caroly says. Until now. Last week Carolyn and a very special organization at ASU known as the National Biomarker Development Alliance put on two “convergence conferences” back-to-back that brought the major stakeholders—pathologists, physicians, patients, tool makers, and regulators—together AND found broad agreement on five basic standards to improve biospecimen handling.

This is indeed huge. Because the President and President Elect of the College of American Pathologists attended the conferences and committed to enforce these five basic standards in all of the labs which they accredit.

In her ebullience, Carolyn is careful to say that this is just the beginning, that the standards must now be written out and optimized. But it’s a very strong beginning which will have ramifications throughout the industry.

Who will be most affected? Patients, Carolyn says. Because tests, such as the popular HER2 assay for breast cancer, will be more accurate.

This story is not only unique in how it will change the course of the industry, but a powerful example of how non-governmental leadership can effect much needed change.

Podcast brought to you by: Quintiles - bringing people and knowledge together for a healthier world.

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Faces of Leadership in Diagnostics: Mara Aspinall

Guest:

Mara Aspinall, Founder, DxInsights

Bio and Contact Info

Listen (2:43) DxInsights and EPEMED

Listen (3:53) Diagnostics 5.0

Listen (2:43) International School of Biomedical Diagnostics

Listen (3:43) FDA Guidance on LDTs: the right time? the right move?

Listen (3:53) Angelina Jolie: a teaching moment

Listen (4:17) What translational gap?

Listen (1:56) Educating payers

You could call her Ms. Dx Education. Mara Aspinall has served as CEO of diagnostics companies big and small, but she’s also spent a great deal of time building a diagnostics community. Some of the early meetings of the Personalized Medicine Coalition took place in her home.

Recently Mara has stepped up her efforts in diagnostics education: that of the industry players, of physicians, and also of the next generation. She’s a co-founder of DxInsights, a new non-profit focused on better education that has already put on their first conference. DxInsights recently announced a partnership with EPEMED, or the European version of the Personalized Medicine Coalition, to create a new Knowledge Center. She is perhaps most proud of helping to create a new International School of Biomedical Diagnostics which has enrolled their first graduate students this year. This school is a first of its kind, pulling faculty expertise from various organizations in Southern Arizona as well as Dublin City University.

“We have medical school students requesting that sequencing--and what I call “Diagnostics 5.0”-- be a part of their curriculum, and it has not been,” she says in today’s interview.

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Mara is a supporter of the FDA’s recent move on LDTs, saying that the current system for diagnostics has not been working. She thinks new regulation that levels the playing field among the various players will help to spur business and innovation because it offers a more predictable path to market.

We end with her thoughts on physician and payer education.

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Janet Woodcock, FDA, on Biomarker Development and the Future of Clinical Trials

Guest:

Janet, Woodcock, MD, Director, CDER, FDA
Bio and Contact Info

Listen (4:41) No agency charged with better translational outcomes

Listen (2:46) How will recent FDA move on LDTs impact biomarker development?

Listen (4:45) Lung Map and the future of clinical trials

Listen (3:07) What about trials of one?

Listen (4:12) Translation not just about getting to Phase I

Listen (1:49) How important are biosampling issues?

Listen (2:50) Diagnosis the foundation of medicine

Listen (2:25) Thoughts on drug pricing debate

Since becoming the Director of the Center for Drug Evaluation and Research at the FDA, Janet Woodcock has been a strong advocate for better science. But the FDA’s job as regulator is to protect and promote health, not particularly to focus on improving the process of drug development. In fact, Janet points out, there is no agency “charged” with better translational outcomes.

For this reason, Janet worked to set up the Critical Path Initiative some years back. This would be a program to bring together a consortia of different organizations to primarily focus on better biomarker development as well as to modernize clinical trials.

Today we ask Janet for an update on the program. Are we getting better biomarkers? And what do the clinical trials of the future look like?

“In the future, we’ll be setting up trials around patients and disease rather than setting up a trial around a drug,” she says in today’s interview. “We have too many questions to answer about treating disease than can be done by serial trials, each one one only addressing a question about a single investigational drug.”

She’s particularly excited about a new Lung Map trial being run by the NCI. This trial is about finding a second line treatment for patients with squamous cell lung cancer. Patients are enrolled into one of five different strata based on the biomarkers of their tumors. This is a trial that can go on and on, where if a patient doesn’t respond to one treatment, this patient can be moved to a different therapy. This type of trial is known as an adaptive trial and has strong support from the FDA.

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What are Janet’s thoughts about "trials of one," where the health of the patient in the trial is more important than some future unknown patients as advocated by former guest on the program, Marty Tenenbaum?

And what does Janet think about the translational work of physician scientists who are enrolling their own patients in clinical trials based on those patients' biomarkers?

We explore with Janet the question of why diagnostics are valued so much lower than therapeutics in our culture. And at the end of the interview she offers some brave, if limited, comments about the current debate over drug pricing.

Podcast brought to you by: National Biomarker Development Alliance - Collaboratively creating standards for end-to-end systems-based biomarker development—to advance precision medicine

U.K. Life Science Update with Eliot Forster, MedCity

Guest:

Eliot Forster, Executive Chair, MedCity Bio and Contact Info

Listen (2:54) Cashing in on illustrious life science tradition

Listen (2:56) Culture of collaboration strong in the UK

Listen (5:01) A growing acceptance of GMOs

Listen (4:59) Did you ever think you would see American companies moving to the UK?

Listen (7:49) How goes the shift toward personalized medicine?

From Darwin's seminal work to the discovery of the structure of DNA to the 100K Genomes Project, the U.K. can boast of an illustrious life science tradition. Today, the U.K. government is backing a major push to cash in on that tradition, seeing life science business--from personalized medicine to genetic engineering—as a a major component of their new economy.

MedCity is a non-profit representing the life science community in London, Oxford, and Cambridge. Eliot Forster, Executive Chair of MedCity, joins us to give an update on the U.K.’s bio-economy. With support from the highest offices in government, the life science community there is benefiting from some important trends. Forster says there is a strong culture of collaboration and a regulatory and tax environment that is favoring more innovation.

Forster chuckles at the thought that some American companies are taking advantage of these new trends in England in the form of tax inversions. Thirty years ago, you wouldn't have predicted it, he says.

The commitment to biotech in the U.K. was on full display earlier this year when Prime Minister David Cameron created the new cabinet role of life sciences minister.

“If you’re looking to do work in the life sciences sector--whether a startup or a subsidiary of a major international—come to London, come to Oxford, come to Cambridge,” says Forster. "You’ll be very surprised by what you find, and pleasantly so."

Podcast brought to you by: Chempetitive Group - "We love science. We love marketing. We love the idea of combining the two to make great things happen for your marketing communications."

Pioneering Use of the Microbiome: Colleen Cutcliffe, Whole Biome

Guest:

Colleen Cutcliffe, CEO, Whole Biome
Bio and Contact Info

Listen (2:32) Do we know enough about the microbiome yet to commercialize it?

Listen (5:58) The Complete Biome Test

Listen (6:50) A higher resolution platform for developing new diagnostics and therapeutics

Listen (3:12) What are the low hanging fruit?

Listen (2:25) Going forward what is the biggest unknown for you?

There is an intense research effort underway to find out more about the human microbiome. But do we know enough yet to commercialize it?

Whole Biome is one of a handful of new startups in the microbiome space. It was founded by three former employees of PacBio who decided, yes, they knew enough to begin offering a product, the Complete Biome Test.

One of those founders, and the CEO of Whole Biome, Colleen Cutcliffe, joins us to tell their story.

As entrepreneur trainer Steve Blank said here in a recent interview, startup phase is that time period where a company searches for a successful business model. That certainly applies to Whole Biome. Colleen says that early on, the team saw that there was a gap in bacterial strain resolution that they could fill with a tool, meaning they could offer a much clearer picture of a person’s microbial community than what has been available to researchers.

“Before we even began developing that tool,” Colleen says, "we went out and we talked to people who were key opinion leaders in the field and had been thinking about the microbiome and different diseases for a long time. And we said, hey we feel this is a gap. Do you think this is a tool that would be useful? And they all said, that would be a great tool. And if you start a company around that, we’d love to work with you. And we started the company and went back to them and asked would you like to work with us now. And every single one of them is partnered with us.”

Just what will be their business model? And what are the low hanging fruit Colleen and her team are pursuing? She answers these questions and more in today’s interview.

The FDA on LDT Draft Guidance Notice to Congress

Guests:

Liz Mansfield, Deputy Office Director of Personalized Medicine, OIR, FDA
Bio and Contact Info

Katie Serrano, Deputy Director of the Division of Chemistry and Toxicology, OIR, FDA
Bio and Contact Info

Listen (4:08) What was submitted to Congress?

Listen (5:09) Why is CLIA not enough?

Listen (4:07) Does the FDA have the resources for this?

Listen (5:54) How will risk be determined?

Listen (2:13) How will you approach panels and whole genome tests?

Listen (2:33) Is NIPT on your radar?

Listen (1:32) What about laboratory developed processes or LDPs?

Listen (1:19) What should labs be doing now?

On July 31, the FDA notified Congress that they will be publishing draft guidance for laboratory developed tests or LDTs.

This means the potential regulation of lots and lots more lab tests. Thousands of diagnostics for diseases like cancer or heart disease or a rare disease.

As with most actions of the FDA, this news has its fans and critics. There are those who feel that a hands off policy of the FDA has fostered innovation and better testing. And there are those who say that some of these tests carry such high risk that they should be regulated as a device or a therapeutic. In any case, others argue, the playing field should be level among test providers.

Here to walk us through what is happening and what will happen is Liz Mansfield and Katie Serrano, both from the Office of Invitro Diagnostics and Radiological health at the FDA.

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The Lowdown on Adaptive Clinical Trials with Don Berry

Guest:

Don Berry, Prof of Statistics, MD Anderson Cancer Center
Bio and Contact Info

Listen (5:39) What is the goal of I-SPY trials?

Listen (8:31) Why were adaptive trials not introduced sooner?

Listen (4:34) What is the latest from I-SPY 2?

Listen (3:06) What about other cancers and diseases?

Listen (3:51) We have barely scratched the surface with biomarkers

Listen (2:34) Thoughts on genetic testing space

Listen (2:35) Trials of one?

The development of therapeutic drugs is a lengthy and costly process and too often ends in failure during clinical trials. So, understandably, there is much interest in improving clinical trial design to take into account the individual biology of trial participants. This more precise approach not only has great potential to improve the chance for the therapy to succeed, it provides better outcomes for patients in the trial.

The I-SPY trials are just such an approach. These national studies are designed to identify biomarkers predictive of response to various therapies throughout the treatment cycle for women with breast cancer. These trials are being called adaptive because there are changes in design throughout the trial based on an examination of accumulated data at various interim points in the trial. A patient may start with one therapy, but end up in the same trial with another therapy.

Don Berry is the founding head of the division for Quantitative Biosciences at MD Anderson Cancer Center. He is one of the principal designers of the I-SPY trials.

Are there trials in progress for cancers other than breast cancer? Will adaptive trials be broadly adopted? How can we develop better biomarkers?

Join us today in learning about this promising new approach to clinical trials directly from one of the principal architects.

Podcast brought to you by: National Biomarker Development Alliance - Collaboratively creating standards for end-to-end systems-based biomarker development—to advance precision medicine

Can We Do DTC Genomics Right? Misha Angrist, Part II

Guest:

Misha Angrist, Author, Assoc. Professor, Duke Institute for Genomic Sciences

Bio and Contact Info

Listen (7:03) Presidential bioethics commissions do not have a good record

Listen (5:06) Questioning FDA priorities

Listen (4:31) Not the best arguments in FDA letter to 23andMe

Listen (3:26) Can we do DTC genomics right?

Listen (4:49) Thoughts on A Troublesome Inheritance

Listen (4:30) Self censorship

Today we continue with part two of our interview with Duke Assoc. Professor and author, Misha Angrist, mostly centered around the issue of regulating genomic medicine.

Misha questions recent FDA actions, such as the clamp down on 23andMe.

"I'm generally pretty hard on the FDA," he says.

Misha has a "complex view" about the FDA's letter last November to 23andMe. While he thinks 23andMe "dropped the ball" and went too far in interpreting consumers' genomic data, he also thinks the FDA used the wrong arguments in their letter ordering the DTC company to discontinue its health related testing.

He lauds 23andMe's hiring of Jill Hagenkord as CMO, saying that she "gets it."

We finish with Misha's response to Nick Wade's new book, A Troublesome Inheritance: Genes, Race, and Human History.

Editor's Note: Since this interview was recorded, the 23andMe blog put out an update saying that the FDA had accepted their submission for a new 510(k) application.

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