Print BCTV: Begging Questions -- Questions SCOTUS left begging for patients, industry in Myriad decision

Begging Questions

Transcript of BioCentury This Week TV Episode 146




Roger Klein, Chair of the Professional Relations Committee, Association for Molecular Pathology (AMP), Bethesda, Md.

Sharon Terry, President and CEO, Genetic Alliance, Washington, D.C.

Michele Wales, Founder and Principal, InHouse Patent Counsel, Potomac, Md.

Rep. Debbie Wasserman Schultz (D-Fla.)



Myriad Genetics Inc. (NASDAQ:MYGN), Salt Lake City, Utah

National Institutes of Health (NIH), Bethesda, Md.

U.S. Patent and Trademark Office, Alexandria, Va.



Steve Usdin, Senior Editor




STEVE USDIN: The Supreme Court says human genes can't be patented. Does the Myriad ruling end the debate? This week, Representative Debbie Wasserman Schultz plus three other perspectives on how the ruling will affect patients.


I'm Steve Usdin. Welcome to BioCentury This Week.


NARRATOR: Your trusted source for biotechnology information and analysis, BioCentury This Week


STEVE USDIN: The Supreme Court has ruled naturally occurring human gene sequences cannot be patented, but synthetic DNA created in the lab can be patented. What's that mean for patients and for biomedical innovators?


The case was brought by patients, academics and testing labs. They argued that a molecular diagnostics company, Myriad Genetics, should not be able to own patents on the BRCA1 and BRCA2 genes, which increase a woman's chances of getting breast or ovarian cancer. The Supreme Court ruled Myriad couldn't patent the genes and several companies immediately began to offer BRCA diagnostics at costs well below Myriad's.


The ruling gives women more choices, but it leaves many questions unanswered. Like where and how the Patent Office should draw the line between discoveries of natural phenomena, which can't be patented, and inventions which can be protected using patents.


To sort through the Myriad decision's meaning for patients and companies trying to create new diagnostics and medicines, we'll hear today from Sharon Terry, a patient advocate and CEO of Genetic Alliance; from Roger Klein of the Association of Molecular Pathology, the lead plaintiff in the Myriad case; and Michele Wales, a biotech patent attorney who's fought for the right to patent genes. First we'll hear from Representative Debbie Wasserman Schultz. I recently spoke with the Florida Democrat who, at age 41, learned she carried the BRCA2 gene mutation. She's one of the leading advocates on Capitol Hill for access to genetic information.


Representative Wasserman Schultz, I wanted to ask you to start with what learning that you carry the BRCA2 gene mutation meant to you and your life, and why it led you to believe that one company shouldn't have a monopoly on the ability to offer BRCA testing?


REP. DEBBIE WASSERMAN SCHULTZ: Well, it was a devastating diagnosis just to be told that I had breast cancer. I was 41 years old. But then on top of that, I received sort of a double whammy news that I also was BRCA2 positive. Which meant that I was faced with making a life altering decision.


I ultimately had a double mastectomy and my ovaries removed, even though I had very early stage breast cancer, because I knew with that genetic result -- test results that the likelihood of occurrence was significant and my odds of getting ovarian cancer as I got closer to 50 were significant as well. When I asked, as I have been taught my whole life, about the possibility of getting a second opinion and what the accuracy of the tests were, I was assured the test was 100% accurate, but that I couldn't get a second opinion because one company had the patent on the gene and on the test. And that a second opinion wasn't possible.


Well, to force women to make life altering decisions based on the results of one test is totally unacceptable. And as a result, I actually passed legislation to require a study by the Patent and Trade Office to make sure that we could get second opinion testing.


And they were coming back with recommendations this summer, actually as the Supreme Court case was handed down. So we're going to see what they recommend and aggressively explore legislation to make sure that one company -- now that the Supreme Court has thrown out genetic patents as unconstitutional -- cannot control a woman's healthcare decisions or anyone's healthcare decisions.


STEVE USDIN: Did the recent Supreme Court decision solve the problem?


REP. DEBBIE WASSERMAN SCHULTZ: It solves a huge part of the problem. Because put in the same situation today, I would be able to get a second opinion, and at least get that peace of mind and know that the steps that were recommended for me to take to prevent recurrence and to reduce the likelihood of ovarian cancer and other types of cancer were the right ones. Or, that I could have learned that I had a maybe lesser type of mutation that would've led me in different direction. As it is now, I had to make life altering decisions on the basis of one test.


STEVE USDIN: Clinicians have told me that simply having competition around offering the test isn't sufficient, because Myriad has a proprietary database that allows them to be able to interpret the results in a way that nobody else can. Is that issue of concern to you? And if it is, is there anything that legislatively might be done about it?


REP. DEBBIE WASSERMAN SCHULTZ: That's a huge issue of concern. The fact that they hold on to all the data from a genetic patent that they never should have been entitled to not only is unbelievably selfish and also fairly unprecedented, because in most cases, you have data sharing even across companies with patents, and Myriad has refused to share that data. Which means that other research that was possible could not be done.


And quite frankly, if the underlying patent was thrown out and deemed unconstitutional, then to me the data should be available and should be made available. And yes, that is something legislatively that we can address.


We're still reviewing the Supreme Court decision and also waiting for the Patent and Trade Office study that was the result of legislation that I passed on the patent reform bill to make sure that if the Supreme Court ruled the other way, that second opinion testing at least was allowed. And we're going to see what the study recommends and explore what our legislative options are.


STEVE USDIN: It doesn't do anybody any good if there are tests that are on the market, if they can't get access to them. And one of the critical issues about access is reimbursement. Are you concerned about reimbursement for molecular diagnostics? And if you are, is there anything that you think that Congress might do about that?


REP. DEBBIE WASSERMAN SCHULTZ: I'm very concerned about reimbursement issues because it's sort of all over the map. If you're a Medicare patient, a Medicare beneficiary, you actually can't get coverage for the test if you don't have cancer. Well, that seems counter intuitive. If you don't have cancer, let's make sure that we know whether or not you're at higher risk.


My own mother is a Medicare beneficiary. She could not get coverage for the test, even though she has a daughter who was diagnosed with breast cancer at 41 and has the gene mutation. She was denied coverage for the test because she herself didn't have cancer. Seems to me that it would cost more to cover her cancer if we waited to see whether she had the gene mutation and didn't allow her to decide to take prophylactic steps to address it, than it would be to simply cover the test.


STEVE USDIN: When we return, a plaintiff in the Myriad case, the head of a genetic information advocacy group, and a biotech patent attorney debate the case. First, a quote from the Supreme Court ruling.




STEVE USDIN: To discuss implications of the Myriad decision, we're joined now by Sharon Terry, president and CEO of the Genetic Alliance; Michele Wales, founder and principal of InHouse Patent Counsel and former head of litigation in intellectual property at Human Genome Sciences; and Roger Klein, chair of the Association for Molecular Pathology's Professional Relations Committee. The AMP was the plaintiff in the Myriad case.


So I want to ask each of you in turn, what does the Myriad decision mean for patients? What does it mean for biotech companies? What does it mean for the labs that perform these tests? To start with you, Sharon, what does the decision mean for patients?


SHARON TERRY: For patients, I think it means better access to tests. Although the ruling itself doesn't give that access and there's a lot of implications, we're excited that -- at least for now -- there seems to be at least a headway for us to make inroads into getting access to tests and making them cost effective.


STEVE USDIN: Michele, for biotech companies?


MICHELE WALES: Well, I think it's always frustrating when the Supreme Court changes a right that has been in existence for 30 years. But because the way in which the Supreme Court did it, the ruling is very narrow, and therefore, it should have very little impact on the biotech industry.




ROGER KLEIN: Well, we were obviously very, very pleased with the ruling. And we see it as a great victory for our patients. We believe that this is going to result in decreased costs, increased access, and increased innovation in diagnostic testing and in discovery.


STEVE USDIN: It's interesting. So Michele, on the one hand, you're saying you didn't think it really has much of an effect. Roger, you're saying it does have an effect. Sharon, you're saying it does have an effect. Let's talk about that.


First, Michele, there was an awful lot that was made of this case. In your opinion, why isn't it a big deal?


MICHELE WALES: Well, I think the concern was that the Supreme Court would eliminate all DNA patents. And that would have been a much bigger deal. But what they effectively did is really only eliminate patent protection to DNA sequences that are found particularly only in nature.


And because of those sequences, the small number of scientists can easily design around effectively the Supreme Court decision and use other sequences in which to make proteins.


STEVE USDIN: So Sharon, what's your response to that? You start out by saying you think it's a win for patients. It's going to give them access to things. Is it going to allow companies to do things that they wouldn't have ordinarily been able to do before?


SHARON TERRY: So I think it's a very complicated decision. And for patients, it's difficult to understand. And I don't mean that we don't have the capacity to do that. But I do mean that I think some things, like the cost of a test, are not necessarily tied to the patent, and that there are a lot of other considerations.


And the other issue for us very much is what about the information that's generated from sequences in individuals? So when you get your BRCA1 or 2 test or another test, what's happening to that variation? Is it being put in open access databases?


STEVE USDIN: Well, let's go to that also, then. So one of the things that clinicians have told me is that Myriad has a lock on a database that allows them to tell patients with more accuracy and to give them a lot more information about what a bracket test means than anybody else does. So even if other companies can offer the test, that's not going to give patients, really, the options that they need. Broadly speaking, what would be your response to that?


SHARON TERRY: So my response is I think that, in part, that's true, because they have a long history, and until 2006, they did put that variation in a public database called ClinVar run by the National Institutes of Health. And that, when they stop doing that, it is true that they have the largest, most comprehensive database with that variation and those variants.


What I think can be done is that other labs can offer the test and begin to build those libraries. In addition, there are newer methods of doing the test. And probably, Roger can speak better than I to that. But they allow the labs to build up the same kinds of databases.


And finally, I think, if all people who were tested -- BRCA1 and 2 and other genes -- actually offered their information to a public database, we would circumvent that completely. You own your own genes. You can do whatever you want with your own variation.


STEVE USDIN: So Roger, what are you hearing from your companies, from you members from labs?


ROGER KLEIN: So I agree with was much of what Sharon said. And I think the efforts that her group is making in terms of trying to get patients to submit their results is really doing a great service. Most patients with BRCA1 and BRCA2 mutations -- most of the mutations actually are mutations that truncate the gene. About 10% or so of patients will end up with what's called a variant of unknown significance.


And those are the variants which we're not sure about what they mean. And this is where Myriad does have an edge. But I think what's going to happen is as more academic medical centers and other entities start performing the testing, they're going to submit their data to public databases. And Sharon's effort is going to help. And I think that there'll be a community wide effort to really disseminate knowledge about the meaning of variants for patients.


STEVE USDIN: So Michele, one of the concerns that I've heard also is that losing patent protection means that companies will shift more to trade secrecy. Is this an example of that?


MICHELE WALES: I think that the database, Myriad's database was never really based on patenting. It was their own trade secret that they had prior to this decision. And it's their trade secret afterwards.


STEVE USDIN: And -- I'm sorry, but we'll come back to it. To sum that up, though, it's clear that Myriad knew their parents we're going to expire in a couple of years and they weren't planning on going out of business.




STEVE USDIN: So they had some way to continue and to protect information. When we come back, we're going to continue our discussion about the future molecular diagnostics. First, here's a look at a quote from the Supreme Court's Myriad decision.






NARRATOR: Now, back to BioCentury This Week.


STEVE USDIN: We're talking with Roger Klein, Sharon Terry and Michele Wales about genetic information, medicine and innovation. Michele, I wanted to ask you to explore a little bit -- when you said that the decision was a narrow decision, one of the distinctions that the Supreme Court made was between a naturally occurring gene sequence and cDNA. It's a synthetic form of DNA.


Very briefly, what is that distinction, and why do you think it's important?


MICHELE WALES: Well, that distinction is found, really, in humans or in other higher animals. And what it just means is that the Supreme Court has excluded the exact gene sequences that are found in humans, but allowing scientists and companies to work on these sequences that they have assembled in the laboratory in which to make therapeutic or commercial products.


The thing that's most concerning, though, about this decision is that that distinction between genomic sequence and cDNA sequence or synthetic sequence is not found in lower organisms like bacterial or microorganisms. And therefore, if there's a new antibiotic, for example, that is discovered from a new bacteria, it is going to be much harder for companies to protect that.


STEVE USDIN: So Roger, you, AMP hadn't asked the Court to make this distinction between gene sequences and cDNA. Do you think that they did the right thing here?


ROGER KLEIN: What I would say is that most of the genetic testing that we do does not involve cDNA. And from that perspective, we feel that this has been a very strong decision on behalf of our patients.


STEVE USDIN: And Sharon, you actually are the co-inventor, co-holder of a patent on genetic innovation. And it involves the gene mutations that cause the disease that your children have. One, why did you decide to patent it? And two, what does this decision mean for that patent?


SHARON TERRY: So we decided to patent it, my husband and I, when we were part of the team that discovered it as non-scientists because we felt it was important that we be stewards for the gene -- that if we didn't patent it, the universities that were involved would and that there might be high cost for the test. There might be high licensing fees.


And we really wanted to shepherd that gene. We license it for $1 to labs so that we are making sure that we are able to get it out there. And the other thing we do is, in response to that licensing, we collect information. And collecting that information gives us a database that's open so that everybody can figure out what's going on when they see mutations in this particular gene, ABCC6.


STEVE USDIN: And does the Supreme Court ruling invalidate that patent that you have?


SHARON TERRY: So it certainly does for the sequence. We do also have cDNA patents, and so it doesn't for that. But I think that what our patenting the gene did was give us the opportunity to be the stakeholder that had the most skin in the game and to keep that gene in play.


STEVE USDIN: So another issue here, really, is: do these kind of gene sequences, are they really relevant today? They were something they were really big, Michele, when you were at Human Genome Sciences. You fought for these kind of genes. And there was a time in the industry when this was thought to be really, really important. Is it still important?


MICHELE WALES: I think that biotech companies and biotech industry as a whole has moved away from relying purely on human gene patents. It's just something that was important in the '90s.


I think now, companies recognizing that these patents are expiring or have expired even, that they had to come up with other ways in which to protect their commercial investments. And they've known this for the last five to seven years -- that this end of the patent terms was very near.


STEVE USDIN: And Roger, very quickly, I guess, you must have a different view, otherwise you wouldn't have thought that this freed up labs to do things that they did before.


ROGER KLEIN: Right. So for those of us who perform clinical diagnostics as well as research, this really opens up a lot of doors and really knocks down, in combination with past precedents, knocks down the intellectual property barriers to working with these genes.


We are on the advent of introducing new sequencing methodologies that allow us to take an entire genome and sequence it in a matter of hours, for a thousand dollars.


STEVE USDIN: So one of the big issues that motivated this case was the idea that women wanted to be able to get second opinions on bracket testing. Most women aren't going to get second opinions if insurance companies aren't going to cover it. And most women aren't going to get tested it all if insurance companies aren't going to cover it.


Where do you think the reimbursement issues lie, and what can be done in the future about it?


SHARON TERRY: So the reimbursement issues lie in the middle of the mess of our healthcare system, and are related and unrelated, really, to the patent issues. And what I've heard from many, many women is the difficulty of not having those tests reimbursed, especially the second opinion ones, made it very difficult for them.


STEVE USDIN: Michele, is it realistic that people are going to be able to get second opinions? And are companies going to create businesses around the idea that they would be providing second opinions?


MICHELE WALES: Well, I think it depends on reimbursement. A company is not going to create a second opinion test if they know that the market is very low, because it's going to be completely out of pocket, especially a test that is so important and has so much technical requirements. It's just not going to be something a company's going to want to develop.




ROGER KLEIN: Well, I think there's going to be multiple providers. And if a woman, before making a very, very serious decision, would feel reassured in getting a second opinion testing, it will be available, although I suspect it will have to be paid for out of pocket.


STEVE USDIN: You represent labs. You're a pathologist yourself. Is it really necessary, is it important for women to have access to second opinions? Are there questions about the sensitivity and specificity of this kind of testing that realistically, the second opinion is going to be different?


ROGER KLEIN: I think in most cases, it is not. Most of the errors, actually, in these types of situations are actually made before the specimen, for example, gets to the laboratory where something's mixed up. It's uncommon that someone will receive an inaccurate technical result from the laboratory. But it's not zero.


STEVE USDIN: Sharon, is it important for women to able to have second opinion tests?


SHARON TERRY: I think overall, yes, it should be part of the healthcare package that we are able to get second opinions. And certainly, I agree with Roger that most of the time, this is not a problem and the technical expertise in the lab is good. But in any case, we should have that right. But there's a lot of rights in healthcare that are just are not straightforward in the U.S. system.


STEVE USDIN: We're going to talk about the bottom line on the future uses of genetic information in just a moment.


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STEVE USDIN: Now, let's get some final thoughts from Michele Wales, Sharon Terry and Roger Klein. Roger, let's pick up on what you were talking about there.


ROGER KLEIN: So as we introduce these powerful new technologies into both the clinic and research, we are confronted with intellectual property barriers. Gene patents potentially stood in the way of us benefiting patients with these new technologies. And we feel that those obstacles have been removed.


STEVE USDIN: And Michele, what do you think? Do companies that are trying to create new genetic tests, do they have the tools that they need to protect their intellectual property now going forward?


MICHELE WALES: I think they do. I think diagnostic companies have relied on other ways to protect their commercial products, not only based on the genomic sequence. So by using method claims and by using other mechanisms on the patent system, they're able to protect their investment.


STEVE USDIN: Sharon, going forward, where do you see this going and where do you see the relevance of the decision on the ability of companies to innovate and for patients have access?


SHARON TERRY: I think the biggest thing is it's opened up the conversation and the public's in the game now. I think we need a revolution, a kind of insurgency around information, and that if genetic information is going to be important going forward -- and I believe it will -- we need to free the data, which is why we've created a campaign to do just that and ask every individual who's been tested to put that information into the commons so that everyone can use it and we get farther faster.


STEVE USDIN: Roger and Michele, if the data is free, if everything's out there in the commons, is there going to be the commercial incentive that labs need, that companies need, to create the new tests that patients need?


ROGER KLEIN: I think that having a database that's publicly available benefits labs and it benefits patients. It allows us to provide better and more effective results to the patients we serve.


STEVE USDIN: And by database, you're talking about databases that correlate the test results with what actually has happened with patients, right?






MICHELE WALES: I see the value in having these databases. I also think, from a business standpoint, if a company has dedicated the resources to create a state of the art database, then they should be entitled to use it.


Now, that doesn't prevent a patient and patient groups from generating their own database by having, for example, the results sent in. But I think a company should be entitled to protect what they've invested.


SHARON TERRY: I don't know if companies don't need the patents so much anymore, like they did in the past. I'm not sure they need this information as much as they did in the past, and that there are whole businesses that can be built around interpreting the information. People have said the thousand dollar genome and the million dollar interpretation. I think we're going to see that.


STEVE USDIN: OK. Well, that's as far as we're going to get today. That's this week's show. I'd like to thank my guests Michele Wales, Sharon Terry, Roger Klein and Representative Debbie Wasserman Schultz.


Remember to share your thoughts about today's show on Twitter. Join the conversation by using the hashtag #BioCenturyTV. I'm Steve Usdin. Thanks for watching.