[caption id="attachment_4013" align="alignleft" width="189"]Eppur si muove, indeed, but to what effect?[/caption]
From time to time, it's nice to rediscover I'm not the only cynic in the room. So I was obviously relieved to see a couple of criticisms of the latest orthodoxy about the future practice of medicine. The question is: Is the Precision Medicine Initiative the real deal or just another government gamble on the ponies?
Rita Rubin writes in the March 17 issue of the Journal of the American Medical Association that the hyperbole surrounding the President's Precision Medicine Initiative (or PMI) has raised a few eyebrows, albeit for different reasons. Rubin cites Jeffrey Matthews, chair of the department of surgery at the University of Chicago, as arguing that the title of the program exceeds the program's likely reach.
Matthews is said to have stated that precision medicine sounds great on paper, but "it is being oversold and overhyped, and it's creating unrealistic expectations on the part of patients and clinicians." Of course PMI is far from alone in this, as the much-ballyhooed Critical Path Initiative and the parallel review program run by FDA and CMS make clear.
Big data maven Eric Topol of the Scripps Translational Science Institute is credited with a skepticism of a different brand. Rubin quotes Topol as saying, "the science is catapulting forward" toward precision medicine, but that genetic sequencing fills in only one layer of the picture.
"If you really want to change medicine, you have to have all the information on the individual," Topol stated, making the argument that even information on the bacterial environment in the gut is necessary to leverage this new medical paradigm. Topol may be right, but one has to ask whether it's even remotely plausible to economically come up with that much information.
Not to take a shot at Topol, but sooner or later, someone will ask whether the relationship between the individual and his or her parents is essential to a true personalized/precision medicine paradigm. That's how we'll know for certain that all this emphasis on precision has gone too far.
The past as epilogue
Another point of consideration is that the PMI calls for the collection of data on a million Americans and the use of smart phones and so on to evaluate the interaction between genes and other factors, such as environmental factors.
We might note that government finally (and mercifully) pulled the plug on the National Children's Study and that bisphenol A has been pretty much exonerated from any culpability where human disease is concerned. The National Children's Study suffered from no shortage of ambition or of bogeymen, and we might ask ourselves if the Precision Medicine Initiative, relying as it will on electronic health record interoperability, is likely to meet the same fate, especially given that lifestyle is still a big – maybe the biggest – factor in declining health.
Time will tell, but I'd point out that betting against government's blind ambitions does not routinely result in a bad day at the horse track.
As those in attendance at ACC 2015 know, Jeff Popma of Beth Israel Deaconess asked publicly whether the 30-day data for Sapien 3 should suffice for approval of the device, but the story grew more interesting today (March 16). I was at a session at ACC dealing with translation of research into clinical practice, and Bram Zuckerman, director of the cardiovascular devices division at FDA’s Office of Device Evaluation, was on the dais. He talked a little about how FDA approaches evidence and so on, and really had little to say that he hasn’t said many times before.
Someone in the audience asked Zuckerman about whether the U.S. premarket review system should not look more like the system at work in Europe, and I followed with a question about whether an approval of Sapien 3 and other TAVR devices would be as a PMA supplement or as a PMA, and whether he could characterize his conversations with industry about PMA supplements for TAVR generally.
That didn’t sit well with Zuckerman, who said “this question is designed to obtain confidential information” about a device application. He went so far as to say that my presence was “an example of the kinds of people” who attend events like ACC 2015, and I suppose I should have been flattered when he described me as “a well-known member” of the press.
I wasn’t aware I am all that well known. After all, Medical Device Daily is a small-volume publication. If you want someone who is well known, try tracking down a name with the New York Times. They’re well known, but I doubt seriously whether more than 1,000 people in this world know who I am.
In any event, Zuckerman’s allegation that I was looking for confidential information is absurd. I asked for some idea of the conversations between FDA and industry on the notion of what might constitute a reasonable set of conditions for the use of a PMA supplement for TAVR. Zuckerman’s confusion about whether I was asking about Sapien 3 specifically is understandable, but he could have just said he couldn’t answer the question due to confidentiality.
That wasn’t good enough, though. He decided to go out of his way in an attempt to embarrass me. For the record, I was embarrassed alright, but not for myself. I found Zuckerman’s paranoia odd, not to mention unbecoming of his self-described status as a “humble public servant.”
I get that everyone has a bad day now and then, but this was way out of line. Bram Zuckerman needs to realize that the media have their place in this world. If he doesn’t like dealing with questions, maybe he should get a job that keeps him safe and sound in a place where no one has the temerity to challenge his authority. Until that day arrives, he might want to consider that it’s not the job of those in government to attempt to bully the press.
Earlier this month, I had the distinct pleasure of having a sit-down interview with Kenneth Mandato, the lead researcher for the migraine study and an interventional radiologist at Albany medical center, during the Society of Interventional Radiologists Annual meeting held here in Atlanta. During the interview we discussed the prevalence of migraines and a rather old (but underutilized) technique to treat these conditions. Mandato and a team of researchers from Albany Medical Center and the State University New York Empire State College (Saratoga Springs, New York), used a technique called image-guided intranasal sphenopalatine ganglion (SPG) blocks to give patients enough ongoing relief that they required less medication to relieve migraine pain.
Mandato said that the study shows that before treatment, patients reported a visual analogue scale of 8.25 with scores greater than four at least 15 days per month. The day after the SPG block patients' visual analogue scores were cut in half, to an average of 4.10 Thirty days after the procedure patients reported an average score of 5.25 a 36% decrease from pretreatment. About 88% of patients indicated that they required less or no migraine medication for ongoing relief.
Mandato seemed pretty happy about the results and the potential to do even more trials in the future to bolster the results. What was so impressive was the number of calls and media requests Mandato managed during his time at the conference. Migraines are a hot topic. Here's hoping interest in Mandato's work continues.
[caption id="attachment_3967" align="alignleft" width="241"]Skating champ Scott Hamilton sounds off on the device tax[/caption]
You don’t see it very much these days – bipartisanship. Yes, federal legislators on both sides of the political spectrum actually agree on something. This time they concur that the medical device tax, one of several funding mechanisms for the Affordable Care Act (ACA), has got to go. They argue that the tax stifles innovation and employment in the medical device industry.
Medical device companies will more likely pay this tax by withholding some investments in research and development and their workforce, rather than passing the cost onto their healthcare customers. Between research and development and employees, the likelier target for cuts is research and development, an area that is more of a delicate balancing act between risk and reward. Companies take a risk that their investment in better medical products will lead to a reward in the future, both for them and patients. It’s a leap of faith, it’s more speculative. There are no guarantees. But what is guaranteed is that according to ACA law, medical device vendors have to pay the medical device tax, and that money has to come from somewhere.
You may ask yourselves: Why would a retired Olympic figure skater care about how a tax impacts medical device companies? The answer is that I am the beneficiary of years of medical innovation, in this case a very specific medical procedure used to treat brain lesions – single-session Gamma Knife radiosurgery.
Several years ago, I was diagnosed with a benign brain tumor and received Gamma Knife radiosurgery. The therapy is incredibly precise and quick. From a semi-circular, helmet-like array around the head, the Gamma Knife focuses 201 beams of low-energy cobalt-60 radiation on the tumor target. When the beams converge on the lesion, the cumulative dose to the tumor is massive and highly conformal. In other words, the radiation dose is confined virtually exclusively to the tumor and not to healthy nearby brain tissue. For me, it meant I received a highly precise treatment without endangering my optic nerve. And I didn’t have to go back to the clinic on multiple occasions for additional treatments.
I’m intensely interested in this issue because Gamma Knife radiosurgery is truly an elegant and efficient procedure that can be used to treat many difficult brain tumor cases, both benign and malignant. It still amazes me that a single 30-minute procedure could have such a profoundly positive effect on my life.
My Gamma Knife radiosurgery experience
In 2004, I began suffering some unusual symptoms. I was listless and lacked the usual levels of energy that had propelled my career as a figure skater. In addition, I experienced some alarming blurriness in my vision and my testosterone levels were extremely low.
MRI and positron emission tomography (PET) scans showed a 2.46 cm mass in my brain, the identity of which was subsequently confirmed by needle biopsy: I had a craniopharyngioma. This is a benign tumor that affects the pituitary gland in the brain.
Any kind of surgery to cut out this mass was out of the question, as part of the tumor was wrapped around the optic nerve of my right eye. Of course, doing nothing would have resulted in losing my vision in that eye over time.
I consulted an authority on craniopharyngiomas, and the physician told me that if I were his brother or son he would recommend Gamma Knife radiosurgery. Such an endorsement coming from an expert convinced me that Gamma Knife was the way to go.
I had Gamma Knife radiosurgery on December 13, 2004. I had 26 “shots,” in which the 201 beams struck the craniopharyngioma from hundreds of different angles. Incredibly, the entire procedure took only 30 minutes, but that is not unusual for many different Gamma Knife cases. And just to reiterate, Gamma Knife radiosurgery is a single-session treatment – it was “one-and-done” – and the only side effect I had from the treatment was a two-Tylenol headache caused by fixation of the stereotactic frame to my head. By the time I went to bed and woke up next morning the headache was gone.
Every six months after radiosurgery I had brains scans, which showed that the tumor was diminishing in size until it became just a little dot and the lesion was no longer threatening my vision. And each time I went in, the results of the visual field tests would come back better and better, and hormone replacement therapy helped me regain the energy I had lost due to the impact of the lesion on my pituitary gland. In short, I was able to resume my life of speaking and authorship fairly quickly.
Fast forward to 2010, and unfortunately the craniopharyngioma had begun growing back, as these tumors often do. However, this time my physician informed me that the lesion was presenting beautifully for a minimally invasive transphenoidal surgical resection that would not endanger my optic nerve. After that operation, the tumor was gone for good.
A vote for more R&D
The epilogue of this story is that in 2004 I was able to take advantage of a truly remarkable single-session therapy that was the only practical option available to me. But Gamma Knife radiosurgery wasn’t developed overnight – it took decades of continuous refinement and millions of invested R&D dollars by its manufacturer (Elekta) to arrive at the level of technology that is capable of delivering such miracles.
Reflecting on my experience I always wonder what new medicines or techniques are now in peril of never seeing the light of day because companies have had to cut back on research and development. Maybe it’s development of a new cancer drug or a more effective pacemaker. It could even be the next generation Gamma Knife system that could be used to benefit untold thousands of additional patients worldwide.
I join with the hundreds of American medical device manufacturers in supporting a repeal of the medical device tax, and call on Congress and President Obama to join this effort. Continuous innovation in medical technology depends on it.
Scott Hamilton is a retired American figure skater, Olympic gold medalist, speaker and author. He won four consecutive U.S. championships, four consecutive World Championships and a gold medal in the 1984 Olympics.
[caption id="attachment_3945" align="alignleft" width="288"]There's someone innuendo![/caption]
There are facts and there’s the stuff that surfaces when facts are in short supply, but as Groucho Marx might have said, facts goes out your front door and rumors come innuendo.
Following are three developments that have emerged over the past two months, but just what do we really know about all these goings-on?
Who’s next at FDA?
It was hard to be in the life science business and not hear about FDA commissioner Margaret Hamburg’s plan to leave the agency by the end of this month, and the news sparked a ton of speculation, especially given that the agency had just hired Robert Califf of Duke University.
Califf – described in the agency’s Jan. 26 statement as “a recognized global leader in cardiology, clinical research, and medical economics” – will lead the personalized medicine effort at FDA, but does that make him a candidate for the next commissioner?
Maybe. But of the past 10 FDA commissioners, only two had prior experience at the agency (Jane Henney and Lester Crawford), and neither was hired for the commissioner’s post from directly within FDA. They were both working elsewhere when they got the bad news.
One difficulty for the next appointee is that he or she can be guaranteed the job only for the next 22 months because there will be a new head honcho at the White House by January 2017. That fact might seem to argue for an in-house candidate, but that would be an exceptional outcome all the same.
The rumor mill is quiet for now, but give it a couple of days.
FDA and LDT regs: Boomerang in the making?
It’s easy to forget that boomerangs were invented quite a while back to bring down a more or less nimble source of protein, but getting hit in the head by one’s own projectile is not unheard of at any stage of social development. Girish Putcha of Palmetto GBA said in a January meeting on FDA regulation of lab-developed tests that the agency’s efforts could push some LDTs to doctors’ offices.
There have been rumblings that industry might sue FDA over its proposed regulation, but the more likely scenario might be that identified by Putcha. Obviously he would know better than I about such things, given that he gets paid to pay attention to the goings-on at clinical labs. That’s not the same as saying it would play out that way, but it wouldn’t be the strangest reaction to FDA’s regulatory moves, either.
Lest the notion be greeted with chortles and skepticism, let’s recall that the calls for healthcare reform in the 1960s were sparked in part by physicians ordering tests conducted at labs owned by (you guessed it) physicians.
Conflicted over conflict of interest?
We’ve heard tons about FDA advisory committees and conflicts of interest, but more data have emerged about the situation, and they don’t favor the furor over conflicts of interest.
A January report addressing those concerns for pharmaceutical advisory committees concluded that there was almost zero evidence that conflicts of interest affected the outcomes of these advisory hearings. In fact, seemingly conflicted panel members were more likely to surprise the authors of the report with their votes than non-conflicted members.
In other words, those with conflicts went out of their way to vote in a way that ran against their financial interests. Is this innuendo finally out the door?