Evaluating New Medical Device Technologies

Aug. 30, 2013 5:01 PM ET
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Contributor Since 2013

When we see a human being suffer from a devastating disease or injury, it is natural for us to be eager to support new technologies that appear to promise meaningful treatment. These also seem like good investments, because, after all, wouldn't a person pay almost anything to walk or hear again, or an insurance company be willing to reimburse a procedure that could save them hundreds of thousands of dollars in future healthcare costs? The difficulty, however, is developing a framework that can effectively assess the potential viability of new technologies, while paring away the hype and grounding evaluation in the facts at hand. There is certainly no perfect recipe to do this. If there were, we'd all be wealthy. There are a few things that should always be considered, however, and a few basic questions that should be asked.

1. Where is the product in its regulatory approval cycle and what animal model was chosen for feasibility?

Early stage technologies, and by this I mean what the industry calls "pre-clinical," meaning animal research, or phase I, meaning in the first human clinical trial, have to be considered high risk. If the product is in animal trials, ask yourself how relevant the animal research model chosen is to people. The farther away from a human being the model is, the less of a reliable predictor of a human response or outcome it may be. Published research papers in search engines like Google Scholar can be very helpful in finding information. In looking at this, remember that even in what appear to be more closely related animal models, such as small monkeys, there can be significant differences in certain functional sub-systems. For example, with severe neurological injuries, such as traumatic brain injury, while animal models are useful, because of variability in neuroplasticity, there continues to be a lack of good consensus regarding their validity as surrogates for human performance and recovery outcomes. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2721779/

2. Were the pre-clinical data collected with good scientific integrity?

Integrity in this instance does not refer to ethics; it speaks to the validity of baseline assumptions that underpin research design. For example, is the sample size adequate to support the conclusions? Given the inherent differences between people and animals, it would be a rare situation that a sample of only a small number of animals would be sufficient to extrapolate with confidence to human beings. Was the study designed with a control arm? Studies in which all animals receive exactly the same treatment are suspect by design. Unless there is a "no treatment" arm of the study that allows you to compare sick or injured animals that receive no experimental treatment to similar animals that did receive the experimental treatment, there is no way to know how much of the recovery is due to the new drug or device, versus how much recovery would have happened spontaneously anyway. Are there other confounders, or extraneous variables in play, that are not controlled for, and what impact might they have on research outcomes? Pulling from the example of primate models above, are some species more neuroplastic (or capable of more spontaneous recovery) than others? Could there be an environmental impact of something like post-treatment care or source of injury, and how was this controlled for in the initial experiments? One thing to consider is that with animal research, the researcher is in charge and in control - of everything. With people, this will not be the case as human beings tend to exercise their "free will" more often than not.

3. What is the current regulatory environment surrounding this product type or disease condition?

One of the first things I do when evaluating an emerging new medical technology is research real and potential competitors. If there are similar products, or products for similar medical conditions on the market, it is important to examine how safe and effective they are perceived to be. Remember the silicone breast implant? Due to public safety concerns, many of which were actually scientifically not supportable, there was a period of many years when even the most exceptionally well-designed and well-researched implant could not get past the FDA. I also consider the vulnerability of the target population for the product. The more vulnerable the population (children, the disabled, Alzheimer's patients), the more scrutiny the product will receive from a regulatory and ethics perspective. This could create significant delays in product evaluation and approval, and may raise the bar considerably for what FDA expects or will accept in research design or conduct. What makes these populations vulnerable is the possibility that they are unable to truly exercise freedom of choice for themselves, and the participant's freedom to choose, without covert or overt coercion, is a fundamental tenant in clinical research. Finally, one exercise I also suggest engaging in is going to clinicaltrials.gov and researching the registered clinical trials for either the given product type or trials that appear to compete for the same patient population. If there appear to be many companies working in a technology space, and most of those companies are early phase, you know that the time is right for a winner to emerge. If there are too many others in the pond, however, or too many other clinical trials competing for the same population, it is possible that research progress will be slower than expected and that the timetables given by the company may be less reliable than they should be.

4. How much self promoting is NewCo doing and how much of that promotion is science and technology based versus designed to pull at the emotional and compassionate heart string?

Good technologies based upon solid scientific foundations with well-designed and well-executed research and clinical trials often speak for themselves. All companies do, and should promote. This is how investors are attracted and clearly that is essential. When I see a company led by an individual who seems to be getting themselves into every nook and cranny of any promotional activity, however, my skepticism antennae go up. Many of the "best of" competitions are little more than opportunities to exploit business relationships. Getting oneself nominated for Most Outstanding Polish American Businessman, for example, is generally a competition related more to who has the best publicist versus who has the best technology. When I hear what sounds like somewhat outrageous claims, such as 100% of animals treated so far have responded, I go back to the beginning of this essay and start asking the hard scientific questions.

As I asserted earlier, there is no perfect framework, but there are clearly a number of things to consider that help to better frame technology evaluation. In the final analysis, when I see a novel technology that looks exciting, that promises a potential treatment/cure for a population that clearly needs one, that appears to exploit emerging technology markets for which there will be competition, and that is based upon solid, well-supported, replicatable valid scientific evidence, it's exciting. When I see a novel technology, however, that appears to be at least as much hype as real science, that has a charismatic leader who works on my heart strings as much as on my intellect, and that appears to be growing in "supporters" much faster than the current regulatory framework or state of the science indicates it should be, I step back. Sometimes things really are too good to be true, or at least, we don't have enough data yet to tell the difference.

This article reflects my opinions only as a 20 veteran of the clinical research industry. Readers should understand that this is a perspective and not rely upon my suggestions as any guarantee of success in technology evaluation.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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