Getting new therapy to patients can be harder than putting a lander on Mars

Getting new medical treatments from the lab benchtop to patients' bedsides can be harder and more expensive than putting a lander on Mars. But it doesn't have to be, says Tiffany Wilson, who heads a nonprofit organization that guides new medical solutions from the lab bench to the bedside.

One difference between landing on Mars and healing heart infarcts is that the first Mars lander touched down in 1971. There's still no therapy on the market to adequately heal muscle tissue killed in a heart attack.

It's not for lack of medical research progress. Indeed, treatments for heart disease have vastly improved since Mars 2 landed. But a promising new therapy can take longer to make it from lab validation to clinicians' hands than a space probe does from prototype to Mars -; and only about 15% of new therapies arrive with patients.

A new device or drug can fail clinically, but more likely, it will run out of finances, miscalculate the market, or collide with regulations. Researchers can plan for these obstacles early on, and the more of them who do, the more good science will make it to patients, according to Tiffany Wilson, CEO of the Global Center for Medical Innovation (GCMI).

GCMI is a nonprofit affiliate of Georgia Tech. It advises researchers who are trying to advance solutions from the lab bench to prescription pads and surgical tables, including product development and preclinical trials.

In this interview, Wilson outlines that stony path.

RESEARCH HORIZONS: What's harder? Getting a new therapy to patients or putting a lander on Mars?

TIFFANY WILSON: It depends. If you follow proper medtech product development pathways, you get better, more predictable outcomes. That also enables failing fast, which is important. If you can realize while you're in the lab that a solution you're working on is unlikely to make it to market, it may lead you to solve the problem another way that will work for clinicians and regulators.

A lot of people continue pushing a technology before sorting that out, and then getting through regulatory compliance and to market becomes more time-consuming, difficult, and expensive. It actually doesn't take much of a financial investment to figure out early if something has a poor chance of making it to market or to develop a plan so that future funds are spent well.

As to being harder than landing on Mars, I can't really say. That's not my world. (She laughs.)

RH: What are some ways of failing fast enough?

WILSON: Don't build things too fast. Validate the unmet clinical need first. Find out about clinical workflow and how health care operates, then maybe decide not to pursue the prototype you had planned but work on a new one instead.

It generally doesn't work to take what was built in the lab and make the same thing with medical-grade materials, and unfortunately, many researchers don't realize this until it's too late.

With the Wallace H. Coulter Department of Biomedical Engineering operated jointly by Georgia Tech and Emory University, we have a wonderful opportunity to engage early. If engineers run into clinical obstacles at Emory, that may indicate that these same obstacles will exist throughout the health care system.

Also, know your competition. Can you develop something novel enough to protect it from their intellectual property claims? Are you more competitive than the current standard of care?

RH: What percentage of therapies make it from the lab bench to the bedside?

WILSON: Eight or nine things out of 10 will fail - if we're talking about going from a very early stage all the way to commercial launch.

RH: Those odds don't sound too horrible.

WILSON: It depends on what you have. Is it a new catheter design with incremental changes, or is it a new CardioMEMS [invasive device to monitor congestive heart failure] that is truly transformational? The odds get slimmer with truly novel, innovative things with no predicate technology. You really need the right team in place to navigate pitfalls associated with that.

A new catheter design would probably fall under class II devices with the FDA. These devices pose little risk and are usually officially classified as non-invasive. Higher-risk, invasive technologies are class III and have to meet much stricter guidelines.

A device doesn't have to mean a machine, by the way. Something like a patch placed on the heart operatively would be a device. An injection into heart muscle might be considered a device or a drug.

RH: How often do things fail medically between the lab and human trials?

WILSON: Successful preclinical trials, that is, testing in large animal models, are a good indicator if medical devices are going to work on human patients. As always, there are exceptions. For example, some early heart valves that worked on large animals didn't work under the stresses of everyday human life or in combination with other medical conditions the patients faced.

Otherwise, there can be hiccups when moving from the lab to preclinical or clinical trials because of anatomical differences that the design of a device doesn't work for. That can mean just going back to do more science. There can also be bureaucratic snags connected with trials, like an investigational plan for the FDA that needs changing.

RH: That sounds reasonable. So, what more serious pitfalls generally stop a new therapy?

WILSON: First, not understanding enough about who your customers are because there are many customers. The clinician is only a part of the equation. There's the patient, who may not want the therapy. The hospital supply chain may not be able to handle it. Regulators may not approve it. How is it delivered? Is it part of a procedure? Many stakeholders need their questions answered.

Another one is not defining the claims in a way that will enable the product to be promoted in the market. Solutions are classified into different categories based on claims and risk, and the regulations and marketing for each path are very different.

Words matter. For example, if I want to market my new catheter as "pain-free," the FDA may want me to conduct an expensive clinical trial, but if I take that same catheter and market it as "low friction," which is why it's pain-free, then I can demonstrate that with simple bench tests. For an incremental innovation, I'm likely going to choose the path of least resistance.

A very serious potential snag is the business model, and startups led by technologists often don't see it coming. It's not what they're used to dealing with. Even for those on the business side, it's tough enough because health care business models and policies are in a state of change.

With all these variables, you have to bring together scientists, clinicians, businesspeople, lawyers, and government regulators, and we have to work together to get new solutions across the finish line. No individual group can do this alone.

Then researchers need to ask if a sizeable enough investment can be mustered to get the technology through clinical trials and then to commercialization. A very high-risk device that clinicians will rarely use does not have great chances at attracting investment.

Find out what frustrations clinicians have with existing solutions and if your solution will make enough of a difference for them to go to their purchasing departments and push to get your solution. In addition, just like with business models, hospitals are rapidly changing how they work, and one hospital may be very different from the next. You have to stay on top of these trends.

RH: Following scenario: Agency funding is coming to an end on a solution that works great on the benchtop. It starts preclinical trials. How likely is it to get funded to marketability?

WILSON: That scenario is very difficult because it's rare for benchtop research to include the risk mitigation that an investor needs. This is a big factor in the discussion of the "Valley of Death." That's the nickname given to the financial chasm between the end of public research funds and the first major private investment funds. Many new solutions die in that space.

Presentations to investors are usually about the great science but don't address market questions, and investors leave the table thinking it's too early to invest, if at all. At a minimum, you need to show them verification and validation that the technology works and success is repeatable. There should be no ambiguity about chances of getting FDA approval and an understanding of where things could go wrong, along with plans for addressing it.

Angel investors will take on more risk because they're interested in engaging with you and in the excitement of doing good. If we could do some of this de-risking already on campus, we might be able to get them to fund development sooner to shorten the Valley of Death.

You want to understand what investors want before you exhaust your grant money. At GCMI, to help with that, we frequently write letters of support for researchers applying for grants, so they have that money, and we provide guidance on the development process.

RH: Since we're on the topic of money, how much does it take?

WILSON: That's an extremely important question because the business side appears to be the biggest challenge to so many researchers, and that can have unintended consequences beyond sacrificing finances. If the business side gets drawn out, a competing lab with a similar product can make it to market first, and then you've also lost time and resources to do your research.

As a rule of thumb, getting a novel device to market takes 10 years of work and about $100 million total investment. For a drug, investments can total $1 billion. Both of those include the money that a device company or pharmaceutical company will pay in costs to get the solution to market.

Pharmaceutical investors face higher risk. They can actually sink their own companies with a single drug failure, but the potential rewards are also much higher.

Also, a researcher doesn't necessarily have to go all the way to commercialization with a drug. If you make it to human trials, many times a pharmaceutical company will scoop it up, sometimes even in preclinical trials.

Device companies usually don't do this. Generally speaking, you need to be generating revenue with your solution before they'll get involved.

RH: Just for comparison's sake, NASA's entire mission budget for the Mars InSight Probe is $814 million, so it appears cheaper than getting a new drug to market.

Those big-money investments are all farther down the road, but serious funding has to happen up front, too, right?

WILSON: Yes, about four rounds of funding before you even get to venture capital.

Getting seed money is pretty easy. That comes from very early investors, who also get an equity stake. The follow-on rounds of funding are really hard. Initially, those usually come from your own personal savings or home equity, friends, family, and angel investors.

Then early-stage venture capital funds can take researchers through clinical trials. Traditional venture capital these days pays for commercialization.

If you can market a solution for veterinary use first, that can help with the funding you need to make it to market for humans. And getting veterinary treatments to market is easier because there are far fewer regulatory and market constraints.

Whichever route you take, many stakeholders are investing heavy amounts of time and money, and to make that count, you should know who has gone before you with similar solutions and how they succeeded or failed.

RH: I've heard people complain that regulations are extremely difficult to satisfy.

WILSON: Regulations have increased over time, but we previously had the wild, wild West -; basically little to no regulation -; until the mid-1970s. That was not great for patients, so regulations got more complicated over time until they became nearly paralyzing.

That began to shift around the last recession in 2008 or 2009, when the FDA started putting in new programs and staff to open doors for innovation and let it happen more quickly and predictably. Now, federal laws and policy work quite well, and regulators want to engage early and often with faculty to answer questions.

You have an opportunity to show regulators that you're committed to quality and due diligence. It also helps the FDA anticipate the pipeline so they can figure out how to deal with truly novel therapies and converging technologies to make sure they get to patients expediently.

RH: Your job sounds pretty tough.

WILSON: I won't argue with that. Actually, it's fun because I have an amazing team at GCMI, and we get to see the newest technology and meet incredibly fascinating people every single day. The research and the studies are just phenomenal.

And it's really wonderful and exciting when we get that research developed into products that benefit patients.

This interview was edited and condensed. It was approved by Tiffany Wilson.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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