Great support can, must be brought to broader swath of biomedical enterprise

Two weeks ago, the NIH and FDA had their say to the Senate Health, Education, Labor and Pensions (HELP) Committee on how to enable those agencies to most effectively speed the development of new drugs and devices. (See BioWorld Today, March 11, 2015.)

This week, at another meeting of the HELP Committee, representatives from academia, private funders and industry itself had a chance to give their perspectives on what they need from the NIH and FDA in order to continue supporting and delivering medical innovation.

Senator Patty Murray (D-WA), the ranking Democrat on the committee, noted in her opening comments that "we can't legislate new cures into existence." But "what we can and must do," she added, is give the biomedical community the tools to discover such cures, and get them to the patients that need them.

The consensus among the biomedical community, at least as represented by Tuesday's panelists, appeared to be that FDA has made progress unevenly in giving them those tools.

Some FDA initiatives, such as the introduction of the breakthrough therapy designation, have been considered successes that have led to a climate of innovation and a willingness on the part of venture capital to invest in those innovations. But other therapeutic areas, and the area of medical devices and diagnostics as a whole, have lagged.

Alexis Borisy, who is a partner at biotechnology venture capital firm Third Rock Ventures, described the diverging fortunes of therapeutics and medical devices to the committee.

"Overall, investments in 2014 in the life sciences sector, both biotechnology and medical devices combined, rose to the highest level since 2008 with $8.6 billion invested into 789 deals," he told the committee.

But those benefits disproportionately accrued to some therapeutic areas – those most likely to be able to get a breakthrough designation for their therapies, specifically oncology and rare diseases. Meanwhile, other areas, including metabolic disorders and neurology, have not profited as much from those changes.

"Medical device and diagnostics did not fare as well, as venture capitalists invested $2.6 billion in private medical device companies in 2014, down more than 27 percent from the 2008 peak of $3.6 billion," Borisy said. "Of even greater concern, first-time investments into medical device companies tell an even starker story. In 2014, there were only 58 medical device companies that raised their first round of venture capital financing, the lowest number of companies since 1995."

Borisy attributed that lack of investment to what he termed a "double jeopardy" in the device space. Here, a lack of regulatory clarity combines with an unclear reimbursement hurdle to leave investors with too many added uncertainties on top of the uncertainty that is inherent in research. Combine all three uncertainties, he said, and "the math just doesn't work and the investments can't flow."

TRIALS, REGISTRIES AND ELECTRONIC DATABASES

Allan Coukell, senior director of health programs for the Pew Charitable Trusts, told the committee in his testimony that the cost of clinical trials cannot be laid at the FDA's door alone. "For many drugs and devices, the clinical effects are difficult to distinguish from the normal variation in outcomes seen in the relevant population of patients," he said. The size of clinical trials is driven not by the approval standard written in statute, but by the difficulty of discerning the effect of the treatment."

That said, there are ways to make trials smaller and less expensive to run. Possible improvements range from centralizing approval of trial protocols so that a single review board could give approval, rather than having to repeat the approval process with every institution participating in a clinical trial.

One approach that could potentially save a lot of money is the use of registries. Coukell gave the particularly dramatic example of the Thrombus Aspiration in ST-Elevation myocardial infarction in Scandinavia (TASTE) trial that looked at whether aspirating blood clots before re-opening a patient's artery with a balloon catheter was beneficial or not. It wasn't, and the researchers found out for a total cost of about $300,000 – about 50 dollars for each of the more than 7,000 enrolled patients.

"Conducting a traditional study of this size in the United States would cost hundreds of millions of dollars, if not more," Coukell noted.

Being able to take advantage of such registries will take improvements in electronic databases. In fact, Coukell said, "being able to get better at extracting information from our health care system" would go a long way toward reducing trial costs.

Michael A. Mussallem, chairman and CEO of device company Edwards Lifesciences, concurred, noting that for his company's transcatheter aortic valve replacement, entering information about the procedure into an electronic database can take longer than the procedure itself.

Beyond the specifics of how to extract data, and how to make it accessible, such changes will take changes in how researchers are trained.

Bruce Sullenger, director of the Duke Translational Research Institute, said one of the things institutions like his need is NIH commitment to "reposition and train" a new generation of biomedical researchers who are "ready to utilize and act upon the genomic and informatics revolution."

In general, training is one of the looming problems of the system.

It needs money. But to attract the best and brightest trainees, it also needs perspectives – and those are sorely lacking in academia right now.

Third Rock's Borisy noted the irony of the current situation as far as employment is concerned. "I am able to hire people that would have been the rising stars," he told the committee, and "this is good in the short-term."

"But," he added, "it is bad for the ecosystem," because as things stand, the best talent, "that used to go into academia," are not there to make the sorts of discoveries that would fuel innovation down the line.

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