From legislation that would establish breakthrough designation for medical devices to a billion-dollar White House initiative on antibiotic-resistant bacteria, pharma and medtech science has risen to the top of the US policy agenda in 2015.
President Barack Obama and congressional leaders have taken advantage of the formal kickoff of a new legislative session this month to tout proposals that seek to speed products to market and develop more individually tailored therapies. As for the background issue of the flat-funding of the National Institutes of Health, more might be revealed when Mr Obama releases his budget request next week (As pharma R&D engine surges US research backing sputters, January 2, 2015).
Fifteen years later
While many of the proposals are at a preliminary stage, a concrete proposal called 21st Century Cures has emerged from the House of Representatives’ Energy and Commerce Committee aimed at improving FDA functions.
The proposal is the product of eight months of bipartisan work, and most significantly throws its weight behind the eagerly awaited priority review plans for medical devices. In May the FDA released guidance on its expedited approval pathway, with EP Vantage calculating that products with the designation would be likely to gain FDA approval in around a year from submission (New FDA expedited review could halve the cost of device development, May 13, 2014).
The committee’s draft calls for creation of a pathway for medical technology aimed at unmet needs, similar to the breakthrough drug designation used in biopharma. To qualify, a device would have to treat a disorder with no approved therapy, or have “significant advantages” over an existing approved therapy.
But then it gets a little woollier: a product could also qualify if it is “otherwise in the best interest of patients” or could reduce the need for hospitalisation and improve the patient’s quality of life. It would be a poor regulatory submission that could not argue at least one of these last two points.
Once the designation is achieved, the FDA would work with medtech companies to aid device development, agreeing trial design at an earlier stage. The proposals encourage the agency to allow shorter or smaller trials, and to compensate by collecting data post-market. As a consequence, a device approved under these rules could not be used as a predicate for subsequent 510(k) applications until these postmarketing studies are complete.
The FDA would also be permitted – or perhaps more accurately, forced – to evaluate a wider range of scientific evidence when considering approval decisions. Devices designated for accelerated review could thus be approved based on surrogate or intermediate clinical endpoints that are “reasonably likely” to predict clinical benefit.
The bill does not yet, but might soon, include details of plans to regulate lab-developed tests (LDTs), according to a placeholder title in the document.
Medtech is not the only focus of the draft legislation. Across all trials, the use of adaptive designs and Bayesian statistical analyses that predict the probability of success earlier in clinical programmes would be encouraged, along with extension of market exclusivity for drugs that have orphan indications added to their labels and new drug formulations, delivery systems or combinations.
This proposal is one of several biomedical developments emerging in the weeks of the new Congress. Another is Mr Obama’s plan, revealed earlier this week, to spend $1.2bn in fiscal 2016 to back research into new drugs and diagnostics to fight antibiotic-resistant bacteria.
The money would primarily be aimed at research-funding agencies NIH and the Biomedical Advanced Research and Development Authority; the Centers for Disease Control and Prevention would receive some backing for resistance monitoring and surveillance, and the FDA would be funded to support evaluation of novel antibacterials.
Mr Obama took the opportunity of his state of the union address to talk up a “precision medicine” initiative, or those therapeutics that can address conditions with a specific genetic cause. While little has been revealed about this, media reports have suggested that it would include the creation of a national “biobank” of genetic information developed from existing databases.
That budget request remains under wraps for now, which means it will not be known until next week how resolute Mr Obama will be on backing his proposals with dollars. Today, however, he will reportedly call for an end to the budget sequester that has led to limited increases in government agency funding, including NIH, for some time (US health agencies gird for cuts in research dollars, August 10, 2012).
Congressional Democrats have withheld their support for the 21st Century Cures initiative in part because it fails to add funding for basic NIH research. Next week will reveal whether Mr Obama is in concert with his legislative allies.