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Casey McDonald is Senior Editor, Pharmaceutical Executive.
Rethinking views on limited-efficacy drug approvals may be in order.
As with so much this past political year, we all witnessed things we would not have thought possible 12 months ago. Experts had to clear the pie from their faces more than once. The main story for pharma was also one of the top issues nationally, the now very real prospect of repealing/replacing Obamacare. Connected, of course, are the industry’s favorite two words, drug pricing, a topic politicians are all to willing to sound off on, though rarely with much more insight than to incite the mob.
In contrast to repeal/replace and drug prices, the post election story that few saw coming is the implication stemming from a possible Trump appointee: the inkling that there might be a challenge to the FDA itself, calling into question its core mission. The healthcare media threw a fit when Jim O’Neill’s name was mentioned. O’Neill, as we now know, comes from the venture capital world as the managing director of Mithril Capital and is connected to the controversial Peter Thiel.
Suggesting a somewhat libertarian lean, O’Neill’s has in the past expressed that FDA ought to approve drugs based on safety alone, easing and speeding up the path to market. Those from venture and tech communities are known to look at healthcare regulatory burdens with a cynical eye. But for those entrenched in the healthcare world, questioning the FDA’s mission to determine safety and efficacy is anathema, downright blasphemous.
The news that O’Neill’s name could be on Trump’s roll call made an earlier panel discussion even more relevant than it had seemed at the time. On December 1, at the Forbes Healthcare Summit in New York, the always ebullient Leonard Schleifer, CEO of Regeneron, stressed the importance of “high bars” for approval while butting heads with Pfizer’s CEO Ian Read. Schleifer’s braggadocious characterization of the company’s science (and disdain for others) came across as supremely confident that the FDA should set bars for approval as high as possible, with Regeneron among the few able to make the leap. The concept of outright eliminating efficacy was not even on the table.
But while many were right to point out that such a change would indeed be a massive shock to the industry, few have connected to the other saga for which industry watchers have blasted the FDA. The approval of Sarepta’s Exondys 51 for a subset of Duchenne muscular dystrophy (DMD) patients came as major headlines for the tragically underserved patient population, but was trashed by those who expect efficacy metrics to be quantifiable. Sarepta’s drug proved very little in terms of efficacy, in a very difficult patient population, where placebo groups are nearly impossible to populate. So the FDA failed, according to many, ceding its objectivity in reverence to a pitiable and animated rare disease advocacy group.
But industry critics failed to connect the next dot, i.e., that the market still worked! Sarepta now faces significant challenges because payers are pushing back. The company’s stock rocketed, doubling after approval, but has fallen back, and is now close to its pre-approval value.
The next step for the FDA, not to mention those in rare disease communities, is anyone’s guess. Few will celebrate the fact that DMD remains a miserable death sentence with minimal options. Do payers want this responsibility and the backlash that comes with refusing an option, even though they can call it unproven?
But the industry, at minimum, needs to be willing to consider different thinking. Many rare diseases seem intractable given the current approval demands. Why not consider for certain diseases lower bars for approval to get drugs to market, then let payers, doctors, and patients sort things out?
Though their voices have rarely been heard at healthcare conferences, there are many of the VC/tech/libertarian ilk who see the FDA fitting the origin story of other bureaucratic regulatory bodies. Starting with a government overreach in reaction to real or perceived disaster, they grow like weeds with few questioning their necessity. A contrarian, outsider taking the helm could be interesting, to say the least.
Casey McDonald is Pharm Exec’s Senior Editor. He can be reached on Twitter at @mcd_casey