Trump signals elimination of ‘burdensome’ drug approval system

The speech, which was also made in conjunction with Rare Disease Day, saw him focus on a range of issues from the economy to education, but healthcare also got a lot of airtime, with him reiterating the need to repeal Obamacare.  However,  some of his toughest words were reserved for the FDA and the drug approval system, which for months now he has signaled is too slow, and could very soon be about to get a new, and possibly radical, commissioner.

In his speech, he said: “We must eliminate the burdensome approval process for life-saving drugs so that more lives can be saved.”  He also made mention of Rare Disease Day and brought into the gallery Megan Crowley, who was diagnosed with the rare condition Pompe disease as a young child.

In a traditional biotech startup story, Crowley’s father John went on to create a new company to help her disease known as Novazyme, and is now chairman and CEO of another rare disease biotech, Amicus Therapeutics.  His startup and its back story also became a 2010 film, Extraordinary Measures, starring Brendan Fraser and Harrison Ford.

Pompe is a neuromuscular disease that can ultimately be fatal, even with the enzyme replacement therapies (ERT) on the market from Sanofi Genzyme. Genzyme bought Novazyme in 2001 for its then early-stage therapies; Genzyme itself was then bought 11 years later by Sanofi.

The pair now market Myozyme (alglucosidase alfa, rhGAA), which was the first drug approved for Pompe disease back in 2006, based on trials from just a few dozen patients and in a review time of less than a year.

Amicus and Crowley are now currently working on early and midstage tests that join its experimental chaperone med with ERTs in the hope of creating a better, new therapy for the condition.

Trump used Crowley’s story as an example of the “unbounded power of a father’s love for a daughter,” but then said: “But our slow and burdensome approval process at the [FDA] keeps too many advances, like the one that saved Megan’s life, from reaching those in need.  “If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan,” he concluded.

The company, which has a $1 billion market cap, was up 3% after hours before Trump’s speech, and up 11% this morning (and also after posting its financials). This will be good news for the biotech, which had a rough 2016 after the FDA said its Fabry disease candidate migalastat did not meet its requirements for a speedy review, and the biotech will also see any approval delayed until at least the end of the decade as it needs to create more data for the drug. Cue its shares down 20% on the news.  However, the drug is approved in Europe, gaining an EMA nod last spring under the name Galafold. It’s unclear whether Trump was making any specific reference to this apparent juxtaposition.

And while backing Trump’s focus on rare diseases, The National Organization for Rare Disorders (NORD) believes a wholesale change in the approval process would not in fact benefit rare disease patients.

It said in a statement: “We agree that FDA review processes can be improved upon to expedite the development and review of orphan drugs. Yet we disagree with the President that restraints must be slashed, or that the approval process at the FDA is preventing advances from reaching those in need.  “Between 2008 and 2013, 87% of the 113 rare disease treatments reviewed by the FDA received an expedited review, compared to 35% of treatments for common diseases.

“Seventy-eight percent of rare disease treatments were approved using one or more flexible development approaches (generally defined as an approach that does not include two adequate and well-controlled trials or uses novel endpoints).  For patients with immediately life-threatening illnesses who cannot participate in clinical trials, the FDA approves 99.5% of all expanded access requests submitted by physicians and companies.  We also believe that the current safety and effectiveness standards for drugs and biologics are crucial to ensuring individuals with rare diseases receive therapies that will positively impact their lives. Our patients deserve the same quality therapies as everyone else, and to weaken the standards will only threaten our population with unsafe, ineffective therapies.”

As with all of Trump’s speeches, a pinch of salt always needs to be taken with just how literal he is being, and it would be extreme to say the least if his administration eliminated the “burdensome” elements of the drug approval process.

What was likely being spoken about was speeding up the system to get more new meds on the market more speedily than they are now, beyond the 21st Century Cures Act provisions, and the fast-track/priority review processes, although an exact plan as to how this would happen has yet to be detailed.  Some saw the speech as a nod to a new commissioner breaking with tradition being on the cards, while others, such as Forbes’ Matthew Herper, said insiders and the industry are still pushing for a more traditional candidate in the form of Scott Gottlieb.  A decision on the commissioner is likely to be made this month.

Analysts at Leerink saw the speech as being broadly positive for biotech. In a note to clients, it said:  “Last night’s presidential speech to the joint sessions of Congress was light on details, and long on rhetoric, but overall should provide reassurance to biopharma industry participants and investors.  The trajectory still seems to be toward less regulation, rather than more, and toward a market-based approach to drug pricing, rather than a federally mandated or regulated one.  This stance, along with apparently continued congressional support for traditional industry priorities, suggests to us that the outlook for innovative medicines producers is relatively favorable, and the fears of the sword of Damocles hanging over investor attitudes should continue to dissipate.”

The firm also noted that Trump’s demand that the FDA review and approval system should be sped up is:  “Probably also to the industry’s advantage, although such a change would inherently lower barriers to entry and increase competitive intensity.”

REFERENCE:  Fierce BioTech; 01 MAR 2017; Ben Adams

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