In his first joint address to Congress, President Donald Trump had some harsh words for the FDA and its drug-approval system, which he has repeatedly said is too slow.
During the speech, Trump acknowledged in the audience Megan Crowley, a college student who has Pompe disease––a rare muscle disorder that can be treated with an expensive enzyme-replacement therapy.
“But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” the president said. “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.”
Crowley’s father, John, created a new company, Novazyme, to help treat her disease. He is now chairman and CEO of the rare-disease biotech Amicus Therapeutics. Genzyme bought Novazyme in 2001 for its then early-stage therapies; Genzyme itself was bought 11 years later by Sanofi. The two companies now market Myozyme (alglucosidase alfa), which in 2006 became the first drug approved for patients with Pompe disease, based on trials in only a few dozen patients, with a nine-month review period. Currently, the average annual cost of treatment with Myozyme is $298,000, according to Sanofi.
While backing Trump’s focus on rare diseases, the National Organization for Rare Disorders (NORD) believes that a wholesale change in the FDA’s approval process would not benefit that patient population. NORD 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.”
“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),” the group noted. Furthermore, “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.”
Under the Orphan Drug Act of 1983, rare diseases are defined as those that affect 200,000 patients. NORD estimates that approximately 30 million Americans have rare diseases.
Analysts at Leerink, a leading investment bank specializing in health care, saw the president’s speech as being broadly positive for the biotech industry.
“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,” the firm told its clients. “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.”
Leerink also noted that Trump’s demand that the FDA’s review system should be sped up is “probably also to the industry’s advantage.”