OR WAIT null SECS
In promising to expedite and simplify the FDA approval process, Donald Trump fails to take account of industry's appreciation an efficient, rigorous FDA regulatory system, writes Jill Wechsler.
In a much-publicized White House meeting late last month with top biopharma executives, president Donald Trump promised to “streamline” FDA operations to speed up approval decisions on new drugs and medical products. Market-based reforms, he said, would inject more competition into prescription drug markets and help lower prices. Leading industry executives smiled and applauded, largely hoping to defuse the President’s call for Medicare drug price negotiations. But the attack on FDA rules and processes could be more damaging.
Heading up an FDA overhaul, Trump said, would be a “fantastic person” to serve as the next FDA commissioner. Several names have been floated, including candidates who talk of approving new therapies based on initial safety testing and letting patients and doctors sort out effective and appropriate uses later.
Trump further promised to cut regulations “at a level that nobody’s ever seen before” to establish “a quick process” for approving new products, while still providing “tremendous protection for the people.” Such statements ignore the fact that industry has worked closely with FDA to accelerate new drug approvals, which now take only 10 months on average and less for breakthrough and priority therapies. Sure there are complaints about too much testing for certain products and delays in final approvals, but extensive praise for fast action where it matters.
In fact, the pharmaceutical industry has long recognized the benefit of an efficient, rigorous FDA regulatory system that brings to market therapies able to demonstrate a certain level of safety and efficacy to gain acceptance from clinicians, patients, and payors that set coverage and reimbursement levels commensurate with value. This costly and complex vetting process sets a highly prized “gold standard” that facilitates market approval around the world; a high bar for FDA approval enhances market position and bolsters exclusivity.
FDA also has speeded up its approval process for generic drugs, largely due to a user fee program adopted five years ago. The additional resources have helped whittle down an enormous generic drug application backlog and help move new and important generic products quickly through the review process. A similar program also supports efficient vetting of new biosimilar products, which promise to provide less costly alternatives to expensive biotech therapies that now account for a growing volume of innovative medicines.
Killing regs, freezing staff
De-regulatory policies backed by the new administration threaten to undermine this efficient FDA oversight process. One proposal requires federal agencies to remove two regulations for every new one proposed. Most FDA regulations are required by law and often take years to develop, review and finalize; a similarly lengthy process is involved in making changes. Cancelling one third of FDA’s regulations would only generate confusion and delay, and blocking new regulations may violate Congressional statutes.
Trump also has instituted a federal hiring freeze, which would be devastating for FDA if fully implemented (there’s an effort to gain a public health exemption but it’s not clear that will apply). FDA has hundreds of unfilled positions, partly because it struggles to hire the scientists and experts it needs to effectively evaluate cutting-edge medical products and to shepherd new candidates through its review process. The “Cures” legislation authorized some helpful changes, but those will be of little value if the agency is forced to scale back its work force. That would make it impossible for FDA to meet the many requirements negotiated under user fee programs for efficiently vetting applications for new drugs, generics, biosimilars and medical devices. And it could limit FDA advisory programs designed to streamline new product testing and development in order to avoid waste and delay. Trump told industry executives that his faster FDA would prevent them waiting for “many, many years” to get new products approved, but just the opposite is more likely if his new policies are implemented.
Trump also blamed FDA regulatory policies for blocking terminally ill patients from accessing potentially life-saving investigational drugs and urged rule changes to grant more compassionate use requests. This ignores the tensions between providing seriously ill patients with unapproved test therapies, and efforts by sponsors to fully test and determine the efficacy and value of such new treatments. State right-to-try laws and manufacturer programs seek to address these challenges, and Trump thinks FDA should do more.
Another difficult challenge for pharma companies is Trump’s focus on creating more jobs in the U.S. by pressuring manufacturers to reduce overseas production and to cut imports. This could be a major problem for pharma, as about 60% of finished drugs are imported, plus 90% of active pharmaceutical ingredients for U.S.-made drugs, which come largely from China and India. A tax on such imports could be devastating to U.S. industry, and could raise costs and drug prices. Tax reform might encourage more investment in U.S.-based operations, but a massive overhaul of international supply chains could damage the global biopharma industry. At the same time, Trump’s travel ban from several Muslim countries has ignited a firestorm from many industry executives and from the biomedical research community – but little comment from pharma leaders.
Instead, drug company CEOs joined Trump at the White House to defuse talk of price negotiations on drugs covered by Medicare. Industry has long shuddered at such a concept as the first step towards government price controls on prescription drugs. Even though such a move runs counter to Republican efforts to reduce government regulation and interference in competitive markets, manufacturers recognize that Trump often follows his own course.
The administration’s choice for FDA commissioner will go far in indicating whether the White House is serious about supporting effective development of important new therapies in the U.S., or is comfortable in heightening uncertainty about how rules and laws will govern biomedical research and production. A first order of business for FDA is to gain Congressional reauthorization of several key user fee programs; delay in this complex process could bring the drug review process to a halt.