When legislators tour their home states and districts, private sector leaders often tell them: We need a commonsense and predictable business environment.
Business environments are shaped by laws, regulations, and market conditions. In particular, they are shaped by a company’s trust in the rules of the road as determined by policymakers, regulators, and judges. Businesses are more likely to succeed when they are confident in the durability of laws and regulations that allow them to invest and bring products to market.
Unfortunately, the regulatory authority of the Food and Drug Administration is under siege. The federal agency responsible for ensuring the safety and efficacy of the nation’s drugs, medical devices, and food supply is being systematically undermined in its efforts to advance and protect public health. As a result, both Americans and the biopharmaceutical companies that provide them with needed care and solutions are losing out.
There are multiple examples to point to. First is the assault on the FDA’s long-standing accelerated approval program. Created in 1992 in the wake of the AIDS crisis, the initiative allows for the timelier approval of drugs that treat serious conditions and fill an unmet medical need. The pathway has successfully provided cancer, sickle cell, and Alzheimer’s disease treatments years earlier than other pathways. Yet some legal and policy figures are sowing distrust and misinformation about the safety and efficacy of these FDA-approved treatments in order to support limiting coverage and access to them.
We are entering dangerous, uncharted territory. Beyond legislative and bureaucratic challenges to the FDA’s authority, a Texas federal judge’s decision on Friday would reverse the FDA’s long-standing approval of mifepristone, a drug that has been on the market for more than 20 years. It’s virtually unprecedented for a court to overrule the FDA’s scientific determinations, which invariably involve complicated risk-benefit decisions that only medical experts should be trusted to make. As top legal scholars have pointed out, the decision could also pave the way for future challenges to the FDA’s past drug approvals.
More broadly, undermining the FDA’s prerogative to approve or reject new drug applications would introduce massive uncertainties into the incentive system that brings lifesaving biopharmaceutical breakthroughs to patients. As the Department of Justice warned prior to the Texas judge’s ruling, questioning the FDA’s authority could mean that drug inventors will be “unable to confidently rely on FDA approval decisions to develop pharmaceutical-drug infrastructure” and invest in new treatments.
But this is only the latest attack on the FDA. Most notably, the Centers for Medicare and Medicaid Services recently restated its April 2022 decision that Medicare would cover monoclonal antibodies targeting amyloid plaque for the treatment of Alzheimer’s only if they have received traditional FDA approval. Drugs receiving accelerated approval would be covered only for patients in specific clinical trials — of which there are currently none. Thus, Alzheimer’s patients, who are running a race against time to lead lives of their choosing, face severely limited access to multiple FDA-approved treatments that could improve their quality of life.
Scientific evidence does not support CMS’s ruling, but just as concerning is the decision’s weakening of trust in FDA decision-making. For years, after FDA scientists rigorously evaluated and approved new drugs and treatments, CMS — as the administrator of a program that supports tens of millions of older adults — covered these critical innovations without rendering its own scientific opinions. Now, the public and their representatives are left wondering who’s really in charge.
There is no doubt that the FDA has gone through challenging times in recent years. As the chief scientific officer and chief policy officer of the nation’s leading biotechnology advocacy organization, we’ve had our fair share of issues with the FDA. However, we live in a world that is both changing and aging. More and more people will need access to proven and groundbreaking cures, treatments, and advancements that allow them to lead happier and healthier lives. They’ll also need access to innovations in food security and nutrition, which fall under the FDA’s purview.
To meet this moment, key decision-makers must focus on empowering the FDA to better support and protect Americans, not on undercutting its authority, creating unnecessary doubt in its science, and hamstringing efforts to get people the care they need. The FDA must be trusted, transparent, and seen as the paramount voice on the safety and efficacy of any particular breakthrough available to patients in the United States.
Let’s restore the agency’s gold-standard stamp and its ability to ensure effective and nimble approvals across disease states, patient classes, and lifesaving innovations. Let’s rebuild its public trust. In doing so, we will create a policy and regulatory environment that allows the FDA, individual people, and businesses to thrive together.
Dr. Cartier Esham is the chief scientific officer at the Biotechnology Innovation Organization (BIO). John Murphy is BIO’s chief policy officer.
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