Several clinicians have asked me, as a regulatory expert, about the drug approval process in the United States and the role that the FDA plays. Bedside clinicians believe that knowing about the FDA could provide them with information that they could use to treat their patients more effectively. This knowledge also would help them understand the implication of the many FDA-related articles published in the popular press. Many professionals, however, do not focus on the activities of the agency that is responsible for ensuring the safety, quality, and efficacy of drugs until it directly affects them, one of their patients, or someone close to them.
Globalization, new types of innovative products, and the digitalization of healthcare present the FDA with new challenges. Increasingly, drugs are being developed overseas and are manufactured with ingredients sourced outside of the United States. Often, commentators complain that the FDA serves as a barrier to innovation because of burdensome requirements and an inefficient review process.1–5 Some people argue that the excessive oversight hurts the ability of companies to bring new drugs to market. They maintain that companies pay too much in user fees to be able to sell their drugs in the United States. As a regulatory professional, I find it important to set the record straight. Whether you are just now learning the truth or have known all along, the following are three of the most common misconceptions about the FDA.
Industry provides all of the funds for the FDA to rush a new drug to market6–10
FACT: The taxpayer also subsidizes the drug approval and monitoring process. The Prescription Drug User Fee Act (PDUFA) of 1992 lets the FDA collect user fees from drug companies. The law was enacted to supplement the FDA's budget outside of appropriations, so drugs can be approved and marketed more quickly. The law is renegotiated and approved by Congress every 5 years. In 1992, the revenue that was collected from PDUFA and added to the FDA's budget accounted for 9.7% of the total funding for the Human Drugs Program.11 For fiscal year 2019, which started October 1, 2018, the requested user fees would provide 43% of the total funding: the fiscal year 2019 budget request was $5.8 billion, of which $2.5 billion is user fees.
The FDA fails to protect and promote the public health, as patients are forced to wait years for access to innovative products1–5
FACT: Patient groups and journalists claim that FDA reviewers spend more time reviewing product applications than do reviewers working at foreign regulatory agencies. In reality, over the past decade, approval times have converged in six major regulatory agencies: FDA, European Medicines Agency, Japan Pharmaceuticals and Medical Devices Agency, Health Canada, Swissmedic, and Australian Therapeutic Goods Administration.12 I believe regulators are gatekeepers and enablers. As gatekeepers, they ensure compliance and protect the public. As enablers, they facilitate drug development and speed the approval of new products so new therapies can reach patients in need.
The cost-effectiveness of a new drug is the FDA's responsibility13–16
FACT: The FDA makes no judgment about the cost-effectiveness of drugs, and the approval process does not require companies to submit evidence that the new drug is better than the products already on the market. FDA's requirements for new drugs focus on product quality, safety, and effectiveness. To prove effectiveness, a company should provide data that support that its product is better than placebo. In other words, the FDA does not require head-to-head comparisons of new drugs with the current standard of care. At times, a drug that has been on the market for years might be more effective than a newly approved drug.
I believe that FDA's gatekeeping and enabling roles are both critical. As healthcare professionals—those working at the patient's bedside as well as in other areas of the translational spectrum—we should advocate that Congress provides more resources to the FDA so the agency can protect the public. We should not fall for arguments that have no basis in fact and are only myths. By better understanding the functions of the agency and its approval process, we can know how this directly affects our patients' safety.