In April of this year, an advisory panel to the Food and Drug Administration (FDA) voted against approval of the drug Eteplirsen, a treatment for Duchenne muscular dystrophy. This came despite the testimonies by patients and their families who believed the drug gave them hope and who voiced their support for the drug’s approval. The conflict between FDA experts and patients underscores the contradictions in the current drug-approval process. In addition, it raises the question of whether the FDA should be in position to prevent well-informed patients from gaining access to drugs. 

Under the 1962 amendment to the Food, Drug and Cosmetics Act, the FDA must ensure both safety and efficacy of drugs. The FDA panel faulted Sarepta, the drugmaker behind eteplirsen, for failing to provide sufficient proof that its treatment is in fact effective. Consequently, the panel recommended against approving the drug.

The fundamental problem with the FDA's approval process is that it is trying to answer two distinct questions: which drugs the agency should permit and which drugs it is willing to recommend. Since the approval process allows only a single answer to both questions, the FDA does not permit any new drug unless it is also willing to recommend it. Consequently, the process needlessly restricts patient access to new drugs.

Safety trials determine which drugs the FDA is willing to allow on the market. The public health disaster in 1937 when the drug Elixir Sulfanilamide fatally poisoned over 100 people clearly demonstrated the need for safety regulation. In the wake of the tragedy, Congress required all new drugs to receive FDA approval before they could be marketed. The safety regulations were further tightened in 1962 after the drug Thalidomide, commonly prescribed to pregnant women to reduce morning sickness, was found to cause birth defects in newborn babies.

The primary aim of this standard is to ensure that new drugs do not lead to adverse effects in patients; it is not concerned with therapeutic qualities of the drugs. For example, under the compassionate use clause, the FDA allows patients access to unapproved drugs that are still undergoing clinical trials. At this stage, the FDA does not have sufficient evidence that the drug would actually help patients. However, the FDA allows expanded access only when there is sufficient data on the experimental drug's safety. It does not allow access to drugs with serious safety concerns.

In contrast, efficacy trials determine which drugs the FDA is willing to recommend. The agency ensures that the new drug is not only safe but also offers substantial health benefits to patients. Since many physicians are too busy to examine the published clinical studies on a new drug's efficacy, they rely on the FDA's seal of approval to tell them which drugs will actually benefit their patients.

The two standards serve different purposes and therefore require different processes. The approval process makes sense when enforcing drug safety — the FDA is correct to keep unsafe drugs from the market. Yet, the same restrictive approach does not make sense in case of the efficacy standard. The problem is that new drugs must demonstrate effectiveness in a broader population — clinical trials testing efficacy typically require thousands of volunteers. Since the FDA does not have specific information on patients' cases, it must ensure the drug's efficacy in a wide variety of cases.

Yet, as the compassionate use cases indicate, there may be valid reasons for physicians to recommend experimental drugs to their patients. There may be no alternative treatments, as in the case of Duchenne muscular dystrophy, or patients may not react well to the existing treatments.

A better approach to ensuring drugs' efficacy would be a certification process. Under such process, the FDA would bestow a seal of approval on drugs that have demonstrated their efficacy. The certification would provide guidance to busy physicians with regards to a new drug's efficacy. At the same time, it would not restrict patients' access to safe drugs. In fact, separating safety and efficacy processes may relieve political pressure from patients' groups on the FDA to approve drugs prematurely.