IOM: Improved Framework Needed For FDA Oversight of Drugs, Devices, Foods

The Food and Drug Administration, in regulating drugs, medical devices, foods, and nutritional supplements, needs to coordinate with other federal agencies and improve its surveillance systems to gain better understanding of the use of biomarkers, according to a new Institute of Medicine report released Wednesday.

Since companies may find it difficult to test their products against actual clinical outcomes to find out if they cure or reduce the risk of disease, these companies often conduct studies measuring the effects of biomarkers. Biomarkers, sometimes described as "biological yardsticks," are factors such as temperature, blood pressure, or cholesterol that can be used as substitutes when reviewing clinical outcomes.

As an example, a tumor size is used as a biomarker when it is used to measure a cancer drug's effectiveness. In addition, blood levels of low density lipoprotein (LDL) cholesterol are often used as a biomarker for the risk of heart disease. Based on these cholesterol levels, drug and food companies make claims about heart health benefits—even if the products have not been shown to actually lower heart disease, according to the report, Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease.

The report calls on Congress to boost the agency's authority to require further studies of drugs and devices—after they are approved—if their approval is based on studies using biomarkers as surrogate clinical outcomes.

The committee called for a new framework that entails validating a biomarker so it could be accurately measured, ensuring that it is associated with the clinical outcome of concern, and confirming that it is appropriate for the proposed use.

Also, the IOM committee observed that no scientific grounds exist now for using different standards of evidence to evaluate the health benefits of food ingredients or medications—given that both can have "significant impacts on individuals' well being," said the committee.

Foods and drugs are regulated differently by the FDA. When the FDA reviews drugs, the safety and efficacy of the entire product is considered; however, when the agency considers foods, the safety of individual ingredients is evaluated rather than the food as a whole, the committee noted.

Despite the common perception that foods present fewer risks to consumers than drugs, food based public health interventions—for example, supplementing milk with vitamin D and fortifying cereal with iron—may pose greater risks than many drugs because the reach of food is so vast. Even minor risks are significant when the majority of the population is exposed to them, the committee said.

FDA has been hampered in its ability to assess the number of health claims being made by food and supplement manufacturers in part because it lacks a process broadly accepted across the regulatory, food, and medical communities to evaluate biomarkers as valid and appropriate measurements to substitute for clinical outcomes, the report noted.

"Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medications, and this committee thinks that should in fact be the case," said committee chair John Ball, who is executive vice president of the American Society for Clinical Pathology, Chicago.