A call for action on biomarkers and the FDA's drug approval process

Molecular biomarker science is being used at every and all stages in the drug development process, but, according to a report from the Manhattan Institute, the “relevant molecular science and practice of molecular medicine have already moved far ahead of the FDA.”

Consequently, according to the report, "Unlocking the Code Health: Bridging the Gap Between Precision Medicine and FDA Regulation," changes in policy are going to be needed if that gap isn’t going to widen.

Paul Huber, JD, PhD, and Paul Howard, PhD, both senior fellows at the Manhattan Institute, wrote that the FDA has been slow to incorporate biomarkers into the regulatory procedures for drug approval, which is slowing the development of safe and effective treatments for many diseases.

Why are biomarkers important for drug development and approval?

Biochemists can design drugs targeted at the biomarkers that propel certain diseases. Other biomarkers can be used to see how diseases are advancing or retreating, which provides early indications of how well a drug performs. In addition biomarkers could help in the development of drugs that could take control of disease-causing molecular pathways before clinical symptoms appear. “The potential benefits are enormous,” Huber and Howard wrote. “For example, according to one estimate, a drug that would delay the onset of Alzheimer’s by five years would save about $367 billion in direct health costs by 2050 while likely extending the life span of millions of patients.”

Accordingly, they suggested that the inability of the FDA to move faster in this area has “serious economic and commercial implications.” Other countries, for example, are already moving ahead on biomarker development to deal with illnesses ranging from cardiovascular disease to psychiatric disorders. “While the debate goes on in America, over a new regulatory framework, other countries are already moving rapidly to translate biomarker science into new protocols for approving drugs and diagnostics,” the authors wrote.

The consequences of delay in this area are dire for patients as well, Huber and Howard added. “Absent a full integration of the best available biomarker science in the drug-approval process, many drugs that could save many lives simply won’t get approved at all.”

The authors suggested that to “get things moving,” Congress needs to step in and create “expert” panels that would be within the purview of the National Institutes of Health and the Institute of Medicine. These panels would develop substantive standards to be used for biomarkers in the drug approval process. Furthermore, expert panels would be formed that would address the statistical tools needed to analyze biomarker data.

The panels would frame standards that reflect the consensus views of the scientific community, Huber and Howard wrote, while the FDA would have the final authority in the drug approval process to “strike the right balance in assessing what is known, and with how much confidence, about the relevant biomarkers and surrogate endpoints used in clinical trials, a drug’s safety and efficacy as established in those trials, the seriousness of the disease, and the availability of other therapies.”

Such a reform effort should, the authors wrote, provide the FDA-approval process with the best available molecular biology, expedite the regulatory process, and ensure that FDA reviews are performed transparently, predictably and efficiently.

Access the report.