In October 2014, the US Food and Drug Administration (FDA) issued a draft guidance outlining its plan to overhaul the regulation of in vitro diagnostics, which are tests conducted outside of a living body to detect or diagnose diseases, conditions and infections. Most in vitro diagnostics, specifically those developed by individual laboratories (known as laboratory-developed tests, or LDTs) for use by clinicians, have historically been exempt from premarket FDA review. As a result, medical-center laboratories have been at the forefront of precision medicine, rapidly developing tests for rare diseases and public health threats, such as HIV.

However, citing the potential for serious harms, the FDA now proposes to use a risk-based framework to determine which LDTs will be subject to regulatory oversight before marketing. Over a 9-year period, requirements for premarket review will be gradually phased in first for high-risk (or class III) LDTs, and then for moderate-risk (or class II) LDTs. Thus, before high-risk LDTs can be marketed or used by clinicians, manufacturers of these tests—like those of other class III devices such as implantable pacemakers—will have to provide evidence of safety, effectiveness and clinical validity.

In our article, we argue that this plan could be modified to minimize delays in market entry of lower-risk tests and those for pressing clinical needs. To promote innovation in precision medicine, the FDA, the CMS and other stakeholders should collaborate to ensure that rigorously validated diagnostic tests are reimbursed commensurately with their benefits to patients.

(cite: Hwang TJ, Lehmann LS, Kesselheim AS. Precision medicine and the FDA's draft guidance on laboratory-developed tests. Nature Biotech 2015; 33(5): 449-451.)