Scientists, policymakers, and medical professionals alike have become increasingly worried about the rise of antibiotic resistance, and the growing number of infections due to bacteria like Clostridium difficile, which cause a significant number of deaths and are imposing increasing costs on our health care system. However, in the last few years, fecal microbiota transplantation (FMT), the transplantation of stool from a healthy donor into the bowel of a patient, has emerged as a startlingly effective means to treat recurrent C. difficile infections. At present, the FDA is proposing to regulate FMT as a biologic drug. However, this proposed classification is both underregulatory and overregulatory. The FDA's primary goal is to ensure that patients have access to safe, effective treatments—and as such they should regulate some aspects of FMT more stringently than they propose to, and others less so. This essay will examine the nature of the regulatory challenges the FDA will face in deciding to regulate FMT as a biologic drug, and will then evaluate available policy alternatives for the FDA to pursue, ultimately concluding that the FDA ought to consider adopting a hybrid regulatory model as it has done in the case of cord blood.

US Food and Drug Administration (FDA) approval of a new drug typically coincides with a period of patent protection, during which the manufacturer will often apply for additional indications to expand the market for the product. For example, the tyrosine kinase inhibitor imatinib (Gleevec; Novartis) was originally approved to treat Philadelphia chromosomepositive chronic myelogenous leukemia, but has since been approved for treatment of other cancers. Many noncancer drugs also follow this pattern, including botulinum toxin A (Botox; Allergan), which was originally approved for the treatment of strabismus and blepharospasm and subsequently approved for treatment of cervical dystonia, cosmetic uses, and chronic migraine.This pattern of additional testing and approvals is common for more expensive on-patent drugs, but new indications are rarely sought for less-expensive generic drugs, for which it is more difficult to profit from the research. This creates a policy conundrum: follow-on innovation for low-cost generic products offers a rare opportunity to simultaneously improve health outcomes and likely reduce health care expenditures, but how could such research be encouraged?

IMPORTANCEState Medicaid programs have reported concerns about rising drug prices and spending, particularly regarding drugs entering the market through the accelerated approval program under the US Food and Drug Administration (FDA). The accelerated approval program enables the FDA to approve drugs on the basis of unverified surrogate end points, meaning that clinical benefits for these products are uncertain at the time of approval. However, state Medicaid programs are legally required to cover these drugs. Little is known about the set of products with accelerated approval over time, their use among Medicaid beneficiaries, or the magnitude of their financial influence on state Medicaid programs. OBJECTIVE To identify the number and class of drugs approved through the FDA's accelerated approval pathway and analyze state Medicaid programs' use and spending on these drugs from 2015 through 2019. DESIGN, SETTING, AND PARTICIPANTS In this cross-sectional study, biannual FDA reports were

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Many state Medicaid officials are concerned about rising prescription drug spending, particularly about drugs approved through the Food & Drug Administration’s (FDA) accelerated approval pathway. This article aims to determine how much of Medicaid programs’ accelerated approval spending is attributable to products that have demonstrated clinical benefits versus those that have not. Our findings provide support for states’ concerns that pharmaceutical companies often fail to complete their required post-approval confirmatory studies within the FDA’s requested timeline. But this article also highlights one issue that policy stakeholders have not yet devoted substantial attention toward: the use of surrogate endpoints involved in the post-approval confirmatory studies for most of the products in our sample. The granularity of our results enables us to analyze the impact of different policy recommendations on both the accelerated approval pathway and Medicaid programs and to inform the current policy debate, suggesting that policy stakeholders might focus attention on products converting their approval on the basis of surrogate outcomes rather than clinical outcomes.