EXPANDED FECAL MICROBIOTA TRANSPLANTATION USE FOR THE TREATMENT OF CLOSTRIDIOIDES DIFFICILE: A STUDY OF THE ECONOMIC AND POLICY IMPLICATIONS
Persing, Nichole M
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Problem Statement: Patients and clinicians face a dire need for access to effective treatments for diseases with treatment-resistant colitis and diarrhea. An efficacious treatment—fecal microbiota transplant— exists but is currently considered third-line therapy in the United States and has limited access due to current U.S. policies and regulations. Methods: This research used a systematic literature review and key informant interviews to identify key issues and barriers in gut biome research, discuss the potential for fecal microbiota transplantation (FMT) as a treatment option, identify other innovative treatments for gut-mediated illness, and evaluate the policies and procedures regulating and impacting the use of FMT. An economic model was developed to explore the cost-effectiveness of expanded use of FMT to treat clostridium difficile infection (CDI) in the United States, as compared to current standard treatment options. Results: A process diagram describes the steps and components that comprise the complete FMT lifecycle, the relevant policy and regulatory governance and oversight in each step, the economic impact, and the stakeholders involved throughout. The systematic literature review showed that although FMT is widely used globally and microbiota therapies are growing, there is not a consistent, widely accepted policy and regulatory approach to stool as a therapeautic treatment. Economic model results showed that the cost effectiveness of CDI and rCDI treatment is largely driven by the efficacy and cost of currently available antibiotics; unless there is a significant decline in antibiotic effectiveness and a significant drop in the cost of FMT, standard antibiotic therapy is the most cost-effective treatment option. Conclusion: FMT and microbiota therapy are promising novel treatment options that will likely become more widely used as understanding of the role of the microbiome in the immune system grows and as the need for non-antibiotic therapies increases. More research is needed on the safety and efficacy of FMT, including unintended and long-term effects, before widespread adoption. The current regulatory framework is not flexible enough to adapt to rapid advances in innovative treatment options, and the risks of declining efficacy of antibiotics in the future necessitates advances in innovative treatment options now.