Access to Chagas disease treatment in the United States after the regulatory approval of benznidazole

Approximately 300,000 persons in the United States (US) are infected with Trypanosoma cruzi, the protozoan that causes Chagas disease, but less than 1% are estimated to have received antiparasitic treatment. Benznidazole was approved by the US Food and Drug Administration (FDA) for treatment of T. cruzi infection in 2017 and commercialized in May 2018. This paper analyzes factors that affect access to benznidazole following commercialization and suggests directions for future actions to expand access. We applied an access framework to identify barriers, facilitators, and key actors that influence the ability of people with Chagas disease to receive appropriate treatment with benznidazole. Data were collected from the published literature, key informants, and commercial databases. We found that the mean number of persons who obtained benznidazole increased from just under 5 when distributed by the CDC to 13 per month after the commercial launch (from May 2018 to February 2019). Nine key barriers to access were identified: lack of multi-sector coordination, failure of health care providers to use a specific order form, lack of an emergency delivery system, high medical costs for uninsured patients, narrow indications for use of benznidazole, lack of treatment guidelines, limited number of qualified treaters, difficulties for patients to make medical appointments, and inadequate evaluation by providers to determine eligibility for treatment. Our analysis shows that access to benznidazole is still limited after FDA approval. We suggest six areas for strategic action for the pharmaceutical company that markets benznidazole and its allied private foundation to expand access to benznidazole in the US. In addition, we recommend expanding the existing researcher-clinician network by including government agencies, companies and others. This paper’s approach could be applied to access programs for benznidazole in other countries or for other health products that target neglected populations throughout the world.Author summary: Less than 1% of persons in the United States with Trypanosoma cruzi infection, the cause of Chagas disease, have received antiparasitic treatment. In this paper, we examine the status of access to benznidazole in the United States following its approval by the FDA for treatment of Chagas disease treatment in 2017 and its commercialization in 2018. We found that the average number of patients who received benznidazole from the manufacturer increased after commercialization from just under 5 (when distributed by the CDC) to 13 patients (when sold in commercial channels) per month. We identified various barriers to access to treatment such as problems in the drug ordering process, low demand from health care providers, inadequate clinical assessment of patient eligibility for treatment, obstacles for patients to make medical appointments or to pay medical costs, and weak coordination among key actors in the health system. To overcome these access barriers, we propose strategic actions that can be taken by the pharmaceutical producer in collaboration with other key actors. Our work contributes to expanding access to Chagas disease treatment in the United States, and our approach could be applied to efforts to expand access to health products for other neglected diseases.