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

• Published in: PLoS neglected tropical diseases 2020-06, Vol.14 (6), p.e0008398
• Main Authors: Yoshioka, Kota, Manne-Goehler, Jennifer, Maguire, James H, Reich, Michael R
• Format: Article
• Language: English
• Subjects: Access; Adaptation; Age Factors; Antiparasitic agents; Benznidazole; Biology and Life Sciences; Centers for Disease Control and Prevention, U.S; Chagas disease; Chagas Disease - drug therapy; Commercialization; Diseases; Distribution; Drug Costs; Drug therapy; Drugs, Investigational; Emergency medical services; Evaluation; FDA approval; Government agencies; Health care; Health care expenditures; Health care industry; Health care services accessibility; Health services; Humans; International organizations; Marketing; Medical treatment; Medicine and Health Sciences; Nitroimidazoles - economics; Nitroimidazoles - supply & distribution; Nitroimidazoles - therapeutic use; Parasitic diseases; Patients; People and Places; Pharmaceutical industry; Protozoa; Public health; Regulatory agencies; Regulatory approval; Social Sciences; Supervision; Tropical diseases; Trypanocidal Agents - economics; Trypanocidal Agents - supply & distribution; Trypanocidal Agents - therapeutic use; Trypanosoma cruzi; United States; United States Food and Drug Administration; Vector-borne diseases; Womens health
• ISSN: 1935-2735; 1935-2727; 1935-2735
• DOI: 10.1371/journal.pntd.0008398

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.