1234. Increasing private sector investment in neglected tropical disease (NTD) research and development: a mixed methods study

BACKGROUND: This study explores the drug development environment for Human African Trypanosomiasis and Chagas disease and investigates ways to stimulate greater investment in the small molecule drug pipeline for NTDs. METHODS: We conducted qualitative case studies of successful public-private partnerships (PPPs) that have supported the research pipeline for treatment of HAT (Fexinidazole & Acoziborole) & Chagas disease (Benznidazole). We conducted semi-structured interviews with 21 key informants. We then performed an economic modeling analysis to evaluate the development costs compared to estimates of potential revenue in order to identify expected private investment returns, inclusive of the contribution of the Priority Review Voucher (PRV) program. We also investigate under what circumstances one can expect positive returns if R&D for NTDs was executed by a private biopharmaceutical firm. We calculate the net present value of R&D costs both for an average NTD drug and use the specific features of these three candidates to estimate the private ROI that would have prevailed for each drug had they been developed by a for-profit entity. RESULTS: The major themes that emerged from the case studies were: (1) the importance of pre-existing philanthropic efforts for a given disease, (2) the desire to align the social goals of the company with its actions, (3) the vital role of PPPs in coordinating development and reducing private-sector risk & (4) the limited role that the PRV played in motivating these activities. Overall, the current NTD R&D landscape is driven by PPPs. The modeling analysis shows why: although the PRV is an important incentive for drug development, it is not sufficient to generate a positive private return for novel drug development when adjusting for capitalized costs and failure risk. However, for a pre-existing, unapproved drug such as Benzinadozole, returns are positive in the current environment (Table 1). We find that both a restricted PRV supply & better estimates of approval probabilities would generate positive returns (Fig 1 + 2). Table 1: Return on investment by small molecule drug [Image: see text] Figure 1. Return on Investment Sensitivity Analysis for Fexinidazole (Source: Authors’ calculations) [Image: see text] Figure 2. Return on Investment Sensitivity Analysis for Acoziborole (Source: Authors’ calculations) [Image: see text] CONCLUSION: We find support for NTD drug development within the private sector, but no novel R&D without nonprofit stewardship. Our findings intend to foster PPPs that stimulate this pipeline from very low current levels. DISCLOSURES: All Authors: No reported disclosures