Since the early 80s, incentives have been introduced to stimulate R&D for rare diseases. We develop a theoretical model to study the impact of push and pull incentives on the intensive and extensive margin of optimal R&D investments. The model describes the mechanisms by which the type of incentives provided may favor R&D for orphan diseases with comparatively high prevalence. In our empirical analysis, we merge data on orphan drug designations by the Food and Drug Administration with Orphanet data on disease characteristics. In line with the theoretical results, we find evidence supporting the idea that the incentives adopted may have contributed substantially to widening the gap between more and less rare diseases classified as orphan. Our theoretical and empirical findings together suggest that, if providing some therapeutic option to patients with very rare diseases is a priority, a revision of the current system of incentives should be considered.

R&D and market size : Who benefits from orphan drug legislation? / S. Gamba, L. Magazzini, P. Pertile. - In: JOURNAL OF HEALTH ECONOMICS. - ISSN 0167-6296. - 80(2021 Dec), pp. 102522.1-102522.22. [10.1016/j.jhealeco.2021.102522]

R&D and market size : Who benefits from orphan drug legislation?

S. Gamba;
2021

Abstract

Since the early 80s, incentives have been introduced to stimulate R&D for rare diseases. We develop a theoretical model to study the impact of push and pull incentives on the intensive and extensive margin of optimal R&D investments. The model describes the mechanisms by which the type of incentives provided may favor R&D for orphan diseases with comparatively high prevalence. In our empirical analysis, we merge data on orphan drug designations by the Food and Drug Administration with Orphanet data on disease characteristics. In line with the theoretical results, we find evidence supporting the idea that the incentives adopted may have contributed substantially to widening the gap between more and less rare diseases classified as orphan. Our theoretical and empirical findings together suggest that, if providing some therapeutic option to patients with very rare diseases is a priority, a revision of the current system of incentives should be considered.
Gumbel distribution; Health inequality; Market size; Orphan drug regulation; Pharmaceutical innovation
Settore SECS-P/01 - Economia Politica
Settore SECS-P/03 - Scienza delle Finanze
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/873631
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