Setti Rais Ali
Chaire hospinnomics – Adresse postale : Hospinnomics - AP-HP, 1, parvis Notre-Dame 75004 Paris
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Travaux en cours
Raïs Ali Setti, Does the Orphan Drug Legislation foster R&D on rare diseases?
The Orphan Drug legislation was introduced in 2000 at the European level and offers supply-side market incentives to stimulate R&D investments in rare diseases arenas. We study the impact of the Orphan Drug legislation by examining the variation in the number of new clinical trials and new academic publications from 1997 to 2015 using a Difference-in-Differences design estimated with a Conditional Fixed-Effect Poisson Model. We find a causal positive impact on the number of clinical trials from 2004 that increases over the 10 years following the legislation. The increase in the number of academic publications following the policy introduction is immediate and persistent.
Keywords: R&D; Policy Evaluation; Pharmaceutical Regulation; Rare Diseases; Orphan Drugs
Raïs Ali Setti, Tubeuf Sandy. (In)equality of opportunity in the allocation of R&D resources for rare diseases
There have been considerable discussions in the philosophical and political economy literature about the role of the welfare state in promoting equity in the provision of certain goods and services. The regulation schemes in pharmaceutical markets impact directly the equity in the distribution of R&D investments and indirectly health status disparities, and treatment and care opportunities for patients with rare diseases. The objectives of this paper are twofold. Firstly, it investigates the level of inequality of opportunity in the allocation of R&D resources within rare diseases arenas. Secondly, it identifies the disease characteristics that appear to lead R&D investments for rare diseases. We assess the inequality in the distribution of R&D investments of pharmaceutical firms according to a number of disease characteristics using non-parametric methods based on cumulative distribution functions, and then complement our approach measuring the level of inequality at disease-level in academic research intensity, number of clinical trials, orphan designations and marketed drugs.
Keywords: R&D; Equality of opportunity; FOSD; Rare Diseases; Orphan Drug