Levers & barriers for orphan drug business development: a systematic literature review

While orphan drug regulations have proven to be an effective tool in stimulating the development of rare disease therapies (de Vrueh, 2014), challenges still remain. Correct diagnosis of a rare disease is often delayed by many years because of lack of physicians and healthcare providers with relevant clinical training and experience in recognizing and treating these disorders, and the few treatment options available are difficult to access and costly (Benjamin et al., 2017; Grossman et al., 2014). At the same time, companies, like Genzyme and Shire, proved that orphan drugs can be the basis of a viable business model (Boon & Broekgaarden, 2013). As the number of orphan drugs is rising, and they are predicted to account for over 20% of all prescription drugs by 2020 (Benjamin et al., 2017), this calls for a better understanding of the situation: are there opportunities that we are missing? The purpose of this poster is to present the results of a review of existing theory and practice concerning the business development of rare disease drugs. We structure our review using the neo-functionalist framework of Groen (2005) which suggests that for a system to be viable, it has to maintain four core functions: goal attainment, pattern maintenance, social network interaction and optimization of gratification. With this approach we analyze strategic, organizational, and economic issues in a network perspective of the orphan drug development processes highlighting the areas of market and government potential and failure in generating solutions for rare diseases. Our search reveals multiple strategic challenges, including the decision making (evaluation frameworks, approval procedures across the globe) and prioritization issues at societal and regulatory levels, but also opportunities linked to harmonization of the approval processes and emergence of new technologies. The economics of orphan drugs is probably one of the most discussed issues. However, it is dominated by discussions on cost-efficiency and reimbursement decisions across countries. Much less attention is devoted to successful business models available in the field, for example, models oriented on repurposing of existing drugs, or the role of non-profits. We systematize the best practices here. At pattern (safety and quality) maintenance level, much work has been done in analyzing the regulatory frameworks (Orphan Drug Act and similar statutes), comparing them across countries and over time, providing the needed understanding of the avenues for their modernization. Recently, also a body of works on best practices for clinical trials (e.g. avoiding parallel trajectories that may hinder the speed of drug development) and sustainable orphan drug development began to emerge. This is a notable move towards a better aligned collaboration in the field. At network level we observe the how and who of multiple stakeholders constituting the ecosystem for the orphan drug development: co-developing clinical trials, building communications, aligning expectations and learning together about the new technological possibilities. In contrast, we also highlight where and how information disconnects hurt knowledge exchange and research development, thus leading to missed opportunities in drug development. With this poster we hope to raise attention about and help with accessing the available best practices in working together towards finding opportunities for orphan drug cost efficient development.