Opportunities and barriers for innovation and entrepreneurship in orphan drug development

Olga A Belousova,Aard J Groen,Aniek M Ouendag

doi:10.1016/j.techfore.2020.120333

Olga A Belousova, Aard J Groen + Show 1 more

Open Access

https://doi.org/10.1016/j.techfore.2020.120333

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Abstract

Orphan diseases pose both a challenge to the global medical community and an opportunity for it to focus on global peace engineering and innovation. Where, any single orphan disease is rare, when taken as a whole they affect more than 250 million people throughout the world. This number by comparison is larger than the global number of cancer and AIDS patients. We add to the literature by mapping the available knowledge in the orphan drug development field and exploring the tensions at play for innovation and entrepreneurship in this field. We further add to the literature by providing a framework to review this field based on social systems theory. Our review highlights the gaps in research and proposes a path forward in understanding of and learning from the orphan drug development field.

Highlights

Rare diseases pose a challenge to society on a global scale
As economic activity often serves to solve problems of adaptation, at a macro we looked for articles covering national budget instruments and concerns about national and societal budget impact of rare diseases
We present the main findings along the AGIL model dimensions

Summary

Introduction

Rare diseases pose a challenge to society on a global scale. most of these diseases have a prevalence of less than 10 patients per 1 million inhabitants (Aymé and Hivert, 2011), together they affect at least 250 million people around the world (Grossman et al, 2014). This means that most of the individuals affected by these dis eases are being deprived of basic needs, such as proper diagnosis, treatment, and a cure (Certo and Miller, 2008), offering opportu nities for global peace engineering and innovation For those of the rare diseases, where medical treatment or cure are possible, challenges for developing them include the limited knowledge available for most diseases, the difficulties of generating adequate ef ficacy and safety data in small populations and the risk of financial unsustainability for both developers and health-care systems (Hechtelt Jonker et al, 2020). Orphan drug (OD) development is an area where governments, patient communities, knowledge institutes, and private actors such as foundations and social impact‒oriented enterprises actively work together on socially accep table, sustainable, and desirable innovation to overcome this global challenge

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