We are a Wellcome Trust funded project, examining the high price of medicines for rare diseases (called 'orphan drugs') and its impact for patients on terms gaining access to relevant treatments in the EU, UK and USA.
Medicines for rare diseases are often expensive to develop and produce due to the small sample sizes of patients with a particular condition in clinical trials. There are also limited potential markets once a medicine has been licensed, making them less commercially attractive. As a result, treatments for patients with a defined rare disease (affects fewer than 1 in 2,000 people) have often seen stifled innovation.
To resolve this, EU, UK, and US legislation defines some treatments for rare diseases as 'orphan drugs'. Once a treatment is designated as an orphan drug, the manufacturer receives various incentives such as tax relief and market exclusivity for set periods of time. Following these regulatory mechanisms, there has been an unprecedented increase in the number of treatments being designated as 'orphan drugs', a process we conceptualise as orphanisation. In examining this process, we address the core research questions:
- To what extent is orphanisation occurring (in the EU, UK, and USA)?
- How is it shaped by different technologies, institutions, and actors?
- What are the implications for industry, health policy, and patients?
The project has four worksteams (WS1-4), each of which addresses a particular aspect of the core research questions:
- Workstream 1 - Chart the industrial development of orphan drugs and associated business strategies
Led by Dr. Jin Ding, Worksteam 1 charts the growing industrial development of orphan drugs and analyse the emergence of new orphan drug business strategies
- Workstream 2 - Map controversies around high orphan drug prices and access
Led by Dr. Matthew S. Hanchard, Workstream 2 seeks to understand the role of different actors in the evolution of orphan drug policy, map the controversy around high prices, and analyse proposal to improve patient access and improve orphan incentives
- Workstream 3 - Patient engagement in drug development and institutional reform
Led by Dr. Eva Hilberg, Worksteam 3 develops a new critical angle that connects forms of patient engagement in drug development as parts of an ongoing dialogue, and also analyses silences and omissions in public debate and processes of institutional reform on pharmaceutical innovation. Furthermore, the workstream also traces the changing use of evidence in initiatives to support drug repurposing.
- Across our workstreams
In drawing together the separate workstreams, Principal Investigator Prof. Paul Martin will assess the implications of orphanisation for health and healthcare systems, and develop proposals for more sustainable models of pharmaceutical innovation. Doing so will serve to develop and evaluate orphanisation as a social science concept.
The research is funded by the Wellcome Trust for five years (2020-2025) under grant reference 219875/Z/19/Z, and based within the iHuman institute at the University of Sheffield (UK). To align with a Wellcome Trust commitment to open access, a majority of our outputs apply a CC-BY public copyright licence, including our dataset and publications.
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