Orphan drugs: high prices, access to medicines and the transformation of biopharmaceutical innovation

‘Orphan drugs’ are a class of medicines that treat rare diseases. They are growing rapidly and they are highly profitable, attracting massive investment from the biopharmaceutical industry and stimulating new business models based on high cost products for niche markets. The very high price of some orphan drugs has led several countries, including the UK, to refuse to pay for them. This has provoked controversy and growing political concern that patients are being denied access to life-saving therapies due to their price. 

A major international debate on how best to improve access is ongoing. This project is innovative in developing a new concept, ‘orphanisation’, to understand the technical, political and regulatory changes associated with orphan drugs. It will ask to what extent is orphanisation occurring in the UK and USA, how is it being shaped by different technologies, institutions and stakeholders, and what are the implications for industry, health policy and patients?