The economics of Orphan drugs: A PhD Project

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PhD Student

Siobhan Bourke

Funder

Bangor University

Project Summary

Orphan drugs is the term applied to drugs that treat rare conditions. These drugs are expensive to develop and supply due to the small numbers of patients who receive these treatments. As such these therapies rarely adhere to existing cost effectiveness criteria, therefore unless the threshold for acceptable treatment is increased, orphan drugs would struggle to get reimbursed on the NHS. Policies have already been developed to increase the incentives for pharmaceuticals companies to produce Orphan Drugs in both the US and Europe.

While these policies have been successful in increasing the number of Orphan Drug on the market, barriers still remain for patient access to these drugs. There are no consistent policy in the UK to determine whether or not Orphan drugs should adhere to the rigor of traditional cost effectiveness standards or whether or not these drugs should have an alternate process for funding within national health care systems. This project will examine the issues of Orphan Drugs in the following areas:

• Society's preferences for funding orphan drugs.
• The cost of Neuromyelitis Optica (NMO) an auto immune condition.

This study is being funded solely by Bangor University and it is hoped that this study will add to the policy debates for funding rare disease treatments.

For further information, please contact Siobhan Bourke at s.bourke@bangor.ac.uk