The impact of orphan drug policies in treating rare diseases

Abstract

This paper is based on Shalini Weerasooriya's Masters dissertation in Public Health at the University of Sheffield. A literature review was conducted to understand the role that orphan drug policies have played in the development of new treatments for rare diseases. The impacts of the policies were categorised as ‘tangible’ or ‘intangible’ and further synthesis identified 10 main themes such as incentives for investment, criteria for eligibility and assessment of drug applications and further guidance to industry during the drug development cycle. The review concludes that whilst policies have contributed positively towards improving the research and development of orphan drugs it has not exhausted its uses and must now shift its focus to facilitating greater accessibility and affordability of the treatments and that stakeholders are essential to the success of this process. Implications for practice are identified, for example the need to further update and refine the policy with changing demographics and advancing technologies and, in particular, greater collaboration and involvement through, for example, evidence based training programmes is recommended. It is concluded that focus must shift to address the gap between having available drugs and being able to access and afford them. F.J.