THE CHANGING LANDSCAPE OF ORPHAN DRUG PROVISION IN THE EU5 AND UNITED STATES

Author(s)

McConkey DDouble Helix Consulting, London, United Kingdom

OBJECTIVES: The global healthcare environment is in a considerable state of flux with many national health systems undergoing some degree of reform. Orphan drugs have historically been funded at a national level through different systems to the rest of the pharmacopeia. With this in mind, the author set out to assess the impact of healthcare reform on how orphan drugs are likely to be provided for in the future. METHODS: This project involved a study of orphan drug provision in the past and more recently, including both primary and secondary research elements. Trends in the changing healthcare market and specific reforms were considered as part of a prospective study on the orphan drug landscape RESULTS:  Orphan drug provision usually utilises a separate infrastructure to manage the allocation of funding, with this system insulated to a degree from general healthcare reform. In countries where rare diseases are treated in specific institutions (e.g. Italy) there is likely to be little or no change in how this is managed. Conversely, in countries where the orphan drug provision is integrated with the healthcare macro-structure, such as in the UK with the former regional Special Commissioning Groups, this will likely change along with structural reforms to the overall system. Finally, in markets where healthcare can be considered predominantly privately funded (e.g. USA), unless there are changes made to legislation, orphan drug provision and funding are unlikely to be significantly affected in the future.  CONCLUSIONS: Despite the issues of tightening budgets, restrictions on prescribing and structural reform, orphan drug provision is unlikely to decrease from current levels, but budgetary constraints may make the future landscape more hostile to drugs for new orphan indications.

Conference/Value in Health Info

2011-05, ISPOR 2011, Baltimore, MD, USA

Value in Health, Vol. 14, No. 3 (May 2011)

Code

PHP41

Topic

Economic Evaluation

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies

Disease

Multiple Diseases

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