OBJECTIVES: Within the EU, pharmaceutical products in development may apply for an orphan product designation if the product can demonstrate potential value in a rare disease indication. Not all products used in rare diseases apply for or receive such a designation. The research question posed was whether gaining an orphan drug designation in the EU is indicative of a rare disease product achieving greater market access success.    METHODS: Products receiving an EU Marketing Authorisation (MA) between 2009-2011 with an orphan designation were compared with those that were approved for at least one indication of use in a rare disease.  Indicative markers of market access success were reviewed, including outputs of product evaluation recommendations from the Scottish Medicines Consortium and HAS in France. Additionally, 16 payers in 4 European countries were interviewed for qualitative insights on how an orphan designation would impact on their evaluation of price.   RESULTS: Fourteen (14) products with orphan-product designation received an EU MA during 2009-2011. Twenty (20) products without orphan-designation but with at least one indication for a rare disease received an EU MA during 2009-2011.  Forty two (42)% of the orphan designated products gaining an EU MA received  a recommendation for use in Scotland, with 67% of non-orphan designated products indicated for a rare disease receiving a recommendation for use. Ninety two (92)% of the orphan designated products were included on the reimbursement list in France, with 89% of non-orphan designated products included on the reimbursement list. Payers indicated that orphan drug status is methodologically difficult to consider in relation to price, but it may have other relevant market access benefits.    CONCLUSIONS: For products with an MA for treating rare diseases, the analysis performed concluded that products receiving an EU orphan drug designation did not demonstrate greater market access success in the selected EU countries using the applied criteria.