Evidence and values: requirements for public reimbursement of drugs for rare diseases--a case study in oncology.

INTRODUCTION: Doubts have been expressed about whether standard methods of health technology assessment are suitable for the evaluation of drugs for rare diseases. Under conditions of rarity, it may be more difficult to conduct large randomized trials in order to gather adequate evidence on efficacy, and the standard methods of economic evaluation may not adequately reflect societal preferences for the treatment of serious and/or …