Review of regulatory recommendations for orphan drug submissions in the Netherlands and Scotland: focus on the underlying pharmacoeconomic esvaluations

Background: Pharmacoeconomic evaluations of new drug therapies are often required for reimbursement or guidance decisions. However, for orphan drugs, country-specific requirements exist. In the Netherlands, orphan drug developers can be exempted from providing a full pharmacoeconomic evaluation, whereas in Scotland, no such exceptions can be made, although additional modifying factors specific to orphan products may be considered. Objective: The aim of the present work was to identify differences in the outcomes of the recommendations for orphan drugs for rare diseases between 2 European countries: Scotland and the Netherlands. Methods: All orphan drug reports to the Dutch Committee for Pharmaceutical Assistance (Commissie Farmaceutische Hulp [CFH]) and orphan drugs guidance issued by the Scottish Medicines Consortium (SMC) through May 2009 were reviewed from the respective organizations' Web sites. The following were gathered from the pharmacoeconomic analyses and recommendations for reimbursement in the Netherlands or guidance for drug use in Scotland: drug indication, outcome of pharmacoeconomic evaluation, recommendation, and exact date. Results: Dossiers for 38 orphan drugs were submitted to the CFH; 37 were submitted to the SMC. Only 1 submission to the CFH included a full pharmacoeconomic analysis; all other reports included only a cost analysis. Twenty-four submissions to the SMC were accompanied by a full pharmacoeconomic evaluation; no information could be obtained for 4 drugs. The remaining reports either did not include a cost-effectiveness analysis or were deemed insufficient by the SMC. Forty-three percent (10/23) of the cost-utility analyses submitted to the SMC reported an unfavorable outcome of more than 30,000 pound/quality-adjusted life-year (QALY) gained; of these, only 2 (20%) reporting incremental cost-utility ratios of 43.717 pound to 81.000 pound were granted a restricted positive recommendation. The CFH gave positive recommendations for reimbursement for 36 of 38 submissions (95%). Of the 37 orphan drugs submitted to the SMC, 19 (51%) received a positive recommendation for use. Seventy-three percent (8/11) of submissions to the SMC with an unfavorable cost-effectiveness estimate (ie, more than 30,000 [(sic)34,000 or US $48,000] per QALY gained) received a negative recommendation for reimbursement. Conclusions: Ninety-five percent of orphan drug submissions were approved for reimbursement in the Netherlands, compared with 51% in Scotland, during the period of interest. Moreover, cost-effectiveness or cost-utility analyses were included in 24 of 37 submissions in Scotland, compared with only 1 of 38 in the Netherlands. (Clin Ther. 2010;32:1651-1661) (C) 2010 Excerpta Medica Inc