4. 54% from the submissions. Bad reimbursement recommendations were made due to a lack of clinical effectiveness (38. 5%), insufficient evidence (30. 8%), multiple reasons (23. 1%), or lack of cost effectiveness/high cost (7. 7%). == Bottom line == The number of DRD submissions to CDR increased since 2013; from 4 to 5 per year between 2004 and 2012, to 10, 9, and 8 in 2013, 2014, and 2015 respectively. More than half of DRD submissions received positive reimbursement recommendation. Poor quality evidence and/or lack of supportive clinical evidence was at least partly responsible for a negative RG14620 reimbursement recommendation in all cases. Although the average cost of DRD treatments was high, high cost was a reason behind a negative reimbursement recommendation in only two (7. 7%) of negative reimbursement RG14620 recommendations. Keywords: Rare diseases; Orphan drugs; Technology evaluation, health; Canada == Background == The pipeline of drugs for rare diseases (DRD) has been increasing as biochemical research has shifted focus from blockbusters to niche drugs [1]. While almost all rare diseases, by definition, affect relatively small populations, the exact number of patients affected and the extent of the severity of the condition used to determine a rare disease is a matter of continuous debate [2]. The regulator in Canada, Wellness Canada, is yet to officially RG14620 choose a definition of a rare disease, although in a 2014 press release, Health Canada defined a rare disease as a life-threatening, seriously debilitating, or serious chronic condition that only affects a very small number of patients (typically less than 5 in 10, 000 persons) [3]. The Canadian Agency for Drugs and Technologies in Wellness (CADTH) is a publicly funded Canadian wellness technology evaluation (HTA) business that evaluates drugs to get reimbursement reasons through its Common Drug Review (CDR) and pan-Canadian Oncology Drug Review [pCODR] processes [4]. Through its admonitory body, the Canadian Drug Expert Committee (CDEC), CADTH offers non-binding reimbursement recommendations to all general public drug plans in Canada except for those in Quebec, which has a separate review process. Overall, CDEC provides reimbursement recommendations under three themes: List, List with clinical criteria or conditions, and do not list. It has been reported that participating drug plans adhere to these recommendations in more than 90% of formulary listings [4]. In 2012, CADTH received a formal mandate from its federal, provincial, and territorial (F/P/T) funders to review DRDs [5]. Prior to 2012, there was no formal distinction given for DRDs and CADTH evaluated such drugs under the same process as non-DRDs. After a formal stakeholder consultation, CADTH chose to keep the evaluation of DRDs under an enhanced CDR process, with more emphasis on engagement of professionals, patient input, and offering opportunities to get manufacturers to engage in dialogue with CADTH earlier in the pre-submission phase [6]. As such, CADTH has Rabbit Polyclonal to GPR37 no formal definition that would make a distinction between DRDs and non-DRDs. This report provides a summary of key characteristics for all DRD submissions that have been filed to get review RG14620 through the CDR process, an analysis of the volume and frequency of DRD submissions, and a detailed examination of the reimbursement recommendations and reasons for reimbursement recommendation that have RG14620 been issued to get DRDs. This will allow us to draw a bigger picture from the fast evolving landscape.