Edition of 04 April 2024

A new article has been published in Expert Opinion on Orphan Drugs, discussing the approval and pricing of orphan drugs in Canada from 2015-2020.

Unlike Europe or the United States, Canada does not offer any incentives to stimulate the development or marketing of drugs for rare diseases. This includes a lack of any type of orphan designation.

In order to understand how rare disease drugs are approved and priced in Canada in the absence of specialised regulatory pathways, the authors identified drugs with orphan designation from the EMA and/or FDA which were also authorised in Canada. They then assessed the time required for processes such as health technology assessment (HTA), price negotiation, and listing in government drug plans.

Overall, the median times for HTAs and price negotiations suggested an approximate delay of one year; however, the delay between marketing authorisation and price negotiation completion averaged over 18 months. This demonstrates a significant disparity between Canada and the United States and some western European countries in timely access to innovative treatments. Problems of equity are also caused by the close ties between listing of orphan drugs in Canadian government drug plans and outcomes of price negotiations and reimbursement recommendations.

In order to address these issues, the authors recommend a comprehensive Canadian orphan drug policy. This strategy would improve Canadians’ access to emerging and innovative therapies, and would also make Canada a more desirable market for drug developers.

An article has been published in the Orphanet Journal of Rare Diseases presenting a pilot study of a telehealth intervention for improving peer relationships among adolescents with neurofibromatosis type 1 (NF1). Based on the outcomes of the study, the programme appears to be effective at improving social skills and knowledge.

NF1 is a rare neurogenetic condition which is associated with specific physical characteristics such as café-au-lait spots and skeletal abnormalities. Many individuals with this disorder report social difficulties, however there is little research on psychosocial interventions to help address these challenges.

In response to this gap, researchers adapted an existing programme which has been validated to address the development of social skills and peer relationships in individuals with autism spectrum disorder and other neurodevelopmental conditions. Among these groups, the Program for the Education and Enrichment of Relational Skills (PEERS®) has been shown to improve social knowledge, responsiveness, skills, and frequency of get-togethers; all of these are particular concerns for people living with NF1.

In this study, the authors delivered the 14-week PEERS® curriculum virtually to 19 adolescents living with NF1 in order to assess its effectiveness for this specific condition. They found that even in the context of mild social difficulties, the programme was successful at improving social functioning among participants. This not only provides evidence for the usefulness of PEERS® as an intervention in NF1, but also demonstrates the potential of telehealth-based interventions in reaching populations for whom face-to-face interventions are not feasible.