English (United Kingdom)

https://curated-unify.zendy.io/wp-json/zendy-region/v1/featured_content/oa?rat=en

https://curated-unify.zendy.io/wp-json/zendy-region/v1/highlighted_journal/

Global: Zendy Plus annual

Presents the access of premium content as premium feature

Premium Content

Presents the keyphrase highlighting as premium feature

Keyphrase Highlighting

Presents the summarisation as premium feature

Summarisation

Insights

Presents the pdf analysis as premium feature

PDF Analysis

Presents the zaia usage as premium feature

ZAIA

Global: Zendy Tools annual

Global: Zendy Free Plan

Global: Zendy Tools monthly

Global: Zendy Plus monthly

Policy decisions related to orphan and ultra-orphan drugs challenge traditional decision-making processes and often frustrate those affected by them. In general, these drugs are associated with significant uncertainties around clinical benefit, 'value for money', affordability, and 'adoption/diffusion', all of which arise from a lack of available high-quality evidence. Increasingly, patients with rare diseases and their families are looking for opportunities to contribute to initiatives aimed at reducing these uncertainties. Therefore, a policy framework for guiding their involvement is needed to optimize the impact of any evidence generated.

Developing a Patient-Directed Policy Framework for Managing Orphan and Ultra-Orphan Drugs Throughout Their Lifecycle