Patient engagement
17 April 2023

How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis

Orphanet J Rare Dis. 2023 Apr 17;18(1):84. doi: 10.1186/s13023-023-02682-w. PMID: 37069597; PMCID: PMC10108537. How do patients and other members of the public engage with the orphan […]
17 April 2023

“I just wanted to speak to someone- and there was no one…”: using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients

Orphanet J Rare Dis. 2023 Apr 17;18(1):86. doi: 10.1186/s13023-023-02680-y. PMID: 37069697; PMCID: PMC10111696. “I just wanted to speak to someone- and there was no one…”: using […]
1 September 2023

Composite endpoints, including patient reported outcomes, in rare diseases

Orphanet J Rare Dis. 2023 Sep 1;18(1):262. doi: 10.1186/s13023-023-02819-x. PMID: 37658423; PMCID: PMC10474650. Composite endpoints, including patient reported outcomes, in rare diseases Authors: Johan Verbeeck 1, Maya Dirani 2, Johann […]
17 October 2023

Parental Intervention Program for Preschool children with Rare Diseases – a mixed methods evaluation of parents’ experiences and utility

Orphanet J Rare Dis. 2023 Oct 17;18(1):327. doi: 10.1186/s13023-023-02935-8. PMID: 37848938. Parental Intervention Program for Preschool children with Rare Diseases – a mixed methods evaluation of […]
17 October 2023

Patient pathways for rare diseases in Europe: ataxia as an example

Orphanet J Rare Dis 18, 328 (2023). https://doi.org/10.1186/s13023-023-02907-y Patient pathways for rare diseases in Europe: ataxia as an example Authors: Julie Vallortigara1, Julie Greenfield2, Barry Hunt2, Deborah […]
13 January 2024

The future of the global clinical trial ecosystem: a vision from the first WHO Global Clinical Trials Forum

Lancet. 2024 Jan 13;403(10422):124-126. doi: 10.1016/S0140-6736(23)02798-8. Epub 2023 Dec 18. PMID: 38128557. The future of the global clinical trial ecosystem: a vision from the first WHO […]