Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium

Orphanet Journal of Rare Diseases

Ciarán Scott¹  Nicolas Sireau¹  Anthony K. Hall²  Mohammed Al-sbou³  Lakshminarayan R. Ranganath⁴  Nicholas P. Rhodes⁴  Christa van Kan⁵  Mattias Rudebeck⁶  Birgitta Olsson⁶ 

[1] AKU Society, CB1 2BL, Cambridge, UK;Cudos, Hoofddorp, The Netherlands;Faculty of Medicine, Mutah University, Karak, Jordan;Liverpool University Hospitals NHS Foundation Trust, Liverpool, UK;PSR Group, Hoofddorp, The Netherlands;Swedish Orphan Biovitrum (Sobi), Stockholm, Sweden;

关键词: Rare diseases;    Drug development;    Alkaptonuria;    AKU;    DevelopAKUre;    Best practice;   

DOI  :  10.1186/s13023-021-02137-0

来源: Springer

【 摘 要 】

New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. Our unique consortium model incorporated several of the identified developments under one project, DevelopAKUre, truly bringing together academia, industry and patient organisations in clinical drug development. We found that the equal partnership between all parties in our consortium was a key success factor creating a momentum based on a strong organisational culture where all partners had high engagement and taking ownership of the entire programme. With an agreed mutual objective, this provided synergies through connecting the strengths of the individual parties. Another key success factor was the central role of the patient organisation within the management team, and their unique study participants’ advocacy role securing the understanding and meeting the needs of the clinical study participants in real-time. This resulted in an accelerated enrolment into the clinical studies with a high retention rate allowing for delivery of the programme with significantly improved timelines. Our project was partly funded through an external EU research grant, enabling our model with equal partnership. Further attention within the community should be given to establishing a functional framework where sustainable funding and risk sharing between private and public organisations allow for our model to be replicated.

【 授权许可】

CC BY