Cure HHT wins inaugural Prix Galien Patient First Award
A patient-led initiative for rare disease hereditary haemorrhagic telangiectasia (HHT) has won the inaugural Prix Galien Patient First Award, which recognises projects that "meaningfully integrate patient voices into research, clinical trials, and improvements in care."
Patient advocacy organisation Cure HHT claimed the prize for its "Nothing About HHT Without Us: A Patient-Governed Evidence Engine for a Disease with No Approved Therapy" campaign, which was supported by Bristol Myers Squibb. The initiative was one of 96 nominations and nine finalists in the category.
In its first year, the Patient First Award was granted to the winning nominee in honour of Michael J. Fox, the actor, author, and patient advocate who launched the Michael J. Fox Foundation in 2000 after publicly disclosing his diagnosis with Parkinson's disease.
"This award belongs to the entire HHT community," said Marianne Clancy, chief executive of Cure HHT. "It belongs to the patients and families who share their experiences, the researchers and clinicians who pursue better answers, and the partners and donors who invest in what comes next."
People living with HHT have blood vessels that have not developed properly, known as arteriovenous malformations (AVMs), and suffer symptoms like regular and persistent nosebleeds, red spots on the body, and anaemia. In some cases, the AVMs can form in organs like the lungs, liver, and brain, leading to low blood oxygen levels and even more serious complications like seizures and strokes.
While there is still no approved therapy for HHT, there is hope that an option may be on the horizon after Swiss biotech Vaderis Therapeutics' AKT inhibitor engasertib showed promising efficacy in a phase 2 trial.
The Patient First Award celebrates initiatives that are led by patient organisations or embed patient voices directly into clinical trial design, recruitment, and execution, integrate lived experience into research priorities, protocols, and outcome measures, and advance patient-centred quality improvement in real-world care delivery.
Other key considerations are fostering transparency, equity, and trust in patient partnerships, and helping to translate research into meaningful improvements in patient outcomes and quality of life, according to the Galien Foundation.
Cure HHT said its campaign "highlights a conviction that has shaped the organisation's work: people fuel progress. The experiences of individuals and families affected by HHT help identify the questions that matter, guide research priorities, and build the evidence needed to move toward better treatments and a cure."
The other winners of this year's Prix Galien in the international category were:
- Daiichi Sankyo and AstraZeneca's HER2-directed antibody-drug conjugate Enhertu (trastuzumab deruxtecan) and Regeneron and Sanofi's IL-4 and IL-13 inhibitor Dupixent (dupilumab) in the Best Biotechnology Product category;
- Evaxion's AI-Immunology platform, which harnesses the power of AI to decode the human immune system and develop novel drug candidates, in the Best Digital Health Solution category;
- MSD's cancer immunotherapy Keytruda (pembrolizumab) and Vertex Pharma's ickle cell disease and beta-thalassaemia gene therapy Casgevy (exagamglogene autotemcel) in the Best Pharmaceutical Product category;
- Immunocore's Kimmtrak (tebentafusp), a treatment for uveal melanoma, in the Best Product for Rare/Orphan Diseases category;
- Edwards Lifesciences' Evoque Transcatheter Tricuspid Valve Replacement System in the Best Medical Technology category; and
- Dren Bio, a developer of therapies for rare blood cancers and other diseases, in the Best Startup category.
