Global Patient Advocacy Lead, Bionical Emas
Bio: Naomi is extremely passionate about ensuring people living with rare conditions gain access to treatments safely and ethically. As a paediatric nurse Naomi spent many years at GOSH London as a senior clinical research nurse caring for rare families on phase 1-3 clinical trials and early access programs. Naomi now leads advocacy at Bionical Emas working closely with our internal team and external patient organisations to ensure everything we do incorporates the patient voice and reduces patient burden.
Learn more about Bionical EMAS by visiting their website.
Q. This will be CamRARE’s 5th summit. What is the best/most impactful/most promising change you’ve seen happen in rare disease progress in the last 5 years?
More Gene Therapies and Early Access Programs happening in more rare conditions.
Q. How has your work impacted rare disease progress over the last 5 years?
Advocating for patients and families supporting and educating them about early access. Providing life-changing medicines through EAPs to rare families all around the world! Hearing the impact of the EAPs is very rewarding and exciting!
Q. What next? What do you want to see happen or change in the next 5 years that will have the most impact on diagnostics, care or treatment for those affected by rare conditions?
More pharma companies offering EAPs and earlier in development, better communication with patient advocacy groups and more safe and efficient medicines becoming available for rare families!