Monday the 28th of February 2022 is Rare disease Day – the day when we raise awareness for rare diseases, generating change for the 300 million people worldwide living with a rare disease, their families, and carers.
The expansion and increased involvement of patients in the drug development process is one of the key factors that have stimulated the development in the rare disease space over the last few years.
Dr Patrick Salmon, MD, Advisory Board Member at NDA and former CHMP and COMP member comments:
“ While working as a regulator and a member of the COMP, I was invited by Eurordis, the European Organisation for Rare Diseases, to contribute to training of patient advocates in the Eurordis Summer Schools. Patient advocates were new to me, but I was overwhelmed by their enthusiasm and desire to understand the regulatory process. I was lucky enough to participate in several summer schools and some of my presentations are still on the Eurordis website. Patient participation in and contribution to drug development and assessment are now important and an expected part of the regulatory process ”.
At NDA, the process to support the development of novel treatments for rare diseases continues.
Dr Laurie Smaldone Alsup, MD, CMO / CSO of NDA commented:
“ We are driven by our passion and commitment to bringing new treatments to people in need. The rare disease landscape is still a vast area of unmet medical need with few options for many serious and life-threatening conditions. We continue to provide leadership in solutions for diseases without regulatory precedent staying at the forefront of novel product development by guiding industry through the regulatory hurdles. Importantly we help to find and listen to the patient voice that has been a crucial contributor to the understanding of these rare conditions.”
Smaldone Alsup continues:
“ I believe we are still at the beginning of addressing rare diseases. With new scientific and technical advances every day we will be turning a corner where many more novel treatments will be discovered to address a range of difficult to treat disorders. We are poised and ready to support these innovators and help enable treatments to reach people in need around the world.”
Johan Strömquist, CEO of NDA Group:
“ At NDA we take great pride in helping companies address large unmet medical need. The need is especially pressing when it comes to rare diseases. ”
“Through the experience and knowledge of our regulatory scientists and clinical experts from agencies and the industry we feel privileged to have made a significant difference for thousands of patients living with rare diseases previously considered impossible to treat.”
For 25 years the NDA Group has supported pharmaceutical and biotech companies around the world who are making great progress in the fight against rare diseases. Providing strategic regulatory guidance across a range of indications.
Developing medicines to treat rare diseases presents scientific and regulatory challenges and over the years NDA has been involved in the successful development of more than 80 products treating patients with an unmet need.