Stockholm, 28 February 2018 – Leading drug development consultancy acknowledges the extraordinary contributions of patients and patient organisations around the world on global Rare Disease Day.

For 20 years the NDA Group has supported pharmaceutical and biotech companies around the world with strategic regulatory guidance across a range of diseases. Increasingly more companies have been trying to solve the puzzles for a diversity of untreated rare diseases. NDA has been there every step of the way.

Over the years NDA has been involved in the development of more than 50 products for individuals with rare diseases and several of the key breakthroughs have been supported by the company.

Johan Strömquist, CEO of NDA Group:
“At NDA we take great pride in helping companies address large unmet medical need. The need is most significant when it comes to rare diseases.”

Strömquist continues:

“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.”

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. Markku Toivonen, MD, Scientific Director at NDA and former Chairman of EMA’s Scientific Advice Working party and former CHMP member comments:

“The development of patient advocacy and the involvement of patients in the development and regulatory process has truly been game changing. At many levels the experience of the patient brings perspectives that no amount of objective, scientific scrutiny can bring to the process. At NDA we take great pride in helping to empower patient advocates through training in regulatory science and medical research with Eurordis, the European Organisation for Rare Diseases. ”

At NDA, the process to support the development of novel treatments for rare disease continues. Dr. Laurie Smaldone Alsup, MD, CSO and COO 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.”

The Author

Johan Strömquist


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