Rare disease day 2017

Are we moving fast enough? ARG talks rare disease clinical trials

By Melissa Fassbender contact

- Last updated on GMT

ARG talks rare disease clinical trials

Related tags: Rare disease

Researchers and patients have more efficient tools for sharing information on rare diseases, and as knowledge continues to spread, rare disease studies will become less rare.

To learn more about rare disease clinical trials, Outsourcing-Pharma.com talked with John Boland, Vice President of Product Development, Atlantic Research Group.

What challenges do rare disease clinical trials pose?

The challenges with rare disease research are many and aren't new, the solutions can just be harder to find. The development challenges don't come solely from there being fewer patients, in many cases the biggest challenge can be that the disease hasn't been extensively studied and understood. 

What unique capabilities does a rare disease CRO need to have?

Flexibility, adaptability, and most importantly engaging a group that possesses creativity and a learning mindset.

View: Infographic: Rare disease research

No two development programs are the same in rare disease and, as a sponsor, you're often "trailblazing," so it is key to have a team who has been through similar challenges before and can translate and apply their learnings to your development program.

How has the demand for rare disease clinical trials evolved over the past five years?

Since the turn of the century, we have seen an increasing trend in the number of orphan-designated products. The FDA Law Blog reported​ FDA received 582 orphan applications in 2016, the most ever.

This trend will continue as scientific and technological advances enable the industry to better utilize mechanisms such as the Orphan Drug Act.

Further, the rise of the Internet and social media​ have given researchers and patients more efficient tools​ for gathering and sharing information, as well as a platform to raise awareness.

Based on the current state of the industry, what do you expect in five years?

Rare disease studies will become less rare ... More companies will utilize orphan/rare pathways to develop more and better therapies for rare conditions where previous market conditions made these advances prohibitive.

Our knowledge base will continue to expand as researchers across the globe are able to collaborate and share data more easily through technology. Patient voices will be heard as advocacy groups continue to refine their use of social media and other platforms.

All of these factors will bring industry, researchers, regulators and the patient community together with a shared goal of easing or eliminating the suffering of patients brought on by these rare diseases.

Why are initiatives, like rare disease day, important to raise awareness?

Knowledge and understanding are the first steps to overcoming any challenge – rare diseases included. The greater the awareness for rare diseases the faster we can development treatment to ameliorate them.

What keeps you up at night?

Are we moving fast enough to help those with few to no options for treatment?

(Feature image: iStock/sudok1)

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