According to a story from Research & Development, increasing collaboration between a variety of different organizations is helping to propel and improve the develop of new drugs for the treatment of rare disease. There are around 7,000 known diseases that are considered rare, and while many of them may only directly impact only a few thousand individuals, the global impact of these diseases as a whole is massive. Anywhere from 25 to 35 million people at any one time are living with a rare disease. Clearly, there is an urgent need to develop new therapies for them.
Difficult Development
Often rare disease drug development requires certain specifications and changes to the development process that pertain to certain aspects of the rare disease in question. An example of this includes patient segmentation, an approach that allow for more targeted clinical trials that take into account specific characteristics based on disease research. However, the patient population for many rare diseases is limited, and many rare disease clinical trials are small. Segmentation can also exacerbate this difficulty.
In Depth Research Necessary
A lot of preclinical research data is necessary before clinical trials can begin, and rare disease research models, usually used with mice, are a vital part of the preclinical research process. However, this also presents a challenge because it can be difficult for an accurate model to be created. As a result, there can be wide variance in how useful a mouse model for a rare disease actually is.
The Power of Partnerships
All of these challenges mean that it is really difficult for a one organization or company to undertake all of the research and preparation on their own. This is why drug developers have increasingly begun to partner with other organizations throughout this process. Partners can include universities, non-profit foundations and organizations, other players in the industry, and research groups. When working together, these entities can more effectively conduct rare disease research, find the funding necessary to do it, and ultimately develop a successful therapy.
Perhaps one of the most vital projects for these collaborators is the development of improve research models. At the end of the day, models that more accurately replicate the internal mechanism and the signs and symptoms of a disease are going to be the ones that work the best and result in the more useful data.
