MMS Holdings says a new support scheme will help trial medicines being developed for ultra-rare diseases.
The US contract research organization (CRO) described the program as a free support aimed at advancing research for ultra-rare diseases.
According to MMS the plan is to select patient advocacy groups with demonstrable need which it will “provide complimentary regulatory assistance.”
The CRO added it will establish an advisory board of experts in regulatory strategy and writing as well as other industry thought leaders, and pharma or biotech Sponsor contributors who will be engaged with support of project selection.
MMS founder and chief scientific officer Uma Sharma said “This program will provide a new perspective on radically new technologies and therapies that may never have made it to the licensing stage, and is set to help improve the lives of many.”
The program will make use of MMS’ internal “volunteer hours” system though which staff are made available for pro bono work according to Alan Nicolle, senior director of the CRO’s operations in Europe and South Africa.
“This program was brought to fruition by a colleague who recommended it as a better way to use pre-allocated volunteer hours. People like this who lead change make MMS a best place to work for those with an innovation-first, Sense of Urgency and Leadership (SOUL) mind-set.”
Ultra-rare
In the US a rare disease is defined as an illness that affects fewer than 200,000 people. In the European Union, a rare disease is defined as one affecting fewer than 5 out of 10,000 members of the general population.
In contrast, formal definitions of an ultra-rare disease are few and far between. In the UK, for example, there is no legal definition but in the early 2000s NICE introduced the informal guideline that an ultra-rare disease has a prevalence less than 1 per 50,000 people.
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