- Of the 7000 identified rare diseases, 50% affect children.
- From this group, 30% of these children die before their 5thbirthday
- Current drug development process takes an average of 14 years and 2 billion dollars to develop a new therapeutic
- In addition, on average it takes 7.6 years in the U.S. and 5.6 years in the UK on average for correct diagnosis. During this time, a patient will be misdiagnosed 2-3 times and sees four different physicians and four different specialists.
- The time it takes to develop a new drug or even the time it takes to accurately diagnose a child is unacceptable which is driving the need to reassess the process and find faster routes from diagnosis to therapeutic options for children.
- We believe our platform will enable information and data sharing across stakeholders and organizations fostering collaboration which builds efficiency and speed to finding a therapy.
- There is such a large unmet need to support patient families and provide resources that bridge the gap to navigating the health care system in rare diseases- more and more foundations are coming to us to utilize the resources so they can move forward in finding a cure for their children when no one else is researching on the disease.
- Non- profit research organization focused on accelerating identification of more immediate therapeutic solutions for kids with rare disease potentially through repurposing currently approved drugs.
- We are unique as we work directly with patient families to empower them with tools to own their data and build patient communities in a way that research and clinical information is captured that can be acted on in an efficient way – addressing the urgency needed.
- The team is made up of scientists and executives that have worked in Pharmaceutical industry but have come together, volunteering their time to drive a new approach that addresses the urgency needed for children.
- The resources are provided through the RARE Hub which is a neutral common computational platform that links all stakeholders – the patient family/foundation, research and clinical to close the gap of time between discovery and actual clinical application.
- Our clinical activities are provided by our partner Agility clinical, together we help families unite patient communities of critical mass and conduct clinical trials. Agility Clinical is the first orphan drug focused clinical trial services organization in the US. Due to experience and innovation in small trial design the organization has become known for successful Orphan/Rare clinical trial design and execution.
- Although we are all volunteers, we do need to raise funding for supporting families and patient communities, the research and clinical activities, and getting new therapies to kids no matter where they are in the world. Help support RARE Science rare disease programs Donate Now
We have a RARE approach of empowering patient families and foundations with the tools/resources they need to drive forward finding a cure. In parallel we assist in building a collaborative strategy across all stakeholder communities by using a neutral common data portal called the RARE Hub.
We have a simple 3 step process:
- Unite the patient community through building registries rich in patient reported information, research and clinical data.
- Unite research and clinical communities specializing in the disease area.
- Unite all stakeholders through activities that foster collaboration building efficiencies to accelerate clinical trials with our partner Agility Clinical, an expert in international rare clinical trials
The tools available through the RARE Hub will enable data sharing across the different stakeholders. In addition, at all times the end goal – a therapy – is the driving force for the implemented strategy focusing on safety, efficacy and the urgency of time.