Hope is perhaps the one thing that 30 million Americans living with a rare disease have to hold on to. On average, it takes more than 7 years and several specialists before a diagnosis is discovered for those who have a rare disease – if one is found at all. Limited resources and funding, insufficient information, little evidence-based medicine, and no treatment or cure remain an all-too-frequent proposition.
The rare-disease world is not for the faint of heart. Rather, it is for those with a deep-rooted sense of commitment, those willing to share their experiences to benefit others, and those that have learned to doggedly pursue the scientific advances that will someday bring new hope to those who wait.
But until a cure or treatment can be found, hope remains the bedrock for those living with rare diseases. It comes in the increased effort and the accelerated progress that we have seen in recent years. The number of submissions, designations and approvals are rising, with more FDA approvals than ever before. Nonprofit agencies and advocacy groups bring rare diseases to the forefront by raising money and awareness, disseminating information, and providing an invaluable connection to thought leaders in this space. Pharmaceutical and biotechnology companies both large and small are researching the field of rare diseases and advancing science with the hope of finding effective treatments or, ultimately, cures. Additionally, it is medical communications companies that enhance patient access to optimal medical treatment by informing, educating, and demonstrating the value of new innovations and perspectives to healthcare professional (HCP) stakeholders.
Download this whitepaper to find out how ClinicalMind is working to bring a first-in-class orphan drug to market >>