Hope for Orphan and Rare Diseases

1. Hope for Orphan and Rare Diseases Orphan diseases are classified as a medical condition affecting less than 200,000 people in the country. Diseases such as Tourette’s syndrome, Lou Gehrig disease, and acromegaly are all rare diseases. There is no question that such medical complications are mostly genetic – the diseases and their symptoms are always uncommon to medical scientists and doctors. And with no major advancements in finding the cure to a lot of these diseases, the problem has become a harrowing struggle for the affected and a daunting ordeal for doctors worldwide. Amalgamate this with inadequate marketing and financial incentives – which show that finding the cure to a rare disease is less significant, the problem will never cease to exist. However, it is safe to say that some of the world’s topmost pharmaceutical companies have taken an interest in developing medications to treat or possibly cure rare diseases. Some Vital Statistics Here are some stats on rare disease research to illuminate the global problem at a much broader level: There are more than 7,000 varying types of rare and orphan diseases and health complications – and more diseases are being discovered every passing day. In the US alone, there are more than 30 million people affected by various rare diseases. If you break this number down – you get 1 affected American out of a 10, this is 10% of the entire country’s population. Europe too has 30 million people living with orphan and rare diseases. According to a global estimate into the issue, it was discovered that more than 350 million are affected on a global scale. As per the report posted by the Kakkis EveryLife Foundation it was revealed that there is no cure or medical intervention or treatment programs for 95% of orphan diseases that are approved by the FDA – which is truly heartbreaking.

2. Rare Disease Clinical Research Network – The INC Research When it comes to streamlined and state of the art research into orphan and rare disease trial and medications, there aren't even a handful of clinical organizations in the world. However, there is one that has been actively indulged. INC Research has played an instrumental role in orphan disease research. The organization has overcome a lot of challenges in its efforts to develop a robust avenue for orphan disease research and over the last decade. It is one of the few clinical organizations that have the experience and the technology required to integrate cutting-edge data management, logistical evaluations and implement a robust strategy. This is something that has helped bring a lot of clinical trial to fruition. One of the best things about their approach to orphan and rare disease research is that they focus on patient experience, which is especially important in the case of medical trials for rare diseases. Partnering with several successful sites and collaborating with the likes of the Society of Clinical Research Sites, which is indeed a big name when it comes to global orphan disease research and medicine – they reached and completed phenomenal milestones.