As a patient recruitment company, it’s elementary (though not always obvious to the casual observer) that, with extremely few exceptions, we deal only with the hard-to-enroll studies. Further, it’s not out of the ordinary for us to tackle the (seemingly) impossible-to-enroll studies. So, rare diseases are not uncommon to us, being that they often fall into the latter category of recruit-ability. Indeed, BBK Worldwide (BBK) has had a great deal of experience with rare disease studies. And, if there’s such a thing as a therapeutic category that inspires us more than any other, it’s this one.
As such, it being Rare Disease Day®, I wanted to share a few thoughts of inspiration.
First, EURORDIS, the organization that founded Rare Disease Day, has designated that the theme of this year’s Day is Care, in an effort to rally everyone in the rare disease community to “join together for better care.” A noble cause and battle cry, to be sure, and not vainglorious, in my experience, despite the relatively few number of people who stand to benefit. Here’s why:
What’s remarkable today, in contrast to, say, 20 years ago, is how much more easily and confidently we all can both join and, as a result, care. The rare disease communities that EURORDIS and so many others have galvanized, and the many advancements that biopharmaceutical start-ups and giants alike have achieved, have defied the odds. Or, better said, they have beaten the odds by fostering a presence and a steadily growing voice and effort that stands on the verge of (and in some cases has actually achieved) breakthroughs of a lifetime, within a lifetime.
What you might intuitively expect to be the mouse that roared in the cacophony of the vast and vocal healthcare industry, the successes of the rare disease community are instead a clarion call for clinical research and development that is readily looked to.
To be sure, the Internet has played a huge role in this. It has been the molecular bond that has joined so many previously isolated patients, parents, physicians, caregivers, and scientists across distance and language to stir something in the industry’s collective conscious: a sense of not only hope, but also destiny – that treatments are not only possible, but probable and, in some cases imminent.
As I write this, I am in fact flying across the Atlantic Ocean to present and consult at an investigators meeting for a clinical trial of an investigational treatment for a rare disease. In days gone by, I would have checked steamer trunks filled with everything BBK could possibly throw at the problem – not least of which would have been some serious strategies and tactics designed to achieve but the preliminary goal of conveying knowledge of and garnering attention for a disease so rare that potential referring physician specialists had never heard of it, let alone encountered a case.
Here’s what I’m bringing with me today: a well-packed carry on of targeted, proven tactics; inspiration; and confidence in medical professionals, a sponsor, and an industry that have indeed joined together for better care, and that are undaunted by the enrollment odds facing them because, in their lifetime, they have seen the world of rare diseases profoundly change for the better.