A rare disease patient, once a negligible habitant hiding in the dark, has now become more comfortable to portray his story, and is now cherished by the empathy of the general community. Technological advancements accompanied by the popularity and impact of social network, the discussion around orphan disease had been made more common. Generation Y, and Z use social media platforms to advocate and share the stories of patients with rare diseases to attract more support and positive recognition from their social peers. The acceptance of patients with incapacitating conditions, followed by the goodwill and social support are empowering rare disease patients to strive to have a life as normal as possible. As more opportunities open for rare disease patients to live normal lives, more awareness and acceptance of orphan disease will raise. Looming on a rare debilitating condition is not the right way to tackle the problem, as it paints it in dark colors. Alternatively, it is best to give the affected people hope and draw from heroes power and hope for a better life is the right way to play it. Orphan patients’ caregivers devote their social and personal life to take care of their beloved ones. If this journey can be accompanied by a bigger pool of people e.g., employers, healthcare professionals and non-profit organization in running initiative to support rare disease patients. This activity can relieve and give hope to this vulnerable community in their challenging journey. Engaging communities through advocacy groups distribution in various geographic location. These groups can circle ideas and activities that might illuminate the opportunities that could give hope and improve rare patients and caregivers’ motivation globally. Empowering the second and third circle of orphan disease patients, is just as important important than treating the patient themselves.

The caregivers are the one that devote their time for providing support and treatments to their beloved ones. They shouldn’t be kept behind in the orphan disease patient journey. They should source their power from an organized global recognition body supporting them in their challenging day to day activities. Governments need to take a more active role in advocating orphan drugs, conditions and success stories to fuel the hope for the next pool of patients that unfortunately will follow, and to enhance the social rare disease awareness. Governments need to recognize orphan disease as a state mission by embracing country/geographic disease programs, to address the country high prevalence rare condition. In Israel, for reference, one would expect the country to invest heavily in Gaucher orphan disease because of the high prevalence of this debilitating condition in Ashkenazi Jews and initiate state programs to provide a viable solution. I strongly believe that if every country would zoom in on their high prevalence and unmet needs, they can have more funds allocation, support, and success in providing outbreaking solutions. It is always easier to engage people to support communities they know and understand than remote communities, the same as diseases, hence a more disease geographic oriental approach should be applied. An important topic that unfortunately has not been addressed and mainly been left behind is supporting weak economies, especially in the context of rare diseases. Without the right diagnostic tools and awareness, developing countries won’t be able to diminish the quantity of their rare disease patients’ pools. For reference, genetic blood testing for pregnant women and population at risk, can prevent the outcome of childbirth suffering from rare disease because of genetic disorder that could be detected by simple blood test. Focusing on vulnerable communities and tribes holding a high chance of having a newborn baby suffering from orphan disease because of his parents’ predictable genetic loads, are in the center of the prevention scheme. Tribes in the sub-Saharan area and Africa have 10-fold more chances to give birth to babies suffering from sickle cell anemia disease than in the global population.

There are approximately 20M people suffering from Sickle Cell Anemia where 80-90% of them are in Africa and the sub-Saharan area, where the mortality rate of children below 5 years is between 50-80%. Those numbers are hard to adjust, nevertheless in large they are preventable. If we would just gather forces to tackle the pathogenesis awareness activities in rural areas, we can save lives and minimize the rare disease patient pools. The circle of impacted people is way beyond the patient itself and can reach as high as 3-fold taking in consideration the caregivers’ devotion to their life to treat their beloved ones. It should be part of orphan drug companies’ endeavor to create awareness and tackle orphan diseases in areas that will not necessarily be the most lucrative in the mission to save lives. I believe this activity should be a fundamental target and a solid KPI in the performance of any orphan drug company. Orphan drug companies should be measured by what they do beyond the creation of shareholders monetary value. It will be great to see this vision become part of the norm of making rare disease patients from neglected to protected by raising the awareness and the importance of applying preventative steps to address rare disease scattered globally.