Rare Disease Day has truly become an epic event. And one need only check the headlines in mainstream media, business publications, pharma and biopharma websites, and various social media platforms to see that Rare Diseases are in the news spotlight like never before. Rare Diseases and Disorders have traditionally been the "ugly-ducklings" of the worlds of disease and chronic illness, and perhaps with good reason: it's easy to be ignored when your disorder affects only 1 person per 10,000, 50,000, 250,000, or million individuals.
In Rare Diseases 2.0, taking a chance that the spotlight will randomly fall upon your particular disorder will no longer be a viable option. There is far too much at stake. The Orphan Drug marketplace is exploding with new firms springing up almost weekly; acquisitions, mergers, and IPOs are all taking place at a frenetic, feverish pace, and new"specialty-drug" ( Rare Disease drug) pharma and biopharma manufacturing facilities are breaking ground across the globe. Stem-cell advances, representing hope for true cures, are also making almost weekly leaps. Genomics and gene-sequencing, which promise new horizons for disease-mapping and precise, targeted-treatments, are also racing ahead at full-speed. This is high-stakes!
So what is a Rare Disease to do in Rare Diseases 2.0? How is a Rare Disease or Disorder, yours in particular, going to set itself apart from the other 7,000 or so, mostly nameless, faceless afflictions that 99.9% of the globe has never heard about? How is your particular disorder going to vie for and win the attention of pharmaceutical and biopharmaceutical companies and corporations, individual investors, investment funds, researchers and scientists? My answer? With a great deal of planning and effort.
Pharma, biopharma, and investors are "courting" Rare Diseases. Where these institutions go, the money follows in the form of research, studies, clinical trials and R&D. In Rare Disease 2.0 it is no longer an option to sit back and hope they will choose your disorder. Individual diseases must take a proactive approach and begin to court the pharmaceutical industry and its investors. The individual disorder must begin to sell itself: "This is who we are. This is the condition which affects us. Take a good look at us, and see how with a partnership, we can establish a win-win formula: new and more effective treatments and cures for us, and a healthy ROI for you." So how can this "marketing" of individual Rare Diseases best be accomplished in 2.0?
1) Rare Diseases must reach out and engage their suitors.
2) Rare Diseases must develop a 21st-century, savvy business mentality.
3) Dynamic and informative sales-pitches must be developed and strategically placed in the public and private realm.
4) Rare Diseases must abandon the "wait and hope" approach and develop a "go out and make it happen" approach. Rare Diseases must up their levels of surveillance; they must become more opportunistic entities.
5) Although Rare Diseases typically have many websites and awareness campaigns by individual patients and advocates, they must also develop a more united, centralized approach and brand. Fragmented messages and branding lead to confusion and can easily be mistaken for disunity and disorganization.
6) Rare Diseases must update and revamp tired, stale, informational resources and websites. These "front-lines" of first-impressions must become modernized, interactive, and attractive.
7) Social Media must be engaged like never before. 99.9% of Rare Disease Facebook groups are closed-groups; obviously there exists a need for privacy. These groups are exceptional resources for member-support, but by their very nature they close the vitality, energy, enthusiasm, hopes, dreams, and aspirations of patients and families from the eyes of those who might desire to invest in the particular disorder. In addition to the invaluable support and community that Facebook provides, it must also become a place to engage the outside world. It must become a sales platform. In 2.0, LinkedIn must also be exploited as it is for other business ventures; Rare Disease has become a business.
8) Twitter must become a stronger platform for engagement. With a following of 500 to 2,000, a Rare Disease patient, advocate, or organization can move mountains. A strategic tweet, re-tweeted 3 or 4 times has the capacity to reach a huge audience. It is crucial to remember that "Big Pharma" is strengthening its presence on Twitter. It is now the "mat" at their collective front door. Twitter must become a platform for businesslike, informative, educational, and sales-oriented messages. Messages that go out regularly, in a coordinated manner, and en-masse. In 2.0, Rare Disease messages sandwiched between your newest recipe for chocolate-chip cookies and what you did last night are no longer an option.
9) Rare Diseases must develop a Civil-Rights-oriented approach. We have been ignored and neglected for too long. We are citizens, we pay taxes, we are part of society, and we have expectations. In 2.0, Rare Diseases must understand that although there is a time for cordiality, and "good manners," there is also a time to put on the gloves and fight.
10) Rare Diseases must engage the political establishment like never before. They must come to a deeper understanding of the relationship between government and industry and realize that political connections, once established, open the doors for funding. Political influence, additionally, is often directly tied to the pharmaceutical industry and research institutions. Rare Diseases must become more adept at opening these doors and exploiting the opportunities which they create.
11) Rare Diseases must become fully-cognizant of the current, unprecedented atmosphere which the media is providing. Rare Diseases must become more adept at courting the media, asking for attention and coverage, and presenting the message.
12) The world as-a-whole is in a period of financial cutbacks and austerity measures. These cutbacks are beginning to affect many disorders. Rare Diseases must develop a militant mentality: "Although we may cost more than other illnesses, you will never take back what we have fought for and gained; you will never take away treatments. You will not re-write history, and we will never allow you to turn back the clock on us!"
For this point one can look to one of my Rare Diseases, Common Variable Immunodeficiency (CVID), and to recent events in which US Medicare and insurance companies have threatened to take away life-saving treatments and retroactively rewrite rules of access. CVID has fought back, with one inspirational individual, Randi Schulman, initiating the fight, turning it into a "grassroots" movement, and leading the way. With mass-petitions, protests, and upcoming media events, one of which will be featured prominently on CNN in coming days, an epic battle is being waged, one which could have implications for other Rare Diseases as well. We have collectively drawn a line in the sand and dared the "Powers-That-Be" to cross it. Slowly and surely, they are backing down. Call it "militancy" or call it "civil-disobedience," it is an integral part of Rare Diseases 2.0.
The era of Rare Disease 2.0 has begun.
Collectively we have gained the attention of those who can influence and impact our futures. Opportunities exist like never before. But with almost 7,000 Rare Diseases, there will not be opportunity for all.
How will your Rare Disease set itself apart in 2.0?
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