No, none of the above scenarios are true.
Sure, it's true though, Rare Disease Day 2014 quickly approaches - an event which has become a global phenomenon. Sure "Rare" communities continue to expand at a quick pace. Sure our voices scream daily on Twitter and Facebook. But something else has happened.
"Big-Pharma," still thought by a certain percentage of the populace as akin to the evils of "Big-Tobacco," has jumped on the Rare Disease bandwagon. And it should be noted rather implicitly, without the addition of the pharmaceutical industry, this sudden "vogue" transformation in Rare Disease awareness could not be taking place. No one can "blame" this phenomenon on the emergence of a Rare Disease "Superstar." We've always had Lou Gehrigs, Michael J. Foxes, Mattie Stepaneks, and Sam Bernses to lead the way. We've always had parades and marches, as well as lobbyists and advocates knocking on Capitol Hill office doors. What we haven't had until now is pharma as such a strong and willing partner; a partner now backed by "uber"-eager boardrooms and hordes of risk-taking investors.
The headlines have screamed this past month. Of a new investment fund for Orphan Drugs and Rare Diseases. Of new Rare Disease drugs in pipelines. Of emerging new companies, corporations, and mega-conglomerates vying to crack the Rare Disease market. Almost daily now, Rare Diseases are featured in mainstream headlines as well as full-page articles on stock-trading websites. Just a very, very small sampling of recent headlines:
‘Orphans’ No More: Renaissance in New Drugs for Rare Diseases"
"Wall Street's Next Bet: Cures For Rare Diseases"
Moderna Signs Rare Disease Pact with Alexion and Launches Onkaido
"Sanofi Strikes $700m Rare Diseases Deal"
"Drug Makers See Profit Potential in Rare Diseases"
"Rare Disease Biotech Ultragenyx Sets Terms for $75 million IPO"
"New Way to Make Big Money in Biotech Stocks Sector"
"NIH and Appistry Partner to Implement Genetic Analysis Pipeline for Undiagnosed Diseases"
"Genzyme Expands Strategic Agreement With Alnylam To Target Rare Genetic Diseases"
My take on the debate? I choose not to examine motives, as ulterior as some suspect them of being. Why pharma and "Wall Street" are on this Rare Disease quest is of no interest to me. The important matter, the only matter as far as I'm concerned, is that what's happening IS happening.
We need treatments. We need cures. We need lives saved; lives of "invisible" individuals relegated to the shadows with disease names that most can't even pronounce. Remember those almost 7,000 Rare Diseases - they are genetic, hereditary and chromosomal. They wipe out immune-systems, they paralyze, they choke, and they send many to early graves. They are ALS, CVID, Batten's, Fragile-X, and more. Our only collective hope of normality and survival rests with the creation of novel pharmaceutical and biopharma products.
The warmth of the spotlight is glaring on Rare Disease at the moment. I'm going to continue to peruse the Web for relevant headlines on a daily - no, make that an hourly - basis. The "Rare" bandwagon is filling up quickly, and I predict that what has gained momentum slowly, and has picked up amazing steam as-of-late, is going to turn into one hell of a ride!
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