Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives for children and other patients with these heartbreaking conditions.
That's the finding of a major examination, published today in the weekly newsmagazine of the world's largest scientific society, of the status of new drugs for the 7,000 conditions that affect 200,000 patients or fewer and fall into the "rare disease" category. Written by senior editor Lisa Jarvis after months of interviews with patients, parents, pharmaceutical industry officials and others, it is the cover story in this week's issue of Chemical & Engineering News (C&EN), which reaches more than 138,000 scientists, policy-makers, educators and others. The 4-part story, and a wealth of associated online-only content, is available at the end of this press release.
"For most of the last century, people afflicted by rare diseases especially the parents and families of young children shared the heartbreak of knowing that medicines to treat their loved ones were little more than a dream," says A. Maureen Rouhi, Ph.D., editor-in-chief of C&EN. "As our story documents in such compelling fashion, that situation is dramatically changing. Pharmaceutical companies are making unprecedented investments in medicines for these enigmatic conditions, popularized in films, and treatments for some are on the way."
Jarvis describes how a combination of factors has coalesced to foster a renaissance in drug development for rare diseases.
Smaller drug companies, for instance, have shown that it is possible to make big profits from sales of medicines for rare diseases, and larger companies have taken notice, Jarvis explains in the article. Patient advocacy groups are ano
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American Chemical Society