Every Drug Will Be An Orphan Drug

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  • 08/22/2012
A study of the economic value relative to the investment in orphan diseases and drugs that target specific subpopulations demonstrates they generate more returns to R&D than the old blockbuster approach..

The article mentions that such drugs can command a high price.  In point of fact -- and this is something worth studying -- most companies that have developed new orphan or targeted therapies underwrite the cost for patients or substantially discount the price.   I don't think this is sustainable because eventually all new medicines will be targeted and likely to be taken orally rather than injected.    The most interesting piece of information in the study is the observation that orphan drugs are more likely to be approved more quickly, have a higher degree of success in getting FDA approval and are more likely to be adopted more quickly.   The reduction in risk and uncertainty is likely to be more valuable to companies than launch price.    Moreover, the shift to oral therapies is probably associated with increased compliance and increased survival.   

You ask Amicus CEO John Crowley about why his firm is determined to turn infusions for Pompe's, Fabry's and Gaucher's diseases into pills and he will tell what it's like to have to spend a day and a half transporting his children to a hospital when they could be taking a pill and be monitored at home through sensors or online tools.    

Indeed, I think were are now in the age of personalized medicine.   The use of biomarkers and sophisticated analytical tools to weed out toxicities specific to subpopulation is becoming standard operating procedure.  Rather than just jumping into development with a target that is biologically active, companies large and small are engaging in what my colleagues and mentors Frank Douglas and Steve Paul have described as value-driven proof of concept.  

  Additionally,  people can now monitor their health at home, through apps and sensors that collect and analyze data in real time and in less time than it takes to check email or text messages.   And people are know sharing information about their 'consumer' experience,  opinions that can shape their care and future product development.  If companies will listen.

As Josh Lederberg often said: science is not linear in its progression.   Lederberg also  noted that for innovation to flourish, social and economic institutions had to change with medical progress as well.   For the most part, despite pushback from those who warned about a 'tsunami' of new biologics that would devour every dollar spent on health care, the future is here.  These new biologics are more targeted and more effective.  They extend life and reduce more expensive hospitalization.   They are solutions that make staying healthier and living longer easier and cheaper to do.   We seek out such products for the same reason smart phones and notebooks are crowding out conventional PC sales:   They make doing what we want easier and more enjoyable.  They enrich life.   We need more articles that track these benefits and more companies that use these values to design and produce new medical products.   And companies will or should be more savvy in how they launch these products in countries where the middle class is growing along with life expectancy.   

Point of care and personalized diagnostics, tools to prevent disease and targeted therapies that ultimately 'cure' by shutting off every avenue of escape for degenerative illnesses that are widely commercialized across the planet.   I think we are getting there.   


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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