Orphan Drugs and Healthy Survivors

Thanks for bearing with the dry, didactic nature of my last post. That skeletal history of orphan drugs set the stage for today's post on "Entering the Age of the $1 Million Medicine."

In a Reuters release, journalist Ben Hirschler discusses how >25% of new medicines approved in the US last year were designated for orphan diseases -- great news for Healthy Survivors with these diseases. 

The model for orphan drug development is based on relatively small, less expensive studies. Government incentives provided by Orphan Drug Act have successfully raised interest by various pharmaceuticals. Any increase in competition that drives down prices is good news for Healthy Survivors, too. 

The problem is the increasing financial pressures globally that are causing governments and insurance companies to balk at costs, such as gene therapy for lipoprotein lipase deficiency [projected to cost ~$1 million/patient when released this summer]. Patients may be unable to afford more effective, less toxic treatments that are not fully covered.

Pharmaceuticals justify the pricing, since a single dose that cures a patient gives little opportunity to recoup their investment, a victim of its own success.

Healthy Survivors care about orphan-drug development because...

• Insights about the diagnosis and treatment of orphan diseases may shed light on common diseases.
• Some drugs that were developed to treat rare cancers have since become treatment for more common diseases.
• The model for fast-track development and approval of orphan drugs may hold valuable lessons for improving the way we develop, release and follow all drugs.

Comments

[Bill Kleine]

I met a young man when my son was competing for a college scholarship. The school had their best and brightest to meet with the twelve candidates and their families. My wife and I sat a while with Andrew and asked about his goals. His story came out a little, and so I shared my cancer story and then he told all about his successful sophomore year with a brain tumor [germ cell]. Andrew graduated a year ago and was given a National Science Foundation grant to pursue his doctorate in engineering, developing nano particle delivery systems small enough to pass through the blood-brain barrier. There are many such stories that drive brilliant young men and women to fight disease rather than pursue money. Those are what interest me. Orphan status helps such people pursue their passion to cure. Pharmaceutical companies are all about the pursuit of profit and "need" incentives to take on the humane.