The premier journal, Science, published a May 2016 article entitled Paying for Future Success in Gene Therapy.  In this article, Harvard University’s, Stuart Orkin, and co-author Phillip Reilly discuss the intersection of money, health and investment in the future. With gene therapy more of a probable reality than a futuristic pipe dream, Orkin and Reilly ask readers to account for the costs and value of such treatment.  They ask provocative questions:

“For a one-time therapy that is the product of a lengthy and expensive preclinical and clinical effort, what is the appropriate price to charge the patient and, ultimately, society? How should that price be determined? Who will pay?”

They remind us that gene therapy and drug advances are emerging at a contentious time, where the ethics of drug pricing may come in conflict with the values of capitalism and private enterprise.  Where is the federal government on this?  According to Orkin and Reilly, we have a problem:

“In drug development, great scientific success creates short-term monopolies limited by patent life, legislative protections, and other forces.”

So what is the problem with this?  Doesn’t profit help fuel the competition for better, more effective products? Well, the rest of the world doesn’t think it is worth it.

“The United States stands virtually alone in letting a free market determine drug prices.”

This is where the “healthcare economists”–I never even knew we have that specialty of economists–come in with their cost-benefit analysis models…and we know what that means. High prices for sick people.

Orkin and Reilly suggest that even a small number of case studies is important to pay for and asks that the companies themselves.  They also suggest that profits from the Orphan Drug Act flow back to companies to develop new therapies for “orphan” diseases and disorders.  Finally, they charge the U.S. National Academy of Medicine (or a similar body) with the task of “exploring new methods to streamline the regulatory process for developing genetic and perhaps other therapies for ultrarare disorders.”

Orkin and Reilly’s suggestions wound ethical and right.  I am wondering, however, what the unintended consequences might be of the kinds of suggestions they recommend.