Still, orphan drugs, which cost up to $600,000 a year, may end up driving a broader discussion of how much is too much. This is an issue “that society is going to have to deal with,” says Mike Scott, who serves on the board of directors of the International Myeloma Foundation. “And that has commercial implications, research implications and societal implications.”

As Coté sees it, the focus should be on getting orphan drugs made, whatever the cost, to repay a long overdue debt. Rare diseases have led to a host of medical knowledge, including the urea cycle, metabolism and blood clotting. “We owe people with rare diseases some therapies,” he argues, “because most of our understanding in medical science is based on rare diseases.”

This article was originally published with the title Adoption Agents.

2 Comments

1. 1. Lynn S 10:43 PM 6/3/10

   Rare diseases frequently open paths to treatments for more prevalent diseases. If one child suffers from a rare condition or loses his life to an orphan disease, the world loses the potential gifts of that human being and others with the same affliction. Analyzing genetics while studying orphan diseases, may lead to new approaches that could be used against more familiar diseases. The possibility of making a spectacular discovery is always a possibility when engaging in any scientific research. It is shortsighted to minimize the value of the scientific process itself which frequently yields unanticipated, accidental benefits while focused on a specific unknown.

   Reply | Report Abuse | Link to this

2. 2. dani.tal@cio-1.com 02:49 PM 9/16/10

   How long is a drug under patent ?
   Will a pharma company develop a drug for 1M patients a year?
   I suggest the following formula:
   
   1,000,000
   N =( ----------------------------) X Standard drug patent years
   Annual number of patients
   
   New drug patent years = N
   
   No need for tax reduction etc.
   
   Dani.Tal@cio-1.com

   Reply | Report Abuse | Link to this