Communication is a two-way street, but unfortunately that is not always the case when finding ways to research rare disease. Sharing of data by researchers does not happen as easily, or as often as you would hope. This is especially true in the realm of medical studies and clinical trials. Far too often information is not widely shared because the companies and/or academic researchers do not want to give up control of what they consider to be proprietary information.

A recent article by Amy Dockser Marcus in the Wall Street Journal goes into great depth about this topic. The article focuses on what parents who have children with a rare and debilitating diseases are doing to change the pattern. These parents hope to have the scientists and researchers share the data in an effort to reduce the amount of children that will have to participate in so many studies. As GIST patients can attest, there are so many hoops to jump through to gain admission, let alone actively take part in a study or trial.

The National Institutes of Health and the Food and Drug Administration are on board with this research model, so changes could be in our future. The Journal article states that some parents have offered to raise money to cover the costs of a medical study. If companies, hospitals and researchers don’t have to eat the total cost of these studies, then efforts to develop treatments for rare diseases may increase.

The Life Raft Group has been involved in this kind of collaborative research for over six years. Research projects are done in groups with group members sharing findings and methods not just with each other but with other Research Team members at quarterly meetings. This allows the researchers to learn from one another and adjust their projects according to new information. The value of teamwork is undervalued in an era where the need for individual advancement often overshadows the greater good.research-rare-disease

In addition, the LRG Research Team is directly funded. Top cancer centers can charge anywhere from 50% to a whopping 75% in indirect costs, leaving the researcher with as little as 25 cents from every dollar donated. While indirect or administrative costs are an unavoidable part of all research, the LRG believes that number is too high, and money allocated to a research effort should go directly into the hands of the researcher. At Pathway to a Cure’s start in 2007, all indirect costs were capped at 10% and within a few years, we were able to lower that number to zero. We can now take that money and feed it into this groundbreaking research.

Click here to learn more about LRG Research. Read the full Wall Street Journal article here.


LRG Staff
Author: LRG Staff