“Fail faster” is a saying in drug research. In 2014, the Tufts Center for the Study of Drug Development estimated that the average cost of bringing a new drug through the development pipeline and Food and Drug Administration (FDA) approval process to market is $2.6 billion. More than 90 percent of all drugs being developed in research labs fail to reach the market. Thus, the earlier researchers realize in the drug development process that the drug is not going to meet its objectives, the quicker they can pull the plug, saving critical time and money that can be invested in a new drug.
The lack of access to patient data
The FDA mandates that new therapies are tested on humans in carefully staged clinical trials for safety, efficacy and dosage. This is typically the most expensive segment of the pipeline. Today, there are no FDA-approved therapies to treat celiac disease, a genetic autoimmune disorder that affects approximately 3 million Americans, or non-celiac gluten sensitivity, which is believed to impact at least that many Americans and likely more. Unfortunately, approximately 30 percent of patients with celiac disease continue to experience symptoms and/or intestinal damage while on the gluten-free diet. Drugs that will improve the quality of life for millions who are now suffering are desperately needed, but researchers must have access to larger and deeper pools of high-quality patient data that will empower them to better target treatments.
The struggle to recruit
In addition to actively advancing the search for treatments and a cure, patients who choose to participate in clinical trials may themselves get some relief from celiac disease and non-celiac gluten sensitivity symptoms. Medical researchers must populate clinical trials with the right quantity and mix of patients (including controls), but many struggle to identify and recruit the individuals needed for their trials, most often because patients and their doctors are not aware of the relevant clinical patient trials available.
This is where disease advocacy organizations like the Celiac Disease Foundation come in. Educating and empowering patients to play an active role in the research and treatment process has proven to be the most efficient path to the development of effective treatments and cures for disease. Widespread patient participation in clinical trials will help accelerate much-needed and long-overdue medical breakthroughs.
Marilyn G. Geller, Chief Executive Officer of Celiac Disease Foundation, [email protected]