Funding Clinical Trials
Clinical trials are an essential step in the road to preventing, treating and, hopefully, curing disease. They test the safety and effectiveness of treatments before they are made available to the public. While clinical trials are necessary to ensure safety, getting a treatment approved by the FDA is a very long and very expensive process.
DEF-funded researchers conduct and participate in many preclinical and clinical trials in an effort to find new treatments and cures for eye diseases, including keratoconus, macular degeneration, glaucoma and retinitis pigmentosa, among others. While outside funding from corporations, the government and other organizations is available for trials, financial resources are scarce, and there are many, often more well-known, diseases “fighting” for a share.
What is a clinical trial?
Clinical trials evaluate the safety and effectiveness of potential treatments before they are approved for the public. They take place after extensive research and other testing is completed to move forward treatments that show the most promise.
According to the US Food and Drug Administration (FDA), “A clinical trial is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to find treatments that work in people and new ways to improve health.
“There are different kinds of clinical trials, including those to study prevention options, new treatments or new ways to use existing treatments, new screening and diagnostic techniques, and options for improving the quality of life for people who have serious medical conditions.”
Clinical trials are generally categorized into three types or phases, which take place after years of preclinical studies. A Phase 1 clinical trial is the first stage of testing in humans, usually with a small group and the primary goal of assessing safety. If the therapy is confirmed as safe, Phase 2 clinical trials commence with a larger group to assess both safety and efficacy. The next step is a Phase 3 clinical trial, which is usually conducted with large groups in multiple locations, in order to definitively assess efficacy and dosing. Because of their size and duration, Phase 3 trials are the most expensive, time-consuming and difficult trials, especially for chronic medical conditions.
Due to the expense – both in dollars and time – of the clinical-trial phases, the massive amount of research done during the “preclinical phase” becomes even more important. It can be especially difficult for researchers to get financing during this time, as it will be many years before funders – including venture capital firms, universities and corporations – see a return on their investment. However, without this necessary stage, no new therapies will advance. It is here that The Discovery Eye Foundation makes a critical difference, by providing pre-clinical “bridge funding” while vision researchers apply to larger funders, such as the National Institutes of Health, to continue their work.
How much do they cost?
The cost of conducting clinical trials is growing at an average rate of 4.6% per year in the United States and is expected to reach $32.1 billion in 2011, reports Drug Discovery & Development magazine. Further, the number of clinical trials is predicted to reach more than 13,000. As a therapy progresses in the clinical trial process, costs* increase:
- Phase 1: Costs range from about just less than $1 million to about $1.25 million.
- Phase 2: Costs range from about $1.25 million to nearly $2.5 million.
- Phase 3: Costs range from about $2.5 million to nearly $4.5 million.
Why do they cost so much?
Clinical trials are labor-intensive, requiring the time and effort of specialized medical and technical personnel. Of course, trial participants’ time is also required, and often compensated. As noted by Foundation Fighting Blindness, clinical tests and exams often consume many hours, multiple evaluations are usually needed during a period of up to three years, each evaluation can cost thousands of dollars, and hundreds of participants may take part in a clinical trial. All this adds up to a very costly process.
And it doesn’t end there. Complying with FDA regulatory requirements, as well as the requirements of other countries’ regulations, is another huge expense. Additional cost factors may include academic trial units and/or contract research organizations, which help run studies and ensure compliance. All this is before costs for producing, manufacturing and marketing successful therapies.
“While the clinical trial process is an arduous and expensive one, it is also necessary,” says DEF Medical Director Anthony Nesburn. “DEF is committed to funding the world’s best vision researchers as they bring promising treatments for eye disease to clinical trials and, eventually, to the public.”
* cost estimates by Foundation Fighting Blindness